This is the fourth talk that I gave in Asia back in May. It was presented at the Konect (Korea National Enterprise for Clinical Trials) 3rd symposia that was held in Seoul at Seoul National University.
Rare Disease Patient Registries:
Key to Drug Development and Access
Tuesday, May 3 @ 12:00 – 1:00 pm EDT
Slides:
USA National Institutes of Health RaDaR
NORD IAMRARE (Pam Gavin, VP)
Canada INFORM RARE (Beth Potter, CHEO; Pranesh Chakraborty, CHEO; Kim Angel, Can MPS Society; John Adams, CanPKU)
GVP stands for Good Pharmacovigilance Practices, which are a set of guidelines and regulatory requirements that provide a framework for the conduct of pharmacovigilance activities. The GVP modules outline specific areas of pharmacovigilance and provide detailed guidance on various aspects. Here are the main GVP modules
Rare Disease Series Slide Deck Part 1.pptxMMS Holdings
This document summarizes an MMS Expert Insights webinar on rare disease research in the United States. The webinar discusses challenges in rare disease research such as limited patient populations and understanding of diseases. It provides an overview of the regulatory history around orphan drugs and incentives in the US. It also discusses how the FDA flexibly applies regulatory standards for rare disease drug development, including allowing single efficacy studies, modified clinical trial designs, and use of existing data in some cases.
ICMR Guidelines Presented By:Raj Kishor [CRC], Tech Observer the Global CRO,I...Raj Aryan
The document provides an overview of the Indian Council of Medical Research (ICMR). It discusses that ICMR was established in 1911 as the Indian Research Fund Association to sponsor and coordinate medical research in India. ICMR is the apex body in India for the formulation, coordination and promotion of biomedical research. It oversees various research activities including communicable diseases, non-communicable diseases, reproductive and child health, and nutrition. ICMR operates numerous research institutes and centers across India focused on specific disease areas. The document outlines ICMR's achievements in developing drugs, vaccines and conducting various research studies to address India's health priorities.
Safety data reconciliation involves comparing safety data between a clinical database and safety database to ensure consistency. Key fields like adverse event term, action taken, causality, and outcome are reconciled. Discrepancies between the databases are identified and queries are issued to sites for resolution. The process aims to clean 100% of agreed upon safety data points and document any acceptable discrepancies.
Pharmacovigilance Process Work Flow - Katalyst HLSKatalyst HLS
Introduction to Drug Safety & Pharmacovigilance Process Work Flow for Pharmaceuticals, Bio-Pharmaceuticals, Medical Devices, Cosmeceuticals and Foods.
Contact:
"Katalyst Healthcares & Life Sciences"
South Plainfield, NJ, USA
info@KatalystHLS.com
Pharmacovigilance involves monitoring approved drugs to detect adverse effects, assess risks, and prevent harm. It aims to improve patient safety by identifying unknown risks from drugs and informing regulatory decisions. Various methods are used, including spontaneous reporting of adverse drug reactions, active surveillance, and observational studies. Stringent pharmacovigilance is important given historical examples of drugs that caused significant harm after approval, demonstrating the need for ongoing monitoring of drug safety.
Rare Disease Patient Registries:
Key to Drug Development and Access
Tuesday, May 3 @ 12:00 – 1:00 pm EDT
Slides:
USA National Institutes of Health RaDaR
NORD IAMRARE (Pam Gavin, VP)
Canada INFORM RARE (Beth Potter, CHEO; Pranesh Chakraborty, CHEO; Kim Angel, Can MPS Society; John Adams, CanPKU)
GVP stands for Good Pharmacovigilance Practices, which are a set of guidelines and regulatory requirements that provide a framework for the conduct of pharmacovigilance activities. The GVP modules outline specific areas of pharmacovigilance and provide detailed guidance on various aspects. Here are the main GVP modules
Rare Disease Series Slide Deck Part 1.pptxMMS Holdings
This document summarizes an MMS Expert Insights webinar on rare disease research in the United States. The webinar discusses challenges in rare disease research such as limited patient populations and understanding of diseases. It provides an overview of the regulatory history around orphan drugs and incentives in the US. It also discusses how the FDA flexibly applies regulatory standards for rare disease drug development, including allowing single efficacy studies, modified clinical trial designs, and use of existing data in some cases.
ICMR Guidelines Presented By:Raj Kishor [CRC], Tech Observer the Global CRO,I...Raj Aryan
The document provides an overview of the Indian Council of Medical Research (ICMR). It discusses that ICMR was established in 1911 as the Indian Research Fund Association to sponsor and coordinate medical research in India. ICMR is the apex body in India for the formulation, coordination and promotion of biomedical research. It oversees various research activities including communicable diseases, non-communicable diseases, reproductive and child health, and nutrition. ICMR operates numerous research institutes and centers across India focused on specific disease areas. The document outlines ICMR's achievements in developing drugs, vaccines and conducting various research studies to address India's health priorities.
Safety data reconciliation involves comparing safety data between a clinical database and safety database to ensure consistency. Key fields like adverse event term, action taken, causality, and outcome are reconciled. Discrepancies between the databases are identified and queries are issued to sites for resolution. The process aims to clean 100% of agreed upon safety data points and document any acceptable discrepancies.
Pharmacovigilance Process Work Flow - Katalyst HLSKatalyst HLS
Introduction to Drug Safety & Pharmacovigilance Process Work Flow for Pharmaceuticals, Bio-Pharmaceuticals, Medical Devices, Cosmeceuticals and Foods.
Contact:
"Katalyst Healthcares & Life Sciences"
South Plainfield, NJ, USA
info@KatalystHLS.com
Pharmacovigilance involves monitoring approved drugs to detect adverse effects, assess risks, and prevent harm. It aims to improve patient safety by identifying unknown risks from drugs and informing regulatory decisions. Various methods are used, including spontaneous reporting of adverse drug reactions, active surveillance, and observational studies. Stringent pharmacovigilance is important given historical examples of drugs that caused significant harm after approval, demonstrating the need for ongoing monitoring of drug safety.
Safety reports rmp risk management plan pharmacovigilanceAzierta
A Risk management plan is a document based on safety profile of medicines that collects all pharmacovigilance activities and it is used to plan and implement measures in order to minimize risks.
This summary explains how to develop a Risk Management Plan according to European regulatory requirements.
Implementation dates and objectives of RMP module V of Good Pharmacovigilance Practices.
The Office of Orphan Products Development (OOPD) at the FDA promotes the development of treatments for rare diseases and conditions. There are more than 6,800 known rare diseases affecting an estimated 25-30 million Americans. The Orphan Drug Act of 1983 provides financial incentives like tax credits, user fee waivers, and exclusive marketing rights for 7 years to encourage development of treatments for rare diseases. The OOPD oversees programs that grant orphan drug designation, provide funding for clinical trials and natural history studies, and award priority review vouchers for rare pediatric diseases.
Literature searches in Pharmacovigilancesamikshagupta
This presentation provides a detailed description of various literature searches performed in pharmacovigilance including search criteria, different search engines etc
An brief introduction to the clinical data management process is described in this slides. These slides provides you the information regarding the data evaluation in the clinical trials , edit checks and data review finally data locking,then the data is submitted to the concerned regulatory body.
Introduction to MedDRA Coding in Drug Safety & Pharmacovigilance Process for Pharmaceuticals, Bio-Pharmaceuticals, Medical Devices, Cosmeceuticals and Foods.
Contact:
"Katalyst Healthcares & Life Sciences"
South Plainfield, NJ, USA
info@KatalystHLS.com
Clinical research involves systematic studies in human subjects to improve quality of life. Clinical trials are a form of clinical research that experimentally tests medications, devices, or biologics to evaluate safety and effectiveness. There are different phases of clinical trials, from small early phase safety studies to larger late phase studies to establish efficacy. Clinical trials require many players including regulators, sponsors, investigators, ethics committees, and participants to design, monitor, and conduct the trials according to strict protocols to safely and effectively evaluate potential new treatments.
MedDRA is a standardized medical terminology used in the pharmaceutical industry and regulatory bodies for coding adverse event reports. It contains over 60,000 terms organized into a hierarchical structure with 5 levels across 26 system organ classes. MedDRA allows for improved data entry of reports and flexible retrieval of information due to its multiaxial structure and guidance documents provide recommendations for its effective use and analysis.
Clinical trials are scientific studies that test new medical treatments or interventions on human volunteers. They are conducted by pharmaceutical companies or contract research organizations to evaluate the effects, risks, benefits, and efficacy of new drugs, biologics, or devices prior to their market release. Major players in a clinical research team include investigators, clinical research associates, coordinators, data managers, statisticians, patients, institutional review boards, and regulatory bodies. Principal investigators are responsible for leading the clinical trial team at each study site and ensuring proper conduct of the trial according to protocol and regulations.
This document defines signal detection as information that suggests a new potentially causal association or aspect of a known association between an intervention and adverse or beneficial events. It discusses the importance of signal detection for identifying safety concerns, improving patient outcomes, and regulatory compliance. Several methods of signal detection are described, including data mining, quantitative statistics, adverse event monitoring, and machine learning algorithms. Challenges and future directions are also outlined.
Safety and Pharmacovigilance System: Oracle Argus Safety SuitePerficient
Oracle's Argus Safety is a comprehensive pharmacovigilance platform designed to help life sciences companies ensure global regulatory compliance, enable faster safety decisions through data insights, and integrate safety and risk management functions. With Argus Safety, companies can manage regulatory reporting requirements, obtain visibility into reporting metrics and compliance, and leverage automation and advanced functionality to maximize case processing efficiency and data access for timely review. Argus Safety also provides integrated risk management capabilities and can be seamlessly connected to signal detection systems to proactively manage product safety.
This journal club presentation summarizes an article that examines the association between women's autonomy and utilization of maternal health services in Nepal. The presentation provides background on high maternal mortality in developing countries like Nepal. It summarizes the article's methods, which used Nepal Demographic and Health Survey data to analyze the relationship between women's autonomy and antenatal care, institutional delivery, and postnatal care. The results found a positive association between women's autonomy and utilization of maternal health services. In conclusion, the presentation emphasizes the need to promote women's autonomy to help ensure effective maternal healthcare.
The slide provides a basic understanding about Clinical Research process and the various Phases of Drug Discovery and Development. It also explains about the various trial designs and techniques in research such as blinding and randomization. It may be useful for giving a basic class for Fourth Year B.Pharm Students.
Introduction to Adverse Drug Reactions in Pharmacovigilance and Drug Safety in Pharma, Biotech, Medical Device, Cosmeceutical and Foods.
Contact:
"Katalyst Healthcares & Life Sciences"
South Plainfield, NJ, USA
info@KatalystHLS.com
Explaining the importance of a database lock in clinical researchTrialJoin
One of the most crucial aspects of research is clinical data management or CDM. Proper CDM will generate results with excellent quality, integrity, and reliability. Quality data is essential in order to support the final conclusions of a certain study.
The person responsible for this area of research is called a clinical data manager. This job position can be filled by a PI, a study coordinator, or a CRA. No matter who fills this position at your site, data management has to be done promptly and correctly in order to generate the best results. Aside from all the other reasons why data management is so important, it’s also what determines the future IP (investigational product) development.
Clinical trials involve testing investigational drugs or treatments on human subjects to determine safety and efficacy. They progress through several phases, beginning with small pre-clinical trials on animals. Phase 1 trials involve 20-50 healthy volunteers to assess pharmacokinetics and safety. Phase 2 trials enroll 50-300 patient volunteers to further evaluate safety and dosage. Phase 3 trials are large randomized controlled trials of 250-1000+ subjects comparing the investigational treatment to standard treatment or placebo. If Phase 3 is successful, the results are submitted to regulatory agencies for approval to market the new drug. Post-marketing Phase 4 trials monitor long-term safety and efficacy.
Clinical data management is the process of collecting, validating, and cleaning data from clinical trials. It aims to ensure data quality and integrity. Key aspects of clinical data management include electronic data capture, establishing data standards, using clinical data management systems, and performing activities like data collection, validation, and discrepancy management. It follows guidelines from organizations like SCDM and regulations like 21 CFR Part 11.
The PV audit ensures that a company’s drug safety and pharmacovigilance operations comply with applicable laws, regulations and guidances worldwide, and compare to best practices for organizations of similar size.
Medical Writing Essential: Reviewing Statisitical Analysis Plansjbarag
Regulatory medical writers are tasked with generating high-quality clinical study reports (CSRs) promptly. To this end, statistical analysis plan (SAP) reviews are essential as they allow medical writers to verify that the SAP contains the information required for the CSR per regulatory guidance. This session will explain how to conduct SAP reviews and how to assess whether data presentations in addition to those proposed are needed for the CSR.
This document discusses the rising costs of medical imaging and strategies to reduce costs. It notes that diagnostic imaging is the fastest growing component of healthcare costs, with up to a third of procedures being inappropriate. Digital imaging technologies can reduce costs compared to conventional methods when accounting for long-term costs. However, overuse of imaging from increased physician requests and patient demands contributes to higher costs. Promoting the appropriate use of diagnostic imaging through guidelines and reducing unnecessary procedures can help lower costs while maintaining quality of care.
The document discusses Hutchinson-Gilford Progeria Syndrome (HGPS), a rare genetic condition that causes rapid, premature aging in children. It notes that HGPS is caused by mutations in the lamin A gene that lead to structural and functional abnormalities in the nuclear lamina. Specifically, it disrupts the biogenesis of lamin A, causing lobulation of the nuclear membrane, diminished replication potential, abnormal chromatin organization, and a slow DNA damage response. This results in premature aging symptoms like lipodystrophy, alopecia, and joint contractures in children. The document explores potential mechanisms of the disease, including effects on nuclear morphology, telomere length, DNA replication, and protein accumulation. It suggests that even
Safety reports rmp risk management plan pharmacovigilanceAzierta
A Risk management plan is a document based on safety profile of medicines that collects all pharmacovigilance activities and it is used to plan and implement measures in order to minimize risks.
This summary explains how to develop a Risk Management Plan according to European regulatory requirements.
Implementation dates and objectives of RMP module V of Good Pharmacovigilance Practices.
The Office of Orphan Products Development (OOPD) at the FDA promotes the development of treatments for rare diseases and conditions. There are more than 6,800 known rare diseases affecting an estimated 25-30 million Americans. The Orphan Drug Act of 1983 provides financial incentives like tax credits, user fee waivers, and exclusive marketing rights for 7 years to encourage development of treatments for rare diseases. The OOPD oversees programs that grant orphan drug designation, provide funding for clinical trials and natural history studies, and award priority review vouchers for rare pediatric diseases.
Literature searches in Pharmacovigilancesamikshagupta
This presentation provides a detailed description of various literature searches performed in pharmacovigilance including search criteria, different search engines etc
An brief introduction to the clinical data management process is described in this slides. These slides provides you the information regarding the data evaluation in the clinical trials , edit checks and data review finally data locking,then the data is submitted to the concerned regulatory body.
Introduction to MedDRA Coding in Drug Safety & Pharmacovigilance Process for Pharmaceuticals, Bio-Pharmaceuticals, Medical Devices, Cosmeceuticals and Foods.
Contact:
"Katalyst Healthcares & Life Sciences"
South Plainfield, NJ, USA
info@KatalystHLS.com
Clinical research involves systematic studies in human subjects to improve quality of life. Clinical trials are a form of clinical research that experimentally tests medications, devices, or biologics to evaluate safety and effectiveness. There are different phases of clinical trials, from small early phase safety studies to larger late phase studies to establish efficacy. Clinical trials require many players including regulators, sponsors, investigators, ethics committees, and participants to design, monitor, and conduct the trials according to strict protocols to safely and effectively evaluate potential new treatments.
MedDRA is a standardized medical terminology used in the pharmaceutical industry and regulatory bodies for coding adverse event reports. It contains over 60,000 terms organized into a hierarchical structure with 5 levels across 26 system organ classes. MedDRA allows for improved data entry of reports and flexible retrieval of information due to its multiaxial structure and guidance documents provide recommendations for its effective use and analysis.
Clinical trials are scientific studies that test new medical treatments or interventions on human volunteers. They are conducted by pharmaceutical companies or contract research organizations to evaluate the effects, risks, benefits, and efficacy of new drugs, biologics, or devices prior to their market release. Major players in a clinical research team include investigators, clinical research associates, coordinators, data managers, statisticians, patients, institutional review boards, and regulatory bodies. Principal investigators are responsible for leading the clinical trial team at each study site and ensuring proper conduct of the trial according to protocol and regulations.
This document defines signal detection as information that suggests a new potentially causal association or aspect of a known association between an intervention and adverse or beneficial events. It discusses the importance of signal detection for identifying safety concerns, improving patient outcomes, and regulatory compliance. Several methods of signal detection are described, including data mining, quantitative statistics, adverse event monitoring, and machine learning algorithms. Challenges and future directions are also outlined.
Safety and Pharmacovigilance System: Oracle Argus Safety SuitePerficient
Oracle's Argus Safety is a comprehensive pharmacovigilance platform designed to help life sciences companies ensure global regulatory compliance, enable faster safety decisions through data insights, and integrate safety and risk management functions. With Argus Safety, companies can manage regulatory reporting requirements, obtain visibility into reporting metrics and compliance, and leverage automation and advanced functionality to maximize case processing efficiency and data access for timely review. Argus Safety also provides integrated risk management capabilities and can be seamlessly connected to signal detection systems to proactively manage product safety.
This journal club presentation summarizes an article that examines the association between women's autonomy and utilization of maternal health services in Nepal. The presentation provides background on high maternal mortality in developing countries like Nepal. It summarizes the article's methods, which used Nepal Demographic and Health Survey data to analyze the relationship between women's autonomy and antenatal care, institutional delivery, and postnatal care. The results found a positive association between women's autonomy and utilization of maternal health services. In conclusion, the presentation emphasizes the need to promote women's autonomy to help ensure effective maternal healthcare.
The slide provides a basic understanding about Clinical Research process and the various Phases of Drug Discovery and Development. It also explains about the various trial designs and techniques in research such as blinding and randomization. It may be useful for giving a basic class for Fourth Year B.Pharm Students.
Introduction to Adverse Drug Reactions in Pharmacovigilance and Drug Safety in Pharma, Biotech, Medical Device, Cosmeceutical and Foods.
Contact:
"Katalyst Healthcares & Life Sciences"
South Plainfield, NJ, USA
info@KatalystHLS.com
Explaining the importance of a database lock in clinical researchTrialJoin
One of the most crucial aspects of research is clinical data management or CDM. Proper CDM will generate results with excellent quality, integrity, and reliability. Quality data is essential in order to support the final conclusions of a certain study.
The person responsible for this area of research is called a clinical data manager. This job position can be filled by a PI, a study coordinator, or a CRA. No matter who fills this position at your site, data management has to be done promptly and correctly in order to generate the best results. Aside from all the other reasons why data management is so important, it’s also what determines the future IP (investigational product) development.
Clinical trials involve testing investigational drugs or treatments on human subjects to determine safety and efficacy. They progress through several phases, beginning with small pre-clinical trials on animals. Phase 1 trials involve 20-50 healthy volunteers to assess pharmacokinetics and safety. Phase 2 trials enroll 50-300 patient volunteers to further evaluate safety and dosage. Phase 3 trials are large randomized controlled trials of 250-1000+ subjects comparing the investigational treatment to standard treatment or placebo. If Phase 3 is successful, the results are submitted to regulatory agencies for approval to market the new drug. Post-marketing Phase 4 trials monitor long-term safety and efficacy.
Clinical data management is the process of collecting, validating, and cleaning data from clinical trials. It aims to ensure data quality and integrity. Key aspects of clinical data management include electronic data capture, establishing data standards, using clinical data management systems, and performing activities like data collection, validation, and discrepancy management. It follows guidelines from organizations like SCDM and regulations like 21 CFR Part 11.
The PV audit ensures that a company’s drug safety and pharmacovigilance operations comply with applicable laws, regulations and guidances worldwide, and compare to best practices for organizations of similar size.
Medical Writing Essential: Reviewing Statisitical Analysis Plansjbarag
Regulatory medical writers are tasked with generating high-quality clinical study reports (CSRs) promptly. To this end, statistical analysis plan (SAP) reviews are essential as they allow medical writers to verify that the SAP contains the information required for the CSR per regulatory guidance. This session will explain how to conduct SAP reviews and how to assess whether data presentations in addition to those proposed are needed for the CSR.
This document discusses the rising costs of medical imaging and strategies to reduce costs. It notes that diagnostic imaging is the fastest growing component of healthcare costs, with up to a third of procedures being inappropriate. Digital imaging technologies can reduce costs compared to conventional methods when accounting for long-term costs. However, overuse of imaging from increased physician requests and patient demands contributes to higher costs. Promoting the appropriate use of diagnostic imaging through guidelines and reducing unnecessary procedures can help lower costs while maintaining quality of care.
The document discusses Hutchinson-Gilford Progeria Syndrome (HGPS), a rare genetic condition that causes rapid, premature aging in children. It notes that HGPS is caused by mutations in the lamin A gene that lead to structural and functional abnormalities in the nuclear lamina. Specifically, it disrupts the biogenesis of lamin A, causing lobulation of the nuclear membrane, diminished replication potential, abnormal chromatin organization, and a slow DNA damage response. This results in premature aging symptoms like lipodystrophy, alopecia, and joint contractures in children. The document explores potential mechanisms of the disease, including effects on nuclear morphology, telomere length, DNA replication, and protein accumulation. It suggests that even
Charlie Keller, a primary care physician at Mercy Clinics, Inc. describes Mercy's experience with shared decision making implementation.
This presentation was part of a Shared Decision Making Month webinar -- Shared Decision Making in the Real World: Stories from the Frontline.
The document discusses medication non-adherence, which is a major problem that impacts health and costs the healthcare system billions annually. It describes various technologies that have been developed to help patients better manage and adhere to their medication schedules, ranging from basic pillboxes and reminders to more advanced automated dispensers and sensors. The document recommends pilot studies be conducted to evaluate the real-world effectiveness of different medication adherence technologies in improving patient outcomes beyond standard discharge instructions alone.
This document discusses medicines optimisation, which aims to identify, intervene, and resolve issues with adverse medication performance, inappropriate prescribing, and non-compliance. The goal is to improve treatment outcomes for patients, improve value for the NHS, and ensure optimal and safe treatment based on evidence. Pharmacists can contribute by ensuring safe prescribing adheres to best practices, assessing drug histories for issues like polypharmacy, optimizing drug therapy, providing medication education, and coordinating information for patients. A case study examines a high-risk elderly patient taking multiple medications and proposes recommendations like reviewing medications like diuretics and shorter-acting benzodiazepines to reduce falls risk.
Palliative care aims to provide relief from the symptoms and stress of serious illness through comprehensive treatment of pain and other physical, psychosocial, and spiritual problems. It can improve quality of life for both patients and families. Palliative care is provided along the illness continuum and can be beneficial at any stage of a serious illness, while hospice care focuses on comfort and support for those nearing the end of life. Both palliative care and hospice aim to improve quality of life, but palliative care is not limited to a prognosis of six months or less.
This document discusses anesthesia considerations for patients with diabetes. It defines diabetes and classifies it into types 1, 2, gestational, and secondary. It describes end-organ complications like cardiovascular, renal, and neurological issues. It also discusses acute complications like diabetic ketoacidosis and hypoglycemia as well as chronic effects of hyperglycemia. The anesthetic management of patients with diabetes focuses on glycemic control and addressing any organ dysfunction or comorbidities.
Translational Medicine: Patterns of Response to Antidepressant Treatment and ...Joanne Luciano
This is a talk I gave at the IEEE Schenectady Section - 17 MAY Membership Meeting.
The mission of my depression research is to help people figure out what they need to help them get out of a depressed state. That is, finding out what is best for them, not what is best for their doctor, friends, therapist, or anyone else. Depression is now a global problem. In the past 15 years it has gotten worse. Depression is complex; it has a wide range of varying symptoms and degrees of intensity. It can be challenging to determine the best course of action, whether medical treatment is necessary, or which of the many treatments (drug and non-drug) is the best match. Many people who are depressed do not get the help they need, and many people receive medications when they are not necessary. My work aims to bring together tools, technology, scientific and medical data and patient experience to help address depression, both personally and globally.
Geriatric Special Focus, Pain Management and Analgesic Prescribing for Advanc...Michelle Peck
Michelle Peck | Geriatric Nurse Practitioner | Health Care Consultant | Professional Speaker | Nursing Faculty| Legal Nurse Consultant | Mindful Geriatrics
In collaboration with Dr. Linh Nguyen, Supportive Medicine at UTHealth Medical School, we have created this slide deck for Advanced Practice Nurses.
Our mission is to simplify the pharmacologic basics of good pain prescribing. We have not provided very much detail about schedule II controlled substances due to the current limitations on Texas Nurse Practitioner prescribing in primary care.
This lecture is designed to meet our Advanced Practice Nursing audience where they are at and provide tools, knowledge and practical tips. Areas where we detect mastery with our polling questions are briefly touched upon and more time and examples are given are to areas of audience identified needs. Prescribing pain medication for Advanced Practice Nurses is dynamic, complex and ever changing
We have also included a special focus (our passion) for pain prescribing in the geriatric population. Beer’s Criteria medications, to be used with caution or avoid completely in geriatrics are mentioned throughout this presentation.
This presentation starts with the audience writing down their biggest fear about pain prescribing. We then categorize these fears, so that throughout our lecture we can give special focus and alleviate fears with practical tips, guidelines and real life examples.
Our objectives are to discuss:
1. Benefits and side effects of common analgesics
2. The impact of patient-related factors on drug selection & dose based on knowledge of patient related changes
3. Medications to avoid, use with caution, explain why
4. Management of pain based on client care goals
We hope you Learn it-Live it-Love it!
A world without (new) drugs – play it before you live itJonas Boström
Gamification has proven successful in motivating behavior change. Examples like Nike+ have engaged millions to improve fitness. Games like "Zombies, Run!" use audio narratives and mission-based gameplay to motivate running. Crowdsourcing projects have harnessed gamers' collaborative skills, as seen when over 20,000 British citizens investigated MPs' expenses claims through an online game. Some examples apply gamification in medicine for patient adherence or cognitive training. Computational protein folding games like Foldit engage players in scientific problem-solving. However, drug discovery could better leverage gamification through more collaborative and intuitive software that captures elements of serious gaming.
The endogenous cannabinoid signaling system is comprised of cannabinoid receptors, endocannabinoid ligands, and proteins involved in endocannabinoid synthesis. The two main cannabinoid receptors are CB1, found primarily in the brain and reproductive systems, and CB2, found almost exclusively in the immune system. Anandamide is the main endogenous cannabinoid ligand and is broken down by the enzyme FAAH. Inhibitors of FAAH are being researched for therapeutic use as they can elevate anandamide levels. Cannabidiol is a non-intoxicating component of cannabis that inhibits FAAH and shows promise for treating conditions like schizophrenia
This document discusses medical marijuana. It provides background on marijuana and its classification. It assesses the efficacy and safety of medical marijuana for conditions like nausea, pain, appetite stimulation, and others. It also explains the legal implications of medical marijuana in the US and in states like Colorado and Illinois that have legalized it. Remaining challenges include a lack of standardized dosing and quality control. The pharmacist's role could include counseling patients, assessing drug interactions, and producing customized dosage forms.
Medication non-adherence is a growing concern, as it is increasingly associated with negative health outcomes and higher cost of care. Tackling the burden of non-adherence requires a collaborative, patient-centric approach that considers individual patient needs and results in intelligent interventions that combine high-tech with high-touch.
Palliative care aims to improve quality of life for patients with life-limiting illnesses through early identification and treatment of pain and other symptoms. Palliative care takes a holistic approach addressing physical, psychosocial and spiritual needs. Dyspnea, or breathlessness, is a common and distressing symptom experienced by over 50% of hospice patients. A thorough history and assessment of dyspnea is important to identify potential causes and guide treatment options. Both non-pharmacological and pharmacological interventions can provide relief, including opioids, benzodiazepines, oxygen, bronchodilators, and corticosteroids. Active management of dyspnea is important during the last hours of life to minimize suffering.
This document discusses cannabis-related disorders. It begins with an introduction to cannabis and its various names. It then defines cannabis-related disorder and discusses its history of use dating back 8000 years. It describes how cannabis is prepared and consumed, as well as its epidemiology. The document outlines diagnostic criteria for cannabis use disorder and intoxication based on the DSM-5. It discusses various health problems associated with cannabis use including withdrawal symptoms, psychosis, anxiety, cognitive impairment, and effects on respiratory and pregnancy outcomes. Treatment involves both pharmacological approaches like benzodiazepines for withdrawal and psychosocial therapies such as CBT, MET, and family intervention.
This document discusses orphan drugs and rare disease drug development. It outlines the challenges in developing drugs for rare diseases, including small patient populations and lack of knowledge. The role of clinical pharmacology in drug development is described, including use of innovative analyses, trial designs, and knowledge management to help overcome challenges. Clinical pharmacology can point the way by applying lessons from oncology and pediatric drug models to the orphan drug context. Collaboration across different stakeholders is needed to develop safe and effective drugs for rare diseases.
Transalational Sciences and Clinical Pharmacology in Orphan Drug DevelopmentE. Dennis Bashaw
This document discusses challenges and solutions for developing orphan drugs and implementing precision medicine programs. It notes that orphan drug development faces resource constraints as trials require more patients than rare diseases have. However, using clinical pharmacology tools allows learning from small populations by incorporating biomarkers, modeling, and innovative designs. The future of drug development lies in going beyond traditional trials to involve patients more and continually learn from individual experiences. Quantitative tools during all stages of development can extract maximum knowledge from minimal data to improve understanding, approval chances, and labeling.
Clinical Pharmacology: Leveraging Science to Provide AccessE. Dennis Bashaw
1) The document discusses the paradigm shift in biomarker development from a "learn and confirm" model to an "identify, confirm, refine, and learn" model to continuously learn from clinical trials.
2) It outlines policies used by the FDA to incentivize orphan drug development, including expedited programs, marketing exclusivity, and fee reductions. International collaboration is important for developing policies tailored to individual countries.
3) Successful partnerships between the FDA, NIH, patient groups, and industry have helped advance rare disease drug development, including through biospecimen repositories and training programs.
This document summarizes regulatory perspectives on approving drugs for rare diseases from a presentation given by an FDA official. It provides an overview of drug development for rare diseases before and after the 1983 Orphan Drug Act. While the Act increased incentives for rare disease drug development, approval challenges remain due to small patient populations and lack of understanding of rare diseases. The FDA aims to apply flexibility in effectiveness standards and innovative trial designs and analyses. Ongoing collaborations between the FDA, industry, academia, and patient groups are needed to continue advancing treatments for rare diseases.
Slides presented by me at the Korean-American Professional Association in Life Sciences (KAPAL) 5th Annual Meeting in Rockville, MD. Slides discuss the recent reorganization of the Center for Drug Evaluation and Research at the FDA
This document provides an overview of the drug development and clinical trial process. It discusses the various phases of drug discovery and development, including pre-clinical research (discovery and pharmacology) and clinical trials (Phase 1-3). Key points covered include the high costs and risks of drug development, clinical trial design and goals for each phase, pharmacokinetics/dynamics studies to understand drug behavior in the body, and factors like safety, efficacy, and regulatory approval. The overall process takes an average of 15 years and $2.6 billion to bring a new drug to market.
The document discusses regulations for clinical trials in India. It begins by explaining that an Investigational New Drug Application (IND) provides an exemption that allows investigational drugs to be transported across state lines for clinical trials. It then describes the process of submitting an IND to the FDA, including providing animal studies data, manufacturing information, clinical protocols, and investigator information. It notes that the FDA has 30 days to review submitted INDs. Finally, it summarizes that in India, an application for clinical trials should be submitted to the DCGI along with chemistry, manufacturing, animal study data and other required documents and trial protocols, and trials can only begin after approval from the DCGI and ethics committee.
This document provides an introduction to pharmacoepidemiology. It defines pharmacoepidemiology as the study of drug use and effects in large populations. It discusses study designs used in pharmacoepidemiology like randomized trials, cohort studies, and case-control studies. It also covers sources of data for pharmacoepidemiology studies like spontaneous reporting, case-control surveillance, and prescription event monitoring. Reasons for conducting pharmacoepidemiology studies include fulfilling regulatory requirements, exploring safety questions, and generating or testing hypotheses.
Evaluation of the evidence of the drug developmentaJaY mIsHrA
This document summarizes the process of drug development, including clinical trials and phases of trials. It discusses key features of clinical trials like randomization and blinding. The four phases of trials are described - phase I evaluates safety, phase II assesses efficacy and dose, phase III confirms therapeutic effects, and phase IV monitors post-marketing use. Observational studies like cohort and case-control designs are also mentioned. Factors that can alter drug pharmacokinetics in disease states like impaired renal or hepatic function are outlined. The history of early drug discovery and development in India is briefly discussed.
Population pharmacokinetics is the study of the sources and correlates of variability in drug concentrations among individuals who are the target patient population receiving clinically relevant doses of a drug of interest
Presentation: The Australian and International landscape - keynote forumTGA Australia
This document summarizes a keynote forum on the Australian and international regulatory landscape presented at the 2017 ARCS Annual Conference. It discusses several topics, including an increasing number of oncology drugs and trials, new pathways for priority and provisional drug approvals in Australia and comparisons to other countries, regulation of biosimilars and orphan drugs, clinical trial complexity, and developments in medical device and software regulation. It also touches on issues of regulatory transparency and uncertainties from Brexit.
This document discusses Phase IV clinical trials, which are post-marketing studies conducted after a drug has been approved. Phase IV trials have several objectives, including assessing long-term risks and benefits of drug use in real-world populations. They can be mandated by regulatory authorities or initiated independently. Regulatory-mandated studies evaluate things like drug interactions, new formulations, or safety in special populations. Independent studies may utilize randomized controlled trial designs to compare drugs or surveillance methods to monitor drug use. Sample sizes are typically larger than Phase III trials to detect rare side effects.
Presentation on drug development challenges and clinical trial optimization. Originally presented at DIA China May 2017. The companion slide set is here as well-Optimizing Clinical Trials with Advanced Tools
This document summarizes a webinar on building Canada's strategy for access to rare drugs. Day 1 focuses on challenges to access. Dr. Cheryl Rockman-Greenberg discusses her work on hypophosphatasia (HPP), a rare bone disease. She outlines the different forms of HPP and clinical studies of asfotase alfa treatment. Asfotase alfa was approved in Canada for pediatric HPP but access challenges remain for adults. Real-world evidence is needed to understand natural history and inform treatment criteria for adults. The HPP patient registry collects global data on treatment effects, safety and outcomes to address knowledge gaps.
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This document discusses Good Pharmacy Practice (GPP) and clinical pharmacy. It provides background on GPP and outlines the key components of a GPP framework, including legal, workforce, and economic considerations. The presentation defines clinical pharmacy as applying scientific principles to patient care and outlines the shift in pharmacy from product-focused to patient-focused care. It also discusses rational prescribing and dispensing, pharmacovigilance, drug information centers, and recommendations for a national GPP framework in Nepal.
DDS personalised medicines M.Pharma 1st Sem Pharmaceutics.pptxkushaltegginamani18
The document discusses personalized medicines and customized drug delivery systems. It defines personalized medicine as using genetic profiling and other individual patient characteristics to guide medical treatment. Customized drug delivery systems aim to optimize drug therapy for each patient by controlling dosage and delivery through technologies like bioelectronic medicines, 3D printing of pharmaceuticals, and telepharmacy.
This document provides an overview of Schedule Y, which establishes requirements and guidelines for conducting clinical trials and obtaining permission to import or manufacture new drugs in India. Schedule Y aims to frame guidelines for clinical research in line with global standards. It outlines application procedures, ethics committee requirements, pre-clinical and clinical data to be submitted, protocol elements, and other rules to ensure safety and efficacy of investigational products. The objectives of Schedule Y are to regulate clinical trials and new drugs in India according to current scenarios and integrate the country into global drug development while improving clinical trial quality.
Similar to Clinical Pharmacology in Orphan Drug Development (20)
The document summarizes the history of dermal absorption assessment at the FDA. It discusses how prior to the 1990s, topical dermal drugs had little direct assessment of in vivo bioavailability and clinical efficacy trials were used instead. In the mid-1990s, the FDA began requiring "maximal use trials" (MUsT) to better understand absorption under conditions maximizing exposure. MUsTs became standard for new drug applications. The document then focuses on sunscreen absorption studies showing circulating levels and the FDA's own studies evaluating absorption of 7 ingredients under MUsT conditions. It concludes MUsTs provide important safety data but the FDA has not said sunscreens are unsafe.
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This is the slide deck used in my webinar that was presented on 2/1/2018 on the FDA Predictive Toxicology Roadmap. It was presented at a webinar hosted by BioIVT in my private capacity and is not an official statement of FDA policy
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This document summarizes four case studies involving issues with bioanalytical method validation:
1) An analyst constructed standard curves inconsistently between assay runs, using a varying number of calibration standards.
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3) Subject concentration-time curves showed implausible "U-shaped" profiles, indicating an unresolved analytical problem.
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This document summarizes four case studies of issues encountered during bioanalytical method validation:
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2) An assay was used for many years without re-validation despite changes in equipment affecting analyte retention times.
3) Some subjects showed non-physiological "U-shaped" concentration-time curves that were accepted without investigation.
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Learning objectives:
1. Define an electrocardiogram (ECG) and electrocardiography
2. Describe how dipoles generated by the heart produce the waveforms of the ECG
3. Describe the components of a normal electrocardiogram of a typical bipolar lead (limb II)
4. Differentiate between intervals and segments
5. Enlist some common indications for obtaining an ECG
6. Describe the flow of current around the heart during the cardiac cycle
7. Discuss the placement and polarity of the leads of electrocardiograph
8. Describe the normal electrocardiograms recorded from the limb leads and explain the physiological basis of the different records that are obtained
9. Define mean electrical vector (axis) of the heart and give the normal range
10. Define the mean QRS vector
11. Describe the axes of leads (hexagonal reference system)
12. Comprehend the vectorial analysis of the normal ECG
13. Determine the mean electrical axis of the ventricular QRS and appreciate the mean axis deviation
14. Explain the concepts of current of injury, J point, and their significance
Study Resources:
1. Chapter 11, Guyton and Hall Textbook of Medical Physiology, 14th edition
2. Chapter 9, Human Physiology - From Cells to Systems, Lauralee Sherwood, 9th edition
3. Chapter 29, Ganong’s Review of Medical Physiology, 26th edition
4. Electrocardiogram, StatPearls - https://www.ncbi.nlm.nih.gov/books/NBK549803/
5. ECG in Medical Practice by ABM Abdullah, 4th edition
6. Chapter 3, Cardiology Explained, https://www.ncbi.nlm.nih.gov/books/NBK2214/
7. ECG Basics, http://www.nataliescasebook.com/tag/e-c-g-basics
Here is the updated list of Top Best Ayurvedic medicine for Gas and Indigestion and those are Gas-O-Go Syp for Dyspepsia | Lavizyme Syrup for Acidity | Yumzyme Hepatoprotective Capsules etc
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1. Clinical Pharmacology in
Orphan Drug Development
KoNECT 3rd Regulatory Symposium
CAPT E. Dennis Bashaw, PharmD.
Director, Division of Clinical Pharmacology-3
Office of Clinical Pharmacology
Office of Translational Sciences
CDER/FDA
2. 2
Disclaimer: The presentation today
should not be considered, in whole or
in part as being statements of policy or
recommendation by the US Food and
Drug Administration.
3. 3
Outline
• Drug Development and Orphan Drugs/Rare
Diseases in the United States: An Overview
• 1983 Orphan Drugs Act-PROBLEM SOLVED?
• Challenges And the Role of the Regulator:
Science, Regulation, and Policy
• Bringing Clinical Pharmacology Tools to Bear
• Concluding Thoughts
5. 5
Pre Orphan Drug Act: 1982
• 1973-1982: 10 new drugs for rare diseases
– Little economic incentive for large pharmaceutical
companies to pursue rare disease indications
• ≈7,000 rare diseases; 25 million people
– In comparison: 67 million American adults (31%)
have high blood pressure—that’s 1 in every 3
people in this room
(http://www.cdc.gov/bloodpressure/facts.htm)
• ~85% of orphan diseases have a genetic basis
• Increasing by ~100 diseases/year
6. 6
Chronology of Drug Development
for Classical and Orphan Drug
Nature Reviews and Drug Discovery, 2003, Volume 2, Page 71
7. 7
Trends in Drug Discovery
Scannell, JW, et al “Diagnosing the decline in pharmaceutical R&D efficiency”
Nature Reviews Drug Discovery, 11:191-200 (March 2012)
An unsustainable
trend for an Orphan
Drug
8. 8
Updated Drug Development Cost Figures
J Health Econ. 2016 May;47:20-33. doi: 10.1016/j.jhealeco.2016.01.012
9. 9
Fast Track Designation
• Drug intended to treat a serious condition
• Nonclinical or clinical data needed to
demonstrate the potential to meet an
unmet medical need.
Breakthrough
• Drug intended to treat a serious condition
• Must be preliminary clinical evidence to
indicate the drug may substantially improve a
clinically significant endpoint compared to
available therapies
Priority Review
• Drug must treat a serious condition and, if
approved, offer a significant improvement in
safety or effectiveness
• Designation assigned only at the time of the
original NDA or efficacy filing
Accelerated Approval
• Drug must treat a serious condition and
generally provide a meaningful advantage over
available therapies
• Must demonstrate an effect on a surrogate
endpoint that is likely to predict a clinical
benefit or on a clinical endpoint
FDA’s “Accelerated” Pathways –
Not Exclusive to Orphan Drugs
Entering Drug
Development cycle
To Market
https://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm358301.pdf
10. 10
Accelerated Pathways Under PDUFA-V
https://www.fda.gov/downloads/forindustry/userfees/prescriptiondruguserfee/ucm552923.pdf
13. 13
How Well Are We Doing?
• In past few years
– ~1/3 of all NME approvals are Orphan products
– 2/3 of therapeutic biological product approvals
• While there has been progress in the general science and
approval of Orphan Drugs….just like an iceberg much more
lies below the surface to be done.
– 7,000 plus indications
• Since 1983
– 4071* drugs with an orphan designation
– 602* drugs approved……..
*as of 4/23/2017
17. 17
Regulatory Challenge
• Orphan drugs held to same evidentiary standard
as non-Orphan drugs
• To be approved in US, Orphan drugs must:
– Demonstrate substantial evidence of
effectiveness/clinical benefit (21CFR 314.50)
– Substantial evidence of benefit requires:
• Adequate and well-controlled clinical study(ies)
– designed well enough so as to be able “to distinguish the effect of a
drug from other influences, such as spontaneous change…, placebo
effect, or biased observation” (§314.126)
18. 18
The REGULATORS “DUAL” Role
National
Authority
Regulations
Science
Conservative Innovation
Clinical
Pharmacology
Regulations provide room for flexibility in the review of treatments for rare diseases
and the application of regulatory standards….i.e., good scientific judgment
20. 20
Challenges in Orphan Disease/
Rare Drug Development
• Large heterogeneity in disease pathophysiology
• Poorly understood natural histories and progression
• Few patients are available conducting clinical trials
• Uncertain appropriate duration of treatment
• Lack appropriate endpoints that predict outcomes
• Large heterogeneity in treatment effects
• Require compromise, innovation and trade-offs
• Make difficult decisions in absence of ideal information
Proper deployment of Clinical Pharmacology in
orphan drug development can extract the most
amount of knowledge from least amount of
information
21. 21
INNOVATIVE ANALYSES
•Improved Computing Resources
•Quantitative drug-disease-trial models
• Exposure-response models
INNOVATIVE TRIAL DESIGNS
• Clinical trial simulations
• Enrichment, adaptive, dose-response
• In Silico
KNOWLEDGE MANAGEMENT
• Leverage prior data
• Locate and observe trends
• Understand the need
Bringing Clinical Pharmacology
Tools to Bear
22. 22
Trends in Orphan Drug Approvals and
Development
Clin Pharmacol Ther. 2012
May;91(5):932-6.
doi: 10.1038/clpt.2012.23.
27. 27
INNOVATIVE ANALYSES
•Improved Computing Resources
•Quantitative drug-disease-trial models
• Exposure-response models
INNOVATIVE TRIAL DESIGNS
• Clinical trial simulations
• Enrichment, adaptive, dose-response
• In Silico
KNOWLEDGE MANAGEMENT
• Leverage prior data
• Locate and observe trends
• Understand the need
Bringing Clinical Pharmacology
Tools to Bear
28. 28
Clinical Pharmacology Studies in an NDA
• Bioavailability (Absolute or Relative)
– Radiolabel
• Dose and/or Dosage Form Proportionality
• Single Dose Pharmacokinetics
• Multiple Dose Pharmacokinetics
• Metabolism Studies
– P-450 Isoenzymes
• Special Populations
– Elderly
– Renal Failure/ Hepatic Failure
– Pediatrics
• Misc.
– Formulation Studies
– Bioequivalency (Clinical vs. To-be-marketed)
– Drug Interaction
– Protein Binding
– Mechanism of Action
– Dissolution
Exposure Response
Dose Response
Dose Titration
Surrogate Endpoint
Pharmacometrics
Pharmacogenomics
29. 29
Orphan Drug Review at the FDA
Eliglustat for Gaucher’s Disease
• Gaucher disease is a rare, autosomal recessive lysosomal storage
disorder caused by a deficiency in the lysosomal enzyme
glucocerebrosidase (or acid-β glucosidase), which catalyzes the
hydrolysis of glucosylceramide (or GL-1) to glucose and ceramide.
This enzyme deficiency results in the accumulation of GL-1,
especially in the liver, spleen, and bone marrow.
http://www.accessdata.fda.gov/drugsatfda_docs/nda/2014/205494Orig1s000ClinPharmR.pdf
30. 30
Eliglustat (Cerdelga®)
• Eliglustat is a selective glucosylceramide synthase inhibitor for
substrate reduction therapy (SRT) to reduce the synthesis and
hence the accumulation of GL-1.
• Received both a Priority Review and Orphan Drug Designation
31. 31
Metabolic Information Available on
Eliglustat Prior to PBPK Modeling
• Metabolized by CYP2D6 (~80%) and CYP3A (~20%)
• High clearance, nonlinear PK: time-dependent CYP2D6 inhibitor
• Clinical drug interaction studies
– With strong CYP2D6 inhibitor paroxetine: AUC increased by ~8-fold
– With strong CYP3A inhibitor ketoconazole: AUC increased by ~4-fold
• Pharmacogenetic effects: PM/EM ~ 8-fold
32. 32
Combining the Workstreams
Pharmacogenomics
Utilize in vitro systems
to identify relevant CPY
polymorphism
PBPK Modeling
Build models based on
observed knowledge with a
“learn and confirm” strategy.
Classical PK/PD
Synthesize the
available PK/PD data
on Drug Metabolism
Develop
Actionable
Information
Informed labeling for the
prescriber
33. 33
Impact of PBPK on Labeling
http://www.accessdata.fda.gov/drugsatfda_docs/label/2014/205494Orig1s000lbl.pdf
34. 34
Impact of PBPK on Labeling
http://www.accessdata.fda.gov/drugsatfda_docs/label/2014/205494Orig1s000lbl.pdf
38. In October 2011, FDA awarded $2 million to launch Centers of
Excellence in Regulatory Science and Innovation at the
University of Maryland and Georgetown University. The
investment is part of FDA’s effort, outlined in the Agency's
strategic plan, to foster a robust, collaborative, regulatory
science culture that enables FDA to address the scientific
challenges presented by revolutions in medical product
development and to improve food safety and quality. In 2014
two new centers were established in collaboration with the FDA.
FDA-Academic Collaboration
41. 41
Policies to Incentivize Orphan Drug
Development - APAC & Major Regions*
POLICY elements USA EU Australia Japan S. Korea Singapore Taiwan
Legal framework
(1983)
(2000)
(1997)
(1993)
(1998)
(1991)
(2000)
Prevalence
(per 10,000)
7.5 5 1 4 4 36 1
Other criteria
Life-
threatening &
chronically
debilitating
Incurable
disease with no
alternative
treatment
No alternative
treatment
Marketing exclusivity 7 years 10 years - 10 years 6 years§
- 10 years
Tax credit/Grants for R&D - - - - -
Accelerated MA procedure ** ** **
Fee reduction - - -
Protocol assistance - - -
*Slide modified from David Tsui (Shire Pharmaceuticals
** For life saving drugs where there is no therapeutic alternative
§ proposal to increase to a maximum of 10 years
43. 43
Development of Safe and Effective Drugs
For ALL Requires a Team Effort
Academia
Industry
International
Collaboration
Patient
Advocacy
FDA Science
& Policy
Benefits
To All
44.
45. 45
Contact Information
CAPT Edward D. Bashaw, PharmD.
Director, Div. of Clinical Pharmacology-3
US FDA
10903 New Hampshire Ave
Building 51, Rm 3134
Edward.Bashaw@fda.hhs.gov
46. 46
Acknowledgements
• The Staff of the Division of Clinical Pharmacology-3
• The Office of Clinical Pharmacology
• The Office of Translational Sciences