Scientific advice is gaining momentum around the world and many processes are evolving
A shift in the regulatory/HTA paradigm is occurring, driven by affordability concerns, efficacy-effectiveness gaps, patient centricity, and early access to important medicines. The value of early integrated scientific advice is to support navigating this paradigm shift, manage uncertainty and de-risk, manage evidence complexity in HTA submissions, and promote company collaboration to eliminate the “silo effect”. The most important reason for seeking integrated scientific advice is to provide support for timely access to innovative medicines.
Transformational medicines may be made available to patients up to 4 years earlier through the Early Access to Medicines Scheme (EAMS) in the UK, Compassionate Access programs or expanded access studies. Obtaining this designation will require integrated early dialogue between the regulatory authorities, payers, and HTA agencies to support these new changes to align the regulatory and HTA bodies earlier.
Today, we’ll discuss the considerations from the regulatory, payer, and HTA perspectives with respect to the overlap and synergies in the consideration of the definition of population, study design and comparators, outcomes, and subgroups. The importance of the value proposition is emphasized in the development of a robust briefing book to provide a framework to develop “value added” questions for the agencies. Additionally, the importance of patient involvement in these early engagements will be discussed to support early alignment of all stakeholders to optimize the clinical development plan and identify potential gaps.
- Dr. Matthew Bending
- Rory Graham
- Manasee Shah
- Dr. Fanta Waterman
- Dave Clark
- Mike Pace
- Dr. Jonathan Watanabe
Dr. Matthew BendingDirector, Head of HTA, Strategy & Communication, Real World Strategy & Analytics, Mapi Group
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 or Real World Strategy & Analytics. Matthew has let the development of early payer engagement services in Mapi and has experience of more than 25 HTA scientific advice engagements across a range of diseases areas. Matthew provides senior leadership for large projects on strategic market access and Health Technology Assessments (HTAs) for pharmaceutical, vaccine and medical device companies.
Rory GrahamSenior Director, EU Regulatory Services, Mapi Group
Mr. Graham has facilitated numerous aspects of drug and medical device development during his 25 year career in the pharmaceutical, biotechnology and device industries. Mr. Graham has held senior positions in the area of regulatory a airs in companies in Europe and Asia-Pacific. He has arranged and directly participated in meetings with many global agencies including FDA, EMA, PMDA, TGA and European National Agencies. He has experience in various therapeutic areas including CNS, Oncology, Hematology, Immunology, Diabetes, Analgesia and Wound-Care. He has achieved orphan designations for pharmaceutical products in the EU, US and Australia. Mr. Graham leads a talented team of regulatory professionals that produce client-specific regulatory and development strategies, as well as solutions for global and local market needs.
Manasee ShahSenior Research Manager, Real World Strategy & Analytics, Mapi Group
Manasee V Shah, MPH, joined Mapi in 2016 and has extensive experience in the areas of health economics, outcomes research, and US market access issues. Prior to joining Mapi, she worked for a US-based market access consultancy, providing strategic advice regarding drugs in a number of therapeutic areas, including pain management, mental health, ophthalmology, and orphan indications. She has experience in conducting market research engagements, creating and implementing HEOR strategy plans, and designing and implementing real world studies. Ms. Shah holds a Bachelor of Arts degree in economics from Northwestern University and a Master of Public Health degree from the Department of Health Management and Policy of the School of Public Health at the University of Michigan where she is also a part-time lecturer.
Dr. Fanta WatermanIndustry Expert
Dr. Waterman completed an MPH and PhD in Public Health at Temple University and a 1-year postdoc with the University of Kentucky College of Public Health in Public Health Systems and Services Research, funded by the Robert Wood Johnson Foundation. Fanta taught as an adjunct Associate Professor within the City University of New York’s Community College System from 2008 until 2013. Fanta has more than 10 years of experience in Clinical Development, COA/PRO, HEOR and Global Market Access., including consulting with KantarHealth and increasing leadership positions in pharma with Pfizer, Genentech/Roche, Boehringer Ingelheim and Avanir.
Dave ClarkChief Development Officer, Ventegra Inc.
David Clark has been involved in managed care pharmacy and evidence base critical appraisal for more than 20 years. His currently the Chief Development Officer for Ventegra, Inc. His previous experience includes President, VisumRx, LLC, President, RegenceRx, Senior Vice President Health Care Services, Regence Blue Cross Blue Shield, and Director Pharmacy Services, Intermountain Health Care. During his career he has been honored to serve as President and Chair of the Board for the Academy of Managed Care Pharmacy and has been appointed to serve on two federal committees, the Effective Health Care Stakeholder Group, Agency for Agency for Healthcare Research and Quality (AHRQ) and the State Pharmaceutical Assistance Transition Commission, Department of Health and Human Services (HHS). He was also selected to serve as a member of the Blue Cross Blue Shield Association National Council of Physician Executives. He received both his Bachelor of Science Pharmacy degree and his Masters of Business Administration from the University of Utah in Salt Lake City, Utah.
Mike PaceSenior Principal, Pricing & Market Access, Commercialisation & Outcomes, ICON
Mike has more than 20 years of operational executive experience within global biopharmaceutical firms and digital health start-up ventures, leading market access strategy, payer and specialty pharmacy account management, contracting, business development, sales and operations functions. In these roles, he and his team launched and enabled market access for dozens of products in the US, across specialty, primary care and orphan conditions, including leading blockbuster brands in the areas of neurology, oncology, cardiology, allergy and ophthalmology. At EMD Serono, he pioneered the outcomes-based contracting effort that led to the first outcomes-based agreement on a specialty medication with a health plan, followed by the first outcomes-based agreement with a pharmacy benefit manager in the United States.
Dr. Jonathan WatanabeAssociate Professor of Clinical Pharmacy, Skaggs School of Pharmacy and Pharmaceutical Sciences, University of California, San Diego
Professor Jonathan Watanabe is a clinical consultant at the San Diego Program of All-inclusive Care for the Elderly (PACE) Clinic in San Ysidro, CA and the Villa Pomerado Skilled Nursing Facility in Poway, CA. He trains students, post-docs, and practitioners in multiple disciplines in clinical research and post-acute care. He is also an investigator on the Health Resources and Services Administration-funded San Diego Geriatrics Workforce Enhancement Program Grant. Dr. Watanabe was the inaugural recipient of the University of Washington/Allergan Health Economics and Outcomes Research Fellowship. He is a Board Certified Geriatric Pharmacist (BCGP).
• High level review of FDA Advice processes • Comparison to international regulatory advice processes • Potential for co-ordination with payer advice
• Discuss the value of engaging stakeholders to obtain early scientific advice with a focus on de-risking clinical development programs • Compare and contrast scientific advice engagements in the EU (formal) versus US (informal) and how formal advice can be utilized in the US • Share how early multi-stakeholder advice can increase patient access
• Cross-functional collaboration between Medical, Market Access, Clinical Development and Regulatory departments to develop efficient and strategic programs for regulatory approval and commercialization • Executing US clinical programs with a global value proposition in mind
• Understand the issues facing payers when making coverage decisions • Discuss how payers may make different coverage decisions based on the same evidence • Share how evidence documenting value might increase early patient access
• Discover how informing innovative contracting through early engagement with key stakeholders benefits commercial market entry and outcomes • Learn why traditional contracting approaches and post-marketing payer engagement may not be enough to address the needs of both payers and pharma manufacturers • Hear about current trends and anticipated innovations, including the use of outcomes-based agreements • Understand the key contracting issues manufacturers should be considering today to optimise the commercial product success.