Access to Experimental Drugs

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Experimental drugs can be a lifeline for those with conditions for which conventional treatments are not working or readily available. At the same time, doctors and patients want to have confidence that they are making safe and informed choices when requesting the use of drugs that have not have fully completed the clinical trial process.  With many doctors turning to experimental treatments for COVID-19 patients, this topic has recently gained renewed attention. Two processes in particular: compassionate use and Right to Try, give patients with serious or terminal conditions the ability to gain access to treatments which have not received FDA approval. These treatments can be potentially lifesaving for those whom conventional treatment is either unavailable or not working.

Compassionate use, formally known as Expanded Access, allows patients access to experimental treatments with approval from their doctor, the FDA, and an Institutional Review Board (IRB). The patient must meet several criteria, including having a serious disease or condition for which no comparable satisfactory treatment is available and being unable to enroll in clinical trials for the drug. If patients decide to pursue compassionate use, they must first speak to their physician, who then files a request to both the FDA and IRB. Eligibility is granted by the FDA if the agency determines that both (1) the benefits of using the experimental treatment outweigh any risks and (2) that its use will not jeopardize ongoing clinical trials. The FDA must respond to such a request within thirty days, and approval has historically been granted in 99% of cases. Secondly, the use must be approved by a registered IRB. Only once both of these steps are complete is the patient allowed access to the drug.

In May 2018 Congress passed the Right to Try Act, establishing a second process for patients to gain access to experimental treatments. Under Right to Try, patients with life-threatening conditions no longer need the approval of the FDA and IRBs to gain access to certain experimental drugs. Those with a terminal illness can submit a request directly to the manufacturer of an experimental treatment with the consent of their doctor. They may then obtain the treatment if the manufacturer approves. Similar to with compassionate use, patients using the Right to Try process must have exhausted all available conventional treatments and be unable to participate in clinical trails involving the experimental drug. The medication must also have completed Phase 1 clinical trials, an additional requirement not present in the compassionate use process. Right to Try has proven controversial in the scientific community since its conception. Proponents claim it cuts red tape patients for terminally ill patients in obtaining treatment, but critics point to the lack of reporting requirements and say that Right to Try removes safeguards in the compassionate use process that ensure patient safety.

While compassionate use and Right to Try have different processes, the programs share several key limitations. Importantly, manufacturers have no obligation to provide experimental drugs under either process, and the FDA has no power to compel companies to do so. Many manufacturers are reluctant to provide patients access to experimental drugs due to concerns that adverse reactions could lead to negative publicity or interfere with the FDA approval process.  Additionally, insurance companies are under no obligation to cover experimental treatments. The fact that many patients must pay out-of-pocket for experimental treatments has led to a stratified system where low-income patients are unable to obtain experimental drugs.

Despite these constraints, both compassionate use and Right to Try continued to be utilized by doctors and patients across the country. Many individuals with severe or terminal illness can’t afford to wait the seven to twelve years it takes for drugs to be approved by the FDA. Through compassionate use and Right to Try, these patients with few options left get a renewed chance.