PRO103 The IMPACT of COVID-19 on the Future Pricing and Market Access of RARE Diseases and CELL-/GENE-Therapies

Objectives: To explore the long-term impact of Covid-19 on the pricing, market access, and payer evidence requirements in rare diseases and cell/gene-therapies Methods: A survey-based Covid-19 payer impact tracker covering US and EU (n= 80 EU, 25 US) identified factors surrounding rare diseases and cell/gene-therapies that are currently, and likely in the future to be, “payer-disruptive” Results: The Covid-19 Payer tracker indicated that, in both US and EU5: • The economic impact of the Corona virus is expected to be the biggest single driver / threat to the healthcare system • Lower impact on rare diseases than on more common disease areas due to there being usually no therapeutic choice • Higher impact on the pricing and market access of cell and gene therapies since the current generation of these: • Are perceived as expensive • Have significant uncertainty around durability / duration of effect • Impact will increase as cell and gene therapy moves to more mainstream diseases where the total budget impact will be much higher and the likelihood of there being (cheaper) therapeutic alternatives may be higher • Surrogate endpoints will increasingly come under-challenge Evidence will be required of link between surrogate end-points, effect size (relative to other often cheaper therapeutic options), and importantly (short-term) costs (drug, healthcare system, and patient costs) • Economic considerations may drive cell-/gene- therapies to be moved later in treatment algorithm because of both upfront cost/expense and long-term durability of effect uncertainty • Time to outcome increasingly important as is avoidance of short term-costs • New assessment and reimbursement models expected including regular (annual) reviews of data and modification of coverage and cost agreements / contracts Conclusions: The long-term economic impact of Covid-19, and affordability considerations, may affect the pricing, market access, positioning, and uptake of cell- and gene-therapies The magnitude of effect will depend on the speed and timing of economic recovery