Why Rare Disease Therapeutics Need Early Market Access Planning

When developing and bringing a therapeutic for a rare disease to market, sponsors should think with the end in mind and plan for payer requirements early in protocol design. The economic and clinical value evidence required by payers for market access differs from the clinical efficacy and safety evidence demanded by regulators. Securing approval but lacking market access results in unrealized revenue and, most importantly, patients left untreated. 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?

In this podcast by Blog Talk Radio, Sangeeta Budhia, VP and Global Head, Pricing and Market Access, and Wyatt Gotbetter, Worldwide Head, Access Consulting, of Parexel discuss 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.