The U.S. 'Right to Try' law, enacted in 2018, has generated significant discussion within the rare disease community, including those affected by inherited retinal diseases (IRDs). This legislation aims to provide a pathway for patients with life-threatening conditions to access investigational treatments that have not yet received full approval from the U.S. Food and Drug Administration (FDA). For individuals and families facing the challenges of IRDs, where treatment options can be limited, understanding this law is crucial.

What is the 'Right to Try' Law?

The federal 'Right to Try' Act, officially known as the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act, was signed into law on May 30, 2018. It allows certain terminally ill patients to seek access to unapproved investigational drugs directly from manufacturers, bypassing the FDA's authorization step that is part of its Expanded Access program (also known as 'compassionate use').

To be eligible under the federal law, a patient must meet specific criteria: they must have a life-threatening disease or condition, have exhausted all approved treatment options, and be unable to participate in a clinical trial involving the investigational drug. A physician must certify this eligibility, and the patient (or their legal representative) must provide written informed consent. The investigational drug must have successfully completed a Phase 1 clinical trial and be under ongoing investigation for FDA approval.

Praise and Criticisms

Proponents of 'Right to Try' laws argue that they remove regulatory burdens and offer timely access to potentially life-saving medications for terminally ill patients, empowering them to make choices about their care when time is critical. They suggest it allows patients and doctors to pursue promising treatments without unnecessary government interference.

However, the law has also faced criticism. Opponents argue that it doesn't guarantee access, but rather a 'right to ask' manufacturers, who are not obligated to provide the drug. Concerns have also been raised about patient safety, as drugs accessed through this pathway have not completed the full FDA approval process, meaning their effectiveness and full safety profile are not yet established. Critics also point out that these laws may shift financial burdens to patients, as insurance companies are not required to cover the costs of experimental treatments, and patients might even lose eligibility for other benefits like hospice care. The National Organization for Rare Disorders (NORD) has expressed disappointment with the federal law, citing concerns about patient protections and the potential for ineffective or unsafe therapies.

Impact on Patients and Families in the IRD Community

For the IRD community, the 'Right to Try' law presents both a glimmer of hope and complex considerations. While it theoretically offers a path to experimental treatments for rapidly progressing or severe forms of inherited retinal diseases where approved therapies are lacking, the practical realities can be challenging. The law does not compel drug manufacturers to provide access, and the financial implications can be substantial. Patients and families must weigh the potential benefits of an unproven therapy against significant risks, costs, and the emotional toll of pursuing treatments outside established clinical trial pathways. It is important to remember that the FDA's Expanded Access program also exists, which allows access to investigational drugs under different criteria, with FDA oversight.

Looking Ahead

Recent developments indicate ongoing efforts to refine and expand 'Right to Try' legislation. The