Adaptive Pathways – Applications for Scientific Insights in Europe
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For the past five to ten years we have witnessed a heightened interest in improving timely access for patients to new medicines. On the one hand, the European Medicines Agency (EMA) started introducing regulatory processes aiming at cutting down lead-time for marketing authorization in indications with high unmet medical needs with the introduction of initiatives such as the Compassionate Use Programs (based on Regulation (EC) No 726/2004), the Conditional Approval Mechanism (based on Regulation (EC) No 507/2006), and the Initiative for Patient Registries launched in September 2015. On the other hand, the United States Food and Drug Administration (FDA) developed four approaches, which were formalized with the adoption of FDA Safety Innovations Act in 2012, known as Priority Review, Breakthrough Therapy, Accelerated Approval, and Fast Track.
Now, the paradigm is rapidly shifting as last year this trend found culminating expressions on both sides of the Ocean when Europe launched the adaptive pathways (AP) approach and the US passed the 21st Century Cures Act. There are elements of similarities and contrasts between the two but the endeavors go in the same direction and require similar efforts from all counterparts; that is:
- a concerted approach during the complete life-cycle of the products (from clinical development to market adoption),
- the need to complement trial data with real-world evidence,
- and the acknowledgement of patients’ centeredness.
Focusing on Europe, according to EMA adaptive pathways is a scientific concept for medicine development and data generation which allows for early and progressive patient access to a medicine and it is based on three principles:
- early involvement of patients and health-technology-assessment bodies in discussions on a medicine’s development;
- gathering evidence through real-life use to supplement clinical trial data; and
- iterative development, which either means:
- approval in stages, beginning with a restricted patient population then expanding to wider patient populations;
- confirming the benefit-risk balance of a product, following a conditional approval based on early data (using surrogate endpoints) considered predictive of important clinical outcomes.
- Prof. Michael Drummond
- Dr. Matthew Bending
- Dr. Will Maier
- Mia Malmenas
- Nahila Justo
- Joakim Ramsberg
- Karolina Antonov
Prof. Michael Drummond
Michael Drummond is Professor of Health Economics and former Director of the Centre for Health Economics at the University of York and part of the team from the University of York that produces independent Technology Assessment Reports for NICE. Professor Drummond has authored three major textbooks and co-authored more than 400 publications in peer-reviewed scientific journals. Professor Drummond has consulted for the World Health Organization, the European Union, and numerous major pharmaceutical companies. He has served the Medicines Commission in the United Kingdom, the Board of the International Society of Technology Assessment in Health Care (ISTAHC) and the Board of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR).
Dr. Matthew Bending
Dr. Matthew Bending is an experienced health economist with over 12 years consultancy experience and is Head of the HTA, Strategy and Communications team and Director of Real World Strategy & Analytics. Matthew has led the development of early payer engagement services in Mapi and has experience of more than 25 HTA scientific advice engagements across a range of disease areas. Matthew provides senior leadership for large projects on strategic market access and Health Technology Assessments (HTAs) for pharmaceutical, vaccine, and medical device companies.
Dr. Will Maier
Mapi's Chief Scientific Officer, Dr. Will Maier provides scientific leadership to Mapi's research and consulting services across a range of scientific areas. Dr. Maier has more than 20 years of drug development and commercialization experience. He served as Senior Director of Epidemiology at GlaxoSmithKline and Elan Pharmaceuticals, and led research groups conducting observational research to support reimbursement, marketing, and drug safety investigations of pharmaceuticals. Dr. Maier is a member of the EMA's European Network of Centres for Pharmacoepidemiology and Pharmacovigilance.
Mia Malmenäs has over 17 years of experience form the pharmaceutical industry analyzing and designing clinical trials, retrospective data sources and observational studies, and validation of Patient Reported Outcomes/Clinical Outcome Assessments. She has published over 10 journal articles and been an author on more than 15 posters. Her publications reflect her research interests in retrospective data analysis as well as study design methodology. Currently, Mia is co-chairing one work stream in the ISPOR Medication Adherence and Persistence Special Interest Group and she has chaired 3 workshops and led one course within the same organization.
Nahila Justo is a PhD candidate at the Karolinska Institute and has a MBA from the Stockholm School of Economics (Sweden), an MPhil in European Affairs from the University of Salamanca (Spain) and a MSc in Economics from the Inter-American Development Bank and the Di Tella Institute (Argentina). Nahila also has undertaken post-graduate courses in Epidemiology and Biostatistics at the IECS Argentina (Instituto de Efectividad Clínica y Sanitaria) and holds a MA in International Relations and a BA in Political Science from National University of Rosario, Argentina. She currently serves as Scientific Director at Mapi Real World Evidence Strategy and Analytics (formerly OptumInsight HEOR).
Joakim Ramsberg, Ph.D., is Principal Secretary in the Government commission on Financing, reimbursement and pricing of pharmaceuticals. He is project director and chief scientific officer at Vårdanalys (The Swedish Agency for Health and Care Services Analysis), where he has lead projects that e.g., evaluate national clinical guidelines, examine disease registries, and assess government reforms to increase access and strengthen psychiatric care. He is a lecturer at the Department of Population Medicine, Harvard Pilgrim Healthcare Institute and Harvard Medical School where he also was the 2015-16 Swedish Harkness Fellow in Health Care Policy and Practice.
Karolina Antonov is Head of Strategy at Läkemedelsindustriföreningen (LIF), the trade association for the research-based pharmaceutical industry in Sweden. With about 85 members and associate companies, LIF represents approximately 80 percent of the total sales of pharmaceuticals in Sweden. LIF’s primary responsibilities are pricing and reimbursement issues, but is involved in many of the activities within pharmaceutical strategy such as managing introductions, adapting pathways, and RED issues. LIF is also a member of the European trade association EFPIA, which works to improve access to medicines for all patients in Sweden.
Challenges and experiences of national implementation of AP
How can secondary data such as Nordic and European registries support AP discussing challenges and opportunities
Discussing both dimensions, the early involvement of patient representatives as an interlocutor in the assessment and the enhanced use of PROs
The adaptive pathway development should be iterative and as such plans for demonstration and change of the value within the evolving data set can/needs be discussed with HTAs. What is the role of integrated scientific advice in adaptive pathways and the key HTA questions and issues considered?
Presentation and discussion of trends that operated in the background and brought this shift, the overall policy in context, comparison with 21st Century Cures and global trends, additional changes we can expect
Consequences downstream: the payer perspective