This document lists publications by Ari Gnanasakthy related to patient-reported outcomes in clinical trials and health economics evaluations. It includes 22 publications ranging from 2005 to 2010 related to establishing minimally important differences in patient-reported outcomes, developing guidance for collecting patient-reported data, evaluating the cost-effectiveness of drugs like valsartan and rivastigmine, and methods for conducting economic evaluations alongside multinational clinical trials. The publications indicate Gnanasakthy has extensive experience in analyzing patient-reported outcomes and health economics data from clinical trials.
Advanced Diagnostics in the Post-PAMA EraJohn Hanna
The document discusses trends impacting molecular diagnostics reimbursement, including:
1) Commercial payers are increasingly adopting strategies like specialty drug carve-outs, step therapies, prior authorization, and utilization management programs to contain costs, similar to strategies used for specialty drugs.
2) Reference pricing and lab benefit management programs that provide price transparency and incentivize use of lower-cost providers have been associated with reduced prices and spending while maintaining patient access.
3) Post-PAMA reforms like ADLT designation and Medicare clinical lab fee schedule updates aim to balance innovation and access for new tests, but challenges remain around coding, rate-setting, and determining what constitutes "new clinical information".
El Estudio que Demostró el Control de la Industria de la Enfermedad sobre la Medicina
Este nuevo estudio, el primero en su tipo, comparó específicamente el nivel de publicidad farmacéutica con la cantidad y tipo de publicaciones acerca de suplementos alimenticios. Los autores revisaron un año de publicaciones de los 11 journals más grandes del mundo como por ejemplo el Journal of the American Medical Association, New England Journal of Medicine, British Medical Journal, Canadian Medical Association, Annals of Internal Medicine, Archives of International Medicine, Archives of Pediatric and Adolescent Medicine, Pediatrics and Pediatric Research y el American Family Physician.
Medication nonadherence cost and noncompliance in clinical trialsSynegys
Drug development has reached over $2.6 B and is driven by a clinical trial's success rate, out-of-pocket study costs and study timescales. However, medication nonadherence is a hidden cost which heavily influences these cost drivers. We discuss how medication nonadherence introduces data variability, requiring trial managers to enrol more patients to maintain statistical power, which in turn extends trial timelines. Cost savings are described based on improving study noncompliance with a compliance tool such as Synegys' mComply. This mHealth tool reduces costs as a result of improved statistical power, lower enrollment and shorter trial duration.
Prospective identification of drug safety signalsIMSHealthRWES
At a time of growing demand for more accurate and timely
drug safety evidence, a landmark study supports the value of
electronic medical records (EmR) for detecting new adverse
reactions.
March 02, 2018
Value-based health care is one of the most pressing topics in health care finance and policy today. Value-based payment structures are widely touted as critical to controlling runaway health care costs, but are often difficult for health care entities to incorporate into their existing infrastructures. Because value-based health care initiatives have bipartisan support, it is likely that these programs will continue to play a major role in both the public and private health insurance systems. As such, there is a pressing need to evaluate the implementation of these initiatives thus far and to discuss the direction that American health care financing will take in the coming years.
To explore this important issue, the Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School collaborated with Ropes & Gray LLP to host a one-day conference on value-based health care. This event brought together scholars, health law practitioners, and health care entities to evaluate the impact of value-based health care on the American health care system.
For more information, visit our website at: http://petrieflom.law.harvard.edu/events/details/will-value-based-care-save-the-health-care-system
This document summarizes information from PORTAL (Program On Regulation, Therapeutics, And Law) about prescription drug prices and value in the US. It notes that prescription drug spending has risen significantly in recent years due largely to high prices of brand name drugs. However, drug prices do not reflect true value, as the FDA approval process does not consider value, payors have limited ability to exclude low-value drugs, and physicians are not required to consider value when prescribing. Potential solutions discussed include value-based contracts, systematic assessment of drug value, and policy changes to promote value-based prescribing.
The survey analyzed treatment patterns for multiple myeloma in Germany between 2008-2011 based on data from 478 patients. Key findings include:
- Bortezomib-chemotherapy regimens are now preferred for first-line treatment regardless of planned autologous stem cell transplantation (ASCT), which was performed in around 30% of eligible patients.
- Thalidomide- and lenalidomide-based therapies are commonly used in the second-line setting in 31% of patients.
- Cytogenetic testing increased from 23% in 2008 to 53% in 2011 and influences treatment decisions, though age and comorbidities remain major factors.
- Supportive care needs decreased
Advanced Diagnostics in the Post-PAMA EraJohn Hanna
The document discusses trends impacting molecular diagnostics reimbursement, including:
1) Commercial payers are increasingly adopting strategies like specialty drug carve-outs, step therapies, prior authorization, and utilization management programs to contain costs, similar to strategies used for specialty drugs.
2) Reference pricing and lab benefit management programs that provide price transparency and incentivize use of lower-cost providers have been associated with reduced prices and spending while maintaining patient access.
3) Post-PAMA reforms like ADLT designation and Medicare clinical lab fee schedule updates aim to balance innovation and access for new tests, but challenges remain around coding, rate-setting, and determining what constitutes "new clinical information".
El Estudio que Demostró el Control de la Industria de la Enfermedad sobre la Medicina
Este nuevo estudio, el primero en su tipo, comparó específicamente el nivel de publicidad farmacéutica con la cantidad y tipo de publicaciones acerca de suplementos alimenticios. Los autores revisaron un año de publicaciones de los 11 journals más grandes del mundo como por ejemplo el Journal of the American Medical Association, New England Journal of Medicine, British Medical Journal, Canadian Medical Association, Annals of Internal Medicine, Archives of International Medicine, Archives of Pediatric and Adolescent Medicine, Pediatrics and Pediatric Research y el American Family Physician.
Medication nonadherence cost and noncompliance in clinical trialsSynegys
Drug development has reached over $2.6 B and is driven by a clinical trial's success rate, out-of-pocket study costs and study timescales. However, medication nonadherence is a hidden cost which heavily influences these cost drivers. We discuss how medication nonadherence introduces data variability, requiring trial managers to enrol more patients to maintain statistical power, which in turn extends trial timelines. Cost savings are described based on improving study noncompliance with a compliance tool such as Synegys' mComply. This mHealth tool reduces costs as a result of improved statistical power, lower enrollment and shorter trial duration.
Prospective identification of drug safety signalsIMSHealthRWES
At a time of growing demand for more accurate and timely
drug safety evidence, a landmark study supports the value of
electronic medical records (EmR) for detecting new adverse
reactions.
March 02, 2018
Value-based health care is one of the most pressing topics in health care finance and policy today. Value-based payment structures are widely touted as critical to controlling runaway health care costs, but are often difficult for health care entities to incorporate into their existing infrastructures. Because value-based health care initiatives have bipartisan support, it is likely that these programs will continue to play a major role in both the public and private health insurance systems. As such, there is a pressing need to evaluate the implementation of these initiatives thus far and to discuss the direction that American health care financing will take in the coming years.
To explore this important issue, the Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School collaborated with Ropes & Gray LLP to host a one-day conference on value-based health care. This event brought together scholars, health law practitioners, and health care entities to evaluate the impact of value-based health care on the American health care system.
For more information, visit our website at: http://petrieflom.law.harvard.edu/events/details/will-value-based-care-save-the-health-care-system
This document summarizes information from PORTAL (Program On Regulation, Therapeutics, And Law) about prescription drug prices and value in the US. It notes that prescription drug spending has risen significantly in recent years due largely to high prices of brand name drugs. However, drug prices do not reflect true value, as the FDA approval process does not consider value, payors have limited ability to exclude low-value drugs, and physicians are not required to consider value when prescribing. Potential solutions discussed include value-based contracts, systematic assessment of drug value, and policy changes to promote value-based prescribing.
The survey analyzed treatment patterns for multiple myeloma in Germany between 2008-2011 based on data from 478 patients. Key findings include:
- Bortezomib-chemotherapy regimens are now preferred for first-line treatment regardless of planned autologous stem cell transplantation (ASCT), which was performed in around 30% of eligible patients.
- Thalidomide- and lenalidomide-based therapies are commonly used in the second-line setting in 31% of patients.
- Cytogenetic testing increased from 23% in 2008 to 53% in 2011 and influences treatment decisions, though age and comorbidities remain major factors.
- Supportive care needs decreased
- The study examined how Medicare Part D enrollment affected out-of-pocket prescription costs and medication use among Medicare beneficiaries with cardiovascular disease. Nearly 49% of these beneficiaries enrolled in Part D plans.
- Those without prior drug coverage reported their monthly out-of-pocket costs decreased the most and were more likely to increase their use of cardiovascular medications after enrolling in Part D.
- Dual-eligible beneficiaries (covered by both Medicare and Medicaid) reported their out-of-pocket costs increased the most and were less likely to use cardiovascular medications after shifting to Part D plans.
Application of Pharma Economic Evaluation Tools for Analysis of Medical Condi...IJREST
Application of Pharma Economic Evaluation Tools for Analysis of Medical
Conditions: A Case Study of an Educational Institution in India
1 Dr. Debasis Patnaik, 2 Ms. Pranathi Mandadi
1Assistant Professor, Department of Economics, BITS-Pilani, K K Birla Goa Campus, Goa, India
2Research Scholar, Department of Economics, BITS-Pilani, K K Birla Goa Campus, Goa, India
ABSTRACT
The basic idea of a QALY is straightforward. The amount of time spent in a health state is weighted by the utility score given to
that health state. It takes one year of perfect health (utility score of 1) to generate one QALY, whereas one year in a health state
valued at 0.5 is regarded as being equivalent to half a QALY. Thus, an intervention that generates four additional years in a health
state valued at 0.75 will generate one more QALY than an intervention that generates four additional years in a health state valued
at 0.5. This paper discusses effect of self-medication on health care taking an educational institution population comprising of
students, teaching and non-teaching staff in 2011.
Keywords: Pharma economics, QALY, measuring clinical and health excellence
This document discusses the use of biomarkers in drug development. It begins by defining biomarkers and different types of biomarkers used in clinical settings. It then discusses how biomarkers can help facilitate drug development by aiding in preclinical and clinical decisions. Molecular biomarkers discovered through genomics and proteomics are highlighted. The document also discusses predictive efficacy testing using gene expression profiles to predict drug class and prioritize drug candidates. It emphasizes that biomarkers have the potential to reduce drug development costs by detecting failures earlier. Overall, the document advocates for the integration of biomarkers throughout the drug development process.
The Value of Targeted Sequencing in Advanced Cancer: DCE to Elicit the Public...Office of Health Economics
This project seeks to elicit the public’s preferences for different features of a genomic test to sequence advanced solid cancer tumours. Understanding the relative preferences for various attributes of targeted testing are useful for determining the value of sequencing approaches, and informing technology adoption decisions. A discrete choice experiment (DCE) survey was designed to assess the preferences of members of the Australian general public for targeted sequencing in advanced cancer. The survey presented respondents with 12 questions in which they had to choose between two unlabelled tests (Test A and Test B). Tests were specified in terms of five attributes: time to receive the test result; cost of the test; likelihood that the test result will lead to a change in treatment; length of time health care professionals spend describing the test; and type of health care team who explains the test result. Respondents were sampled from an online panel and also completed questions related to demographic and socio-economic factors, experiences of cancer and familial history. We found that cost, timeliness, expertise/location and likeliness of changing treatment regimes were identified as attributes of genomic sequencing that are most valuable to a sample of the public. These results will ultimately be compared with the results of an ongoing DCE being conducted with patients with advanced cancer who are undergoing sequencing.
Author(s) and affiliation(s): Paula Lorgelly (OHE), Grace Hampson (OHE), James Buchanan (Oxford), Melissa Martyn (MGHA), Jayesh Desai (PeterMac), Clara Gaff (MGHA), and iPREDICT MGHA Flagship collaborators
Conference/meeting: EuHEA 2018
Location: Maastricht, the Netherlands
Date: 12/07/2018
This document discusses clinical practice guidelines for oncology treatment. It defines what guidelines are and their purpose in helping clinicians make complex treatment decisions. Guidelines should be based on systematic reviews of evidence, developed by multidisciplinary experts, and provide recommendations along with ratings of evidence quality. The document notes limitations of guidelines and the need for them to be relevant to the local healthcare system and population. It advocates for guidelines developed through a transparent process with management of conflicts of interest.
This study analyzed prescription drug utilization data from a Medicare drug discount card program and employer-insured plans to estimate the extent to which seniors filled prescriptions in Canada prior to Medicare Part D. The authors found that those living closer to the Canadian border had lower rates of filling prescriptions domestically, suggesting some crossed the border for cheaper drugs. The effect was greater for brand drugs and those without insurance coverage. Living 10% closer to the border was associated with 1.15% and 0.80% fewer generic and brand prescriptions filled domestically. The results indicate that while Medicare Part D likely reduced cross-border drug purchases, some seniors may still obtain medications from Canada to save on costs.
This document discusses the importance of patient involvement in health technology assessment (HTA). It notes that HTA evaluates new drugs and medical technologies but typically does not incorporate patient perspectives on factors like quality of life. The document advocates that patients should play a role in HTA processes by providing input on treatment outcomes, tolerability, and other real-world considerations to ensure funding decisions align with patient values and needs. It provides examples of how patients could be engaged as representatives, through surveys or public input, and highlights the need for HTA to consider individual patient impacts rather than just average population effects.
Genetic Testing Reduces Specialty Drug SpendWellDyne
An award-winning WellDyneRx study, recognized by the Academy of Managed Care Pharmacy, found that pharmacogenomics screening saved self-funded employers 5 percent in specialty drug claim costs.
In a research report by Berdud, M., Drummond, M. and Towse, A. (2018), a reasonable price for an orphan drug was established based on the proposition that rates of returns from investments in developing orphan drugs should be no greater than the industry average (for all drugs). At the 2018 EuHEA conference held in Maastricht, The Netherlands, 11-14 July, Mikel showed (i) how the reasonable price should be established and (ii) how NICE's cost-effectiveness threshold should be adjusted to ensure a reasonable price for an orphan drug. In slides results are discussed and conclusions showed too.
Author(s) and affiliation(s): Mikel Berdud, PhD (OHE); Prof. Mike Drummond (University of York); Prof. Adrian Towse (OHE)
Conference/meeting: EuHEA 2018
Location: Maastricht, The Netherlands
Date: 12/07/2018
New technologies like drug-eluting stents and implantable cardioverter-defibrillators significantly increased healthcare costs for Medicare beneficiaries between 2003-2006, adding nearly $4 billion annually. While these technologies provided benefits, they disproportionately drove up costs for lower-acuity patients. The study found that 43% of increased costs from drug-eluting stents were for patients without acute coronary syndromes, and growth in costs from implantable cardioverter-defibrillators for non-hospitalized heart failure patients was minimal. Technologies that increase spending most for low-acuity cases may not be providing optimal value.
Measuring the Relationship between Innovative Drugs and AE_2015Jonathan Bryan
This study used logistic regression to analyze the relationship between drug innovation classes and reported adverse events. It utilized a previous study's classification of 645 drugs from 1987-2011 into first-in-class, advanced-in-class, or addition-to-class based on FDA review designations. Adverse event data was obtained from FAERS reports from 2004-2014. The results found that total adverse events per drug followed a power law distribution, with most drugs having few events but some outliers with many. Logistic regression found a weak relationship between adverse events and innovation classes, except for more advanced-in-class drugs having higher lifetime adverse event totals. This highlights the need for more systematic drug outcome data to develop better models of drug
The document discusses medication reconciliation, which is defined as a process to obtain and document a complete list of a patient's pre-admission medications and reference this list when writing admission, transfer, and discharge orders. It provides background on medical errors from medication issues and the importance of medication reconciliation. It then describes the medication reconciliation process, which involves verifying the patient's medication list with them and reconciling the list at discharge.
The Cancer Drugs Fund in practice, under the new frameworkAlex Diamantopoulos
A review of the process and criteria used for consideration of treatments under the CDF framework. This work describes the extent of evidence collection while in the Fund.
The document summarizes rationing strategies for Viagra adopted in four countries:
1) In the US, decisions were diffused with no national policy, so access depends on location and insurance. Some states excluded it while others allowed limited prescriptions.
2) In Germany, courts played a major role in shaping policy, ruling individual cases for reimbursement when ED causes health issues but not to enhance function. No clear guidelines resulted.
3) In the UK, the NHS excluded Viagra initially but later allowed exceptions for severe ED after assessment. Usage has remained low due to cultural norms and cost deterrents.
4) In Sweden, regional councils made independent decisions but generally allowed reimbursement after assessment, and
This budget impact analysis estimates the costs of treating heterozygous familial hypercholesterolemia (HeFH) patients with alirocumab over one year within a hypothetical commercial health plan. The analysis found that treating an estimated 1,120 HeFH patients with alirocumab would result in a total drug cost of $11.7 million for the health plan, or $0.976 per member per month. Sensitivity analysis showed that the model was most sensitive to changes in the wholesale acquisition cost of alirocumab. While alirocumab is more effective than other options, its high cost needs to be weighed against potential long-term outcomes benefits for formulary decision making.
WHITE PAPER: What’s Next in US Payor Communications: The Impact of FDA's Prop...Nathan White, CPC
This white paper from ApotheCom discusses new FDA guidance on communicating healthcare economic information to payors as outlined in the 21st Century Cures Act. Key points:
- The Cures Act establishes guidelines for communicating information on the economic impact of drugs and devices to payors.
- In response, FDA issued draft guidance to clarify what can be communicated about approved drugs to payors.
- The guidance defines payors as including formulary committees and outlines standards for ensuring communicated analyses are scientifically valid and relate to approved drug indications.
- The changes aim to increase transparency while allowing industry to demonstrate treatment value to payors.
Discovery of Drug and Introduction to Clinical Trial_Katalyst HLSKatalyst HLS
Introduction to Discovery of Drug and Introduction to Clinical Trials in Pharmaceuticals, Bio-Pharmaceuticals, Medical Devices, Cosmeceuticals and Foods.
American Journal of Medical Quality-2015-Palacio-1062860614568646Joseph Messina
This document describes a study that evaluated the implementation of a point-of-care medication delivery system (POCMDS) in a primary care-led health system serving vulnerable populations. The study used a mixed-methods approach including interviews, site visits, patient surveys, and claims data to assess the reach, effectiveness, adoption, implementation, and maintenance of the POCMDS intervention based on the RE-AIM and CFIR frameworks. Key findings included that POCMDS has been implemented in 23 practices across 4 states, with high rates of patient satisfaction reported. Facilitators of implementation included leadership commitment, a culture of prevention, and a sustainable business model.
The document summarizes research that shows the benefits of clinical pharmacists in intensive care units (ICUs). Several studies found that ICUs with clinical pharmacist services had lower mortality rates, shorter hospital stays, fewer medication errors and adverse drug events, lower infection rates, and reduced costs compared to ICUs without these services. Specifically, one study found a 2.4-day decrease in length of stay and a reduction in mortality from 8.28% to 6.61% with a clinical pharmacist-led antimicrobial control program. Another study found that mortality rates were 23.6% higher in ICUs without clinical pharmacists for nosocomial infections. The inclusion of clinical pharmacists in direct patient care in the ICU has
The document discusses 3 Ferrari models: the Ferrari 308 from the 1970s-80s with a 3-liter, 8-cylinder engine and glass reinforced plastic body; the Ferrari 328 of the 1980s, which was an updated version of the 308 with a 3.2-liter engine; and the Ferrari 348 of 1989-1995, which replaced the 328 with a larger size and improved visibility and noise reduction.
- The study examined how Medicare Part D enrollment affected out-of-pocket prescription costs and medication use among Medicare beneficiaries with cardiovascular disease. Nearly 49% of these beneficiaries enrolled in Part D plans.
- Those without prior drug coverage reported their monthly out-of-pocket costs decreased the most and were more likely to increase their use of cardiovascular medications after enrolling in Part D.
- Dual-eligible beneficiaries (covered by both Medicare and Medicaid) reported their out-of-pocket costs increased the most and were less likely to use cardiovascular medications after shifting to Part D plans.
Application of Pharma Economic Evaluation Tools for Analysis of Medical Condi...IJREST
Application of Pharma Economic Evaluation Tools for Analysis of Medical
Conditions: A Case Study of an Educational Institution in India
1 Dr. Debasis Patnaik, 2 Ms. Pranathi Mandadi
1Assistant Professor, Department of Economics, BITS-Pilani, K K Birla Goa Campus, Goa, India
2Research Scholar, Department of Economics, BITS-Pilani, K K Birla Goa Campus, Goa, India
ABSTRACT
The basic idea of a QALY is straightforward. The amount of time spent in a health state is weighted by the utility score given to
that health state. It takes one year of perfect health (utility score of 1) to generate one QALY, whereas one year in a health state
valued at 0.5 is regarded as being equivalent to half a QALY. Thus, an intervention that generates four additional years in a health
state valued at 0.75 will generate one more QALY than an intervention that generates four additional years in a health state valued
at 0.5. This paper discusses effect of self-medication on health care taking an educational institution population comprising of
students, teaching and non-teaching staff in 2011.
Keywords: Pharma economics, QALY, measuring clinical and health excellence
This document discusses the use of biomarkers in drug development. It begins by defining biomarkers and different types of biomarkers used in clinical settings. It then discusses how biomarkers can help facilitate drug development by aiding in preclinical and clinical decisions. Molecular biomarkers discovered through genomics and proteomics are highlighted. The document also discusses predictive efficacy testing using gene expression profiles to predict drug class and prioritize drug candidates. It emphasizes that biomarkers have the potential to reduce drug development costs by detecting failures earlier. Overall, the document advocates for the integration of biomarkers throughout the drug development process.
The Value of Targeted Sequencing in Advanced Cancer: DCE to Elicit the Public...Office of Health Economics
This project seeks to elicit the public’s preferences for different features of a genomic test to sequence advanced solid cancer tumours. Understanding the relative preferences for various attributes of targeted testing are useful for determining the value of sequencing approaches, and informing technology adoption decisions. A discrete choice experiment (DCE) survey was designed to assess the preferences of members of the Australian general public for targeted sequencing in advanced cancer. The survey presented respondents with 12 questions in which they had to choose between two unlabelled tests (Test A and Test B). Tests were specified in terms of five attributes: time to receive the test result; cost of the test; likelihood that the test result will lead to a change in treatment; length of time health care professionals spend describing the test; and type of health care team who explains the test result. Respondents were sampled from an online panel and also completed questions related to demographic and socio-economic factors, experiences of cancer and familial history. We found that cost, timeliness, expertise/location and likeliness of changing treatment regimes were identified as attributes of genomic sequencing that are most valuable to a sample of the public. These results will ultimately be compared with the results of an ongoing DCE being conducted with patients with advanced cancer who are undergoing sequencing.
Author(s) and affiliation(s): Paula Lorgelly (OHE), Grace Hampson (OHE), James Buchanan (Oxford), Melissa Martyn (MGHA), Jayesh Desai (PeterMac), Clara Gaff (MGHA), and iPREDICT MGHA Flagship collaborators
Conference/meeting: EuHEA 2018
Location: Maastricht, the Netherlands
Date: 12/07/2018
This document discusses clinical practice guidelines for oncology treatment. It defines what guidelines are and their purpose in helping clinicians make complex treatment decisions. Guidelines should be based on systematic reviews of evidence, developed by multidisciplinary experts, and provide recommendations along with ratings of evidence quality. The document notes limitations of guidelines and the need for them to be relevant to the local healthcare system and population. It advocates for guidelines developed through a transparent process with management of conflicts of interest.
This study analyzed prescription drug utilization data from a Medicare drug discount card program and employer-insured plans to estimate the extent to which seniors filled prescriptions in Canada prior to Medicare Part D. The authors found that those living closer to the Canadian border had lower rates of filling prescriptions domestically, suggesting some crossed the border for cheaper drugs. The effect was greater for brand drugs and those without insurance coverage. Living 10% closer to the border was associated with 1.15% and 0.80% fewer generic and brand prescriptions filled domestically. The results indicate that while Medicare Part D likely reduced cross-border drug purchases, some seniors may still obtain medications from Canada to save on costs.
This document discusses the importance of patient involvement in health technology assessment (HTA). It notes that HTA evaluates new drugs and medical technologies but typically does not incorporate patient perspectives on factors like quality of life. The document advocates that patients should play a role in HTA processes by providing input on treatment outcomes, tolerability, and other real-world considerations to ensure funding decisions align with patient values and needs. It provides examples of how patients could be engaged as representatives, through surveys or public input, and highlights the need for HTA to consider individual patient impacts rather than just average population effects.
Genetic Testing Reduces Specialty Drug SpendWellDyne
An award-winning WellDyneRx study, recognized by the Academy of Managed Care Pharmacy, found that pharmacogenomics screening saved self-funded employers 5 percent in specialty drug claim costs.
In a research report by Berdud, M., Drummond, M. and Towse, A. (2018), a reasonable price for an orphan drug was established based on the proposition that rates of returns from investments in developing orphan drugs should be no greater than the industry average (for all drugs). At the 2018 EuHEA conference held in Maastricht, The Netherlands, 11-14 July, Mikel showed (i) how the reasonable price should be established and (ii) how NICE's cost-effectiveness threshold should be adjusted to ensure a reasonable price for an orphan drug. In slides results are discussed and conclusions showed too.
Author(s) and affiliation(s): Mikel Berdud, PhD (OHE); Prof. Mike Drummond (University of York); Prof. Adrian Towse (OHE)
Conference/meeting: EuHEA 2018
Location: Maastricht, The Netherlands
Date: 12/07/2018
New technologies like drug-eluting stents and implantable cardioverter-defibrillators significantly increased healthcare costs for Medicare beneficiaries between 2003-2006, adding nearly $4 billion annually. While these technologies provided benefits, they disproportionately drove up costs for lower-acuity patients. The study found that 43% of increased costs from drug-eluting stents were for patients without acute coronary syndromes, and growth in costs from implantable cardioverter-defibrillators for non-hospitalized heart failure patients was minimal. Technologies that increase spending most for low-acuity cases may not be providing optimal value.
Measuring the Relationship between Innovative Drugs and AE_2015Jonathan Bryan
This study used logistic regression to analyze the relationship between drug innovation classes and reported adverse events. It utilized a previous study's classification of 645 drugs from 1987-2011 into first-in-class, advanced-in-class, or addition-to-class based on FDA review designations. Adverse event data was obtained from FAERS reports from 2004-2014. The results found that total adverse events per drug followed a power law distribution, with most drugs having few events but some outliers with many. Logistic regression found a weak relationship between adverse events and innovation classes, except for more advanced-in-class drugs having higher lifetime adverse event totals. This highlights the need for more systematic drug outcome data to develop better models of drug
The document discusses medication reconciliation, which is defined as a process to obtain and document a complete list of a patient's pre-admission medications and reference this list when writing admission, transfer, and discharge orders. It provides background on medical errors from medication issues and the importance of medication reconciliation. It then describes the medication reconciliation process, which involves verifying the patient's medication list with them and reconciling the list at discharge.
The Cancer Drugs Fund in practice, under the new frameworkAlex Diamantopoulos
A review of the process and criteria used for consideration of treatments under the CDF framework. This work describes the extent of evidence collection while in the Fund.
The document summarizes rationing strategies for Viagra adopted in four countries:
1) In the US, decisions were diffused with no national policy, so access depends on location and insurance. Some states excluded it while others allowed limited prescriptions.
2) In Germany, courts played a major role in shaping policy, ruling individual cases for reimbursement when ED causes health issues but not to enhance function. No clear guidelines resulted.
3) In the UK, the NHS excluded Viagra initially but later allowed exceptions for severe ED after assessment. Usage has remained low due to cultural norms and cost deterrents.
4) In Sweden, regional councils made independent decisions but generally allowed reimbursement after assessment, and
This budget impact analysis estimates the costs of treating heterozygous familial hypercholesterolemia (HeFH) patients with alirocumab over one year within a hypothetical commercial health plan. The analysis found that treating an estimated 1,120 HeFH patients with alirocumab would result in a total drug cost of $11.7 million for the health plan, or $0.976 per member per month. Sensitivity analysis showed that the model was most sensitive to changes in the wholesale acquisition cost of alirocumab. While alirocumab is more effective than other options, its high cost needs to be weighed against potential long-term outcomes benefits for formulary decision making.
WHITE PAPER: What’s Next in US Payor Communications: The Impact of FDA's Prop...Nathan White, CPC
This white paper from ApotheCom discusses new FDA guidance on communicating healthcare economic information to payors as outlined in the 21st Century Cures Act. Key points:
- The Cures Act establishes guidelines for communicating information on the economic impact of drugs and devices to payors.
- In response, FDA issued draft guidance to clarify what can be communicated about approved drugs to payors.
- The guidance defines payors as including formulary committees and outlines standards for ensuring communicated analyses are scientifically valid and relate to approved drug indications.
- The changes aim to increase transparency while allowing industry to demonstrate treatment value to payors.
Discovery of Drug and Introduction to Clinical Trial_Katalyst HLSKatalyst HLS
Introduction to Discovery of Drug and Introduction to Clinical Trials in Pharmaceuticals, Bio-Pharmaceuticals, Medical Devices, Cosmeceuticals and Foods.
American Journal of Medical Quality-2015-Palacio-1062860614568646Joseph Messina
This document describes a study that evaluated the implementation of a point-of-care medication delivery system (POCMDS) in a primary care-led health system serving vulnerable populations. The study used a mixed-methods approach including interviews, site visits, patient surveys, and claims data to assess the reach, effectiveness, adoption, implementation, and maintenance of the POCMDS intervention based on the RE-AIM and CFIR frameworks. Key findings included that POCMDS has been implemented in 23 practices across 4 states, with high rates of patient satisfaction reported. Facilitators of implementation included leadership commitment, a culture of prevention, and a sustainable business model.
The document summarizes research that shows the benefits of clinical pharmacists in intensive care units (ICUs). Several studies found that ICUs with clinical pharmacist services had lower mortality rates, shorter hospital stays, fewer medication errors and adverse drug events, lower infection rates, and reduced costs compared to ICUs without these services. Specifically, one study found a 2.4-day decrease in length of stay and a reduction in mortality from 8.28% to 6.61% with a clinical pharmacist-led antimicrobial control program. Another study found that mortality rates were 23.6% higher in ICUs without clinical pharmacists for nosocomial infections. The inclusion of clinical pharmacists in direct patient care in the ICU has
The document discusses 3 Ferrari models: the Ferrari 308 from the 1970s-80s with a 3-liter, 8-cylinder engine and glass reinforced plastic body; the Ferrari 328 of the 1980s, which was an updated version of the 308 with a 3.2-liter engine; and the Ferrari 348 of 1989-1995, which replaced the 328 with a larger size and improved visibility and noise reduction.
My video, "Who Am I?" will fill you in on exactly who I am, and who I want to become. I used PowerPoint to complete this video for my Pathways Seminar class at HHHS.
This document discusses the perceived value of different types of communications from financial advisors according to clients. It also outlines some advantages that a financial reporting tool called Frontex Reporter can provide to financial dealerships, including increasing revenue and optimizing client relationships. A hypothetical scenario is presented showing how the tool could generate hundreds of thousands of dollars in additional yearly revenue for a dealership with 80 advisors, depending on the percentage increase in portfolio size it enables.
Rise to the Challenge: Creating a High-Stakes Graduation Project Program to D...Michelle Fossum
Rise to the Challenge: Creating a High-Stakes Graduation Project Program to Demonstrate Excellence in Information Literacy and Independent Learning will engage participants in creating a graduation project in any school setting.
Presented at AASL National Conference, October 2011
Andrew Mitchell introduces himself and provides details about his life, including where he lives in Horsham, Pennsylvania. He discusses his future goals of becoming a lawyer, then a judge, and getting into politics. He also outlines his interests, passions, struggles, and dreams of having a family and supporting them.
This document provides information on applying the vertex formula to problems involving projectile motion. It discusses key concepts like projectiles, parabolas, maximum and minimum heights. Examples are provided for using the vertex formula (-b/2a) to calculate the time a projectile reaches its highest point and the maximum height achieved for scenarios like firework displays and baseballs thrown vertically into the air.
This document provides information about applying the vertex formula to problems involving projectile motion. It discusses key factors like initial height, maximum height, time, and velocity that are involved when a projectile is launched. Examples are provided about using the vertex formula -h = -16t^2 + v0t + h0 to calculate the time a projectile reaches its highest point and what that maximum height is for scenarios like fireworks launching and baseballs being thrown upward.
A 35-year-old pregnant woman at 41 weeks gestation was scheduled for a cesarean section due to fetal bradycardia. During the procedure, the anesthesiologist was unable to intubate the patient and her oxygen saturation dropped to 55%. A size 4 proseal laryngeal mask airway was inserted and her saturation increased to 98%. Intraoperatively, the patient developed laryngospasm. The document discusses management of the difficult airway and questions regarding the use of general anesthesia versus other techniques for this patient.
This document discusses the perceived value of different types of communications from financial advisors according to clients. It also outlines the advantages of using FRONTEX Reporter software, including better client engagement and education, time savings, lower costs, and attracting higher net worth clients. An example return on investment calculation is provided, assuming costs savings from administrative time and increased portfolio revenues, showing ROI increases from 2,375% to 4,375% for advisors using the software.
This document provides an overview of geography and sights to see in Greece. It discusses regions such as North Greece, Thessaloniki, Athens, the Peloponnese peninsula, Greek islands including Ionian, Dodecanese, and Crete. Specific locations mentioned include Kastoria, Ioannina, Kavala, Vikos gorge, Pindos mountains, Meteora, Athens museums, Acropolis, Plaka neighborhood, Olympia archaeological site, and Knossos archaeological site on Crete.
This document provides information on applying the vertex formula to problems involving projectile motion. It discusses key concepts like projectiles, parabolas, maximum and minimum heights. Examples are provided for using the vertex formula (-b/2a) to calculate the time a projectile reaches its highest point and the maximum height achieved for scenarios like firework displays and baseballs thrown vertically.
Patient-Reported Outcomes in Cancer Care - Zeena NackerdienZeena Nackerdien
PROs should be integral to evidence-based cancer care. Here I summarize the latest expert opinions on the subject in the form of a conversation between two oncologists. This information should be helpful to healthcare practitioners and patients alike. As always, please consult your own medical doctors for case-by-case advice.
Application of Pharma Economic Evaluation Tools for Analysis of Medical Condi...IJREST
ABSTRACT
The basic idea of a QALY is straightforward. The amount of time spent in a health state is weighted by the utility score given to that health state. It takes one year of perfect health (utility score of 1) to generate one QALY, whereas one year in a health state valued at 0.5 is regarded as being equivalent to half a QALY. Thus, an intervention that generates four additional years in a health state valued at 0.75 will generate one more QALY than an intervention that generates four additional years in a health state valued at 0.5. This paper discusses effect of self-medication on health care taking an educational institution population comprising of students, teaching and non-teaching staff in 2011.
Keywords: Pharma economics, QALY, measuring clinical and health excellence
Podium Presentation Midwest Social and Administrative Conference,Chicago,2008aramasa3
The document describes a study that developed and tested a survey instrument to measure patient satisfaction with pharmacy services among HIV-infected patients receiving care from either a mail-order or community pharmacy. Exploratory factor analysis established the construct validity of the survey and showed two key factors. Reliability testing found high internal consistency. The survey can be used to compare patient satisfaction across different pharmacy settings and inform efforts to improve patient-reported outcomes.
This summarizes the outcomes of a 6-month pharmacist-provided diabetes management program for employees of the City of Toledo. The program showed improvements in clinical outcomes like A1c and blood pressure. It also improved humanistic outcomes such as patient satisfaction, knowledge, and adherence. Economic outcomes like healthcare utilization and costs improved as well, with a 62.69% reduction in total costs. The study demonstrates positive short-term outcomes across clinical, humanistic and economic domains from a pharmacist-led diabetes management program.
This document discusses regulatory inefficiencies surrounding companion diagnostics and laboratory developed tests (LDTs) in the United States. It uses the case study of Genentech's drug MPDL3280A and its companion diagnostic to show that the FDA thoroughly regulates companion diagnostics but provides no oversight of clinical validity for LDTs. This allows multiple competing diagnostic tests to be used without proof of efficacy. The document also compares healthcare systems and technology assessment processes in the US, UK, and France to illustrate decentralized decision making in the US compared to centralized bodies in other countries.
Is complementary and alternative medicine (CAM) cost-effective? a systematic ...home
Whereas the number and quality of economic evaluations of CAM have increased in
recent years and more CAM therapies have been shown to be of good value, the majority of CAM
therapies still remain to be evaluated
This document discusses using real world data from healthcare databases to support adaptive biomedical innovation. It outlines four key principles - meaningful, valid, expedited, and transparent evidence (MVET) - that are necessary to generate evidence from healthcare databases that is fit for decision making. Meaningful evidence requires using relevant and high quality data sources to answer the research question. Evidence should be generated and shared in a transparent manner while protecting patient privacy. Following MVET principles can help produce rigorous evidence from real world data to support faster access to new medications through adaptive pathways, while maintaining evidentiary standards.
A Catalyst For Transforming Health Systems And Person-Centred Care Canadian ...Becky Gilbert
This document presents a Canadian national position statement on patient-reported outcomes (PROs) from a steering committee of PRO experts.
The key points are:
1. PROs capture the patient perspective on health and quality of life and can improve care, but their use is not consistent in Canada. This position statement aims to support greater PRO implementation.
2. The position statement was developed through an expert consensus process and stakeholder feedback. It contains recommendations in 4 areas: patients and families, health policy, clinical implementation, and research.
3. The overarching recommendations are that resources should be invested to integrate PROs into care, a national PRO body is needed for guidance, and PRO tools must
Pharmaceutical Comparative Effectiveness Research AbstractLona Vincent
This document summarizes several research studies and articles in the field of health economics and outcomes research. It discusses trends in the use of electronic medical records in outcomes research from 2001-2010, with the number of studies increasing over time. It also reviews characteristics of homeless patients using inpatient and emergency services compared to non-homeless patients. Additionally, it compares the role of health technology assessment systems in reimbursement and market access between Turkey and Poland.
ISSN 2347-2251
It appears that you're describing the scope of a scientific journal. This journal covers a wide range of topics related to both Pharmaceutical Sciences and Biological Sciences of the scopus database journal.
Indo-American Journal of Pharma and Bio Sciences is an international, online, English-language journal that publishes articles on pharmaceutical and biological sciences of the ugc carelist journals.
This document summarizes a study that evaluated the knowledge, attitudes, and practices of oncology health professionals in Australia regarding complementary and alternative medicine (CAM). The study surveyed 99 oncology physicians, nurses, and pharmacists. It found that the professionals had moderate knowledge of CAMs but felt unprepared to advise patients due to a lack of expertise. While they acknowledged potential benefits of CAMs, they also expressed safety concerns. Fewer than 40% of patients were open to discussing CAMs with their providers, hindered by a lack of scientific evidence and guidelines. The study reveals a need for more CAM education for oncology clinicians to improve patient-provider discussions and decision-making regarding CAM use.
ISSN 2347-2251
Manuscripts should be carefully checked for grammatical and punctuation errors. All papers undergo peer review. Please note that all articles published in this journal represent the opinions of the authors and do not necessarily reflect the official policy of the Journal of Indo-American Journal of Pharma and Bio Sciences of the journals to publish paper.
Scientific development is an ever-evolving journey, driven by the exchange of data and ideas among researchers across the globe.One such remarkable publication dedicated to facilitating this exchange within the fields of Pharmacy and Bio Sciences is the Indo-American Journal of Pharma and Bio Sciences of the published research.
Patient-centered medical homes (PCMHs) are intended to actively provide effective care by physician-led teams, Where patients take a leading role and responsibility. Objective: To determine whether the Walter Reed PCMH has reduced costs while at least maintaining if not improving access to and quality of care, and to determine
whether access, quality, and cost impacts differ by chronic condition status. Design, setting, and patients: This study
conducted a retrospective analysis using a patient-level utilization database to determine the impact of the Walter Reed PCMH on utilization and cost metrics, and a survey of enrollees in the Walter Reed PCMH to address access to care and quality of care. Outcome measures: Inpatient and outpatient utilization, per member per quarter costs, Healthcare Effectiveness Data and Information Set metrics, and composite measures for access, patient satisfaction, provider communication, and customer service are included. Results: Costs were 11% lower for those with chronic conditions compared to 7% lower for those without. Since treating patients with chronic conditions is 4 times more costly than treating patients without such conditions, the vast majority of dollar savings are attributable to chronic care.
Module 4 Submodule 4. 2 Final June 2007Flavio Guzmán
The document discusses key considerations for clinicians in evaluating the results of drug clinical trials. It emphasizes the importance of critically assessing trial design, results, and whether the findings warrant changing clinical practice. Clinicians are advised to consider factors like trial methodology, potential biases, statistical/clinical significance, applicability to their patients, and safety. The document provides guidance on how to distinguish high-quality trials that produce useful results for patient care.
The document summarizes research on the benefits of clinical pharmacists participating as members of medical teams. Several studies found that including clinical pharmacists reduced mortality rates in hospitals and improved outcomes across disease states. Pharmacists improved medication management by addressing drug-related problems, which led to decreased mortality for conditions like heart attacks. Their interventions enhanced clinical outcomes for diabetes, cardiovascular disorders, and other conditions. Effective implementation of these pharmacy services requires support from healthcare organizations and infrastructure support within facilities.
1) The document discusses improving outcomes and endpoints in cancer trials by better defining what is important to measure, making endpoints more understandable for patients, and advancing endpoints to reflect changes in trial design and treatments.
2) It notes that endpoints need to show clinically relevant benefits to patients, and that improvements in trial design should be accompanied by improvements in available endpoints.
3) Stakeholders including clinicians, patients, and regulators must work together to determine the best approach for research that ensures accountability and optimizes the use of resources.
A Study on Patient Satisfaction towards Cancer Hospitalijtsrd
A cancer diagnosis places considerable stress on patients and their families. They find themselves discomfort with the strange health system making serious decisions with long term consequences living with uncertainness about the nature, cause and indefinite progress of the disease living with a disrupted family, work, social life and facing the possibility of becoming increasingly dependent on others. Dhivya. S | Harshath. S "A Study on Patient Satisfaction towards Cancer Hospital" Published in International Journal of Trend in Scientific Research and Development (ijtsrd), ISSN: 2456-6470, Volume-3 | Issue-6 , October 2019, URL: https://www.ijtsrd.com/papers/ijtsrd29274.pdf Paper URL: https://www.ijtsrd.com/management/consumer-behaviour/29274/a-study-on-patient-satisfaction-towards-cancer-hospital/dhivya-s
A Study on Patient Satisfaction towards Cancer Hospital
Ari Refs
1. Ari Gnanasakthy - Publications
(1) Doward LC, Gnanasakthy A, Baker MG. Patient reported outcomes: looking beyond the
label claim. Health and Quality of Life Outcomes 2010;8.
Abstract: The use of patient reported outcome scales in clinical trials conducted by the
pharmaceutical industry has become more widespread in recent years. The use of such
outcomes is particularly common for products developed to treat chronic, disabling
conditions where the intention is not to cure but to ameliorate symptoms, facilitate
functioning or, ultimately, to improve quality of life. In such cases, patient reported
evidence is increasingly viewed as an essential complement to traditional clinical
evidence for establishing a product's competitive advantage in the marketplace. In a
commercial setting, the value of patient reported outcomes is viewed largely in terms of
their potential for securing a labelling claim in the USA or inclusion in the summary of
product characteristics in Europe. Although, the publication of the recent US Food and
Drug Administration guidance makes it difficult for companies to make claims in the USA
beyond symptom improvements, the value of these outcomes goes beyond satisfying
requirements for a label claim. The European regulatory authorities, payers both in the
US and Europe, clinicians and patients all play a part in determining both the availability
and the pricing of medicinal products and all have an interest in patient-reported data that
go beyond just symptoms. The purpose of the current paper is to highlight the potential
added value of patient reported outcome data currently collected and held by the industry
for these groups
(2) Mordin M, Lewis S, Gnanasakthy A, Demuro-Mercon C, Copley-Merriman K, Fehnel S.
Patient-Reported Outcomes in Product Development Guidance. Value in Health
2010;13(3):A17-A18.
(3) Gnanasakthy A, DeMuro C, Mordin M, Copley-Merriman K, Mauskopf J. The Role of the
Patient Voice in Health Technology Assessment. Value in Health 2010;13(3):A19.
(4) Mordin M, Clark M, Siersma C, Copley-Merriman K, Gnanasakthy A. Impact of the Fda
Draft Guidance on Patient Reported Outcomes (Pro) Label Claims for Approved Drug
Products in the Us: Has It Made A Difference? Value in Health 2009;12(3):A29.
(5) Gnanasakthy A. Controlling measurement error of patient-reported-outcomes during the
implementation stage of clinical trials. Value in Health 2008;11(3):A178.
(6) Revicki DA, Gnanasakthy A, Weinfurt K. Documenting the rationale and psychometric
characteristics of patient reported outcomes for labeling and promotional claims: the PRO
Evidence Dossier. Quality of Life Research 2007;16(4):717-23.
Abstract: The Food and Drug Administration (FDA) and European Medicines Agency
(EMEA) are willing to consider including information on patient reported outcomes
(PROs) in product labeling and advertising. Pharmaceutical industry researchers must
provide sufficient evidence supporting PRO benefit before an approval may be granted.
This report describes the purpose and content of a PRO Evidence Dossier, which
consists of important information supporting PRO claims. The dossier should be
completed by pharmaceutical industry or other researchers to document the planning of
the PRO assessment strategy, psychometric evidence, desired target labeling
statements, and the clinical trial evidence of PRO benefits. The systematic reporting and
documentation of information on the rationale for including PROs, rationale for the
selection of specific PRO instruments, evidence on the psychometric qualities of the PRO
measures, and guidelines for interpreting PRO findings will facilitate achieving a PRO
labeling or promotional claim. Combining all the relevant information into a single
document will facilitate the review and evaluation process for clinical and regulatory
reviewers. The PRO Evidence Dossier may also be helpful to industry and academic
2. researchers in identifying further information that will need to be developed to support the
clinical development program and the PRO endpoints
(7) Lamotte M, Annemans L, Gnanasakthy A, Khan Z. A health-economic evaluation of the
real life long-term consequences of an angiotensin II receptor blocker (ARB) in the
management of post-MI patients. Case study on valsartan in Canada. European Heart
Journal 2005;26:612-3.
(8) Wonder M, Backhouse M, Gnanasakthy A. At what cost? A review of drug acquisition
cost issues in modelled economic evaluations. Value in Health 2005;8(3):384.
(9) Reed SD, Anstrom KJ, Bakhai A, Briggs AH, Califf RM, Cohen DJ, Drummond MF, Glick
HA, Gnanasakthy A, Hlatky MA, O'Brien BJ, Torti FM, Tsiatis AA, Willan AR, Mark DB,
Schulman KA. Conducting economic evaluations alongside multinational clinical trials:
Toward a research consensus. American Heart Journal 2005;149(3):434-43.
Abstract: Demand for economic evaluations in rnultinational clinical trials is increasing,
but there is little consensus about how such studies should be conducted and reported.
At a workshop in Durham, North Carolina, we sought to identify areas of agreement
about how the primary findings of economic evaluations in multinational clinical trials
should be generated and presented. In this paper, we propose a framework for
classifying multinational economic evaluations according to (a) the sources of an
analyst's estimates of resource use and clinical effectiveness and (b) the analyst's
method of estimating costs. We review existing studies in the cardiology literature in the
context of the proposed framework. We then describe important methodological and
practical considerations in conducting multinational economic evaluations and summarize
the advantages and disadvantages of each approach. Finally, we describe opportunities
for future research. Delineation of the various approaches to multinational economic
evaluation may assist researchers, peer reviewers, journal editors, and decision makers
in evaluating the strengths and limitations of particular studies
(10) Yost KJ, Sorensen MV, Hahn EA, Glendenning GA, Gnanasakthy A, Cella D. Using
multiple anchor- and distribution-based estimates to evaluate clinically meaningful
change on the functional assessment of Cancer Therapy-Biologic Response Modifiers
(FACT-BRM) instrument. Value in Health 2005;8(2):117-27.
Abstract: Objective: The interpretation of health-related quality of life (HRQL) data from
clinical trials can be enhanced by understanding the degree of change in HRQL scores
that is considered meaningful. Our objectives were to combine distribution-based and two
anchor-based approaches to identify minimally important differences (MIDs) for the
27-item Trial Outcome Index (TOI), the seven-item Social Well-Being (SWB) subscale,
and the six-item Emotional Well-being (EWB) subscale from the Functional Assessment
of Cancer Therapy-Biological Response Modifiers (FACT-BRM) instrument. Methods:
Distribution-based MIDs were based on the standard error of measurement.
Anchor-based approaches utilized patient-reported global rating of change (GRC) and
change in physician-reported performance status rating (PSR). Correlations and
weighted kappa statistics were used to assess association and agreement between the
two anchors. FACT-BRM changes were evaluated for three time periods: baseline to
month 1, month 2 to month 3, and month 5 to month 6. Results: Association between
GRC and change in PSR was poor. Correlation between the anchors and HRQL change
scores was largest at month 1 and decreased through month 6. Combining results from
all approaches, the MIDs identified were 5-8 points for the TOI, 2 points for the SWB
subscale, and 2-3 points for the EWB subscale. Conclusions: We combined
patient-reported estimates, physician-reported estimates, and distribution-based
estimates to derive MIDs for HRQL outcomes from the FACT-BRM. These results will
enable interpretation of treatment group effects in a clinical trial setting, and they can be
used to estimate sample size or power when designing future studies
2
3. (11) Reed SD, Friedman JY, Velazquez EJ, Gnanasakthy A, Califf RM, Schulman KA.
Multinational economic evaluation of valsartan in patients with chronic heart failure:
Results from the Valsartan Heart Failure Trial (Val-HeFT). American Heart Journal
2004;148(1):122-8.
Abstract: Background The Valsartan Heart Failure Trial (Val-HeFT) compared valsartan
versus placebo in 5010 patients taking prescribed background therapy for New York
Heart Association class II to IV heart failure. Valsartan reduced the risk of heart failure
hospitalization and improved clinical signs and symptoms of heart failure. We sought to
compare resource use, costs, and health outcomes among patients taking prescribed
therapy for heart failure and randomly assigned to receive valsartan or placebo. Methods
Measures of resource use were based on data collected during the trial. Unit cost
estimates were collected from individual countries and converted to 1999 US dollars.
Total costs were estimated for hospitalizations, inpatient and outpatient physician
services, ambulance transportation, deaths outside the hospital, and outpatient
cardiovascular medications. Results Mean follow-up was 23 months. Mean costs for
heart failure hospitalizations were $423 lower among patients receiving valsartan (95%
CI, -706 to -146). Mean total costs were $9008 for patients receiving valsartan and $8464
for patients receiving placebo, a net incremental cost of $545 (95% CI, -149 to 1148),
including the cost of valsartan. There was an overall reduction in total costs of $929 (95%
CI, -3243 to 1533) among patients not receiving an ACE inhibitor at baseline but a slight
increase in costs of $334 (95% CI, -497 to 1199) among those receiving an ACE inhibitor
without a p-blocker and a $1246 increase (95% CI, 54 to 2230) in patients receiving both
an ACE inhibitor and a beta-blocker at baseline. Conclusions Valsartan provided clinical
benefits at a mean incremental cost of $285 per year during the trial. In patients not
taking ACE inhibitors, valsartan was economically attractive, increasing survival while
reducing or marginally increasing overall costs
(12) Smith DG, Gnanasakthy A, Ouednau K. Cost-effectiveness of blood pressure and urinary
albumin control in diabetics with an angiotensin II receptor antagonist and a calcium
channel blocker: Pharmacoeconomic analysis of the Marval trial-the case of Germany.
Value in Health 2003;6(6):681.
(13) Delea TE, Jacobson TA, Edelsberg JS, Gnanasakthy A, Oster G. Fluvastatin for the
prevention of cardiac events following successful first percutaneous coronary
intervention: analysis of cost-effectiveness based on the lescol intervention prevention
study (LIPS). European Heart Journal 2003;24:246.
(14) Friedman JY, Curtis L, Gnanasakthy A, Whellan D, Schulman KA. Investigator and site
characteristics of the navigator trial. Value in Health 2003;6(3):335-6.
(15) Reed SD, Friedman JY, Velazquez EJ, Gnanasakthy A, Califf RM, Schulman KA.
Cost-effectiveness of valsartan in patients not receiving angiotensin-converting enzyme
inhibitors at baseline. European Heart Journal 2002;23:137.
(16) Reed SD, Friedman JY, Gnanasakthy A, Schulman KA. Comparison of hospital costing
methods in an economic evaluation of a multinational clinical trial. International Journal of
Technology Assessment in Health Care 2003;19(2):396-406.
Abstract: Objectives: To develop and evaluate strategies for estimating hospitalization
costs in multinational clinical trials. Methods: Hospital cost estimates for eleven
diagnoses were collected from twelve countries participating in a trial of therapies for
congestive heart failure. Estimates were combined with U.S.-based diagnosis-related
group weights to compute country-specific unit cost estimates for all reasons for
hospitalization. Variations of hospital costing methods were developed. The unit cost
method assigns a country-specific unit cost estimate to each hospitalization. The other
methods adjust for length of stay using a daily cost (DC) estimate for each diagnosis,
based on either the mean length of stay (DC-mean method) or the median length of stay
3
4. (DC-median method) for each diagnosis in each country. Additional modifications were
explored through adjustment of the distribution of daily costs incurred during a hospital
stay. Results: The mean cost for all hospitalizations was $10,242 (SID, 10,042) using the
unit cost method, $10,242 (SD, 12,760) using the standard DC-mean method, and
$13,967 (SD, 18,762) using the standard DC-median method. In comparisons of costs for
all 5,486 hospitalizations incurred by a subset of 2,352 patients in the trial, the unit cost
method provided 92% power to detect a $1,000 cost difference. The standard DC-mean
method provided 76% power, and the standard DC-median method provided 44% power.
Conclusions: Hospital costing methods that adjust for differences in length of stay require
a significantly larger sample to attain comparable statistical power as methods that
assign unadjusted unit cost estimates to hospitalization events
(17) Fiedman JY, Curtis L, Gnanasakthy A, Schulman KA. Physician characteristics of the
navigator trial predict average length of stay (ALOS) in acute coronary syndrome (ACS).
Value in Health 2002;5(6):478.
(18) Reed SD, Friedman JY, Velazquez EJ, Gnanasakthy A, Califf RM, Schulman KA.
Valsartan is cost-effective in heart failure patients not receiving ACE inhibitors.
Circulation 2002;106(16):4.
(19) Friedman J, Weinfurt K, Curtis L, Gnanasakthy A, Schulman K. Development of a method
to increase power in economic analyses of multinational trials. Circulation
2002;106(16):167.
(20) Hahn EA, Gnanasakthy A, Glendenning GA, Sorensen MV, Odom L, Cella D. Using
patient- and clinician-rated anchors to evaluate change on the functional assessment of
cancer Therapy-Biologic Response Modifiers. Clinical Therapeutics 2002;24:13.
(21) Backhouse ME, Gnanasakthy A, Schulman KA, Akehurst R, Glick H. The development of
standard economic datasets for use in the economic evaluation of medicines. Drug
Information Journal 2000;34(4):1273-91.
Abstract: Regulatory imperatives and competitive forces have led pharmaceutical
companies to focus on extending the scope of their randomized controlled trials (RCTs)
to collect data to address economic as well as safety and efficacy questions. To guide
researchers, a significant literature exists on various methodological aspects of
conducting economic evaluations within an RCT framework. Little has been published
however, about data handling issues surrounding the management of the economic data
collection effort. Thus, the objective of this paper is to present an overview of a project
conducted in order to tackle various aspects of the economic data management process
with a view to improving the efficiency of study conduct. The project outputs include case
report forms (CRF), guidelines for CRF completion, and templates for producing
statistical analysis plans and study protocols. Directions for further research are
highlighted
(22) Hauber AB, Gnanasakthy A, Mauskopf JA. Savings in the cost of caring for patients with
Alzheimer's disease in Canada: An analysis of treatment with rivastigmine. Clinical
Therapeutics 2000;22(4):439-51.
Abstract: Objective: To estimate per-patient potential cost savings using rivastigmine in
the treatment of Alzheimer's disease (AD) in Canada. Background: In recent years, new
members of a class of pharmaceuticals known as cholinesterase inhibitors have been
introduced for the treatment of patients with AD. Two recent studies conducted in the
United Kingdom acid the United States estimated potential cost savings from the new
cholinesterase inhibitor rivastigmine. The present study combined the
disease-progression model used in those 2 studies with Canadian costs to estimate
per-patient potential savings resulting from the treatment of AD in Canada. Methods:
Efficacy data from 2 pivotal, phase III clinical trials of rivastigmine were used in a hazard
4
5. model of disease progression to estimate Long-term differences in cognitive functioning
between patients receiving rivastigmine and patients receiving no treatment. We used the
Mini-Mental State Examination (MMSE) score as our measure of disease progression.
We also used Canadian costs of AD care, estimated as a function of MMSE score, to
estimate cost savings experienced by treated patients compared with patients receiving
no treatment. All costs and cost savings are presented in 1997 Canadian dollars. We
used a societal perspective in this analysis. Results: Rivastigmine was estimated to delay
the transition to more severe stages of AD by up to 188 days for patients with mild AD
after 2 years of treatment. For patients with mild-to-moderate and moderate disease, this
delay was estimated to be 106 and 44 days, respectively. For patients with the mild stage
of AD, estimated average daily cost savings (excluding the cost of rivastigmine) ranged
from Can $0.45 per patient per day at 6 months to Call $6.44 per patient per day after 2
years of treatment. For all patients, these estimated average daily cost savings ranged
from a low of Can $0.71 per patient per day after 6 months of treatment to a high of Can
$4.93 per patient per day after 2 years. Conclusion: On average, treatment with
rivastigmine yields savings in the direct cost of caring for AD patients that exceed the
cost of the drug after 2 years of treatment
(23) Hauber AB, Gnanasakthy A, Snyder EH, Bala MV, Richter A, Mauskopf JA. Potential
savings in the cost of caring for Alzheimer's disease - Treatment with rivastigmine.
Pharmacoeconomics 2000;17(4):351-60.
Abstract: Objective: To estimate savings in the cost of caring for patients with Alzheimer's
disease (AD) during 6 months, 1 year and 2 years of treatment with rivastigmine. An
intermediate objective was to estimate the relationship between disease progression and
institutionalisation. Design and setting: We assessed the relationship between
Mini-Mental State Examination (MMSE) score and institutionalisation using a piecewise
Cox proportional hazard model. To estimate cost savings from treatments lasting 6
months, 1 year and 2 years, estimates of the probability of institutionalisation were
integrated with data from two 6-month phase III clinical trials of rivastigmine and a hazard
model of disease progression. Main outcome measures and results: Our data suggest
that savings in the overall cost of caring for patients with mild and moderate AD can be
as high as $US4839 per patient after 2 years of treatment. Furthermore, the probability of
institutionalisation increases steadily as MMSE score falls. Among our study individuals,
age, race, level of education and marital status were significant predictors of
institutionalisation, whereas gender had little effect. Conclusions: Using rivastigmine to
treat AD results in a delay in disease progression for patients who begin treatment during
the mild or moderate stages of the disease. By delaying the probability that a patient will
be institutionalised, the cost of caring for AD patients can be significantly reduced
(24) Kopelman P, Maclaughlin B, Norris L, Gnanasakthy A, Wilson K. Randomized,
Double-Blind, Placebo-Controlled, Parallel-Group Safety Study to Assess the Effect of
Single Doses of Vigabatrin on Blood-Glucose Levels in Insulin-Treated Diabetic Subjects.
Epilepsia 1995;36:S107-S108.
(25) Dallabernardina B, Fontane E, Vigevano F, Fusco L, Torelli D, Galeone D, Buti D,
Cianchetti C, Gnanasakthy A, Iudice A. Efficacy and Tolerability of Vigabatrin in Children
with Refractory Partial Seizures - A Single-Blind Dose-Increasing Study. Epilepsia
1995;36(7):687-91.
Abstract: The efficacy and tolerability of vigabatrin (VGB) in children with refractory partial
epilepsy were assessed in a single-blind, add-on, fixed-sequence, placebo-controlled
trial. After 1-month observation, the patients entered a 7-month treatment period that
involved administration of placebo for 1 month followed by VGB at the initial dosage of 40
mg/kg/day, to be increased to 60 and 80 mg/kg/day at 2-month intervals if seizures
persisted. Of the 46 children enrolled in the study, 7 dropped out prematurely due to lack
of efficacy of the drug (n = 6) or increased seizure frequency (n = 1). In 11 patients who
either became seizure-free (n = 3) or improved markedly (n = 8), treatment was
5
6. completed at a dose <80 mg/kg/day. The average number of seizures per month in the
39 patients who completed the study decreased from 97 during placebo to 21, 12, and 9
after 2, 4, and 6 months of VGB treatments respectively (p < 0.0001 at each time).
Response to VGB remained statistically significant when dropouts were included in the
evaluation. The number of patients who had >50% reduction in Seizure frequency after 2,
4, and 6 months was 28, 33, and 35, respectively. Eight patients became seizure-free
during the last 2 months of VGB treatment (3 at 40, 3 at 60, and 2 at 80 mg/kg/day, as
compared with none during placebo treatment). Serum levels of associated antiepileptic
drugs (AEDs) showed no significant changes, except for serum phenytoin (Pi-IT)
concentration, which significantly (p < 0.01) decreased after VGB treatment. Increased
appetite and sedation were observed in 17 and 11% of cases, respectively. VGB is
effective in the management of refractory partial epilepsy in children, and in some
patients a positive dose-response relationship appears to occur over the assessed
dosing range
(26) Dubrey SW, Gnanasakthy A, Stein WK, Song JG, Hardman T, Hynd J, Noble MIM.
Enoximone in Chronic Stable Angina - A Double-Blind Placebo-Controlled Cross-Over
Trial. Journal of Cardiovascular Pharmacology 1994;23(4):532-8.
Abstract: Enoximone, a phosphodiesterase inhibitor (PDEI), has both positive inotropic
and vasodilatory properties. We examined the effect of a single oral dose of enoximone
as compared with placebo on myocardial ischaemia and global left ventricular (LV)
function using both exercise ECG and Doppler measurements of aortic blood flow,
respectively. Twenty patients (16 men, 4 women) with a mean age of 59 years and stable
angina were studied. Total exercise duration was significantly longer after enoximone as
compared with placebo treatment, with a mean difference of 22.8 s (p = 0.003). Times
(mean +/- SD) to onset of angina and development of significant ST-segment decrease
were similar after placebo (454 +/- 101 and 352 +/- 155 s, respectively) or enoximone
(500 +/- 155 and 413 +/- 192 s, respectively), although both showed trends in favour of
enoximone. As compared with placebo, significantly higher heart rate (HR) was
measured for enoximone both at rest (75 +/- 18 vs. 90 +/- 22 beats/min, p < 0.01) and on
recovery from exercise (81 +/- 18 vs. 89 +/- 19 beats/min, p < 0.05). Enoximone had no
significant effect on systolic or diastolic blood pressure (SBP, DBP) or rate-pressure
product (RPP) generated at rest or during exercise. Changes in both acceleration and
velocity of aortic blood flow during exercise were similar after administration of
enoximone or placebo. We showed that a single oral dose of enoximone is well tolerated
in patients with ischaemic heart disease, improving both exercise capacity and favourably
influencing ST-segment changes with no increase in adverse events or significant
haemodynamic disturbances
(27) Loeb C, Iudice A, Perucca E, Gnanasakthy A, Angeleri F, Scarpino O, Avanzini G, Binelli
D, Baruzzi A, Procaccianti G, Bergamasco B, Bianco C, Canger R, Mai R, Diperri R,
Pisani F, Cocito L, Manfredi M, Faedda MT, Mutani R, Gianelli M, Tartara A, Tassinari
CA, Michelucci R, Zappoli R et al. Single-Blind, Placebo-Controlled Multicenter Trial of
Vigabatrin in the Treatment of Epilepsy. Italian Journal of Neurological Sciences
1992;13(9):741-7.
Abstract: A single-blind, placebo-controlled multicenter trial of vigabatrin was carried out
in 101 epileptic patients (mostly with partial seizures) refractory to conventional therapy.
The study design included four consecutive periods: (i) an observation phase (run-in), (ii)
a placebo period, (iii) fixed-dosage add-on vigabatrin (2 g/day) and (iv) dose titration (up
to a maximum of 4 g/day) to optimize clinical response. Each period lasted 8 weeks,
except for the titration phase, which could be extended to 16 weeks. 90 patients
completed the trial. Eleven dropped out, one patient developing absence status and 4
cases showing an increased seizure frequency. In the patients completing the trial, the
median number of seizures/month decreased from 16 (inter-quartile range 8-34) during
placebo to 5 (2-10) during the last 8 weeks on vigabatrin (p<0.0001). Both partial and
generalized tonic clonic (mostly secondary) seizures were significantly reduced. A greater
6
7. than 50% reduction in seizure frequency (compared to placebo) was observed in 60
patients. Sedation and weight gain were the most frequently reported adverse events
(28) Morant SV, Gnanasakthy A. A New Approach to the Mathematical Formulation of
Lactation Curves. Animal Production 1989;49:151-62.
(29) Webster AJF, Saville C, Church BM, Gnanasakthy A, Moss R. Some Effects of Different
Rearing Systems on Health, Cleanliness and Injury in Calves. British Veterinary Journal
1985;141(5):472-83.
(30) Webster AJF, Saville C, Church BM, Gnanasakthy A, Moss R. The Effect of Different
Rearing Systems on the Development of Calf Behavior. British Veterinary Journal
1985;141(3):249-64.
(31) Maughan GL, Cooke DA, Gnanasakthy A. The Effects of Soil-Applied Granular
Pesticides on the Establishment and Yield of Sugar-Beet in Commercial Fields. Crop
Protection 1984;3(4):439-50.
(32) Agha M, Gnanasakthy A. A Cluster-Analysis of the Effects of Storage Mites As Allergens
in Relation to Certain Occupations and Living-Conditions. Clinical Allergy
1981;11(5):499-504.
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