The federal Right to Try Act was passed in May of 2018. The Act provides federally-protected rights for access to potentially life-saving investigational drugs prior to FDA final approval. These protected rights are for patients in the United States who suffer from life-threatening diseases or conditions (severely debilitating). Many of these ill patients have tried approved drugs on the market but have not had any success in relieving their severe symptoms and/or disease progression. These patients continue to seek novel treatments including participation in clinical trials that may hold promise as a new therapeutic treatment for their disease or condition.
Right to Try allows patients access to treatments that are in the FDA’s drug approval pipeline and have demonstrated safety in a phase 1 study. These investigational drugs are only available to a select few clinical trial participants, and many will not even qualify for a trial. Additionally, the Act allows any eligible patient in the United States to appeal directly to drug manufacturers for access to these investigational drugs. Patients and their doctors work with the company that is developing a drug or biologic to request access to the drug treatment. The manufacturer is not required to provide access as they alone make this decision. There is no involvement of the FDA in the process as they are prevented from reviewing or approving any Right to Try request1.
The Act formally enables eligible patients the right to appeal directly to a drug manufacturer for access to an investigational drug. If the company grants the request, the patient provides informed consent before the product is used2. Under Right to Try, patients work hand in hand with their physicians to determine a course of treatment that fits their individual health needs. The Right to Try Act was intended to remedy some of the inequities created by the FDA’s approval process, which often ignores the rights of terminally ill patients making decisions about their own care3.
Over the coming weeks we will be covering Right to Try topics that we hope will educate, simplify, and empower patients with information about eligibility criteria, investigational drugs, stem cell treatments, and the role of regenerative medicine under the Right to Try Act.