Right-to-try legislation and access to investigational drugs

Right-to-try legislation and access to investigational drugs

Right-to-try legislation is a legal framework that allows patients with life-threatening diseases or conditions to access investigational drugs that have not been approved by the Food and Drug Administration (FDA) but have completed Phase 1 clinical trials. The aim of right-to-try legislation is to provide an alternative pathway for patients who have exhausted all approved treatment options and are unable to participate in clinical trials. Right-to-try legislation has been enacted at the state and federal levels in the United States, with varying provisions and implications.

The FDA regulates investigational drugs through its expanded access program, which allows patients to request access to investigational drugs outside of clinical trials under certain conditions. The FDA reviews each request and grants approval if the potential benefits outweigh the potential risks, the patient cannot obtain the drug through another means, and the access does not interfere with the development or approval of the drug. However, some critics argue that the FDA’s expanded access program is too cumbersome, restrictive, and slow, and that patients should have more autonomy and choice in their treatment decisions.

The Right to Try Act of 2017 (P.L. 115-176) was signed into law on May 30, 2018, as a federal right-to-try legislation. The act allows eligible patients to access eligible investigational drugs without FDA approval or oversight, as long as they have written informed consent from their treating physician and the drug manufacturer agrees to provide the drug. The act also provides certain protections for the parties involved, such as limiting the liability of manufacturers, sponsors, physicians, and hospitals, and prohibiting the use of clinical outcomes from right-to-try patients to delay or adversely affect the review or approval of the drug by the FDA.

The Right to Try Act has generated mixed reactions from stakeholders and experts. Supporters of the act claim that it gives hope and freedom to terminally ill patients who have no other options, and that it respects their right to try potentially life-saving treatments without bureaucratic interference. Opponents of the act contend that it undermines the FDA’s authority and role in ensuring the safety and efficacy of drugs, and that it exposes vulnerable patients to harm and exploitation by unscrupulous manufacturers or providers. They also point out that the act does not address some of the major barriers to accessing investigational drugs, such as the availability and affordability of the drugs, and the lack of evidence on their benefits and risks.

The impact of the Right to Try Act on patient access to investigational drugs is unclear. According to the FDA, as of February 2021, it has received only one annual summary report from a manufacturer who provided an investigational drug under the act. The FDA does not have any other information on how many patients have used or requested to use investigational drugs under the act, or what outcomes they have experienced. Some observers suggest that the low uptake of the act may indicate that most patients and manufacturers prefer to use the FDA’s expanded access program, which provides more regulatory oversight and guidance. Others argue that more time and data are needed to evaluate the effectiveness and implications of the act.

References:

– Right to Try | FDA. (n.d.). Retrieved February 23, 2021, from https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/right-try
– Sheikh, H. Z. (2021). help write my research essay on Expanded Access and Right to Try: Access to Investigational Drugs (CRS Report No. R45414). Retrieved from Congressional Research Service website: https://crsreports.congress.gov/product/pdf/R/R45414
– Dresser, R. (2019). The Right-to-Try Movement: A Cure Worse Than The Disease? Perspectives in Biology and Medicine, 62(2), 221–238. https://doi.org/10.1353/pbm.2019.0034