Parallel Session 6: How to pay for universal access to medicines for rare diseases and by whom? The great conundrum

The dramatic rise in the number of medicines receiving regulatory approval with a rare disease / orphan designation is both encouraging and worrying. It is encouraging because it shows that scientific advances, government policies and market conditions have collectively stimulated the research and development of new therapies to address previously neglected health needs. It is also worrying because introducing these medicines to the health systems at prices at least in the hundreds of thousands has already resulted in deep financial impacts, even for the wealthiest countries. With diverse disease profiles and a collective 300 million people living with rare diseases globally, the sector is broad. Providing universal coverage for even a fraction of the medicines with well-established efficacy and safety to people across countries of all incomes would be tremendous, if not impossible. It is also important to recognize the significant skills and infrastructure investments required to integrate these new medicines into health systems. Finally, the exorbitant pricing of rare disease medicines has a human cost as it causes significant psychological distress to patients and families, as well as to decision makers who are constrained by financial resources available to them but unable to meet the demand. In this session, the panel will discuss questions such as whether the market has been over-incentivised, whether universal coverage would be realistic under the status quo, and what it would take to better prepare health systems and policies to meet the pace of development and public expectations for access to medicines for the treatment of rare diseases.