Philanthropy’s Role in Catalyzing New Uses for Approved Treatments

Abstract

Foundations, nonprofits, non-governmental organizations, and other philanthropic organizations are frequently involved in supporting drug development. Cures Within Reach, a US-based nonprofit leader of testing already approved therapies for new indications in unsolved diseases, has been providing seed funds for first-in-human clinical trials for over 15 years. Philanthropy plays a critical role in risk mitigation of investigator-initiated trials, funding proof-of-concept, pivotal clinical trials as well as later-stage trials. Efforts such as these are complementary to the pharmaceutical industry’s portfolio life cycle management and complementary to governments’ funding of translational medicine initiatives. Results from smaller, earlier trials provide the data needed for industry, government agencies, and others to fund the larger confirmatory trials. Together, these data build evidence for publication, allow clinicians to make their own decisions with their patients (off-label use), or support regulatory approval by the regulatory agencies. In addition, these efforts are not a question of commercial opportunity via intellectual property versus charity, as these are not mutually exclusive—and often are not yet clearly known at earlier clinical trial stages.