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Sometimes, and not automatically. The movement behind the phrase argues that a therapy proven in more than one rare disease could reach a larger group of patients than any one of those diseases offers alone. A bigger patient base could, in principle, spread the fixed costs of developing a drug across more sales. That is an economic hypothesis, not an established result. Each disease still needs convincing evidence of benefit and acceptable safety, and approval, reimbursement and commercial viability do not carry over automatically from one indication to another.
Where the idea comes from
The clearest statement of the thesis is a 2020 review in Metabolic Brain Disease, “One drug to treat many diseases: unlocking the economic trap of rare diseases,” by Karolina Pierzynowska, Teresa Kamińska and Grzegorz Węgrzyn, published September 14, 2020. The authors hypothesize that one effective medicine for several rare diseases might increase market demand and lower the average fixed cost each manufacturer carries. They call for further preclinical and clinical studies to test whether this can work. The paper is a hypothesis-generating review. It does not show that the model has made rare medicines broadly profitable.
The “economic trap” is simple to state. Each rare disease has a small potential population, while research, clinical development and regulatory work can be substantial. When only a few patients can use a product, its cost and risk are hard to recover. Finding therapies that are useful across several diseases is the proposed way out, because it enlarges the potential population for a single product and spreads fixed costs over it.
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“One drug, many diseases” covers several related strategies. They share a goal, but each solves a different problem, and none guarantees the others.
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Repurposing an existing medicine
Repurposing means investigating a medicine that already exists for a new disease or group of diseases. Prior knowledge about a compound may help development move faster, but a new indication still needs evidence suited to that use. NCATS’s 2025–2030 strategic plan includes high-throughput screening to identify new uses for existing drugs among its platform tools.
Shared-mechanism development
This approach selects diseases that share biology, even when their genetic diagnoses differ, and tests whether a treatment aimed at the common mechanism helps each group. A shared molecular pathway can justify running one trial across those groups. It does not guarantee a shared clinical response, because patients with the same underlying mechanism may still differ in symptoms, progression or how they respond to a drug. The clearest current description of this approach is the SIMPATHIC consortium’s work on rare neurological, neurometabolic and neuromuscular diseases, covered below.
Reusable platform technologies
A platform is a common tool or modular approach intended to be adapted across diseases. NCATS’s plan names research platforms, gene-targeted therapies and repurposing as routes to address multiple diseases, and it includes models for predicting drug response. A platform can streamline how candidates are made or tested. That does not prove any particular candidate works in more than one disease. Infrastructure can be shared; clinical benefit cannot be assumed to be.
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A basket trial tests one product across several diseases or disease subtypes under one protocol. Sites, oversight and parts of the protocol can be reused, and in suitable settings a control group may be shared too. The design, and why it is harder than it sounds, is covered in the next section.
What regulators say about shared trials
FDA’s recap of master protocols sorts these designs by what is being tested:
- Basket trial: one product evaluated in multiple diseases or disease subtypes.
- Umbrella trial: multiple products evaluated for one disease.
- Platform trial: multiple products evaluated across one or more diseases over time.
FDA says master protocols can share infrastructure and oversight and may use participants more efficiently than separate trials, in appropriate circumstances. The same recap lists the hard parts: choices about control groups, consent, blinding, adaptive design, multiplicity (the statistical problem of testing many hypotheses at once), safety evaluation and how interim results are communicated. The agency also notes that in some rare-disease settings there may not be several drugs ready for phase 3 testing, which limits when umbrella or platform designs are suitable.
A shared protocol does not erase disease-specific evidence requirements. Three questions stay separate for each disease:
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- Scientific: is the treatment effect and safety profile credible for each condition, or for a biologically justified subgroup?
- Statistical: how are results interpreted when they are pooled across groups that may respond differently?
- Operational: can enough patients and qualified sites be recruited for each arm or cohort?
Why the numbers drive the debate
Public funding has started to address the same problem. ARPA-H announced its THRIVE program on July 9, 2026, saying it would award up to $160 million to advance personalized curative medicines for rare genetic diseases. The announcement states that “about 95% of rare diseases have no approved medicines.” That figure belongs to ARPA-H and its July 2026 announcement. It is not a peer-reviewed prevalence estimate or an independent count. The $160 million is a maximum award amount, not reported spending or the value of any drug. Public money signals policy interest and investment; it does not show that private-market economics now work.
NCATS takes the same view of the status quo. Its 2025–2030 strategic plan says: “Developing one drug for one disease is inefficient, particularly with the extremely small number of approved treatments reaching patients per year.” That is an agency strategy statement, not evidence of a commercial result.
Three examples and how much they prove
Genistein: a laboratory illustration, not a treatment
The 2020 review uses genistein, a small molecule, as an example. It reports effects in experimental models of mucopolysaccharidoses, Huntington’s disease and Alzheimer’s disease, with findings drawn from cell and animal models. The authors state explicitly that it is not possible to predict whether genistein will be effective in humans. Read it as an illustration of the cross-disease argument. It is not a demonstrated human treatment for any of these conditions, and it should not be taken or bought on the strength of this example.
SIMPATHIC: an organized shared-mechanism effort
An international consortium established in 2023 is pursuing repurposing for groups of rare neurological, neurometabolic and neuromuscular diseases that share clinical symptoms or molecular pathomechanisms despite different genetic diagnoses. Its 2025 paper, “Accelerating drug repurposing for rare diseases by exploiting SIMilarities in clinical and molecular PATHology,” describes running preclinical and clinical development in parallel. It also lists the obstacles it expects: heterogeneous patient groups, suitable models and endpoints, business and intellectual-property arrangements, and regulatory submissions. This is evidence of an organized approach, not yet proof of a profitable therapy.
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THRIVE is a current example of publicly funded platform development for rare genetic disease. Program performers are to pursue modular approaches and trials that span treatments and diseases. ARPA-H Director Alicia Jackson, Ph.D., said in the July 9, 2026 announcement: “By transforming precision genetic medicines through platforms that can test multiple treatments for multiple diseases in a single clinical trial, THRIVE reflects the kind of groundbreaking health innovation needed to help children and families facing life-threatening genetic diseases.”
THRIVE Program Manager Daria Fedyukina, Ph.D., made the case from the patient side in the same announcement: “Our current system is built to develop one drug for one disease tested in clinical trials designed specifically for that drug and that disease. Patients with rare genetic diseases, especially children, cannot wait for this legacy approach, it’s too slow and unsustainable.” That is an advocacy statement by the program manager, not an independent economic evaluation. THRIVE is an active research program, not an approved product.
Independent reader supportYour contribution helps us test, update, and keep practical guides available for everyone.Six questions to ask about any multi-disease claim
The sources identify these biological, trial, regulatory and business dimensions. They do not provide a universal scoring method, so treat this list as a way to read claims rather than a ranking.
Quick Recap
- Is the shared biology a mechanism or only a symptom? A shared pathway is a stronger rationale for testing than a shared symptom.
- What is the asset? A repurposed drug, a disease-specific asset and a reusable platform carry different risks and different evidence burdens.
- What evidence exists in models and in people for each disease?
- Can endpoints and patient groups be harmonized without hiding real differences?
- Can trial infrastructure actually be shared? Shared sites and oversight have to be operational, not nominal.
- How are intellectual property, indication-specific evidence, payer coverage and patient access handled?
What to watch next
- Whether THRIVE-funded work reports results disease by disease rather than as a combined package.
- Whether shared endpoints and models from consortium efforts such as SIMPATHIC are published and reused by other groups.
- Whether basket and platform designs described by FDA are used in rare diseases where enough patients and qualified sites exist.
- Whether incentives for rare-disease products change in your market. Orphan designation and related incentives differ by country and shift over time, so check the current rules of your country’s regulator.
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