What Is "Right to Try," and Does It Actually Help Patients Access Stem Cell Therapy?

The federal Right to Try Act, signed into law in 2018, lets certain seriously ill patients request access to investigational drugs outside of a clinical trial — but it does not require any company to provide that access, and federal data show it has been used only a handful of times per year since passage. This article explains what the law actually does and does not do, how it differs from the FDA's long-standing Expanded Access ("compassionate use") pathway, what happened to the state-level Right to Try laws that preceded it, and — critically for readers researching stem cell clinics — why "we operate under Right to Try" is a phrase that should raise questions rather than reassure, since almost no regenerative-medicine products meet the law's requirements and several stem cell businesses have invoked the law's language while offering products that were never eligible for it.

What the Federal Right to Try Act Actually Says

The Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act was signed on May 30, 2018. Per the FDA's own summary, it lets a patient request an investigational drug directly from a manufacturer, without FDA involvement, if the patient (1) has a life-threatening disease, (2) has exhausted approved options and cannot join a relevant clinical trial, and (3) gives written informed consent (fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/right-try, 2024). The product itself must also qualify: per the Congressional Research Service, it must have completed Phase 1 testing and remain under an active investigational new drug application with ongoing studies, with no clinical hold or discontinued development (Expanded Access and Right to Try: Access to Investigational Drugs, CRS R45414, everycrsreport.com/reports/R45414.html, 2018). Completing Phase 1 only establishes basic safety in a small cohort — it says nothing about whether a treatment works.

The most consequential clause is one the FDA states plainly: "the Right to Try Act does not require a sponsor to provide an eligible investigational drug to an eligible patient." Manufacturers retain full discretion to say no — for cost, liability, limited supply, or to protect an ongoing trial's data.

How This Differs From FDA Expanded Access

Expanded access ("compassionate use") is the decades-old FDA pathway that already let physicians request investigational products outside trials. Per the CRS, expanded access requires a physician request, FDA sign-off, and typically institutional review board (IRB) oversight; Right to Try removes the FDA and, usually, the IRB, substituting a physician certification and a company's willingness to participate (everycrsreport.com/reports/R45414.html, 2018). Expanded access also carries stricter adverse-event reporting; Right to Try relies on lighter annual manufacturer summaries.

Supporters framed Right to Try as cutting red tape, but the Baker Institute at Rice University notes the FDA already approved over 99% of expanded access requests, and by 2015 had trimmed its own application to about 45 minutes of paperwork (The Impact of Right to Try Laws on Medical Access in the United States, Baker Institute, bakerinstitute.org/research/right-try-unproven-drugs). FDA review was rarely the bottleneck — manufacturer willingness was, and remains, the real constraint under both pathways.

State Right to Try Laws and the Federal Law

Colorado passed the first state right-to-try law in May 2014, based on Goldwater Institute model legislation; by August 2018, 41 states had similar statutes (Right-to-try law, Wikipedia, en.wikipedia.org/wiki/Right-to-try_law, accessed 2026). These varied laws had no binding effect on FDA-regulated manufacturers, since drug approval is federally governed. The 2018 federal act created one nationwide pathway; while it didn't formally preempt state statutes, it effectively superseded them for FDA-regulated products. Since 2018, the Goldwater Institute has also promoted "Right to Try 2.0" — Right to Try for Individualized Treatments Acts, now in at least 17 states — covering gene therapies and other products that haven't even completed Phase 1, an even lower bar than the federal law (Wikipedia, en.wikipedia.org/wiki/Right-to-try_law, accessed 2026).

How Often Is Right to Try Actually Used?

Here the law's name and its footprint diverge sharply. STAT, citing FDA data, found the federal pathway supported access to only 4 products in 2023 and 12 total between 2018 and 2022 (Trump gave patients a "right to try." It hasn't helped them, STAT News, statnews.com/2024/08/02/trump-gave-patients-right-to-try-it-hasnt-helped-them/, 2024). Early implementation reporting summarized by Wikipedia put the number even lower at the start: as of June 2019, only two patients nationally had obtained a product through the federal pathway, one of them Matt Bellina — one of the law's own namesakes — who received a dose from BrainStorm Cell Therapeutics before the company stopped granting further requests (en.wikipedia.org/wiki/Right-to-try_law, accessed 2026). The other namesake, ALS patient Frank Mongiello, never accessed treatment through the law bearing his name.

BrainStorm's case is instructive for regenerative medicine specifically, since NurOwn is a mesenchymal stem-cell-derived therapy for ALS. STAT reported in 2018 that BrainStorm planned to charge roughly $300,000 per patient for NurOwn under Right to Try, with its CEO stating "we have to have an incentive" — unlike expanded access, which historically limited sponsors to cost recovery (Here come the right-to-try profiteers. The FDA is powerless to stop them, STAT News, statnews.com/2018/06/20/right-to-try-opportunism/, 2018). Even that access proved short-lived: BrainStorm later stopped providing NurOwn to Right to Try patients altogether, citing an inability to find "a practical funding solution" (NurOwn, for ALS, Not Available Under Right to Try Act, BrainStorm Says, ALS News Today, alsnewstoday.com/news/als-therapy-nurown-not-available-under-new-policy-brainstorm-decides/). NurOwn then failed its Phase 3 trial and was never FDA-approved — a sequence that captures why advocates and bioethicists remain skeptical of the law.

Does Right to Try Actually Deliver Stem Cell Treatments?

Almost never, for structural reasons. Few stem cell products meet the federal criteria at all: they must be under an active FDA investigational new drug application with completed Phase 1 data, which excludes most products sold at U.S. stem cell clinics — typically same-day "minimally manipulated" procedures marketed as exempt from FDA regulation, not as investigational drugs in the IND process. A UC Irvine study led by bioethicist Leigh Turner found U.S. businesses marketing non-FDA-approved stem cell products grew roughly four-fold from 2016 to 2021 — from about 351 businesses/570 clinics to about 1,480 businesses/2,754 clinics, concentrated in California, Texas, and Florida (news.uci.edu/2021/11/04/businesses-selling-non-fda-approved-stem-cell-products-grew-four-fold-in-five-years-uci-study-says, 2021). Turner has separately documented that many such clinics claim their products don't require FDA approval at all — a posture that sits awkwardly beside any simultaneous claim of operating "under Right to Try," since that law only applies to products moving through the FDA's investigational-drug framework.

Patient advocates coined a blunter label years before the federal law passed. Attorney Beth Roxland, discussing early state statutes, told CIRM's blog they amounted to a "right to beg," creating only a right to request a product with zero obligation on the manufacturer to comply — and said she knew of no substantiated case where a company provided access solely because a right-to-try law existed ('Right To Try' laws called 'Right To Beg' by Stem Cell Advocates, CIRM: The Stem Cellar, blog.cirm.ca.gov/2016/12/19/right-to-try-laws-called-right-to-beg-by-stem-cell-advocates, 2016).

Why Some Clinics Invoke "Right to Try" Anyway

Even where a product plainly doesn't meet federal eligibility, "Right to Try" shows up in marketing because it sounds like official legal sanction. Bioethicists have flagged a recurring pattern: some direct-to-consumer stem cell businesses invoke the law's name in patient-facing materials to lend an unproven treatment a veneer of federal legitimacy, regardless of whether the product and patient actually meet the statute's requirements, or whether the "manufacturer" (often the clinic itself, using the patient's own cells) is even the kind of FDA-regulated sponsor the law contemplates. Cell Stem Cell examined this pattern directly, analyzing how stem cell businesses reference right-to-try laws in marketing and flagging the resulting oversight gaps (Stem Cell Businesses and Right to Try Laws, Cell Stem Cell, PubMed 31491393, 2019).

Bottom Line

If a clinic tells you it "operates under Right to Try," don't take that as proof of legitimacy — ask it to show you, specifically: the FDA investigational new drug (IND) number the product is filed under; documented Phase 1 safety data; and written confirmation from the product's actual manufacturer (not the clinic itself) that it has agreed, in this instance, to provide the product to you under the federal statute's request-and-consent process. If the clinic cannot produce an IND number and instead describes its product as "not requiring FDA approval" because it is minimally manipulated or autologous, then Right to Try is not the relevant framework at all — the clinic is operating outside FDA's investigational-drug pathway entirely, which is a different and generally less-regulated situation, not a more legally sanctioned one. Also ask what happens if the product doesn't help, who bears the cost (many Right to Try and clinic arrangements bill patients directly, sometimes tens of thousands of dollars), and whether accepting the treatment could affect insurance or hospice eligibility. Given that FDA's own reporting shows only a handful of products nationwide have ever moved through the federal Right to Try pathway, treat any stem cell clinic's invocation of the law as a claim to be independently verified, not a credential to be trusted at face value.