The Food and Drug Administration's expanded access program and state right-to-try laws seek to provide terminally ill patients with access to experimental drugs.
Elizabeth Dzeng, MD, PhD, assistant professor at the University of California San Francisco School of Medicine, and scholar Amy E. Scharf, penned a Health Affairs blog providing a detailed breakdown of the expanded access program and right-to-try laws.
Here are eight things to know.
Compassionate Use
1. The FDA's expanded access — or compassionate use — program allows patients with serious or life-threatening illnesses to use drugs not yet approved by the agency.
Physicians file paperwork with the FDA to request access to an experimental drug on behalf of the patient, according to the report. To qualify for expanded access, a patient must be suffering from an illness in which "there is reasonable likelihood that death will occur within a matter of months or in which premature death is likely without early treatment," according to FDA regulations. The patient must also have already tried all other treatment options and be ineligible to participate in the experimental drug's clinical trial.
2. In response to concerns regarding physicians being deterred from applying for compassionate use due to the application's complicated nature, the FDA simplified the document in June 2016 by creating a patient-specific format and scaling back the number of questions from 26 to 11.
3. The agency's Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research received 1,757 total expanded access requests during fiscal year 2016. CDER received 1,554 of those requests and approved 99.4 percent. CBER received the remaining 203 requests and granted 96.6 percent, reports Regulatory Focus.
4. While the FDA can grant a patient access to the experimental drug, it cannot force drug companies to supply the drug to the patient, according to the report. Drugmakers must consider numerous factors when deciding whether to dispense their drugs, including supply constraints, financial implications and impacts on regulatory matters if poor patient outcomes occur, according to the Health Affairs blog post.
Right-to-try laws
5. Thirty-three states have enacted right-to-try laws, which allow patients to receive unapproved treatments directly from drugmakers, according to Regulatory Focus. The laws aim to accelerate patients' access to experimental treatments by bypassing any involvement from the FDA.
6. There are two bills in the Senate and House of Representatives that seek to enact a federal right-to-try law, which would bar the FDA from denying patients access to an experimental drug. Vice President Mike Pence in February met with families advocating for federal right-to-try legislation and said he and President Donald Trump supported the bills.
7. Like the compassionate use program, state right-to-try laws do not require drug companies to supply their experimental therapies to patients. No published evidence exists showing these state laws have successfully expanded patient access to experimental drugs since drugmakers are not required to release data on how many requests they receive or grant, according to the report.
8. Opponents of the state-level legislation believe right-to-try laws are unconstitutional since they are preempted by the FDA's federal authority over drug regulation, according to the report. Proponents of the laws argue that individual states have the constitutional right to offer additional or heightened protections of individual rights, including medical rights, according to the report.
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