Frequently Asked Questions
What is the Life Sciences Rare Disease Tracker?
The Life Sciences Rare Disease Tracker is a data‑driven dashboard that measures reductions in rare disease investment, research, and development. Particular attention is paid to the impact of the Inflation Reduction Act’s provisions on orphan drug development, but the tracker is designed to educate policymakers, stakeholders, and the public about all policies that negatively impact rare disease R&D, stifle innovation, and delay treatments. The Life Sciences Rare Disease Tracker builds on Incubate’s main Life Sciences Investment Tracker, which monitors the impact of the Inflation Reduction Act on investment and R&D across all disease areas.
How is information for the tracker selected?
The tracker includes data from SEC filings, earnings reports, company press releases, and federal clinical trials data. The FDA database on orphan designations and approvals is used to validate whether companies are engaged in rare disease research. More information on our methodology is available here.
How often is the tracker updated?
The tracker is updated on a monthly basis based on data from SEC filings, quarterly earnings reports, company announcements, the federal clinical trials database, and FDA data on orphan drug designations and approvals.
Does the tracker solely focus on the United States? Are there global impacts beyond what the tracker captures?
The tracker is intentionally scoped to identify impacts to companies doing business in the United States, but the full effects of policies that threaten rare disease research reach much further. R&D decisions at global companies often hinge on U.S. policy because the United States remains the largest rare-disease market and often sets regulatory and policy precedents. As a result, some large multi-national company announcements are included in the tracker.
How do the tracker’s findings in rare disease research compare to findings across the broader life sciences sector?
The tracker puts firm data behind a well-known concern: when policy changes undermine the expected return on investment in biopharma R&D, capital dries up, drug pipelines shrink, and researchers exit the field. The tracker documents this pattern across the industry — from discovery to late-stage clinical trials — and rare disease research is no exception. Even incremental policy shifts can sour investor sentiment, forcing companies to make tough decisions to cut their losses and walk away from promising research.
How is the Inflation Reduction Act influencing investment changes in rare disease?
Enacted in 2022, the Inflation Reduction Act includes provisions that established a process for Medicare to negotiate a “maximum fair price” for certain drugs. The law includes an orphan drug exclusion, designed to maintain investment in rare disease research by exempting orphan drugs from price negotiations. However, the exclusion is only eligible for drugs approved for one rare disease indication. If a drug receives FDA approval to treat a second rare disease, it is no longer excluded from federally set prices. This narrow exclusion discourages follow-on indications and research into whether a drug may successfully treat multiple rare diseases.
How do we know that these changes are the result of the U.S. policy environment rather than market drivers?
Policy doesn’t happen in a vacuum. The U.S. policy environment directly influences the market. In some cases, companies have directly attributed cutting rare disease R&D to the IRA. In other cases, we can connect the dots between a company publicly stating that the IRA may force them to reconsider their research portfolios and then, within weeks or months, subsequently discontinuing research for reasons other than safety, efficacy, or clinical concerns.
Macro investment trends also illustrate how policies like the IRA are driving decisions in the market. For example, rare disease companies lost more than $1.2 billion in value between December 2020 and February 2025. Small molecule companies similarly saw reduced investment, even while many other research areas saw increased investment.
Why does the tracker count discontinued research programs, but not new research programs that started in the same timeframe?
The tracker is designed to measure attrition, not activity. A canceled study or shelved program leaves a paper trail that can be tracked and verified. By contrast, new research programs are often announced with little financial detail, may be contingent on later milestones, and frequently overlap with projects that were already in motion before the policy shock we are tracking. Including them would blur a clean signal with a noisy one — we’d be mixing solid, confirmed losses with uncertain, hard-to-track beginnings.
