Why Rare-Ultra Orphan Therapies need early Market Access planning and innovative solutions | Ascella Health

Success in launching new therapies for rare diseases (RDs) implies the ability for the manufacturer to achieve a level of reimbursed price and a level of market access that are commercially viable on the market. Access to RD therapies is challenging because the legal and policy frameworks may be absent, funding may be insufficient and/or payers do not see the justification with the prices for these therapies. The industry has, however, a real opportunity to partner with healthcare stakeholders.  There needs to be continued support to provide the education towards payers, drive evidence-based pricing, and innovative contracting, and address the multiple hurdles of access for these unique therapies. But how do we price and how do we gain access on the promise of long-term value when we don’t have the long-term data on these new therapies?  That is why all stakeholders need to address early market access planning, including inclusive data collection, payer models and the impact of new regulations and innovative trial design on data collection and ongoing evidence provision.             Podcast Participants: Jim Clement – Partner & Board Member at Coeus Holdings                                        Donovan Quill – Executive Vice President at Terebellum