"The Use of Patient Preference & Experience Data in FDA Regulatory Decision Making"
When: Monday, January 29, 2018
Carrie Kuehn, M.A., M.P.H., L.P.D, RAC, Senior Managing Scientist, Exponent, Inc.
Jordana Schmier, M.A., Senior Managing Scientist, Exponent, Inc.
This session will review the recent policy developments at FDA with regard to the use of patient preference and patient experience data in regulatory decision-making. FDA has made it a priority to incorporate the patient voice in the review of new therapeutics, including drugs, biologics, and medical devices. The agency is also encouraging the use of patient-based data in the development of new medical products and the design of clinical trials. New guidance documents are now available, and FDA continues to hold frequent webinars, meetings, and workshops for the purpose of making clear their intentions and expectations for this new regulatory process. However, there are many aspects of this regulatory process that remain yet to be determined. By the conclusion of this session, participants will have insight into how and why types of data can be gathered and how to present these findings to the appropriate audience. An analysis of the various stakeholders and goals of the patient engagement from each one’s perspective will help participants to interpret and frame their findings. The implications for FDA’s intense focus on patient-based data for the development and commercialization of new therapeutics will be discussed. In addition, recent experiences and lessons learned will be shared within the context of this rapidly evolving regulatory landscape.
Dr. Carrie Kuehn is a regulatory consultant in Exponent’s Biomedical Engineering practice. She is regulatory affairs certified (RAC) in U.S. FDA medical device and pharmaceutical regulatory affairs. An epidemiologist by training, Dr. Kuehn has an extensive background in observational research in a variety of therapeutic areas. Recently, Dr. Kuehn completed her Doctor of Law and Policy degree from Northeastern University where she focused her research on the emerging policy process of using patient-based data to inform FDA regulatory policy and decision-making. Dr. Kuehn is a Lecturer in Northeastern University’s Regulatory Affairs Program and brings nearly 20 years of diverse training and experience to her consulting practice.
Ms. Jordana Schmier has worked in the outcomes research field for more than 15 years. She has substantial expertise in quality of life assessment and instrument validation, outcomes research, modeling, and health economics. She has published and presented studies in medical areas including asthma and other respiratory conditions, oncology, infectious diseases, psychiatry, hypertension, and neurological and digestive diseases. Particular areas of interest include compliance with treatment, electronic data collection, and the use of novel methodologies in patient preference and utility assessment. Prior to joining Exponent, Ms. Schmier was a project manager at MEDTAP International, Inc., (now Evidera), where her work included quality of life and pharmacoeconomic study design and implementation as well as study and site management.
Link To Archive: https://www.ispor.org/education/Webinars/CRC-PPdata/CRC-PPdata.html
"The Importance of Critically Analyzing Assumptions in Pharmacoeconomic Models: A Case Study in Modeling a Comparison of Diabetes Treatments"
When: Wednesday, December 13, 2017, 12:00-1:00pm CST
Speaker: Harry J. Smolen, MS, President & CEO, Medical Decision Modeling Inc.
This approximate 30-min presentation will address a recent pharmacoeconomic model publication and how a critical analysis of its basic model assumptions substantially alter the publication’s conclusions.
Harry J. Smolen, MS, Medical Decision Modeling Inc., President and CEO since 1994, Mr. Smolen has successfully led hundreds of projects and grants including commercial modeling and evidenced-based projects with leading pharmaceutical, biotechnology, and medical diagnostic and device companies, as well as university medical research centers. He has been Principal Investigator for six National Institutes of Health (NIH) research and development grants. Each of these projects and grants involved the integration, analysis, and presentation of clinical, economic, and humanistic evidence to research and quantitatively analyze the benefits, risks, and costs of pharmaceuticals, biotechnologies, or medical devices. Mr. Smolen is the founder of MDM, and has over 20 years of experience applying computer simulation and other methodologies to analyzing the effectiveness and cost-effectiveness of healthcare interventions. He also has over 25 years of technical writing experience. Mr. Smolen is the Past President of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Chicago Region Chapter. He has been a study section member of the following review groups: NIH’s Healthcare Delivery and Methodologies; NIH’s Surgical Sciences, Biomedical Imaging, and Bioengineering; NIH’s Biomedical Computing and Health Informatics; and the US Army Medical Research and Materiel Command. He is an active member in ISPOR, as well as a frequent journal reviewer for Value in Health, Medical Decision Making, Pharmacoeconomics, and Health Policy. He earned an MS from Purdue University in Industrial Engineering with a concentration in healthcare computer simulation and a BS from the University of Southern California in Electrical Engineering with a specialization in computer systems. Mr. Smolen is pursuing (part-time) a PhD in Industrial Engineering with a concentration in Operations Research at Purdue University. In pursuit of his PhD, he completed the coursework required for a master’s degree in applied statistics.
PowerPoint is posted as an attachment.
Savoring Life: A Comparison of Value Frameworks"
When: November 15, 2017
Dr. Daniel Touchette, PharmD, MA, Associate Professor, University of Illinois at Chicago College of Pharmacy
Discussion on estimating the costs and benefits of pharmaceuticals, along with a summary and critique of current methods and frameworks for assessing value.
Daniel R. Touchette, PharmD, MA, FCCP
Event Archive Link: https://www.ispor.org/education/Webinars/MidwestChapter/Value-Frameworks-Comparison/Value-Frameworks-Comparison.html
**MIDWEST REGIONAL CHAPTER - OCTOBER 2017 LUNCH-N-LEARN WEBINAR**
“Methods for population-adjusted indirect treatment comparison (ITC) of time-to-event outcomes”
When: Wednesday, October 18, 2017
Speaker:Vardhaman Patel, MS, PhD, Scientist, Modeling & Meta-Analysis, Pharmerit International
The presentation will cover: (a) population-adjusted ITC methods such as matching-adjusted indirect treatment comparisons (MAIC) and simulated treatment comparisons (STC); and (b) digitization of time-to-event curves from trial publications to conduct population-adjusted ITC.
Vardhaman Patel, PhD, is a scientist in the Modeling and Meta-Analysis group with Pharmerit International (Bethesda, MD). He received a PhD degree with a major in pharmacoeconomics and minor in pharmacoepidemiology from the University of Illinois at Chicago (UIC). Prior to joining Pharmerit, Vardhaman worked at Eisai Inc., where he extensively developed and adapted core/global budget impact and cost-effectiveness models for countries in Europe and Asia. In addition, he conducted meta-analyses and indirect treatment comparisons, developed the economic sections of dossiers and BaseCase interfaces for economic models. Vardhaman is experienced in the design and execution of medical chart reviews, systematic literature reviews, research proposals, and observational studies using claims data. He has co-authored several publications and peer reviewed manuscripts. His prior areas of experience include soft tissue sarcoma, epilepsy, acute coronary syndrome, thrombocytopenia, thrombosis and atopic dermatitis. Vardhaman is passionate about enhancing patients’ access to medicines by demonstrating the product value to payers using economic models.
Link To Archive:
“Precision medicine and its applications in HTA and clinical decision making”.
Date: Thursday, September 28, 2017
Time: 12:00 pm CST
We will discuss how rich clinical, economic, and genomic data can be used to identify best treatment target population, and the most appropriate treatment for each individual. We will also give examples to illustrate how the application of these methods can help with HTA negotiation and improve clinical practice.
Speaker: Eric Q. Wu, PhD, Managing Principal, Analysis Group, Inc.
Dr. Wu is a health economist specializing in health economics, outcomes, and market access issues related to pharmaceuticals and biologics. He has broad experience consulting to pharmaceutical and device companies, payers, and government agencies in North America, Europe, and Asia. Dr. Wu has extensive experience in the development of health economics and outcomes research strategy, analysis of medical claims and clinical trial data, patient chart review, patient surveys and physician surveys, and economic
When: June 29, 2017
Director, Neurological Institute Center for Outcomes Research and Evaluation,
Patient-reported outcomes are taking an increasingly important role in medical care. In addition to their use at the patient level to monitor progress of individual patients, patient-reported data at an aggregate level have significant theoretical potential for use in research, quality initiatives and operational analyses. Systematic collection of patient-reported information as part of routine care presents several challenges. This presentation will outline the Cleveland Clinic experience with the collection and use of patient-reported information.
Key Learnings & Takeaways:
At the end of the session participants will be able to:
· Discuss reasons for the increased use of patient-reported data in healthcare
· Describe how patient-reported outcomes data can be integrated into ambulatory clinical workflows· List examples of how patient-reported data aid in clinical care
· Identify opportunities to use patient-reported data to generate new knowledge
Irene Katzan, MD, MS is a neurologist and Director of the Cleveland Clinic Neurological Institute Center for Outcomes Research and Evaluation. She has a background in evaluating the outcomes of care in neurological diseases and in modifying systems to optimize patient management across healthcare venues. She is also the Medical Director for Patient-Entered Data at Cleveland Clinic and led the development and implementation of the patient-entered data initiative to collect patient-reported outcomes information at the point of care. Currently health status information is collected electronically in over 100,000 clinical encounters each month at Cleveland Clinic.
• Patients with low comorbidity medication adherence experienced more unhealthy days than those with high comorbidity medication adherence.
Steven Kymes, Ph.D.; Director U.S. Health Economics and Outcomes Research, Lundbeck
Adherence to medical therapy has long been recognized as a major public health problem, and there are numerous studies to demonstrate that patients who are adherent to therapy have better health and economic outcomes than patients who are not. However, these cost studies have been traditionally been conducted in a cross-sectional fashion comparing medical spending of those who are adherent to medication to those who are not in the same period---thus there is limited generalizability of these studies to interventions implemented to improve medication adherence. We examined the impact of change in adherence behavior over two consecutive years on medical spending in a cohort of commercially insured patients with 1) Diabetes; 2) Hypertension; or 3) Hypercholesterolemia (high cholesterol) .
Key Learnings & Takeaways:• Participants will gain an understanding of that whether a patient is becoming adherent or becoming non-adherent affects the magnitude of savings (or extra spending) experienced within the health system.
Dr. Steven Kymes is the Director of U.S. HEOR at Lundbeck, an international pharmaceutical company providing therapies for treatment of diseases of the central nervous system. He received his doctorate in health services research from the Saint Louis University School of Public Health in 2001. After graduation, he joined the faculty of the Washington University School of Medicine (St. Louis) where he was the founding director of the Center for Economic Evaluation in Medicine. He left academic life in 2012 to join CVS Health as an Executive Advisor in their Enterprise Analytics group. There he led development predictive metrics for targeting of pharmacy interventions and worked to improve estimates of the value of improved medication adherence. He left CVS in early 2016 to join Lundbeck.
Use This Link To Access The Archive:
Speaker: Dr. Lisa Sharp, University of Illinois at Chicago
Time: 6:30 pm – 8:30 pm Central Time
Location: University of Illinois at Chicago, College of Pharmacy, Room: PHARM 212/216
833 S. Wood Street, Chicago, IL 60604
JANUARY 2016 LUNCH-N-LEARN WEBINAR
, Partner, Access Market Intelligence, and The National Institute of Collaborative Care
“Market Access in US & Europe”
December 1, 2015 (Tuesday)
Speaker: Professor Mondher Toumi, University of Aix-Marseille & CEO at Creativ-ceutical
Time: 3:00–6:00 pm Central Time
Unlike marketing authorization for pharmaceuticals, mainly regulated at the European level by EMA, pricing and reimbursement decisions in Europe are managed by individual Member States. Health care services are generally covered by a single public health insurer operating under the Ministry of Health supervision. As a monopoly buyer, this situation provides a leading position for the public health insurer to set reimbursement conditions. Therefore, based on each country’s set of regulations, processes, and values, wide variations exist in pricing and reimbursement decisions of pharmaceuticals. Using up-to-date governmental regulation sources this course will discuss health technology decision-making processes for reimbursement decisions for pharmaceuticals in France, Germany, Hungary, Italy, Poland, Spain, Sweden, and the UK. The course will describe these reimbursement systems, as well as compare, and bring into contrast their key characteristics. We will along the whole course put the European findings in perspective with the US system.
Course learning objectives:
After the completion of this course, participants will be able to:
|President||Cynthia Holmes, MSP, PharmD|
|President-Elect||Gebra Cuyún Carter , MPH, PhD|
|Past President||Lesley-Ann Miller, MS, PhD, MBA|
|Secretary/Treasurer||Annette Champion, MBA|
|Director of Programming||Vanessa Perez, MS, PhD|
|Director of Communications||Nancy Kadish, MS|
Contact InformationISPOR Midwest Regional Chapter Office