Scientific advice is gaining momentum around the world and many processes are evolving
Mapi, now a member of ICON, offers Integrated Scientific Advice (ISA) workshops and consulting services to support manufacturers in optimizing their product development programs to generate evidence that is relevant to regulators, HTA bodies, and payers for timely patient access.
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
- Dr. Amina Udechuku
- Dr. Sabine Latour
- Dr. Jacoline Bouvy
- Guy Sherwin
Dr. Matthew Bending, PhD, MSc, BSc Econ.Director, 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 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 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 Graham, BPharm MBASenior 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.
Dr. Amina Udechuku, PhD, BSc.Senior Research Manager, Real World Strategy & Analytics, Mapi Group
Dr. Udechuku is an evidence-based pricing and market access consultant with over 9 years of expertise in HTA engagements in both healthcare academia and strategic consulting. Amina has experience with a wide variety of health economics and outcome research (HEOR) projects for pharmaceutical, vaccine, and medical device companies. Amina specialises in Health Technology Assessment (HTA) submissions, integrated scientific advice engagements with HTA and regulatory agencies, and value communication strategy and methods. Amina provides leadership on projects in antimicrobials, oncology, neurological disorders, mental health disorders, cardiology and various orphan diseases.
Dr. Sabine Latour, MDGlobal Market Access Director, Debiopharm
Dr. Sabine Latour is a physician with diverse pharmaceutical experience through her work for major pharma, medical device companies, and start-ups. Specializing in value identiﬁcation, as well as clinical and economic evidence generation, Dr. Latour earned her medical degree and completed courses in Pharmaceutical Medicine and Health Economics. She leads international, strategic discussions with regulatory and reimbursement authorities, and payer bodies, both individually, with country HTA entities (GBA, NICE, TLV, AIFA, HAS), and also through joint scientiﬁc council with EMA and HTA entities
Dr. Jacoline Bouvy, PhDScientific Adviser, NICE
Dr. Jacoline Bouvy is a Health Economist specialising in the interface between marketing authorisation and health technology assessment (HTA) of medicines. She works at the National Institute for Health and Care Excellence (NICE) in London within the Science, Policy & Research team for several research projects on topics such as adaptive pathways and big data for better outcomes in Alzheimer’s disease. Before joining NICE, Jacoline worked at the European Medicines Agency where she was involved in the EMA registries initiative. Before that, she held postdoctoral positions at Erasmus University Rotterdam and Utrecht University in the Netherlands where she worked on various drug regulatory science and health economics topics.
Guy Sherwin, BSc. HonsPrincipal, EU Pricing and Market Access, ICON Commercialisation & Outcomes
Guy Sherwin is Principal Consultant on EU Pricing and Market Access for ICON Commercialisation & Outcomes. He has spent over six years advising on global pricing, market access and commercialisation strategy, specializing in early access programs and managed entry agreements to support market access. Mr. Sherwin consults with clients across a wide variety of therapeutic areas including cardiovascular disease, oncology, metabolic disorders, ophthalmology and orphan diseases.
Welcome and value of multi-stakeholder scientific advice
- Welcome from the ICON/Mapi team
- A high-level introduction to the value of multi-stakeholder scientific advice
- An overview of the workshop agenda and objectives
The evolution of the regulatory scientific advice landscape
- Overview of European regulatory scientific advice procedures
- The role of enhanced scientific advice within the EMA PRIME initiative
- A review of the EMA Adaptive Pathways process from a regulatory perspective and the role of wider stakeholder advice opportunities in the new drug development paradigm
Overview of integrated HTA and regulatory processes and key considerations
- What is integrated scientific advice?
- What are the key HTA scientific advice processes?
- What are the key HTA considerations in scientific advice?
- Current and future trends in integrated scientific advice
Adaptive Pathways: Key considerations from a NICE perspective
- NICE have been a participant in the EMA adaptive pathways pilot and a partner/work package lead in the innovative Medicines Initiative (IMI) funded ADAPT-SMART project (launched in 2015) which is a multi-stakeholder platform for coordinating adaptive pathways activities in Europe. This session will discuss:
- Early dialogue with HTA bodies and use of real-world evidence to supplement RCT data are key components of adaptive pathways but how might the concept work in practice?
- Post-launch evidence generation is foreseen to reduce uncertainty, but how ready are Europe's healthcare systems to utilise additional evidence generation for outcomes-based managed entry agreements>?
Outcomes-based agreements and innovative contracting for biopharmaceuticals
- Evolution in pharma contracting approaches to address the increasing challenges in reimbursement of new products
- Current trends and anticipated innovations, including the use of outcomes-based agreements
- Key considerations to be incorporated in a structured approach for designing innovative contracting agreements
Industry learnings from seeking integrated scientific advice ro reimbursement and timely patient access
- The importance of early engagement with HTA bodies and payers from an industry perspective
- Overcoming internal hurdles within the company: achieving marketing authorisation is not a license to sell
- PRIME, adaptive pathways, and orphan drug status: what are the implications for reimbursement?
- Key differences between US and Europe and how to address these differences
Roundtable discussion panel on multi-stakeholder integrated scientific advice
- What factors influence the company decision to undertake an adaptive pathways approach with enhanced early multi-stakeholder dialogue? What are the implications for pricing, reimbursement and patient access?