Finding a development path for promising drugs
The Milken Institute is out with a new report pitching a path forward for medicines that could offer hope to patients but have been shelved for business reasons.
“Drugs that can improve people’s lives should not be sitting on drug company shelves,” wrote the institute’s researchers in the report, titled, “Creating a Nonprofit Marketplace for Shelved Drugs: Lessons from a Pilot Project.” “Finding a development path for promising drugs, regardless of their commercial potential, is simply the right thing to do for society.”
The U.S. National Institutes of Health (NIH) estimates (PDF) 80% of all drugs that enter human testing are never approved for use. Weak efficacy or safety concerns are not the only reasons Big Pharmas ditch drugs along the way. Assets can also be shelved simply because the company does not see a profitable path forward—pharma is a business, after all.
But that means patients who could benefit continue to wait for new treatments. Milken is proposing a way to move these promising but not overly profitable therapies over to advocacy groups and other nonprofits that can serve as “matchmakers” to connect discontinued drug assets and capital sources.
This model has worked before—and sometimes major companies don’t need a lot of prodding, either. Earlier this month, Moderna, awash with cash from its COVID-19 vaccine, donated a rare disease therapy for Crigler-Najjar syndrome Type 1, an extremely rare condition that can lead to brain damage, to the Institute for Life Changing Medicines.
The NIH has a program to pick up discontinued drug products through the National Center for Advancing Translational Sciences called the New Therapeutic Uses program, which has helped support continued development of a handful of therapies for new indications in eight therapeutic areas.
Partners include Johnson & Johnson’s Janssen pharmaceutical unit, AstraZeneca, Pfizer and Sanofi, which had collectively made 26 assets available, according to a 2015 fact sheet. The companies donated materials for treatments in Type 2 diabetes, acute myeloid leukemia, the aggressive brain tumor glioblastoma and Chagas disease.
The companies provided the drug product at no cost as well as early human safety data that allowed the agency to advance further testing quickly. This pilot program confirmed the feasibility of repurposing discontinued therapies.
Nonprofits have also been involved in separate approaches. The Children’s Tumor Foundation and CureSearch for Children’s Cancer joined with The Milken Institute in 2019 to develop a new nonprofit marketplace for discontinued drugs called the Bridge initiative. The marketplace helps bring together patient advocacy groups and nonprofit organizations that can activate their networks of industry players and investors to develop discarded therapies.
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SOURCE: Fierce Biotech
How to rescue Big Pharma drug castoffs from the scrap bin through nonprofit partnerships: report
Sep 27, 2021 8:00am