INTRODUCTION TO COMPASSIONATE USE
A Lesser - Known Way for Patients to Access Experimental Treatments
(CurityMD’s educational series on clinical research, part 7)
At CurityMD, we are always busy analyzing and studying research in thousands of different rare diseases. This is work being done by some of the most innovative physicians, scientists, and teams in the world, who have spent years (or even decades) trying to better understand the risks, causes, and pathways of rare conditions. Since they live at the frontier of medicine, many times their work can leave patients, family members and advocates slightly behind in the trail of new science.
Decoding research can be challenging, especially when patients are trying to understand and access new treatments, including experimental treatments that are not yet FDA approved, but which are being studied in partnership with patients and hospitals in “clinical trials.” Towards the end of clinical trials (see our earlier posts for descriptions on Phase 1, Phase 2, Phase 3 and Phase 4 studies), something called compassionate use may become an option. This is an FDA-permitted process where a single physician can petition both the FDA and the company who makes the drug or treatment to provide access for a single patient to an experimental therapy even though the patient is not a candidate or cannot get enrolled in any clinical trial.
Compassionate Use (also called “named patient programs”) are closely-monitored ways for a physician to request an experimental (pre-approval) drug for a specific (“named”) patient for a specific period of time. This is often selected as an option when the condition or the patient has an immediate, severe health risk and/or no other therapeutic options (for example, a patient may have already failed other approved treatments, or may not be eligible for an ongoing clinical trial for one or more reasons).
In compassionate use, a drug company often pays for all costs of treating the patient, in part because the company cannot charge for the drug if it is not FDA approved to treat that patient.
There is a significant amount of paperwork required for compassionate use, and it may not be accessible to all patients or physicians in all cases where a drug is in development or not approved. It is also important to note that compassionate use is not a way for large numbers of patients to access an experimental drug; because there is so much work involved, it is challenging for all parties - hospitals, doctors, regulators and drug makers - to support large numbers of patients in compassionate use programs. Still, it may be worth talking with your provider to learn more about whether this is an option to pursue.
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