How Can We Shape HTA with Real-World Evidence to Encourage Rare Disease Innovation?

Speaker(s)

Moderator: Josie Godfrey, MA, JG Zebra Consulting, Hammersmith, UK
Speakers: Pall Jonsson, BSc, PhD, Data and Analytics, National Institute for Health and Care Excellence (NICE), Manchester, LAN, UK; Elena Nicod, PhD, Dolon Ltd, London, UK; Mark Larkin, PhD, MA, Vitaccess Ltd, Oxford, UK; Anthony Hatswell, PhD, Delta Hat Ltd, Nottingham, UK

Presentation Documents

Rare diseases pose unique challenges in health technology assessment (HTA), where stakeholders must make decisions in the face of greater uncertainty in the evidence base than is seen in more prevalent diseases. Health-care systems must address the large unmet need in rare disease patient populations while maintaining equity of access for them; however, challenges in terms of data, methodology and process implementation remain. Real-world data is a key source of evidence to reduce this uncertainty, complementing limited clinical trial data. Evidence derived from real-world data on natural history, comparative effects, resource use, quality of life etc. may be able to answer questions which are crucial for decision making. In recent years, certain HTA agencies have been active in trying to incorporate real-world evidence (RWE) and adjustments for rare diseases into their assessments. Advances have been made in methods and process guidelines, but have these led to meaningful changes in wider and faster access for patients? Are further theoretical advances required, or are there other, practical barriers to implementation? In which areas could RWE be transformative, and where will other solutions be required? How will the upcoming EU Joint Clinical Assessment process approach this issue? This symposium includes speakers with deep experience in a variety of aspects of HTA, who will discuss the current status of appraisal frameworks for rare disease treatments, how far RWE can go to resolve some of the uncertainties faced by HTA decision makers, and what the future may hold.

Sponsored by Biogen

Code

136

Topic

Health Technology Assessment