An Ethical Option for Shelved Drugs

A key priority in today’s investment world is corporate adherence to environmental, social, and governance (ESG) requirements. A company’s ESG score serves as a marker of the organization’s values and as a disclosure mechanism for investors to consider. Many companies now consider ESG scores when making strategic choices, and my group has identified a tangible option.

In 2019, the Children’s Tumor Foundation and CureSearch for Children’s Cancer, launched the Bridge initiative (1) in partnership with FasterCures, a division of the Milken Institute. Bridge is a neutral information and matchmaking forum for connecting shelved drug assets (SDAs) with partners that have the expertise and capital needed to develop those medicines.

New Life for Shelved Drug Assets
SDAs are promising medicines that have strong phase 1 safety data and pharmaceutical characteristics but have been deprioritized and “shelved” by their sponsor organizations for under five years for non-technical/scientific reasons. Those can include corporate strategy, reorganization, and commercial or marketing issues. Oncology drug assets also might be shelved because they fail to reach endpoints that are required for regulatory approval of malignant-cancer treatments.

The Bridge team believes that all relevant SDAs for pediatric and/or rare diseases should be repositioned for pediatric and/or rare diseases. Despite efforts from governments around the world — and incentives such as the Orphan Drug Act and the rare pediatric voucher program from the US Food and Drug Administration (FDA) — rare diseases and pediatric cancers continue to suffer from a lack of industry attention. Among other concerns, companies cite difficulties in patient recruitment, stringent toxicology requirements, and (more specific to rare diseases) a lack of well-defined, standardized clinical-trial endpoints.

The Bridge team is taking essential but challenging steps to build partnerships for development and commercialization of SDAs to treat pediatric and rare tumors. This nonprofit initiative is approaching biopharmaceutical companies, helping to identify promising SDAs, developing an externalization framework, and matching validated drugs to new development and investment partners.

“FasterCures is thrilled to [help] create a path to development for promising drugs for pediatric tumors,” says Esther Krofah, executive director of FasterCures. “Through this partnership, we are evaluating the potential for a nonprofit approach to accessing and revitalizing discontinued drugs.”

“When drug programs are discontinued or not pursued for business reasons, there can be missed opportunities for children,” says Kay Koehler, CureSearch president and chief executive officer. “This innovative project enables us to offer biopharmaceutical companies an alternative, pediatric-focused path forward for those discontinued drugs.”

The Children’s Tumor Foundation is proud to partner with FasterCures and CureSearch in expanding a collaborative network for accelerating treatments to patients. The recent FDA approval of the first-ever treatment for the genetic disorder neurofibromatosis (NF), AstraZeneca’s Koselugo (selumetinib), shows what is possible through a united effort of all stakeholders: companies, foundations, and patients.

Volunteer advisors to the Bridge team include scientific, clinical, and business experts who believe in the opportunity to make a true impact for patients: D. Wade Clapp (Indiana University School of Medicine and Riley Hospital for Children), Elizabeth Fox (St. Jude Children’s Research Hospital), Kevin Grimes (Stanford University School of Medicine), Chris Lee (Palladium), Freda Lewis-Hall (formerly of Pfizer), Baiju Shah (FasterCures), Kathy Wanner (CureSearch and Fairway Capital Management), Brigitte Widemann (National Cancer Institute), Andrew Kung (Memorial Sloan Kettering Cancer Center), and Craig Thomas (National Center for Advancing Translational Sciences).

What It Means for ESG
FasterCures published a paper in 2021 that summarizes what has been done so far, including lessons learned (2). Together with scientific advisors, we have assembled a list of molecular targets that are relevant for pediatric cancers and NF. If your biopharmaceutical company is willing to explore opportunities in these areas, please contact us.

For the investment community, we are asking bold and innovative risk-takers to join us and invest in valuable shelved drug assets. And with financial analysts, we want to explore an opportunity to incentivize the biopharmaceutical industry to collaborate with Bridge. We advocate introducing an ESG measure that reflects the number of valuable but discontinued medicines for high unmet medical needs released by each company in the sector. The higher that number is, the better the overall ESG score would be, and thus the more attractive a company might be to responsible investors.

References
1 BRIDGE Initiative: Creating Pathways to Innovation. The Milken Institute: Santa Monica, CA; https://milkeninstitute.org/programs/bridge-initiative.

2 Choe SH, et al. Creating a Nonprofit Marketplace for Shelved Drugs: Lessons from a Pilot Project. The Milken Institute: Santa Monica, CA, 2021; https://milkeninstitute.org/report/shelved-drugs-nonprofit-marketplace.

Annette Bakker, PhD, is president of the Children’s Tumor Foundation, 697 Third Avenue, Suite 418, New York, NY 10017; 1-212-344-6633; https://www.ctf.org.