Traditionally, oncology drug developers have regarded the phase I clinical trial as having a utility limited to the assessment of safety, tolerability, and pharmacokinetics and pharmacodynamics of a drug.
In this issue of Peer Perspectives in Oncology, renowned oncology investigator Dr. Daniel Von Hoff describes how biotech Chief Medical Officers can design better oncology phase I trials to glean meaningful efficacy data by using the patient as his or her own control. He further explains how a CMO can gather evidence showing a drug changes the natural history of a patient’s disease, demonstrating improved care and a stronger case for moving a drug into phase II.
Clinical trials are facing rising costs and complexity due to difficulties recruiting and retaining patients. There is a growing recognition in the pharmaceutical industry that patient engagement must be improved by making trials more patient-centric and reducing the burden on participants. This will involve reforming traditional trial procedures and moving trials closer to patients in their homes and communities through virtualization, telemedicine, and community partnerships. Viewing patients as collaborators rather than just subjects and integrating their needs and perspectives into trial design is seen as key to improving development times and success rates.
The document reviews 25 studies that assess medication errors in pediatric patients during prescribing, dispensing, and drug administration processes. The studies found that the combined medication error rate was highest for prescribing errors (17.5%) and administration errors (20.9%), while the rate was lowest for dispensing errors (6.5%). The review concludes that medication errors are common in healthcare systems and that the medication process is prone to errors, especially during prescribing and administration.
This document presents the 2012 World Heart Federation criteria for echocardiographic diagnosis of rheumatic heart disease. An international panel of experts developed evidence-based guidelines to standardize the identification of rheumatic heart disease using echocardiography. The guidelines define three categories - 'definite RHD', 'borderline RHD', and 'normal'. Criteria for 'definite RHD' include pathological mitral or aortic regurgitation accompanied by specific morphological changes to the valves. 'Borderline RHD' includes minor abnormalities that do not meet criteria for 'definite RHD'. The standardized criteria aim to allow consistent identification of rheumatic heart disease globally to facilitate screening and secondary prophylaxis programs.
Detecção pré natal de cc resultado de programa preliminargisa_legal
This study evaluated the effectiveness of a national prenatal screening program for congenital heart disease (CHD) introduced in the Netherlands in 2007. The study found that the overall prenatal detection rate of severe CHD increased from 35.8% before the screening program to 59.7% after. Some specific types of CHD saw even larger increases, such as hypoplastic left heart syndrome which increased from 54.1% to 97.6% detection. Additionally, late referrals after 24 weeks gestation decreased by 24.3% following the screening program. This is the largest study to investigate prenatal detection rates of severe CHD in an unselected population, and found the screening program led to remarkably high detection compared to
Prenatal diagnosis of critical congenital heart disease reduces risk of death...gisa_legal
This meta-analysis examined outcomes for newborns with critical congenital heart disease who received prenatal vs postnatal diagnoses. It analyzed data from 8 studies including 1,373 total patients. When excluding patients who were high risk or received comfort care, newborns with prenatal diagnoses were significantly less likely to die before planned cardiac surgery than those with postnatal diagnoses (pooled odds ratio 0.26). Specifically, of the 1,316 cases deemed standard risk and planned for surgery, preoperative death occurred in 0.7% with prenatal diagnosis vs 3.0% with postnatal diagnosis. The study concludes that for newborns most likely to benefit from treatment, prenatal diagnosis can reduce the risk of death prior to
Circulation 2015-criterios de jones reviewgisa_legal
This document revises the Jones criteria for diagnosing acute rheumatic fever to better align with current evidence and international guidelines. It recognizes that acute rheumatic fever remains a serious health problem globally. The revisions define high-risk populations, acknowledge variability in clinical presentation among these groups, and include Doppler echocardiography as a tool for diagnosing cardiac involvement even without overt symptoms. This represents the first major revision to the Jones criteria by the American Heart Association in over 20 years and applies their classification system for recommendations and evidence levels.
This document proposes a risk-based monitoring approach for clinical trials based on statistical evidence about the effects of errors and error corrections. The analysis shows that errors have minimal effects on study results and conclusions, and this effect diminishes as study size increases. On average, less than 8% source document verification is adequate to ensure data quality, with perhaps higher rates needed for smaller studies and virtually 0% for large studies. It is recommended that monitoring focus on data clarification queries of highly discrepant data, rather than just focusing on key outcomes.
This document discusses patient-reported outcomes and quality of life measures that are important for evaluating the impact of disease and treatment from the patient's perspective. It provides background on common quality of life domains assessed and examples of generic, disease-specific, and symptom-specific patient-reported outcome measures. The document also describes Myeloma UK's experience developing a myeloma-specific patient-reported outcome scale called MyPOS to capture aspects beyond just symptoms that are important to myeloma patients' quality of life.
Clinical trials are facing rising costs and complexity due to difficulties recruiting and retaining patients. There is a growing recognition in the pharmaceutical industry that patient engagement must be improved by making trials more patient-centric and reducing the burden on participants. This will involve reforming traditional trial procedures and moving trials closer to patients in their homes and communities through virtualization, telemedicine, and community partnerships. Viewing patients as collaborators rather than just subjects and integrating their needs and perspectives into trial design is seen as key to improving development times and success rates.
The document reviews 25 studies that assess medication errors in pediatric patients during prescribing, dispensing, and drug administration processes. The studies found that the combined medication error rate was highest for prescribing errors (17.5%) and administration errors (20.9%), while the rate was lowest for dispensing errors (6.5%). The review concludes that medication errors are common in healthcare systems and that the medication process is prone to errors, especially during prescribing and administration.
This document presents the 2012 World Heart Federation criteria for echocardiographic diagnosis of rheumatic heart disease. An international panel of experts developed evidence-based guidelines to standardize the identification of rheumatic heart disease using echocardiography. The guidelines define three categories - 'definite RHD', 'borderline RHD', and 'normal'. Criteria for 'definite RHD' include pathological mitral or aortic regurgitation accompanied by specific morphological changes to the valves. 'Borderline RHD' includes minor abnormalities that do not meet criteria for 'definite RHD'. The standardized criteria aim to allow consistent identification of rheumatic heart disease globally to facilitate screening and secondary prophylaxis programs.
Detecção pré natal de cc resultado de programa preliminargisa_legal
This study evaluated the effectiveness of a national prenatal screening program for congenital heart disease (CHD) introduced in the Netherlands in 2007. The study found that the overall prenatal detection rate of severe CHD increased from 35.8% before the screening program to 59.7% after. Some specific types of CHD saw even larger increases, such as hypoplastic left heart syndrome which increased from 54.1% to 97.6% detection. Additionally, late referrals after 24 weeks gestation decreased by 24.3% following the screening program. This is the largest study to investigate prenatal detection rates of severe CHD in an unselected population, and found the screening program led to remarkably high detection compared to
Prenatal diagnosis of critical congenital heart disease reduces risk of death...gisa_legal
This meta-analysis examined outcomes for newborns with critical congenital heart disease who received prenatal vs postnatal diagnoses. It analyzed data from 8 studies including 1,373 total patients. When excluding patients who were high risk or received comfort care, newborns with prenatal diagnoses were significantly less likely to die before planned cardiac surgery than those with postnatal diagnoses (pooled odds ratio 0.26). Specifically, of the 1,316 cases deemed standard risk and planned for surgery, preoperative death occurred in 0.7% with prenatal diagnosis vs 3.0% with postnatal diagnosis. The study concludes that for newborns most likely to benefit from treatment, prenatal diagnosis can reduce the risk of death prior to
Circulation 2015-criterios de jones reviewgisa_legal
This document revises the Jones criteria for diagnosing acute rheumatic fever to better align with current evidence and international guidelines. It recognizes that acute rheumatic fever remains a serious health problem globally. The revisions define high-risk populations, acknowledge variability in clinical presentation among these groups, and include Doppler echocardiography as a tool for diagnosing cardiac involvement even without overt symptoms. This represents the first major revision to the Jones criteria by the American Heart Association in over 20 years and applies their classification system for recommendations and evidence levels.
This document proposes a risk-based monitoring approach for clinical trials based on statistical evidence about the effects of errors and error corrections. The analysis shows that errors have minimal effects on study results and conclusions, and this effect diminishes as study size increases. On average, less than 8% source document verification is adequate to ensure data quality, with perhaps higher rates needed for smaller studies and virtually 0% for large studies. It is recommended that monitoring focus on data clarification queries of highly discrepant data, rather than just focusing on key outcomes.
This document discusses patient-reported outcomes and quality of life measures that are important for evaluating the impact of disease and treatment from the patient's perspective. It provides background on common quality of life domains assessed and examples of generic, disease-specific, and symptom-specific patient-reported outcome measures. The document also describes Myeloma UK's experience developing a myeloma-specific patient-reported outcome scale called MyPOS to capture aspects beyond just symptoms that are important to myeloma patients' quality of life.
This document provides information about refractive surgery and the science behind it. It discusses corneal optics, measurement of wavefront aberrations, corneal biomechanics, corneal imaging technologies like topography and tomography, laser biophysics, and corneal effects of refractive surgery procedures. It is intended to increase physicians' ophthalmic knowledge through review and study of this material.
This document discusses challenges in interpreting health-related quality of life (HRQOL) findings across cancer clinical trials. Two hypothetical trials that tested the same treatment showed conflicting HRQOL results. This was because the trials may have assessed different HRQOL areas, endpoints, handling of missing data, and analysis populations. To address this, international collaboration is needed to set standards for HRQOL analyses in cancer trials. This would help ensure HRQOL questions, designs, analyses and interpretations are consistent across trials.
Guideline on patient safety and well being in clinical trialsTrialJoin
Patient safety is and should be the number one priority in clinical research. Human participants in a clinical trial are necessary in order to improve and develop new therapies for certain conditions and advance the understanding of how a condition can be treated. Such medical advancements wouldn’t be possible without human subjects as participants. However, even though we all know the importance of clinical trials in the advancement of medicine, most people still have some misconceptions regarding this topic.
The feeling of being treated as a ‘’guinea pig’’ and fear of potential risks and side effects are still common among patients who suffer from a certain condition. Such misinformation and misconceptions regarding clinical trials can unnecessarily prevent patients from finding a potential cure or relief, and can also decrease the number of enrolled patients in studies.
In order to clarify these misunderstandings, we’ve decided to tell you everything there is to know about patient safety in clinical trials. We hope that with this information we can help you to better understand patient safety in clinical trials so that you gain more insight and start considering clinical trials as valid options that can help you improve any condition.
This document discusses biobanks and registries, their value for research, and opportunities for patient involvement. It describes how biobanks store biological samples and associated data to support research. Registries collect standardized clinical data on patient populations over time. Both require governance and quality management. The document presents two cases where patient organizations were involved in biobank and registry co-creation and governance to help advance research.
Evidence-based medicine (EBM) or evidence-based practice (EBP) aims to apply the best available evidence gained from the scientific method to clinical decision making. It seeks to assess the strength of evidence of the risks and benefits of treatments (including lack of treatment) and diagnostic tests. Evidence quality can range from meta-analyses and systematic reviews of double-blind, placebo-controlled clinical trials at the top end, down to conventional wisdom at the bottom.
Development of Biosimilar Products: Determinants of SuccessAjaz Hussain
After leaving FDA my focus has been on practicing QbD to deliver, in highly uncertain business environments, complex products and the necessary scientific evidence. This presentation comments on firm's ability to successfully develop and introduce into markets.
This document discusses challenges in patient recruitment for clinical trials in central nervous system (CNS) disorders. Key challenges include the cognitive impairments many CNS patients experience, lengthy diagnostic assessments required, and low patient awareness of clinical trials. It also outlines opportunities for expanding CNS trials in Southeast Asia, where populations have high rates of CNS disorders and costs are lower. MC SMO aims to help sponsors overcome recruitment barriers and take advantage of the region's potential through their CNS investigator site network and services addressing feasibility, planning, and site management.
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.
Development of the A-DIVA Scale (Medicine) (1) - KopieLisette Puijn
This study developed and validated the A-DIVA scale, a predictive scale to identify adult patients at high risk of difficult intravenous access based on 5 clinical factors. Researchers prospectively observed 1063 adult patients undergoing a procedure requiring IV access. They found that 17% had failed first attempt cannulation. Through logistic regression, they identified 5 independent predictors of difficult access - poor vein palpability/visibility, history of difficult access, unplanned surgery, and small vein size. These factors were used to create the additive A-DIVA scale, which classified patients into low, medium, and high risk groups with failure rates of 5%, 37%, and 93% respectively. The A-DIVA scale provides a reliable method to prospectively
Martha Schmidt has over 20 years of experience in clinical research, drug development, and the medical field. She has worked for several large pharmaceutical companies, managing clinical trials and ensuring regulatory compliance. She is knowledgeable about all phases of drug development and has experience in clinical oncology. Schmidt has extensive expertise in clinical data review and analysis. She is passionate about assisting with product liability and medical malpractice litigation through case review and serving as an expert witness.
Overview of the Patient-Centered Outcomes Research Institute (PCORI), how PCORI views Patient-Centered Outcomes Research and how this is related to PCORI’s major funding mechanisms.
The editorial discusses the publication of results from the REVIVE clinical trials that tested the drug levosimendan for acute decompensated heart failure. The trials were completed over 7 years ago but results were only recently published. The editorial argues that this late publication fulfills critical obligations to study participants who consented to the trials, and to clinical colleagues who need complete trial results to inform decisions. While levosimendan provided some benefits like reduced need for rescue interventions, it also increased risks like hypotension and arrhythmias. The editorial maintains researchers and sponsors have a duty to publish full trial results in a timely manner.
This document summarizes Dr. Michelle Campbell's presentation on developing patient-reported outcomes (PROs) for clinical trials from patient input. The presentation covered FDA's Patient-Focused Drug Development initiative which aims to systematically gather patient perspectives to inform drug development. It discussed bridging from initial patient input to selection of appropriate clinical trial endpoints, including developing new PRO measures. Dr. Campbell emphasized that developing valid PROs requires input from patients and takes time, and outlined FDA pathways for providing advice on PRO development and review.
This document summarizes Canada's rare disease research platforms and their work in addressing unmet medical needs. It describes three key parts of their orchestrated rare disease pipeline: 1) rare disease gene discovery which has identified causes for over 400 rare diseases, 2) disease phenotyping and mechanistic studies across multiple model systems, and 3) therapeutic discovery and validation efforts including drug repurposing and target identification. The overall goal is improving diagnosis and treatment for thousands of rare disease patients through shared expertise and resources across Canada.
The document discusses how patients can find, select, and shape clinical trials in order to identify research that is most beneficial. It emphasizes that patients should: understand their treatment options and scientific landscape; carefully evaluate trial design and motivations; and recognize that various stakeholders have competing interests. The goal is not just increasing the number of trials, but improving trial quality so they answer patients' needs. Patients are encouraged to engage with research groups long-term to help guide trial design toward options that offer meaningful benefit.
Seventh Annual Next Generation Dx SummitJaime Hodges
The Next Generation Dx Summit (www.nextgenerationdx.com), entering its seventh year, brings together more than 800 diagnostics professionals from across the world, providing comprehensive programming and valuable networking opportunities. Spanning from clinical diagnostics to business strategy, this year’s expanded program encompasses predictive cancer biomarkers, companion diagnostics, infectious disease, point-of-care, pharmacy-based diagnostics, cell-free DNA, commercialization, cancer immunotherapy, and reimbursement. With widespread coverage of all the most relevant diagnostics topics, the Next Generation Dx Summit promises to be a must-attend event to hear the latest announcements and developments in this rapidly evolving field.
The document summarizes the educational and professional experience of Somayeh Ranbalooch. She obtained bachelor's and associate degrees in medical laboratory science in 2014 and 2006. During her studies, she worked as an intern at a hospital and blood transfusion organization, gaining experience in hematology, immunoassay, biochemistry, and microbiology. While working at the blood transfusion organization, she acquired extensive experience diagnosing coagulation disorders and working with coagulation testing devices. She also has experience with cancer treatment research using cord blood samples and training medical students. Overall, her background is in coagulation disorders and cord blood research, but she is open to new opportunities in laboratory methods.
EMR as a highly powerful European RWD sourceIMSHealthRWES
This document discusses leveraging electronic medical record (EMR) data from multiple countries in Europe to improve clinical trial design and patient recruitment for a cardiovascular drug trial. Specifically:
EMR data from France, UK, Italy, Germany and Spain was used to 1) characterize and quantify the number of potential patients meeting the inclusion criteria, and 2) identify and pre-select high-potential clinical trial sites. This approach helped clarify the definition of "statin-intolerant patients" and ensured the number of required trial sites would be sufficient. Using real-world data in this way enhanced the clinical trial strategy and avoided potential delays or additional costs associated with insufficient patient recruitment.
Emerging diagnostic technologies proving the clinical application through g...Lyssa Friedman
Next Generation Sequencing is an exciting new technology for diagnostics companies. But is it right for all products and for all companies? This presentation was delivered via Webinar for a IVD audience for Q1 Productions, March 25, 2014.
This document discusses the career of a medical technologist. It begins by providing background on medical technologists and their important role in healthcare. They work in clinical laboratories and help diagnose patients by analyzing samples. The career is growing due to retiring medical technologists, but faces shortages due to lack of new entrants. The document then discusses the author's reasons for choosing this career and outlines some of the responsibilities, stressors, ethics and safety issues involved in the work. It concludes by noting projections for continued growth in jobs and opportunities in the field.
SciTech Development LLC - Intelligent Technology to Solve Unmet Clinical Needs - Often the difference between success and failure is the dedication and persistence of the executive team. SciTech Development’s principal asset, fenretinide, is guided by a deeply experienced team in the broad portfolio of pharmaceutical development, clinical strategy, and scientific formulation.
This document discusses patient-centered drug development and clinical trials. It defines patient centricity as meaningfully including patients, particularly in trial protocol design, and linking clinical trial goals with patients' needs and experiences. It emphasizes measuring what matters to patients. While patient centricity is seen as important, there is no consensus on how to measure it. The document explores definitions of patient centricity, challenges in measuring it, examples of companies working to include the patient voice, and the need for new approaches to drug development that place greater focus on the patient experience.
This document provides information about refractive surgery and the science behind it. It discusses corneal optics, measurement of wavefront aberrations, corneal biomechanics, corneal imaging technologies like topography and tomography, laser biophysics, and corneal effects of refractive surgery procedures. It is intended to increase physicians' ophthalmic knowledge through review and study of this material.
This document discusses challenges in interpreting health-related quality of life (HRQOL) findings across cancer clinical trials. Two hypothetical trials that tested the same treatment showed conflicting HRQOL results. This was because the trials may have assessed different HRQOL areas, endpoints, handling of missing data, and analysis populations. To address this, international collaboration is needed to set standards for HRQOL analyses in cancer trials. This would help ensure HRQOL questions, designs, analyses and interpretations are consistent across trials.
Guideline on patient safety and well being in clinical trialsTrialJoin
Patient safety is and should be the number one priority in clinical research. Human participants in a clinical trial are necessary in order to improve and develop new therapies for certain conditions and advance the understanding of how a condition can be treated. Such medical advancements wouldn’t be possible without human subjects as participants. However, even though we all know the importance of clinical trials in the advancement of medicine, most people still have some misconceptions regarding this topic.
The feeling of being treated as a ‘’guinea pig’’ and fear of potential risks and side effects are still common among patients who suffer from a certain condition. Such misinformation and misconceptions regarding clinical trials can unnecessarily prevent patients from finding a potential cure or relief, and can also decrease the number of enrolled patients in studies.
In order to clarify these misunderstandings, we’ve decided to tell you everything there is to know about patient safety in clinical trials. We hope that with this information we can help you to better understand patient safety in clinical trials so that you gain more insight and start considering clinical trials as valid options that can help you improve any condition.
This document discusses biobanks and registries, their value for research, and opportunities for patient involvement. It describes how biobanks store biological samples and associated data to support research. Registries collect standardized clinical data on patient populations over time. Both require governance and quality management. The document presents two cases where patient organizations were involved in biobank and registry co-creation and governance to help advance research.
Evidence-based medicine (EBM) or evidence-based practice (EBP) aims to apply the best available evidence gained from the scientific method to clinical decision making. It seeks to assess the strength of evidence of the risks and benefits of treatments (including lack of treatment) and diagnostic tests. Evidence quality can range from meta-analyses and systematic reviews of double-blind, placebo-controlled clinical trials at the top end, down to conventional wisdom at the bottom.
Development of Biosimilar Products: Determinants of SuccessAjaz Hussain
After leaving FDA my focus has been on practicing QbD to deliver, in highly uncertain business environments, complex products and the necessary scientific evidence. This presentation comments on firm's ability to successfully develop and introduce into markets.
This document discusses challenges in patient recruitment for clinical trials in central nervous system (CNS) disorders. Key challenges include the cognitive impairments many CNS patients experience, lengthy diagnostic assessments required, and low patient awareness of clinical trials. It also outlines opportunities for expanding CNS trials in Southeast Asia, where populations have high rates of CNS disorders and costs are lower. MC SMO aims to help sponsors overcome recruitment barriers and take advantage of the region's potential through their CNS investigator site network and services addressing feasibility, planning, and site management.
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.
Development of the A-DIVA Scale (Medicine) (1) - KopieLisette Puijn
This study developed and validated the A-DIVA scale, a predictive scale to identify adult patients at high risk of difficult intravenous access based on 5 clinical factors. Researchers prospectively observed 1063 adult patients undergoing a procedure requiring IV access. They found that 17% had failed first attempt cannulation. Through logistic regression, they identified 5 independent predictors of difficult access - poor vein palpability/visibility, history of difficult access, unplanned surgery, and small vein size. These factors were used to create the additive A-DIVA scale, which classified patients into low, medium, and high risk groups with failure rates of 5%, 37%, and 93% respectively. The A-DIVA scale provides a reliable method to prospectively
Martha Schmidt has over 20 years of experience in clinical research, drug development, and the medical field. She has worked for several large pharmaceutical companies, managing clinical trials and ensuring regulatory compliance. She is knowledgeable about all phases of drug development and has experience in clinical oncology. Schmidt has extensive expertise in clinical data review and analysis. She is passionate about assisting with product liability and medical malpractice litigation through case review and serving as an expert witness.
Overview of the Patient-Centered Outcomes Research Institute (PCORI), how PCORI views Patient-Centered Outcomes Research and how this is related to PCORI’s major funding mechanisms.
The editorial discusses the publication of results from the REVIVE clinical trials that tested the drug levosimendan for acute decompensated heart failure. The trials were completed over 7 years ago but results were only recently published. The editorial argues that this late publication fulfills critical obligations to study participants who consented to the trials, and to clinical colleagues who need complete trial results to inform decisions. While levosimendan provided some benefits like reduced need for rescue interventions, it also increased risks like hypotension and arrhythmias. The editorial maintains researchers and sponsors have a duty to publish full trial results in a timely manner.
This document summarizes Dr. Michelle Campbell's presentation on developing patient-reported outcomes (PROs) for clinical trials from patient input. The presentation covered FDA's Patient-Focused Drug Development initiative which aims to systematically gather patient perspectives to inform drug development. It discussed bridging from initial patient input to selection of appropriate clinical trial endpoints, including developing new PRO measures. Dr. Campbell emphasized that developing valid PROs requires input from patients and takes time, and outlined FDA pathways for providing advice on PRO development and review.
This document summarizes Canada's rare disease research platforms and their work in addressing unmet medical needs. It describes three key parts of their orchestrated rare disease pipeline: 1) rare disease gene discovery which has identified causes for over 400 rare diseases, 2) disease phenotyping and mechanistic studies across multiple model systems, and 3) therapeutic discovery and validation efforts including drug repurposing and target identification. The overall goal is improving diagnosis and treatment for thousands of rare disease patients through shared expertise and resources across Canada.
The document discusses how patients can find, select, and shape clinical trials in order to identify research that is most beneficial. It emphasizes that patients should: understand their treatment options and scientific landscape; carefully evaluate trial design and motivations; and recognize that various stakeholders have competing interests. The goal is not just increasing the number of trials, but improving trial quality so they answer patients' needs. Patients are encouraged to engage with research groups long-term to help guide trial design toward options that offer meaningful benefit.
Seventh Annual Next Generation Dx SummitJaime Hodges
The Next Generation Dx Summit (www.nextgenerationdx.com), entering its seventh year, brings together more than 800 diagnostics professionals from across the world, providing comprehensive programming and valuable networking opportunities. Spanning from clinical diagnostics to business strategy, this year’s expanded program encompasses predictive cancer biomarkers, companion diagnostics, infectious disease, point-of-care, pharmacy-based diagnostics, cell-free DNA, commercialization, cancer immunotherapy, and reimbursement. With widespread coverage of all the most relevant diagnostics topics, the Next Generation Dx Summit promises to be a must-attend event to hear the latest announcements and developments in this rapidly evolving field.
The document summarizes the educational and professional experience of Somayeh Ranbalooch. She obtained bachelor's and associate degrees in medical laboratory science in 2014 and 2006. During her studies, she worked as an intern at a hospital and blood transfusion organization, gaining experience in hematology, immunoassay, biochemistry, and microbiology. While working at the blood transfusion organization, she acquired extensive experience diagnosing coagulation disorders and working with coagulation testing devices. She also has experience with cancer treatment research using cord blood samples and training medical students. Overall, her background is in coagulation disorders and cord blood research, but she is open to new opportunities in laboratory methods.
EMR as a highly powerful European RWD sourceIMSHealthRWES
This document discusses leveraging electronic medical record (EMR) data from multiple countries in Europe to improve clinical trial design and patient recruitment for a cardiovascular drug trial. Specifically:
EMR data from France, UK, Italy, Germany and Spain was used to 1) characterize and quantify the number of potential patients meeting the inclusion criteria, and 2) identify and pre-select high-potential clinical trial sites. This approach helped clarify the definition of "statin-intolerant patients" and ensured the number of required trial sites would be sufficient. Using real-world data in this way enhanced the clinical trial strategy and avoided potential delays or additional costs associated with insufficient patient recruitment.
Emerging diagnostic technologies proving the clinical application through g...Lyssa Friedman
Next Generation Sequencing is an exciting new technology for diagnostics companies. But is it right for all products and for all companies? This presentation was delivered via Webinar for a IVD audience for Q1 Productions, March 25, 2014.
This document discusses the career of a medical technologist. It begins by providing background on medical technologists and their important role in healthcare. They work in clinical laboratories and help diagnose patients by analyzing samples. The career is growing due to retiring medical technologists, but faces shortages due to lack of new entrants. The document then discusses the author's reasons for choosing this career and outlines some of the responsibilities, stressors, ethics and safety issues involved in the work. It concludes by noting projections for continued growth in jobs and opportunities in the field.
SciTech Development LLC - Intelligent Technology to Solve Unmet Clinical Needs - Often the difference between success and failure is the dedication and persistence of the executive team. SciTech Development’s principal asset, fenretinide, is guided by a deeply experienced team in the broad portfolio of pharmaceutical development, clinical strategy, and scientific formulation.
This document discusses patient-centered drug development and clinical trials. It defines patient centricity as meaningfully including patients, particularly in trial protocol design, and linking clinical trial goals with patients' needs and experiences. It emphasizes measuring what matters to patients. While patient centricity is seen as important, there is no consensus on how to measure it. The document explores definitions of patient centricity, challenges in measuring it, examples of companies working to include the patient voice, and the need for new approaches to drug development that place greater focus on the patient experience.
This document discusses patient-centered drug development and clinical trials. It defines patient centricity as meaningfully including patients, particularly in designing trial protocols, and linking clinical trial goals with patients' needs and experiences. It emphasizes measuring what matters to patients. While patient centricity is seen as important, there is no consensus on how to measure it. The document explores how companies are working to include the patient voice, such as through patient advisory boards and surveys to capture patient perceptions and experiences in clinical trials. It argues that simply talking about patient centricity is not enough, and that meaningful action is needed to achieve it.
Immuno-Oncology Course, organized by Healthcare Education ServicesJames Prudhomme
The Immuno-Oncology one-day course provides an overview of the rapidly evolving subject of immune-oncology. Delegates are offered a thorough understanding of the basics of tumor immunology as well as the essentials of immunotherapy and its application in cancer medicine. Examples of both how biologics work in the practice of oncology and of the challenges presented are demonstrated.
The program has been developed specifically to support the needs of the pharmaceutical, biotechnology and medical technology industry personnel. It is ideal for individuals with little understanding of the immunotherapy of cancer and those with an existing basic knowledge. Detailed presentations and discussion with Healthcare’s experienced and knowledgeable faculty enable thorough insight to this important subject area.
The document provides an update on the progress and accomplishments of the Nemours Center for Childhood Cancer Research (NCCCR). Key points include:
- NCCCR has established collaborations with other institutions to integrate childhood cancer research efforts and leverage resources to become a national leader.
- Major accomplishments include establishing monthly research translation meetings between clinicians and researchers, and creating one of the few pediatric tumor banks in the US to facilitate biomarker discovery.
- The vision is to develop strategies to advance childhood cancer screening, diagnosis, treatment and prevention, with a focus on identifying biomarkers, developing drug delivery approaches, and discovering new drugs.
James I. Merlino is acolorectal surgeon and thechief exper.docxvrickens
James I. Merlino is a
colorectal surgeon and the
chief experience officer at
the Cleveland Clinic.
Ananth Raman is the UPS
Foundation Professor of
Business Logistics at Harvard
Business School.
HEALTH CARE'S
SERVICE FANATICS
How the Cleveland Clinic leaped to
the top of patient-satisfaction surveys
by James I. Merlino and Ananth Raman
THE CLEVELAND CLINIC has long had a reputation for medical excel-
lence and for holding dov în costs. But in 2009 Delos "Toby" Cos-
grove, the CEO, examined its performance relative to that of other
hospitals and admitted to himself that inpatients did not think
much of their experience at its flagship medical center or its eight
community hospitals—and decided something had to be done.
Over the next three years the Clinic transformed itself. Its overall
ranking in the Centers for Medicare & Medicaid Services (CMS) sur-
vey of patient satisfaction jumped from about average to among the
top 8% of the roughly 4,600 hospitals included. Hospital executives
from all over the world now flock to Cleveland to study the Clinic's
practices and to leam how it changed.
The Clinic's journey also holds lessons for organizations outside
health care—ones that until now have not had to compete by cre-
ating a superior experience for customers. Such enterprises often
have workforces that were not hired with customer satisfaction in
mind. Can they improve the customer experience without jeopar-
dizing their traditional strengths? The Clinic's success suggests that
they can.
The Cleveland Clinic's transformation involved actions any
organization can take. Cosgrove made improving the patient ex-
perience a strategic priority, ultimately appointing James Merlino,
a prominent colorectal surgeon (and a coauthor of this piece), to
io8 Harvard Business Review May 2013
n
HEALTH CARE'S SERVICE FANATICS
lead the efiFort. By spelling out the problems in a sys-
tematic, sustained fashion. Merlino got everyone in
the enterprise—including physicians who thought
that only medical outcomes mattered—to recognize
that patient dissatisfaction was a significant issue
and that all employees, even administrators and
janitors, were "caregivers" who should play a role in
fixing it. By conducting surveys and studies and so-
liciting patients' input, the Clinic developed a deep
understanding of patients' needs. It gave MerUno a
dedicated staff and an ample budget with which to
change mind-sets, develop and implement processes,
create metrics, aind monitor performance so that the
organization could continually improve. And it com-
municated intensively with prospective patients to
set realistic expectations for what their time in the
hospital would be like.
These steps were not rocket science, but they
changed the organization very quickly. What's more,
fears expressed by some physicians that the initia-
tive might conflict with efforts to maintain high qual-
ity and safety standards and to further reduce costs
turned out to be unfounded. Du ...
The document summarizes an upcoming conference on patient-centered clinical trials taking place October 19-20, 2015 in Philadelphia. It will feature over 30 industry experts and leaders from pharmaceutical companies such as Johnson & Johnson, Pfizer, AstraZeneca, and Google. The goal of the conference is to discuss how to better involve patients in the design and conduct of clinical trials to improve recruitment, retention, and trial success by putting the patient voice at the core. It will provide a forum for stakeholders from industry, patient advocacy groups, and regulators to collaborate on developing a framework for patient engagement in clinical trials.
Global Medical Cures™ | Paving way for Personalized Medicine (2013) Global Medical Cures™
Global Medical Cures™ | Paving way for Personalized Medicine (2013)
DISCLAIMER-
Global Medical Cures™ does not offer any medical advice, diagnosis, treatment or recommendations. Only your healthcare provider/physician can offer you information and recommendations for you to decide about your healthcare choices.
The 10 best medical & clinical laboratories to watch for 2019insightscare
SCL Healthcare Central Laboratory (SHC C-LAB) is the largest clinical laboratory and reference lab in Korea, founded in 2006 with a vision of promoting healthcare. The lab focuses on clinical trials, research, and new drug development. SHC C-LAB prides itself on maintaining high quality standards through validated testing methods and specialized data management. The lab utilizes advanced equipment and takes safety seriously. CEO Kyoung Ryul Lee emphasizes meeting client needs through collaboration and communication. SHC C-LAB aims to become a global leader and continue innovating healthcare.
Patient safety has always been the industry’s focus during clinical trials. However, a recent spate of well-publicized patient safety issues have increased public scrutiny and the biotechnology, pharmaceutical and CRO industries' desire to improve study quality, resulting in larger, longer, more expensive trials. In this Q&A, James T. Gourzis, M.D., Ph.D., discusses issues affecting patient safety, including factors that have launched safety to the forefront; what to look for in evaluating CRO excellence; unique oncology considerations and the ramifications of the rare toxicity; optimizing the Data Monitoring Committee; budget decisions that affect patient safety and the evolution/future of FDA requirements.
This corporate presentation outlines Aura Biosciences' novel targeted therapy approach using viral-like particles to treat cancer. Their lead product, AU-011, is being developed for the treatment of ocular melanoma, an orphan disease with no approved therapies. Preclinical data shows AU-011 effectively targets and kills tumor cells through a unique mechanism of action. Aura plans to initiate clinical trials in Q1-2/2016 to obtain proof of concept data and accelerate approval. If approved, AU-011 could be the first FDA-approved treatment for the primary tumor in ocular melanoma patients.
This document contains a summary of Timothy Chinyereugo Ekwebelem's qualifications and experience. He has over 6 years of experience as a clinical research associate conducting clinical trials and research. He holds a Master's degree in Clinical Drug Development and has published articles in journals. He is seeking a challenging position in drug development, clinical research, or regulatory affairs.
MedicalResearch.com: Medical Research Exclusive Interviews February 10 2015Marie Benz MD FAAD
This document summarizes several interviews from the MedicalResearch.com website. In the first interview, Dr. Tang discusses a study finding that high levels of TMAO, a gut bacteria byproduct, is linked to both heart and kidney disease. The second interview discusses a study finding that a simple combination of sofosbuvir and ribavirin can effectively treat hepatitis C in HIV patients with few drug interactions. The third interview discusses a study showing that a brief information sheet improved contraceptive knowledge in women taking isotretinoin.
Dr Dev Kambhampati | FDA- Paving the Way for Personalized MedicineDr Dev Kambhampati
This document discusses personalized medicine from a regulatory perspective. It begins by defining personalized medicine as tailoring medical treatment to an individual's characteristics, needs, and preferences. The concept has existed for hundreds of years but recent advances in genomics, medical imaging, and other areas now enable a more personalized approach. The FDA plays a unique role in advancing personalized medicine by helping to bring these new medical products to market and evolving its regulatory processes in response to scientific developments. The document outlines FDA's efforts to define regulatory pathways, collaborate on research, and apply new knowledge to product reviews to help usher in this new era of individualized healthcare.
9 of 13 I VALUE-DRIVENThe Ali & Science of Evidence-Based .docxsleeperharwell
9 of 13 I VALUE-DRIVEN
The Ali & Science of Evidence-Based Care
RESEARCH BY MATTHEW WEINSTOCK
T
he shift to a value-driven delivery model hinges on a
key element: patients' achieving the best possible out-
comes. The linchpin to that is ensuring that clinicians
regularly follovi best practices and adhere to evidence-
based protocols.
"If this [transformation] is about value and value equals qual-
ity divided by cost, then it makes sense that you look at the evi-
dence," says Joseph Pepe, M.D., CEO of Catholic Medical Center,
Manchester, N.H.
Pepe, who served as CMC's chief medical officer for 12 years
before moving into the chief executive role in 2012, acknowledges
that one of the biggest stumbling blocks to instituting evidence-
based practice more broadly is the fear that it is "cookbook medi-
cine." That's a passé notion, he says. Evidence-based care is not
only about following results from the most recent clinical studies,
but blending that with a patient's values and desires, as well as
relying on a physician's judgment.
"Physicians have gotten a bad rap," says Jean Slutsky, direc-
tor of the Center for Outcomes and Evidence at the Agency for
Healthcare Research and Quality, when talking about the percep-
tion that doctors routinely reject the move toward evidence-based
care. "Physicians are lifelong learners. The very nature of what
they do is about learning."
A 2008 AHRQ handbook on implementing evidence-based
care supports the notion that this is not a completely rigid process.
It defines evidence-based care as "the use of current best evidence
I ABOUTTHISSERIES I
H&HN created this exclusive Fiscal Fitness series with the support of the VHA last year to highlight strategies
hospitals are using to improve quality of care while increasing efficiencies and reining in expenses. In 2013,
the series will focus on organizations that are demonstrating high-value health care
with measurable results. Follow the Fiscal Fitness series in our magazine, in our
e-newsletter H&HN Daily and on our website at www.hhnmag.com/fiscalfitness.
in conjunction with clinical expertise and patient values to guide health
care decisions." That definition first was popularized by David Sackett,
a Canadian doctor, in a 1996 British Medical Journal editorial. "Good
doctors use both individual clinical expertise and the best available
external evidence, and neither alone is enough," he argued.
For example, Slutsky says, the evidence may suggest that a patient
be put on a certain medication. Best practice may be to prescribe one
pill a day for 10 days. In a shared-decision model, which is also a critical
part of the process, the physician and patient would discuss the best
option available — perhaps it is using a different drug on the formulary
that's more affordable but requires the patient to take the medication
three times a day.
Another factor to consider: "What level of risk for side effects is
the patient wilHng t.
Patient engagement in clinical trials Martin Kelly
The document discusses improving patient engagement in clinical trials through digital methods. Only 2-5% of cancer patients currently participate in clinical trials. The top 3 solutions identified to engage patients were: 1) a clinical trial finder for patients to inform them of available trials, 2) virtual clinical trials conducted entirely online, and 3) tools to personalize clinical trials to individual patient needs/preferences. The document reviews several companies providing these types of solutions and proposes an experiment partnering with a CRO to test if a digital intervention increases patient enrollment and retention in a clinical trial compared to a control group without a digital intervention.
Aida Ibrahim Farghly is an experienced pharmacist seeking a job in regulatory affairs or healthcare. She has a bachelor's degree in pharmacy from Cairo University and is working towards a PhD. Her experience includes managing pharmacies, working as a clinical pharmacist, teaching health courses, and currently leading regulatory affairs for a pharmaceutical company. She is skilled in communication, research, analytics, and innovation with a focus on patient care.
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.
Ozempic: Preoperative Management of Patients on GLP-1 Receptor Agonists Saeid Safari
Preoperative Management of Patients on GLP-1 Receptor Agonists like Ozempic and Semiglutide
ASA GUIDELINE
NYSORA Guideline
2 Case Reports of Gastric Ultrasound
Title: Sense of Smell
Presenter: Dr. Faiza, Assistant Professor of Physiology
Qualifications:
MBBS (Best Graduate, AIMC Lahore)
FCPS Physiology
ICMT, CHPE, DHPE (STMU)
MPH (GC University, Faisalabad)
MBA (Virtual University of Pakistan)
Learning Objectives:
Describe the primary categories of smells and the concept of odor blindness.
Explain the structure and location of the olfactory membrane and mucosa, including the types and roles of cells involved in olfaction.
Describe the pathway and mechanisms of olfactory signal transmission from the olfactory receptors to the brain.
Illustrate the biochemical cascade triggered by odorant binding to olfactory receptors, including the role of G-proteins and second messengers in generating an action potential.
Identify different types of olfactory disorders such as anosmia, hyposmia, hyperosmia, and dysosmia, including their potential causes.
Key Topics:
Olfactory Genes:
3% of the human genome accounts for olfactory genes.
400 genes for odorant receptors.
Olfactory Membrane:
Located in the superior part of the nasal cavity.
Medially: Folds downward along the superior septum.
Laterally: Folds over the superior turbinate and upper surface of the middle turbinate.
Total surface area: 5-10 square centimeters.
Olfactory Mucosa:
Olfactory Cells: Bipolar nerve cells derived from the CNS (100 million), with 4-25 olfactory cilia per cell.
Sustentacular Cells: Produce mucus and maintain ionic and molecular environment.
Basal Cells: Replace worn-out olfactory cells with an average lifespan of 1-2 months.
Bowman’s Gland: Secretes mucus.
Stimulation of Olfactory Cells:
Odorant dissolves in mucus and attaches to receptors on olfactory cilia.
Involves a cascade effect through G-proteins and second messengers, leading to depolarization and action potential generation in the olfactory nerve.
Quality of a Good Odorant:
Small (3-20 Carbon atoms), volatile, water-soluble, and lipid-soluble.
Facilitated by odorant-binding proteins in mucus.
Membrane Potential and Action Potential:
Resting membrane potential: -55mV.
Action potential frequency in the olfactory nerve increases with odorant strength.
Adaptation Towards the Sense of Smell:
Rapid adaptation within the first second, with further slow adaptation.
Psychological adaptation greater than receptor adaptation, involving feedback inhibition from the central nervous system.
Primary Sensations of Smell:
Camphoraceous, Musky, Floral, Pepperminty, Ethereal, Pungent, Putrid.
Odor Detection Threshold:
Examples: Hydrogen sulfide (0.0005 ppm), Methyl-mercaptan (0.002 ppm).
Some toxic substances are odorless at lethal concentrations.
Characteristics of Smell:
Odor blindness for single substances due to lack of appropriate receptor protein.
Behavioral and emotional influences of smell.
Transmission of Olfactory Signals:
From olfactory cells to glomeruli in the olfactory bulb, involving lateral inhibition.
Primitive, less old, and new olfactory systems with different path
These simplified slides by Dr. Sidra Arshad present an overview of the non-respiratory functions of the respiratory tract.
Learning objectives:
1. Enlist the non-respiratory functions of the respiratory tract
2. Briefly explain how these functions are carried out
3. Discuss the significance of dead space
4. Differentiate between minute ventilation and alveolar ventilation
5. Describe the cough and sneeze reflexes
Study Resources:
1. Chapter 39, Guyton and Hall Textbook of Medical Physiology, 14th edition
2. Chapter 34, Ganong’s Review of Medical Physiology, 26th edition
3. Chapter 17, Human Physiology by Lauralee Sherwood, 9th edition
4. Non-respiratory functions of the lungs https://academic.oup.com/bjaed/article/13/3/98/278874
micro teaching on communication m.sc nursing.pdfAnurag Sharma
Microteaching is a unique model of practice teaching. It is a viable instrument for the. desired change in the teaching behavior or the behavior potential which, in specified types of real. classroom situations, tends to facilitate the achievement of specified types of objectives.
share - Lions, tigers, AI and health misinformation, oh my!.pptxTina Purnat
• Pitfalls and pivots needed to use AI effectively in public health
• Evidence-based strategies to address health misinformation effectively
• Building trust with communities online and offline
• Equipping health professionals to address questions, concerns and health misinformation
• Assessing risk and mitigating harm from adverse health narratives in communities, health workforce and health system
- Video recording of this lecture in English language: https://youtu.be/kqbnxVAZs-0
- Video recording of this lecture in Arabic language: https://youtu.be/SINlygW1Mpc
- Link to download the book free: https://nephrotube.blogspot.com/p/nephrotube-nephrology-books.html
- Link to NephroTube website: www.NephroTube.com
- Link to NephroTube social media accounts: https://nephrotube.blogspot.com/p/join-nephrotube-on-social-media.html
Basavarajeeyam is an important text for ayurvedic physician belonging to andhra pradehs. It is a popular compendium in various parts of our country as well as in andhra pradesh. The content of the text was presented in sanskrit and telugu language (Bilingual). One of the most famous book in ayurvedic pharmaceutics and therapeutics. This book contains 25 chapters called as prakaranas. Many rasaoushadis were explained, pioneer of dhatu druti, nadi pareeksha, mutra pareeksha etc. Belongs to the period of 15-16 century. New diseases like upadamsha, phiranga rogas are explained.
Local Advanced Lung Cancer: Artificial Intelligence, Synergetics, Complex Sys...Oleg Kshivets
Overall life span (LS) was 1671.7±1721.6 days and cumulative 5YS reached 62.4%, 10 years – 50.4%, 20 years – 44.6%. 94 LCP lived more than 5 years without cancer (LS=2958.6±1723.6 days), 22 – more than 10 years (LS=5571±1841.8 days). 67 LCP died because of LC (LS=471.9±344 days). AT significantly improved 5YS (68% vs. 53.7%) (P=0.028 by log-rank test). Cox modeling displayed that 5YS of LCP significantly depended on: N0-N12, T3-4, blood cell circuit, cell ratio factors (ratio between cancer cells-CC and blood cells subpopulations), LC cell dynamics, recalcification time, heparin tolerance, prothrombin index, protein, AT, procedure type (P=0.000-0.031). Neural networks, genetic algorithm selection and bootstrap simulation revealed relationships between 5YS and N0-12 (rank=1), thrombocytes/CC (rank=2), segmented neutrophils/CC (3), eosinophils/CC (4), erythrocytes/CC (5), healthy cells/CC (6), lymphocytes/CC (7), stick neutrophils/CC (8), leucocytes/CC (9), monocytes/CC (10). Correct prediction of 5YS was 100% by neural networks computing (error=0.000; area under ROC curve=1.0).
Basavarajeeyam is a Sreshta Sangraha grantha (Compiled book ), written by Neelkanta kotturu Basavaraja Virachita. It contains 25 Prakaranas, First 24 Chapters related to Rogas& 25th to Rasadravyas.
ABDOMINAL TRAUMA in pediatrics part one.drhasanrajab
Abdominal trauma in pediatrics refers to injuries or damage to the abdominal organs in children. It can occur due to various causes such as falls, motor vehicle accidents, sports-related injuries, and physical abuse. Children are more vulnerable to abdominal trauma due to their unique anatomical and physiological characteristics. Signs and symptoms include abdominal pain, tenderness, distension, vomiting, and signs of shock. Diagnosis involves physical examination, imaging studies, and laboratory tests. Management depends on the severity and may involve conservative treatment or surgical intervention. Prevention is crucial in reducing the incidence of abdominal trauma in children.
Dehradun #ℂall #gIRLS Oyo Hotel 8107221448 #ℂall #gIRL in Dehradun
The Lost Art Of Phase I Oncology Trials
1. Medelis, Inc.
4105 N 20th St Ste 215
Phoenix, Arizona 85016
Phone: 602.840.1101
www.medelis.com
The Lost Opportunity in
Phase I Oncology Trials
A Q&A with renowned oncology investigator Daniel D. Von
Hoff, M.D., who advocates for a phase I approach that looks
beyond toxicity and gleans meaningful efficacy data, creating
a more compelling rationale for further investment and
improved patient care.
2. Welcome
This presentation is based on our December 2008
issue of Peer Perspectives in Oncology, a free Q&A
series focused on issues that face Chief Medical
Officers today: rising costs, optimum patient accrual,
targeted therapeutics, patient safety, FDA
regulations, efficacy, budgets, and timelines.
You can sign up to receive an email notice for future
issues at www.medelis.com/oncology_abstracts.html.
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.
3. The Lost Opportunity in Phase I Oncology Trials
Traditionally, drug developers have regarded the phase I trial as
having a utility limited to the assessment of safety, tolerability,
and pharmacokinetics and pharmacodynamics of a drug.
In this issue of Peer Perspectives in Oncology, renowned
oncology investigator Dr. Daniel Von Hoff describes how Chief
Medical Officers can design better oncology phase I trials to
glean meaningful efficacy data by using the patient as his or her
own control. He further explains how a CMO can gather
evidence showing a drug changes the natural history of a
patient’s disease, demonstrating improved care and a stronger
case for moving a drug into phase II.
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.
4. About Dr. Daniel Von Hoff
Daniel D. Von Hoff, M.D. is Senior Investigator and Head of Translational Research at the Translational
Genomics Research Institute's (TGen) Translational Drug Development Division and Head, Pancreatic
Cancer Research Program in Phoenix, Arizona. He also serves as Chief Scientific Officer for U.S. Oncology
and the Scottsdale Clinical Research Institute, and is a founding shareholder and advisory board member
of Medelis.
Dr. Von Hoff's major interest is in the development of new anticancer agents, both in the clinic and in the
laboratory. He and his colleagues were involved in the beginning of the development of many of the
agents we now use routinely, including mitoxantrone, ludarabine, paclitaxel, docetaxel, gemcitabine, CPT
11, gefitinib and others. At present, he and his colleagues are concentrating on the development of
molecularly targeted therapies.
Dr. Von Hoff's laboratory interests and contributions have been in the area of in vitro drug sensitivity
testing to individualize treatment for the patient. He and his laboratory are now concentrating on
discovery of new targets in pancreatic cancer. Dr. Von Hoff has published more than 529 papers, 129
book chapters, and more than 891 abstracts.
Dr. Von Hoff was appointed to President Bush's National Cancer Advisory Board from June 2004 March
2010. He is the past President of the American Association for Cancer Research, a Fellow of the American
College of Physicians, and a member and past board member of the American Society of Clinical
Oncology. He is a founder of ILEX Oncology, Inc. (recently acquired by Genzyme). He is founder and
Editor Emeritus of Investigational New Drugs The Journal of New Anticancer Agents as well as the
EditorinChief of Molecular Cancer Therapeutics. He is also proud to have been a mentor and teacher for
multiple medical students, medical oncology fellows, graduate students, and postdoctoral fellows.
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.
5. Dan, you believe that many CMOs today are missing a valuable opportunity to gain
more meaningful data from phase I oncology trials. That seeing each patient as his
or her own control may hold a key to later success and create a more compelling
story for management & investors. Can you describe in detail what you mean?
Yes, and it’s very simple. Typically, a CMO sees phase I as a
toxicity trial, not a therapeutic trial, because of course it isn’t
randomized. But the doctors at the bedside and the patients
themselves don’t see it as purely a toxicity trial. We’re looking for
improvement and survival. And even during phase I, we can glean
important efficacy clues by using the patient as his or her own
control.
Recently, at ACR, I reported on findings involving nine patients in a
phase I trial showing dramatic tumor shrinkage with no side effects
with an oral agent. Clearly the drug did something. It slowed down
the disease and the patients benefited. In fact, the drug changed
the natural history of that patient.
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.
6. How should this information be used?
It can be a key part of the story you tell to
sponsors and investors when raising money.
You show how long the patient was on a prior
therapy and now how long she is on your
new therapy. The longer she is on a therapy,
the more the drug is doing something —
otherwise, you wouldn’t keep the patient on
the drug.
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.
7. In the context of a trial, it seems counter
intuitive to look at the individual patient.
The reason it’s so important to look at each individual
patient is because each patient’s tumor has a
different natural history. Everyone’s cancer is
different in heterogeneity and tempo, or natural
history, and the patient’s immune system. We may
not know all those variables, but we do know that if
the cancer changes, as measured by increased time
on therapy, we must be doing something right.
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.
8. Do you systematically track this information
for each patient?
Yes. In fact, I now recommend including this
kind of information — time to progression on
each drug — in the protocol so it becomes
part of each patient’s data bank.
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.
9. How should a CMO incorporate this data into
the protocol?
You establish it as an endpoint and it is very
convincing. So in addition to all the other
endpoints that measure antitumor activity in
a classic phase I trial, you add a line that
specifies that one of the secondary endpoints
is the time patients are on treatment with a
new agent versus the time they were on their
“justprior drug.”
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.
10. You refer to measuring “time on therapy” as opposed
to “time to progression?” What’s the distinction?
With “time to progression” you need to be
taking regular measurements such as scans.
In comparison, “time on therapy” takes
everything into consideration.
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.
11. That turns it into a more subjective, general
indicator. Is that good?
Yes, because it takes into consideration clinical
judgment, or people’s observational powers, which as
it turns out in medicine, can be more effective than
scans. Sometimes we look at a patient and think,
“Boy, he doesn’t look too good.” He says he’s fine,
but maybe he’s lost a few pounds. He doesn’t have
that twinkle in his eye, or he seems to be giving up.
What those signs mean to me is the tumor is
generating cytokines.
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.
12. And therein lies the art of the matter: the
power of observation.
What makes a clinical investigator great at his
or her trade is the ability to observe keenly.
In addition to the usual scans, it takes clinical
judgment into consideration, and that should
never be underestimated. Heck, Grandma
knew when you didn’t look good. She
watched you grow up. Careful observation is
critical at every stage.
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.
13. Are CMOs starting to do this as a matter of course — using time
on drug to demonstrate improvement, then using that data to
make a case to potential sponsors?
I have written about this but have never seen a CMO
plot time on a new drug versus the time on a justprior
therapy to build a story for raising money. This idea of
using the patient as their own control is a lost concept
in drug development. Dr. Bob Temple at the FDA, an
icon in clinical trial design, calls it a lost art. He’s
referring to the ability to document changes in the
natural history of a patient’s tumor, and how this
information can give you a sense of whether the drug
will work.
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.
14. Does the approach of using a patient as their own control
still work in the case of cyotoxic drugs, which kill cells
without decreasing tumor size?
Even if an agent isn’t shrinking the tumor, it may be keeping it stable
for long periods. If you plot the time a patient is on a new drug versus
the time they were on the therapy they just progressed on, you
can gauge whether an agent changed the natural history of the
patient’s tumor. And, if at the higher doses, you have more people
with longer time to progression as compared to lower doses, then
you have a trend that your drug is actually doing something. If the
patient had been on the next drug for only one month, then you
know it didn’t do anything for them. But if you can beat what the
patient just had, you really have a triumph.
CMOs and others just aren’t giving this information enough value.
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.
15. At what point would you feel validated to invest more
deeply in a particular therapy based on information
deduced from using the patient as their own control?
If you treat 30 patients and 30% stay on a
new therapy for a longer time than the just
prior drug they had progressed on, then that
would justify a deeper investment. Patients’
tumors grow at an inexorable, ever
quickening rate. If you find an agent that can
taper that growth, then it is probably doing
something and should be pursued.
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.
16. Why isn’t this a more common practice?
It’s not how people report. Instead of time on
a therapy, they report stable disease over
time. Unless they know how fast the tumor
was growing in the first place, there is no
reference point. If a patient has a short time
to progression when you put them on study,
they have a fastgrowing tumor. If you slow
its growth, you can tell right away by
comparing time to progression.
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.
17. So the phase I shouldn’t just focus on
maximum tolerable dose?
Exactly. It’s an opportunity to look for therapeutic effect
as well. If it’s there, patients benefit and you can hit it out
of the park.
I presented at the recent AACR meeting showing a
response in 89% of patients with basal cell carcinoma to
an oral Hedgehog inhibitor from Genentech. This was a
standard phase I, single agent, so it was pretty
remarkable. The second presentation I made was in
pancreatic cancer and it involved a 70% response rate.
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.
18. So the payoff for CMOs changing how they
see the phase I could be substantial.
There’s no question in my mind. If a CMO
started comparing time on new drug versus
time on justprior therapy, the rewards are
there for the patients and for the progression
of the drug. All it takes is a more proactive
approach to the phase I.
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.
19. About “Peer Perspectives in Oncology”
In Peer Perspectives in Oncology, Medelis brings together some of the
industry’s most respected researchers to talk about the issues facing
Chief Medical Officers today. They’re issues we all face on a daily basis:
Rising costs. Optimum patient accrual. Targeted therapeutics. Patient
safety. FDA regulations. Efficacy. Budgets. Timelines. In this Q&A
series, we’ll discuss these challenges with leading experts who deliver
practical, frontline insights gleaned from years of experience bringing
new drugs to market.
To download additional issues in the series, please visit
www.medelis.com/oncology_abstracts.html.
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.
20. About Medelis
Medelis, Inc. is a singlesource provider for oncology CRO and drug
development services, providing a total solution for biotechnology and
pharmaceutical companies seeking rapid drug development and approval.
Medelis' medical founders, team physicians and clinical trial management
physicians are internationallyrecognized oncology thought and opinion leaders
who understand the future of personalized medicine and threshold of credibility
trials. Offerings include strategic plans for regulatory approval from phase I
through NDA and complete clinical trial design, management and execution.
Medelis is privatelyheld and located in Phoenix, Arizona with other U.S.
locations in Nashville, Boston and Reno. Medelis Europe oversees projects for
European & Asian sponsors and is headquartered in Port Vendres, France.
Medelis provides a full range of oncology contract research & drug development
services from preclinical through NDA. Download our abstracts or read our blog at
www.medelis.com.