Washington has pledged to boost biomedical research funding, and that's good news for patients. But lawmakers should also be working to maximize the bang for their research buck.
Federal agencies have increasingly realized that an excellent way to do so is by working closely with the private sector research foundations that best know the disease of interest -- and the journeys patients face.
Health nonprofits have long been expert advocates for their patient membership. They still are. But more recently, patient-centered research foundations have been moving toward a more active role in medical research. Staffed with former R&D veterans, they are increasingly devoting resources to facilitating progress from initial discovery to clinical benefits for patients.
They provide seed and bridge funding, forge collaborations, and invest their expertise to accelerate approvals. By testing nascent therapies before taxpayers begin funding them, these nonprofits also help ensure that public research dollars get spent more efficiently and with less risk.
Most importantly, these public-private partnerships provide a model for how Washington can work directly with the nation's top disease experts to spur breakthrough medical research -- which could save the lives of millions of patients in years to come.
Bringing foundations into the research and development ecosystem has delivered outsized results. My organization partners with a Defense Department research fund -- Congressionally Directed Medical Research Programs -- to research neurofibromatosis and schwannomatosis, a group of rare genetic disorders that cause tumors to grow on nerves throughout the body. This work also provides clinical insights for the military on phantom limb pain and how wounds heal.
Our public-private partnerships led to the first approved therapy for NF after decades of limited progress -- an accomplishment that likely would have been impossible absent such collaboration. In a more recent CDMRP-vetted partnership project, CTF provided $2 million in funding to researchers developing a blood test that predicts the risk of malignant tumors in certain patients with NF.
This approach is by no means exclusive to our foundation. Other nonprofits are also building public-private partnerships. Cold Spring Harbor Laboratory, for instance, has collaborated with government partners on research to determine molecular changes that cause ovarian and breast cancers.
Private-public partnerships also play a critical role in developing therapies for rare diseases. Although roughly 10% of Americans live with one, around nine in 10 rare diseases have no approved medical treatment. Some nonprofits have embraced a strategy of "venture philanthropy" to support researchers pursuing riskier novel approaches. Nonprofits like the American Epilepsy Society are seed-funding small grants to help researchers get started and compete for larger grants.
At CTF, our seed grants have helped researchers obtain at least $38 million in follow-up funding. Collectively, nonprofits now make up the fourth-largest source of biomedical funding, contributing $3.8 billion annually.
But funding is just one part of the solution. Rare disease research can be stymied by small patient populations with multi-organ challenges and progressive disease manifestations. Clinical trials are often difficult to design and execute. Tissue and data silos sometimes prevent scientists from building on past work.
Foundations have responded by creating centralized clinical databases where researchers can quickly recruit trial participants and share their data in real time. The Multiple Myeloma Research Foundation, for example, tracked treatment and outcome data for about 1,000 myeloma patients over eight years. Grants through foundations and federal agencies are often conditional on participation in such databases.
Foundations are also encouraging greater scientific collaboration through "platform" trials, in which researchers collaborate to test multiple therapies for efficacy against each other at the same time. A platform trial from the Leukemia & Lymphoma Society helped sequence genomes for over 1,400 blood cancer patients to pinpoint the best treatment for each patient.
This model shows promise on an international scale. A unique public-private partnership between Europe's pharmaceutical trade association and the European Union is funding its own set of groundbreaking platform trials. One such trial on neurofibromatosis aims to spur new treatments and chart the course of NF over time.
In some cases, foundations are also investing in providing pharmaceutical companies with a specialized service offering disease expertise and easy connections to clinic networks, preclinical hubs, platform trials, and the like.
As Congress discusses its 2025 budget, lawmakers must not forget how effective public-private partnerships have been at leveraging donor and taxpayer dollars. Just as government functions best when lawmakers work together across the political aisle, science progresses faster when the public and private sectors can pool resources, share knowledge, and collaborate on developing revolutionary treatments.
Annette Bakker is president of the Children's Tumor Foundation.
