Improving market access to rare disease therapies: A worldwide perspective with recommendations to the industry

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 global market. Access to RD therapies is challenging in many countries 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 systems to address these issues, for example, through education towards payers, responsible and evidence-based pricing, and innovative contracting. Such support is particularly needed in middle-income and emerging markets, where it will contribute to growth in RD therapy coverage.