Right to Try has mostly been a moral cause with little practical effect. Signed into federal law in 2018, and into 41 state laws, it allowed terminally ill patients to use doctor-supervised investigational treatments. In practice almost no one used it.¹

That is starting to change. State medical access frameworks like Montana's SB 535, or the stem cell laws of Florida, Utah and Texas, are overcoming barriers that prevent patients from access.

Which means that an old objection to Right to Try is about to resurface: giving patients early access pulls them out of clinical trials and starves the evidence base we use to tell whether drugs actually work.²

This article argues that this objection has it backwards. These state programs can improve clinical trials: get data from excluded patients, sharpen the trials that follow, get more funding for the sponsors, and engage more patients to partake in IND-registered trials.

However, if the FDA needs to act now and issue a policy of enforcement discretion - otherwise the opportunity will be lost: more credible actors are deterred from participating in state programs, and patients are left to die without a chance to try.

The Clinical Trial Bottleneck

Trial costs are infamously high at upwards of $1 billion, and approval timelines of more than decade. A company with a post-Phase-1 therapy has a 10% chance of final approval.

Yet ClinicalTrials.gov data show that at least two-thirds of clinical trials fail not because of negative safety or efficacy results. The typical reason is insufficient patient recruitment, often in combination with sponsors running out of money.³ This is a catch-22: without data, the company can't raise money to continue; without money, it can't collect data.

The failure rate of post-phase-1 trials comes down to the cost of data collection.⁴

Complementary State Pathways for In-Human Data

Under state law, this is the practice of medicine rather than human-subjects research. It generates outcome data, not trial data, and does not require IRB review.⁵

There are two big improvements to complement the federal pathway.

Broad Access for Excluded Patient Groups
IND-registered trials typically have long lists of eligibility criteria, and they keep adding. Some protect patients or keep the efficacy signal clean; many are superfluous.

I've seen this before. I ran hundreds of survey-design studies in market research across almost any industry you can think of. I watched scientists tighten criteria to reduce “data noise” because it’s easy to see, while the real cost of lost generalizability is nearly invisible. The FDA is aware of this problem and advocating for fewer restrictions in a clear-eyed 2018 guidance.⁶

The cost lands on patients. For example: Sarah Nauser is a former police officer and ALS patient. Sarah was disqualified because her breathing capacity was 42% against a typical 65% cutoff. Even if her lung function were better, she was diagnosed too late - her symptoms had begun three years before diagnosis, but most trials require a two-year window.⁷

On X/Twitter, Sarah reported: “I understand trials need structure and safety measures, but it’s hard not to wonder why those of us with the most to gain are so often excluded.”

Sarah’s situation is not unusual. In 38 ALS trials, 60% of patients are excluded from the day they're diagnosed. Once you count those who die or progress before enrolling, real-world exclusion could be closer to a 80–96% rate seen in fields such as psychiatry.⁸ Even for those who make it into a trial, it's a coin flip whether they get the drug or the placebo.

ALS is not a special case: in Alzheimers, over 90% of real-world patients are excluded from the leading trials; in glioblastoma, a uniformly fatal brain cancer, roughly 60% are.⁹

Patients Become Collaborators, Their Experience Informs Trial Design
Suppose a company decides to make post-Phase-1 therapy available for early use in Montana.

There are two possibilities: (a) it doesn't work, so the company abandons the program early or pivots, so wasteful cost and time to the trial system is avoided; or (b) it shows promise, so we see more patient participation, investor funding, and a push to take it all the way to FDA approval and insurance coverage. Now we have more data on dosing, side effects, and endpoints, which would improve the trial's success rate substantially.

Yes, there will be problems, as with any new modality. Patients may be disappointed when the company says “enough of early access, we need patients for controlled trials now”. But isn’t that better than the status quo of depressed patients and funding-strapped sponsors?

The challenge is to communicate well and with empathy towards patients. It could be an opportunity to make the system more honest, and more patient-centric.

Trials Are Moving Abroad

Access is also shrinking because the trials themselves are leaving. China has now passed the U.S. in annual clinical-trial count, and the U.S. share of new trials has fallen from 49% in 2015 to 33% in 2025 while China's climbed from 4% to 30%. The draw is speed and cost: a Phase 1 trial runs about seven months in China versus seventeen or more in the U.S., at roughly a 30-50% lower cost.¹⁰ A trial running in Beijing is not available to almost every American patient.

Better Access, Better Data, Trials Stay in US - Everyone Wins

In the state-access model, everyone wins. Trials stay in the US. Patients get earlier access. Biotechs get data to break the catch-22 and funding to continue promising programs. Pharma gets a better-oiled acquisition pipeline. The evidence base for public health decisions improves.

To succeed, the FDA needs to do two simple things, neither requiring new legislation: (a) issue a safe-harbor policy for state medical access programs under “enforcement discretion,”¹¹ since the perceived legal risk is otherwise too high for serious providers; and (b) develop guidelines for accepting outcome data from these state programs into INDs.

The objection was that Right to Try works against good evidence. Done right, it may be the biggest opportunity to improve evidence generation for the benefit of American patients.

Sources

1. https://x.com/RighttoTryGuy/status/2052955728865006041
2. For example, the expanded access program explicitly requires patients to be ineligible for trials to receive access to investigational treatments https://www.ecfr.gov/current/title-21/chapter-I/subchapter-D/part-312/subpart-I
3. NLM analysis of 905 terminated trials with posted results: 68% stopped for reasons other than trial data (lead reason: insufficient enrollment) vs. 21% based on efficacy or toxicity. [Williams et al., PLOS ONE 2015.] A larger NLP analysis of 28,561 stopped trials agrees directionally, only a minority stop for a negative efficacy or safety result. [Razuvayevskaya et al., Nat Genet 2024;56(9):1862–67.]
4. https://www.macroscience.org/p/to-get-more-effective-drugs-we-need
5. Treatment under Right to Try is "practice," not "research," under the Belmont Report's distinction and the Common Rule's definition of research (45 CFR 46.102(l)); FDA guidance confirms individual Right to Try requests do not require IRB review or approval.
6. https://www.fda.gov/media/134754/download
7. https://x.com/SarahNauser/status/2055785638742708617?s=20
8. van Eijk et al., Neurology 2019;92(5):e451–e460: applying the criteria of 38 ALS trials to a 2,904-patient incidence cohort, 59.8% were excluded at diagnosis (14–95% across trials). The authors note this underestimates real-world exclusion — 15–24% of diagnosis-eligible patients die before typical enrollment (6–9 months out), and more progress past the criteria, plausibly pushing real-world exclusion toward the 80–96% reported in other fields.
9. In real-world memory-clinic cohorts, single digits qualify for the leading anti-amyloid trials: 4.3% of dementia patients (Austrian psychiatric memory clinic), 6–8% (Alzheimer Center Amsterdam), and 3–6% (Swedish tertiary memory clinic, the lower figure after APOE ε4/ε4 exclusion), i.e., 90%+ excluded. In newly diagnosed glioblastoma, ~57–63% of real-world patients are ineligible for phase III trials.
10. China overtook the U.S. in annual clinical-trial count in 2024: 7,100 vs. 6,000 trials registered (WHO International Clinical Trials Registry Platform, reported by Axios, May 2025). Industry figures presented in 2025 put the U.S. share of newly initiated trials at 33% (down from 49% in 2015) and China's at 30% (up from 4%), with Phase 1 trials averaging ~7 months in China vs. 17+ in the U.S. at 32–52% lower cost (Clinical Trials Arena, 2026, reporting Outsourcing in Clinical Trials East Coast).
11. https://www.pharmexec.com/view/fda-power-de-bottleneck-early-stage-clinical-trials