How to rescue Big Pharma drug castoffs from the scrap bin through nonprofit partnerships: report

If a potentially promising treatment sits idle in a Big Pharma's stockroom, shouldn’t patients have access to it?

That’s a question asked by nonprofit think tank The Milken Institute, which is out with a new report Friday 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.

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Companies review their pipelines routinely and place therapies on the scrapheap when they do not expect a huge return on investment or when the likelihood of success seems slim. These are difficult decisions, typically coming to light as Big Pharmas report earnings each quarter.

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.

Milken says that “in an ideal world,” companies would offer up these candidates for out-licensing, donation or divestiture to new owners that could move them along. This isn’t so easy, because of “significant informational, operational, and cultural barriers within drug companies [that] can undermine even the best-intentioned efforts,” the report said.

“Prominent among these barriers is a deeply rooted cultural mindset to bring assets in and not let assets out,” Milken said.

There’s also a challenge in offering up resources to catalogue the data needed to package up a therapy for transfer. And while there’s plenty of money to go around in the biopharma universe—from angel investors, venture capital, nonprofit organizations and more—many of these parties are not familiar with one another, which hinders dealmaking.

RELATED: NIH backs pharma giants and academics' projects to revive R&D castoffs

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.

Scientist Susannah Huth works at the National Center for Advancing Translational Sciences' Medicinal Chemistry Lab. (Daniel Soñé Photography/National Institutes of Health) 

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.

In one example, the Bridge initiative was approached in 2020 by one of the top 20 pharmaceutical companies with an oncology asset that had been shelved for strategic reasons. The unnamed company had tried but failed to out-license the asset, so a nonprofit approach was next. The parties signed a confidentiality agreement, with the company providing a “data room” loaded with information on the potential therapy.

“From the company’s perspective, a challenge was that only one individual possessed detailed knowledge of the drug’s development program,” Milken said. “It was essential to the company that we minimize the burden on that person’s time.”

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The Bridge initiative developed two possible tracks for the medicine: the “donation model,” where the title for the asset would transfer to the Bridge initiative to shop around to investors; and the “consignment model,” where the company would keep the title while the Bridge folks acted as an intermediary to connect interested investors.

The report did not specify which option the company chose but said a number of lessons have been learned already. They've discovered that a “C-suite champion” from the company is a must to ensure there’s enough institutional support to move a shelved therapy into the hands of new investors.

“We had promising discussions with individuals at multiple drug companies, but this enthusiasm often did not carry over to the level of the C-suite. Without a C-suite champion, the barriers may be too high to overcome,” the report said.

Companies with a commitment to corporate responsibility and ESG have been particularly interested in the Bridge initiative. But before drugmakers are willing to give a therapy away, they tend to exhaust all out-licensing possibilities, according to the Milken report.

“To ensure that all good science enters development, industry, investors, and nonprofits must continue to work synergistically in experimenting with myriad approaches to support the financing and development of discontinued assets,” the report concluded. “Patient-focused organizations understand the barriers to externalizing discontinued assets and are eager to work with companies to navigate these obstacles.”