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Developing a treatment for several rare diseases could enlarge the group of patients who may benefit and spread some fixed development costs. But that is an economic hypothesis, not a proven route to profitability: each disease still needs credible evidence that the treatment is safe and works for its patients, and approval or payment for one use does not automatically extend to another.
What does “one drug, many rare diseases” mean?
The phrase describes a family of strategies for testing whether a medicine, biological target, development tool, or clinical-trial infrastructure can serve more than one rare condition. The approaches are related, but they address different bottlenecks.
| Approach | What is shared | What it can help with | What it does not establish |
|---|---|---|---|
| Drug repurposing | An existing medicine is investigated for a new disease or group of diseases. | Prior knowledge about a medicine may inform development for a new use. | Past use does not by itself prove benefit or acceptable safety for a new disease. |
| Shared-mechanism development | A biological target or disease process is shared across conditions, which may have different genetic diagnoses. | It can identify groups of patients who may be suitable for the same therapeutic approach. | A shared mechanism does not guarantee the same clinical response. |
| Reusable platforms | Tools or modular methods for making, screening, or evaluating treatments are adapted across diseases. | They may make parts of research and development reusable. | A reusable platform is not proof that a particular medicine works in multiple diseases. |
| Basket trials and other master protocols | A trial protocol or infrastructure is used across multiple diseases, products, or both, depending on the design. | In suitable settings, some oversight, sites, protocol elements, or controls may be shared. | A common protocol does not erase the need to interpret benefit and safety for each disease or justified subgroup. |
The distinctions matter. A platform can speed or standardize how candidates are developed without providing evidence of a shared treatment effect. A shared biological rationale may support testing several groups together, but it is not a substitute for the results.
Why are rare-disease drugs difficult to make profitable?
Research, clinical development, and regulatory work can require substantial resources, while the potential patient population for any one rare condition may be small. The 2020 review by Karolina Pierzynowska, Teresa Kamińska, and Grzegorz Węgrzyn describes this as an economic trap: a developer may face significant costs and risks for a medicine that could serve relatively few people.
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The proposed economic mechanism is straightforward. If one intervention can credibly help patients across several conditions, the potential population grows; if development work can also be reused, some fixed costs might be spread across more uses. Whether those gains outweigh the added costs of studying, securing authorization for, and supporting each use is an open commercial question. The review proposes a hypothesis to test, not evidence that multi-disease development has broadly made rare medicines profitable.
Can a shared mechanism mean the same treatment will work?
It can provide a reason to investigate, not a conclusion. Conditions may share a molecular pathway or a broader disease process while differing in which patients are affected, how the disease progresses, or how an outcome should be measured. A trial therefore needs a defensible explanation for grouping patients and evidence that its results apply to each condition or biologically justified subgroup.
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The practical questions include whether the mechanism is well supported in the relevant patients, whether available laboratory or animal models represent those patients, and whether researchers can choose meaningful outcomes without hiding important differences among diseases. The 2025 SIMPATHIC review describes an international consortium, established in 2023, pursuing repurposing across rare neurological, neurometabolic, and neuromuscular diseases with shared clinical symptoms or molecular pathomechanisms despite different genetic diagnoses. It also identifies heterogeneous patient groups, suitable models and endpoints, business and intellectual-property arrangements, and regulatory submissions as implementation challenges. The consortium illustrates an organized research strategy, not a proven commercial success.
What can a basket trial do—and what can it not do?
The FDA defines a basket trial as one that evaluates a product in multiple diseases or disease subtypes. It distinguishes this from an umbrella trial, which evaluates multiple products for one disease, and a platform trial, which can evaluate multiple products across one or more diseases over time. These are forms of master protocol: shared trial structures that may permit efficient use of sites, oversight, protocol elements, or participants in appropriate circumstances.
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Sharing infrastructure does not make the scientific questions identical. Investigators still need to decide how to select controls, obtain informed consent, preserve blinding where appropriate, handle adaptive changes and multiple comparisons, assess safety, and communicate interim findings. Recruitment is another constraint: rare populations may be dispersed, and suitable sites and enough eligible participants may be difficult to assemble. The FDA also notes that some rare-disease settings may not have multiple drugs ready for phase 3 testing, limiting the fit of certain umbrella or platform designs.
In practice, a shared protocol is useful only if its components can be shared without undermining interpretation. The treatment effect and safety profile must remain credible for the disease groups the evidence is meant to cover; a positive result in one group cannot simply be assumed to establish benefit in another.
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What do current examples show?
Genistein: a preclinical illustration
The 2020 review discusses genistein as a small-molecule example with reported effects in experimental models involving mucopolysaccharidoses, Huntington’s disease, and Alzheimer’s disease. These are cell and animal-model findings. The review says it is not possible to predict from them whether genistein will be effective in humans; they are not evidence of an established treatment for those diseases.
NCATS: a platform and repurposing strategy
The U.S. National Center for Advancing Translational Sciences (NCATS) 2025–2030 strategic plan argues that developing one drug for one disease is inefficient given the small number of approved treatments reaching patients each year. It points to research platforms, models for predicting drug response, gene-targeted therapies, and high-throughput screening for new uses of existing drugs as ways to address multiple diseases. This is an agency strategy, not proof that the methods have produced a profitable commercial model.
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THRIVE: public investment in modular development
In its July 9, 2026 announcement, the Advanced Research Projects Agency for Health (ARPA-H) said it would award up to $160 million to advance personalized curative medicines for rare genetic diseases through its THRIVE program. “Up to” is the program’s announced maximum award amount, not a report of money already spent or the commercial value of a medicine. The same announcement says that “about 95% of rare diseases have no approved medicines”; that is ARPA-H’s stated figure in this announcement, not an independent count established here.
THRIVE is described as supporting modular approaches and trials spanning treatments and diseases. It demonstrates federal investment and policy interest in reusable development, not an approved product or evidence that the economics have been solved. Public funding can support work that may not otherwise attract investment; it does not, by itself, establish private-market viability.
How to judge whether a program could work
A proposal should be assessed across its biology, evidence, trial design, and route to patient access—not just by how many diseases it names. Useful questions include:
- Mechanism: Do the conditions share a well-supported biological process, or only broad symptoms?
- Intervention: Is the project repurposing an existing medicine, developing a treatment for a shared target, or building a platform to adapt across diseases?
- Evidence: What is known from models and from people for each proposed disease or subgroup?
- Trial design: Can patient groups and outcomes be harmonized without obscuring meaningful differences? Which protocol elements, sites, oversight, or controls can genuinely be reused?
- Execution: Are enough eligible patients and qualified sites available, and can the study evaluate safety and results clearly across groups?
- Translation: How will intellectual property, evidence for each indication, payer coverage, and patient access be handled?
These are decision questions, not a validated scoring system. A program that shares biology or infrastructure may still encounter separate evidence, regulatory, reimbursement, and commercial barriers for each intended use. The underlying promise is plausible: serve a larger population and reuse some development work. Whether a specific program can do both—and deliver treatments patients can access—has to be demonstrated.
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