Right to Try Laws
Right to try laws are a category of healthcare policy in the United States designed to provide terminally ill patients with access to experimental treatments that have not yet received full approval from the Food and Drug Administration (FDA). These laws aim to allow patients to bypass the traditional regulatory approval process for drugs, biologics, and devices, offering a pathway to therapies when all approved options have been exhausted and participation in clinical trials is not possible. The intent of right to try laws is to empower patients and their physicians to make decisions about unapproved treatments, particularly for life-threatening conditions.56, 57
History and Origin
The movement for right to try laws gained significant traction in the United States starting in 2014, with Colorado being the first state to adopt such a law.55 The impetus for these laws often stemmed from patient advocacy groups and public policy think tanks who argued that the existing regulatory frameworks, while designed for safety, could impede access for patients facing terminal illnesses.54 The concept was supported by the idea that if patients have the right to make end-of-life decisions, they should also have the right to try potentially life-extending treatments.
This state-level momentum culminated in the passage of the federal Trickett Wendler, Frank Mongiello, Jordan McLinn and Matthew Bellina Right to Try Act, which was signed into law on May 30, 2018. This federal legislation created a uniform system, allowing patients with life-threatening diseases to seek access to investigational treatments nationwide, provided certain criteria are met and the drug has completed at least Phase I testing.53 The law fundamentally altered the landscape by allowing patients and their doctors to work directly with a pharmaceutical industry company to request access without requiring prior FDA permission for each individual patient's use.52
Key Takeaways
- Right to try laws allow terminally ill patients to access investigational drugs, biologics, or devices that have completed Phase I clinical trials but are not yet fully approved by the FDA.51
- These laws are intended for patients who have exhausted all approved treatment options and are unable to participate in existing clinical trials.50
- The federal Right to Try Act, signed in 2018, provides a pathway that largely bypasses the FDA's typical review process for individual patient requests, although manufacturers are required to submit annual summaries to the FDA.49
- A core principle is the belief that patients with life-threatening conditions should have greater autonomy in seeking experimental treatments.48
- Access under right to try laws requires agreement from the patient, their physician, and the drug manufacturer, and does not compel manufacturers to provide the treatment.46, 47
Interpreting Right to Try Laws
Right to try laws are interpreted as a legislative effort to grant patients facing life-threatening conditions greater autonomy in pursuing experimental medical products. These laws aim to create a direct channel between patients, their physicians, and drug manufacturers, reducing perceived bureaucratic hurdles in accessing unapproved therapies. The key is that the drug must have successfully completed a Phase I trial, meaning it has demonstrated initial safety in humans, and is still undergoing active drug development.44, 45
The effectiveness of right to try laws is often measured by how many patients successfully gain access to these treatments and whether those treatments yield positive outcomes. However, it is crucial to understand that these laws do not mandate that a pharmaceutical company provide the drug, nor do they guarantee efficacy or safety.42, 43 The decision to provide an investigational product ultimately rests with the manufacturer.41 Patients considering this pathway should engage in thorough discussions with their healthcare providers regarding the potential benefits, risks, and the unproven nature of the therapy. This includes understanding the potential impact on ongoing care and insurance coverage, as these experimental treatments and associated care may not be covered.40
Hypothetical Example
Consider a patient, Sarah, who has been diagnosed with a rare, aggressive form of cancer. She has undergone all standard chemotherapy and radiation treatments, but her disease continues to progress, and her doctors have informed her that there are no further approved options. Sarah's physician determines that she is not eligible for any ongoing clinical trials due to specific health criteria or geographical limitations.
Sarah's doctor learns about an investigational drug being developed by a biotechnology firm. This drug has successfully completed Phase I trials, demonstrating an acceptable safety profile, and is currently in later stages of testing for other indications. Under a federal right to try law, Sarah and her physician can directly contact the biotechnology firm to request access to this experimental drug. If the firm agrees, and Sarah provides informed consent acknowledging the unproven nature and potential risks of the treatment, the drug can be administered without seeking individual patient expanded access authorization from the FDA. This pathway allows Sarah to pursue a potential treatment that would otherwise be unavailable, reflecting the intent of right to try laws to provide options for patients with terminal diagnoses.
Practical Applications
Right to try laws primarily apply within the healthcare sector, offering a pathway for individuals with life-threatening conditions to access investigational medical products. While not directly a financial instrument, these laws have implications for the pharmaceutical industry and biotechnology companies. For these firms, participating in right to try provisions can be a complex decision involving considerations of public perception, resource allocation, and potential legal liabilities, even with the liability protections offered by the federal law.38, 39
The laws are a facet of healthcare legislation that touches upon patient rights and access to care. They can influence a company's approach to drug development and engagement with patient communities. While the intent is to offer hope, the actual number of patients who have received drugs through these pathways remains relatively small compared to those accessing treatments via the FDA's expanded access program. One study reviewing the federal Right to Try Act's implementation found that, by 2022, only a limited number of requests had been reported to the FDA.37 For investors, understanding these policies provides context for the regulatory environment that drug developers operate within, influencing factors like investment risk and potential market opportunities.
Limitations and Criticisms
Despite their compassionate intent, right to try laws face several limitations and criticisms from various medical and ethical perspectives. A primary concern is that these laws may offer false hope to desperate patients, as they do not compel manufacturers to provide investigational drugs.35, 36 Many companies may be unwilling to provide unapproved treatments due to limited supply, liability concerns, or the potential for negative outcomes that could impact future regulatory approval of their products.33, 34
Critics also highlight potential patient safety issues, arguing that bypassing FDA oversight removes a crucial layer of protection.32 While a drug must have completed Phase I clinical trials (focused on safety), its efficacy has not yet been established at this stage.31 There are also concerns about medical ethics, particularly regarding truly informed consent when patients are in vulnerable, life-threatening situations, and the financial implications.29, 30 Right to try laws generally do not require insurance companies to cover the costs of these experimental treatments or any adverse events, potentially leading to significant healthcare costs for patients and their families.28 Some medical professionals argue that the existing FDA expanded access program is a more robust and safer pathway for patients seeking investigational treatments.27
Right to Try Laws vs. Expanded Access Programs
Right to try laws and the FDA's Expanded Access Program (also known as "compassionate use") both aim to provide patients with access to investigational drugs outside of traditional clinical trials, but they differ significantly in their regulatory oversight and process.25, 26
| Feature | Right to Try Laws | Expanded Access Programs |
|---|---|---|
| Regulatory Involvement | Largely bypasses direct FDA involvement for individual patient requests. Patients and physicians work directly with the manufacturer. Manufacturers must submit annual summaries to the FDA.23, 24 | Requires FDA review and authorization for each request. The FDA often responds to emergency requests within hours and non-emergency requests within days.22 |
| Institutional Review Board (IRB) Oversight | Generally, no explicit requirement for IRB review and approval for each request under federal law.21 | Requires IRB review and approval of the treatment protocol and informed consent, except in emergency cases where it must be reported afterwards.20 |
| Drug Development Stage | Applies to drugs that have completed Phase I clinical trials.19 | Typically applies to drugs that have progressed further in drug development, often Phase 2 or 3, where more is known about efficacy and safety.18 |
| Manufacturer Obligation | Manufacturers are not obligated to provide the drug.17 | Manufacturers are not obligated to provide the drug, but the FDA can inquire if access is denied.16 |
| Liability Protections | Federal law grants broad liability protection to manufacturers and prescribers, with exceptions for willful misconduct or gross negligence.15 | The FDA explicitly states that adverse event data from expanded access is considered in context and is unlikely to negatively impact product development.14 |
| Cost Recovery | Manufacturers may charge for the direct costs of manufacturing the drug, but not profit.13 | Manufacturers may charge for the direct costs of manufacturing, but not profit.12 |
The core distinction lies in the FDA's direct oversight and review process. While right to try laws expedite the administrative pathway by removing the FDA from individual approval, expanded access retains the FDA's review, which some argue provides additional patient protection and data collection.10, 11
FAQs
What type of patients are eligible for right to try laws?
Patients eligible for right to try laws must typically have a life-threatening disease or condition, have exhausted all approved treatment options, and be unable to participate in an ongoing clinical trial for the investigational drug.8, 9
Do right to try laws guarantee access to experimental drugs?
No. While right to try laws create a pathway, they do not compel a pharmaceutical company to provide access to its investigational drug. The decision to supply the drug ultimately rests with the manufacturer.6, 7
Are investigational drugs accessed through right to try laws proven to be safe and effective?
No. Drugs accessed through right to try laws have only completed Phase I clinical trials, which primarily assess safety. Their efficacy (whether they work as intended) has not yet been established by the FDA. Patients must understand and accept these unproven aspects.4, 5
Do insurance companies cover treatments obtained through right to try laws?
Typically, no. Right to try laws do not mandate that insurance companies cover the costs of investigational drugs or the medical care associated with their administration. This can lead to significant healthcare costs for patients and their families.2, 3
How does right to try affect the broader public health system?
Right to try laws influence the broader public health system by introducing an alternative pathway for accessing experimental treatments, which can spark debate over regulatory balance between speed of access and patient safety. While intended to provide more options, their actual impact on overall patient outcomes and data collection for future drug approvals is still being assessed.1