As the medical community searches for the best and most effective COVID-19 treatments, many patients are seeking life-saving treatment with drugs approved for compassionate use. The decision by patients, doctors, and pharmaceutical companies to make drug therapies available before FDA-approval has a long history in the U.S, and it’s drawing more attention now than ever due to the coronavirus pandemic.
What is Compassionate Use?
Compassionate use, also known as expanded access, is the use of therapeutic drugs that have not yet received FDA approval.
According to the FDA, compassionate use offers a way for patients with “an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product for treatment” when no other therapy options exist.
Under normal circumstances, investigational drugs are only available to clinical trial participants. Expanded access and compassionate use make it possible for other high-risk patients to use investigational drugs under specific conditions. This requires the active cooperation of the FDA, drug companies, and health care providers.
The History of Compassionate Use
The concept of compassionate use originated in clinical trials. During Phases II and III, clinical trial research teams request volunteers with specific diseases and conditions in order to test the efficacy of their investigational drugs compared to existing treatments. This gives sick participants early access to therapeutic drugs that would otherwise be unavailable until they were approved by the FDA.
However, not all patients with all diseases can participate in clinical trials, leaving many patients unable to take advantage of potentially life-saving treatments. This led the FDA to begin facilitating access to investigational drug therapies in the 1970s. By 1987, regulations were enacted to create a pathway for access to unapproved drugs outside of the clinical trial setting.
Today, the FDA’s compassionate use program is available to patients who meet the following qualifications:
- No approved treatments are available in the patient’s home country
- If there are approved treatments available, the patient has tried them, and they were ineffective
- The patient is not eligible to enroll in an active clinical trial
Eligible patients can request approval for expanded access to drugs through their physicians. A licensed physician must determine whether an investigational drug is available on a compassionate use basis, then contact the drug manufacturer and submit an application to the FDA. If the drug company and FDA both provide preliminary approval, an Institutional Review Board (IRB) reviews the expanded access protocol before the FDA grants final approval.
The “Right to Try” Act of 2018
In addition to the FDA’s Expanded Access program, terminally ill patients can also gain access to investigational drugs through President Trump’s Right to Try Act passed in 2018.
According to the White House, the Right to Try Act amends Federal law to allow certain unapproved, experimental drugs to be administered to terminally ill patients who have exhausted all approved treatment options and are unable to participate in clinical drug trials.
The law seeks to provide faster, easier access to experimental drugs in cases when the FDA’s expanded access program takes too long. Drugs made available through the Right to Try Act must adhere to the following guidelines:
- Successfully passed Phase I clinical trial, which tests safety
- Manufacturer of the drug must report to the FDA on any use of the drug on a “Right to Try” basis
Overall, President Trump approved the Right to Try Act as a way to supplement the FDA’s compassionate use application by giving terminally ill patients new avenues to make healthcare choices that could save their lives.
Hospitals Currently Allowing Hydroxychloroquine as a Compassionate Use Drug
As Americans face the COVID-19 pandemic without any FDA-approved treatment drugs, the medical community has responded by launching dozens of new clinical trials for potential treatments and vaccines. In the meantime, the FDA has granted hydroxychloroquine compassionate use status in treating coronavirus.
This means that hydroxychloroquine is officially available through the Expanded Access program and Right to Try Act. COVID-19 patients with serious or life-threatening cases of the virus now have access to a potentially life-saving treatment, and many hospitals have stockpiled hydroxychloroquine to ensure rapid treatment.
Governor Andrew Cuomo of New York said on April 6 that state officials have allowed use of the drug in combination with the antibiotic Zithromax in hospitals “at their discretion.” Other hospitals in Michigan, Louisiana, and states across the country have also reported promising anecdotal results from patients treated under compassionate use.
Other Drugs Used For Compassionate Care
Dozens of drugs have been used for compassionate care in the past and ultimately approved for front line care.
For example, the pharmaceutical drug Belatacept was available through compassionate use from July 2008 to June 2011 for high-risk renal transplantation patients. Belatacept was approved by the FDA on June 15, 2011 and is now sold as Nulojix to weaken the body’s immune system and keep it from rejecting a transplanted kidney. Nearly a decade later, studies show that Belatacept significantly improves renal function in kidney-transplant recipients.
Other approved drugs previously available through the Expanded Access program include:
- Gemtuzumab Ozogamicin for adult acute myeloid leukemia
- Intravenous fish oils for patients with parenteral nutrition liver injury
- Cabozantinib for medullary thyroid cancer
- Migalastat for Fabry disease
- Teprotumumab for thyroid eye disease
- Voxelotor for Sickle Cell disease
At the moment, there’s one other drug also approved for compassionate use treatment with patients fighting extreme cases of coronavirus: remdesivir. This antiviral drug is believed to prevent infected cells from reproducing. The drug’s manufacturer, Gilead Sciences Inc, is donating at least 1.5 million doses of the experimental drug for use in compassionate use and expanded access cases, which could treat up to 140,000 patients.