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

Medicine Access @ Point of Care

FrançoisLucas¹ 

关键词: Rare disease;    orphan drug;    managed entry agreement;    innovative contracting;    market access;    emerging markets;    middle-income countries;   

DOI  :  10.1177/2399202618810121

学科分类：医学（综合）

来源: Sage Journals

【 摘 要 】

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.

【 授权许可】

CC BY-NC