Right to Try Act: Key Facts for Patients
Empowering terminally ill patients with access to experimental treatments outside clinical trials.
The Right to Try Act, enacted in 2018, provides a federal pathway for certain patients with life-threatening conditions to access investigational drugs, biologics, or devices that have not yet received full FDA approval. This law aims to offer hope when standard treatments fail, bypassing some traditional clinical trial requirements while maintaining safety safeguards.
Historical Background and Purpose of the Law
Prior to the federal Right to Try Act, individual states began enacting similar legislation starting around 2014 to address frustrations with lengthy FDA approval processes. These state laws permitted terminally ill patients to request experimental therapies that had passed Phase I safety trials. The federal version standardized this approach nationwide, signed into law by President Trump on May 30, 2018, to ensure consistency and reduce barriers for interstate access.
The core purpose is to grant eligible patients an additional option beyond FDA’s Expanded Access program, which requires agency approval. Right to Try streamlines access by limiting federal involvement, focusing instead on agreements between patients, physicians, and manufacturers. However, it does not guarantee access, as participation remains voluntary for drug makers.
Defining Eligible Patients: Strict Criteria Apply
To qualify under the Right to Try Act, patients must meet precise criteria outlined in the law. A physician in good standing—meaning licensed and not compensated directly by the drug manufacturer—must certify eligibility.
- Life-threatening disease or condition: Defined per FDA regulations (21 CFR 312.81), this includes illnesses where the likelihood of death is high within months or where permanent impairment is expected without intervention.
- Exhausted approved options: Patients must have tried all FDA-approved treatments reasonably available and suitable, interpreting ‘exhausted’ as having received or declined safe standard therapies.
- Inability to join clinical trials: The patient cannot enroll in an ongoing trial for the drug, due to location, eligibility exclusion, or trial closure.
- Informed consent: Written consent from the patient or their representative, detailing risks, benefits, lack of FDA approval, and potential costs.
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These requirements ensure the law targets those with no viable alternatives, preventing premature use of unproven therapies in curable cases.
Qualifying Investigational Treatments
Not all experimental drugs qualify. The Act specifies drugs, biologics, or devices that meet development milestones, balancing innovation with preliminary safety data.
| Requirement | Description |
|---|---|
| Phase I Completion | Safety testing in humans finished, establishing basic tolerability. |
| Ongoing Pivotal Trial | Active Phase II or III trial forming the basis for FDA approval under an Investigational New Drug (IND) application. |
| Active Development | Manufacturer pursuing approval; not discontinued or on hold. |
| Not FDA-Approved | No approval for any use; remains investigational. |
This framework prioritizes agents with promising efficacy signals from advanced trials, excluding early-stage or abandoned projects.
The Role of Physicians and Manufacturers
Physicians initiate the process by certifying patient eligibility and obtaining consent. They must discuss uncertainties, including unknown side effects and lack of proven benefits. Importantly, the law shields doctors acting in good faith from liability, disciplinary action, or professional repercussions.
Manufacturers hold the key decision: they may voluntarily provide the treatment at no cost, for payment, or decline entirely without penalty. The Act explicitly states no liability for refusals, protecting companies from coerced distribution that could jeopardize FDA approval or trials. Patients cannot sue for denial of access.
Financial Realities: Who Pays?
Right to Try does not mandate insurance coverage. Health insurers, Medicare, Medicaid, or other programs have no obligation to reimburse. Patients often bear full costs, which can reach tens or hundreds of thousands of dollars for a single course.
- Manufacturers may offer free compassionate use, but this is discretionary.
- No federal funding or subsidies exist under the Act.
- Potential loss of hospice eligibility or other benefits if treatment extends life unexpectedly.
Patients should consult financial advisors and explore FDA Expanded Access as an alternative, which sometimes includes cost waivers.
Comparing Right to Try with FDA Expanded Access
| Aspect | Right to Try | FDA Expanded Access |
|---|---|---|
| FDA Approval Needed | No | Yes (individual or emergency) |
| Patient Eligibility | Terminal, exhausted options, no trial access | Similar, plus serious unmet need |
| Drug Stage | Post-Phase I, active pivotal trial | Any stage if IND active |
| Manufacturer Consent | Required, voluntary | Required |
| Reporting to FDA | Annual summaries | Immediate safety reports |
| Cost Coverage | None required | None required |
Right to Try offers faster entry without FDA gatekeeping but less oversight. Expanded Access provides more structured data collection, potentially benefiting future approvals.
State Variations and Federal Preemption
Over 40 states have Right to Try laws, often mirroring federal provisions but with nuances like misdemeanor penalties for officials blocking access or added protections for heirs against debt. The federal Act preempts conflicting state rules, ensuring uniformity for interstate drugs. State laws may offer extras, such as insurer authorization (not requirement) or broader device inclusion.
Real-World Usage and Outcomes
Since 2018, Right to Try requests remain low—fewer than 100 annually per FDA reports—partly due to stringent criteria and manufacturer caution. Outcomes vary: some patients report prolonged survival, but rigorous efficacy data is scarce without trial controls. Critics note risks of adverse events from unproven agents, while proponents highlight autonomy.
Recent developments include ‘RTT 2.0’ discussions emphasizing independent physician certification to avoid conflicts.
Steps to Pursue Right to Try Access
- Confirm diagnosis and exhaustion of options with your oncologist or specialist.
- Verify drug eligibility via manufacturer’s website or ClinicalTrials.gov.
- Discuss risks; obtain written informed consent.
- Contact manufacturer directly for compassionate use request.
- Secure funding or negotiate free provision.
- Monitor progress and report serious adverse events to FDA as required.
Document everything; consider legal counsel for complex cases.
Frequently Asked Questions (FAQs)
Can any patient with cancer use Right to Try?
No, only those with life-threatening stages who meet all criteria, including physician certification.
Does Right to Try guarantee drug access?
No, manufacturers can decline without liability.
Will insurance cover Right to Try treatments?
Typically not; patients pay out-of-pocket.
How does it differ from clinical trials?
No trial enrollment needed, but lacks structured monitoring and data contribution.
Are there updates to the law in 2026?
As of early 2026, core provisions unchanged, but monitor FDA for guidance.
Ethical Considerations and Patient Advocacy
Right to Try embodies patient autonomy but raises debates on balancing hope against hype. Advocates like Goldwater Institute pushed for it to counter ‘FDA paternalism.’ Ethicists urge clear risk communication to avoid false hope. Patients should weigh emotional, financial tolls alongside potential benefits.
Resources: FDA.gov Right to Try page, patient advocacy groups like Triage Cancer.
References
- Understanding the Right to Try Act — Caplan AL, et al. National Library of Medicine. 2020-07-28. https://pmc.ncbi.nlm.nih.gov/articles/PMC7416898/
- Right to Try — U.S. Food and Drug Administration. 2023-05-05. https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/right-try
- Right to Try State Laws — Triage Cancer. 2024-01-15. https://triagecancer.org/state-laws/righttotry
- Right-to-try law — Wikipedia (informational). 2025-12-10. https://en.wikipedia.org/wiki/Right-to-try_law
- Right to Try Policy and Guidance — UCLA Office of Research Administration. 2022-06-01. https://ora.research.ucla.edu/OHRPP/Documents/Policy/8/Right_To_Try_Policy_and_Guidance.pdf
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