From Discovery to Access: How Innovation and Affordability Work Together

During Rare Disease Week, the Senate Special Committee on Aging held a hearing focused on the urgent need to strengthen the United States biomedical innovation ecosystem to benefit patients.

Lawmakers, advocates and industry leaders underscored a shared reality: for individuals living with rare and ultra-rare diseases, innovation represents both hope and the possibility of future access to treatment.

This moment calls attention to the scale of unmet medical need and to the policy decisions that determine whether future therapies will be discovered, developed and ultimately made affordable.

Ranking Member Kirsten Gillibrand (D-NY) spoke to how the FDA’s current approach is failing patients with rare diseases - pointing to hesitation within the agency, limited communication among stakeholders, insufficient use of patient experience in real‑world evidence reviews and last‑minute shifts in trial design expectations.

She cautioned that without greater consistency and predictability, drug sponsors will continue to struggle to secure FDA approval and may move their clinical trials abroad, including to China.

Also at the hearing, Bradley Campbell, President and CEO of Amicus Therapeutics, noted that the United States is not fully keeping pace with its scientific potential. He stressed that Congress has an important opportunity to strengthen policies that support the long-term, high-risk research essential to rare disease innovation.

His remarks underscore a broader point: to understand how patients gain access to therapies, it is necessary to look at the entire lifecycle of medical innovation.

Discovery: Investing in Uncertain Science

Every therapy begins with basic research and early experimentation. In rare diseases, where patient populations are small and scientific understanding may be limited, the scientific and financial risks are especially high. Advancing a scientific hypothesis into a viable treatment requires sustained funding and a willingness to navigate uncertainty.

Intellectual property protection provides the stability needed to attract investment from researchers, universities and biotechnology companies. Without a reliable framework, fewer high-risk ideas would advance, and many potential breakthroughs would remain unexplored.

Development: Sustaining Progress Through Complexity

Turning a discovery into an approved medicine is a long and resource-intensive process. Clinical trials must be carefully designed, regulatory standards must be met and scientific setbacks are common.

Predictable intellectual property protections help companies raise capital, form strategic partnerships and commit to multi-year research programs. This continuity is especially important for smaller biotechnology firms that drive innovation in rare and ultra-rare conditions.

Market Entry, Follow on Innovation, and Affordability

Once a therapy reaches patients, innovation continues through new indications, improved formulations and next generation approaches. Defined exclusivity periods provide clarity on both protection and the eventual transition to competition.

As more manufacturers enter the market, competition expands supply and can place downward pressure on prices, increasing patient access. The policy framework that supports early discovery also creates a pathway to competitive markets and long-term affordability.

A Continuous Ecosystem for Patients

Annie Kennedy, Chief Mission Officer of the EveryLife Foundation for Rare Diseases, emphasized that despite decades of Congressional progress, fewer than 5% of rare diseases have an FDA‑approved treatment, making clear how much unmet need remains.

Rare Disease Week serves as a reminder that patients are waiting not only for current treatments but also for future breakthroughs.

Policies that support each stage of progress, from laboratory research to market competition, strengthen the entire biomedical innovation ecosystem.

When the system recognizes the interdependence of innovation and affordability, promising ideas can move forward, therapies can reach patients and access can expand as markets evolve.