A new BioSpace article highlights an urgent challenge: maintaining America’s leadership in developing rare disease treatments amid increasing global competition.
Meeting that challenge will require policies that support the full life sciences innovation ecosystem – including pro-competitive mergers and acquisitions (M&A), which help connect rare disease innovation to the resources and capabilities needed to reach patients.
The unmet need: More than 30 million Americans live with a rare disease. Yet fewer than 10% of the 10,000 known rare diseases have an approved treatment. Developing rare disease treatments requires groundbreaking science to address diseases that are often not well understood.
A recent National Security Commission on Emerging Biotechnology (NSCEB) report identifies additional barriers slowing progress – with important implications for America’s continued leadership in rare disease innovation:
- Limited investment in early-stage research and development
- Gaps in data on rare disease progression
- Regulatory and clinical trial frameworks not designed for rare diseases
- Challenges manufacturing and scaling highly specialized treatments
The challenge: Rare diseases present significant clinical and commercial uncertainty, particularly for small and emerging companies. While many of these companies are pursuing rare disease therapies, they often operate without a profit and lack the infrastructure, experience and resources to carry a highly specialized therapeutic candidate through late-stage clinical trials and regulatory approval.
Where M&A fits in: M&A allows these companies to connect with experienced companies that have the clinical development capabilities, regulatory expertise and manufacturing infrastructure needed to advance a potential medicine. It also often serves as an anticipated exit point, helping pre-profit, innovative companies attract the investment they need to pursue high-risk, early-stage science.
The data: Recent research underscores the critical role of M&A in bringing new treatments to patients. Drug candidates developed by a smaller company that undergoes M&A with a company that has previously launched a medicine are:
- Five times more likely to launch as novel medicines that address an unmet need or improve upon existing treatments; and
- Nearly 24 times more likely to launch with a new mechanism of action.
The policy imperative: The NSCEB report calls for an all-of-government strategy to strengthen rare disease innovation, including more flexible regulatory pathways, modern clinical trial designs and sustained support for research and manufacturing. Balanced competition policy and antitrust enforcement are an essential part of that strategy.
To accelerate new treatments, compete globally and preserve America’s life sciences leadership, policymakers must recognize and support the unique role of pro-competitive M&A in helping companies of all sizes transform scientific breakthroughs into medicines for patients with the greatest unmet needs.