Co-Founder/President, Friedreich's Ataxia Research Alliance, (FARA) P.O. Box 1537, Springfield, VA 22151, USA.
Curr Top Med Chem. 2014;14(3):313-8. doi: 10.2174/1568026613666131127154903.
The business model for medical therapy development has changed drastically. Large companies that once conducted their own Research and Development (R&D) and funded all the preclinical studies, all phases of clinical development and marketing of the products are increasingly turning to others for more and more of the earlier work in hopes of being able to in-license a de-risked program well downstream, take it through the final phases of clinical development and into the marketplace. This new paradigm has required patient-advocacy foundations, especially in the rare-disease space, to become far more effective in building relationships with all the players along the therapy-development pathway -- academic scientists, government agencies, other foundations with overlapping interests, biotechs, small biopharmaceutical entities and even the larger industry companies. From the perspective of the patient-advocacy community, these increasingly essential public-private partnerships have taken on the nature of what could be called joint-venture philanthropy and involve a broad spectrum of collaborations and financial relationships between foundations and industry partners that are not without concerns about potential conflicts of interest.
医学治疗开发的商业模式已经发生了巨大变化。曾经自行进行研发并为所有临床前研究、所有临床开发阶段以及产品营销提供资金的大型公司,越来越多地将更多的早期工作外包给其他公司,希望能够获得风险降低的项目,并将其推进到临床开发的最后阶段并推向市场。这种新模式要求患者倡导基金会,尤其是在罕见病领域,与治疗开发路径上的所有参与者建立更有效的关系——学术科学家、政府机构、具有重叠利益的其他基金会、生物技术公司、小型生物制药实体甚至更大的行业公司。从患者倡导团体的角度来看,这些日益重要的公私合作伙伴关系具有所谓的合资慈善事业的性质,涉及基金会和行业合作伙伴之间广泛的合作和财务关系,这些关系并非没有潜在利益冲突的担忧。