Compassionate Use
What Is Compassionate Use?
Compassionate use, often referred to by the Food and Drug Administration (FDA) as "Expanded Access," is a regulatory pathway that allows patients with serious or immediately life-threatening diseases to access investigational medical products (drugs, biologics, or medical devices) outside of clinical trials when no comparable or satisfactory alternative medical treatments are available. This framework falls under healthcare policy and regulatory approval, providing a potential avenue for patients who have exhausted standard treatment options. Compassionate use allows individuals to receive treatments that are still in the drug development process and have not yet received full market authorization.
History and Origin
The concept of providing experimental treatments to desperately ill patients has roots dating back to the 1970s. However, the formalization of compassionate use, or expanded access, gained significant momentum in the United States during the HIV/AIDS crisis of the 1980s.7 Patient advocacy groups, facing a devastating epidemic with limited approved treatments, exerted considerable pressure on the FDA to make promising experimental drugs more widely available.6 This activism led to the establishment of formal regulations in 1987, creating a structured pathway for access to unapproved drugs.5 Since then, the FDA has continued to refine its Expanded Access program, streamlining the application process to facilitate access for patients in urgent need.4
Key Takeaways
- Compassionate use allows patients with serious or life-threatening conditions to access unapproved medical products outside of clinical trials.
- It is typically pursued when no alternative satisfactory treatment options exist, and the patient is ineligible for ongoing clinical trials.
- The process requires agreement from both the treating physician and the manufacturer, along with FDA authorization.
- The FDA's Expanded Access program has a high approval rate for requests once submitted.
- The goal is to offer potential treatment when standard care has failed, balancing patient needs with the integrity of the overall drug development process.
Interpreting Compassionate Use
Compassionate use is not a substitute for formal clinical trials, which remain the primary method for establishing the safety and efficacy of new medical products. Instead, it serves as a safety net for patients in dire situations. When evaluating a compassionate use request, the FDA considers criteria such as the seriousness of the patient's condition, the lack of alternative therapies, the potential patient benefit versus the potential risk-benefit analysis of the investigational product, and whether providing the product would interfere with ongoing clinical investigations. The process involves a careful balancing act between immediate patient needs and broader public health interests.
Hypothetical Example
Imagine a patient, Sarah, diagnosed with a rare, aggressive form of cancer for which all standard medical treatments have failed, and she is not eligible for any ongoing clinical trials due to her specific disease markers or advanced stage. Her oncologist learns of an experimental drug being developed by a pharmaceutical industry company that has shown promising results in early-stage trials for a similar type of cancer.
The oncologist, in consultation with Sarah and her family, determines that this experimental drug represents the last potential option. The oncologist then contacts the drug manufacturer to inquire about compassionate use. If the manufacturer agrees to provide the drug, the oncologist submits an Expanded Access request to the FDA, detailing Sarah's condition, previous treatments, and the rationale for seeking the investigational drug. After reviewing the case, the FDA authorizes the compassionate use, allowing Sarah to receive the unapproved medication. This process provides Sarah with a chance at treatment while acknowledging the drug's unproven status.
Practical Applications
Compassionate use programs primarily show up in the healthcare and pharmaceutical industry sectors, impacting patient advocacy, clinical practice, and regulatory affairs. For pharmaceutical companies, navigating compassionate use requests is a critical aspect of their investor relations and public image, as it demonstrates a commitment to patient welfare. It can also influence a company's market access strategies as they consider the broader implications of providing unapproved therapies. The processes involved, including agreement from the manufacturer and regulatory approval from the FDA, highlight the regulatory complexities for manufacturers.[3](https://www.raps.org/news-and-articles/news-articles/2014/2/regulatory-explainer-fda-s-expanded-access-(compa)
Limitations and Criticisms
Despite its humanitarian intent, compassionate use faces several limitations and criticisms. A primary concern is that accessing unapproved drugs through this pathway does not guarantee effectiveness or safety; patients may experience significant side effects without any therapeutic benefit.2 Manufacturers are not obligated to provide investigational products, and their ability to do so may be limited by drug supply, potential interference with ongoing clinical trials, or the financial burden of providing the drug. The costs associated with administering the drug and patient care are typically not covered by insurance or the manufacturer, placing a substantial financial strain on patients and their families. Furthermore, the Abigail Alliance v. von Eschenbach case highlighted the legal limitations, as courts have affirmed that there is no constitutional right to access unapproved drugs.1 Striking a balance between patient desperation and sound ethical considerations remains a continuous challenge.
Compassionate Use vs. Right-to-Try Laws
Compassionate use is often confused with right-to-try laws. While both aim to provide access to experimental treatments for patients with serious or terminal illnesses, they operate under different regulatory frameworks.
| Feature | Compassionate Use (Expanded Access) | Right-to-Try Laws |
|---|---|---|
| Oversight | Requires Food and Drug Administration (FDA) approval. | Bypasses FDA approval, allowing direct negotiation between patient/physician and manufacturer. |
| Intent | Provides a regulatory pathway for individuals in urgent need. | Aims to assert a patient's right to try experimental treatments without federal regulatory hurdles. |
| Ethical Review | Generally requires Institutional Review Board (IRB) approval. | Does not require IRB review at the federal level, though some state laws may differ. |
| Manufacturer Role | Manufacturer must agree to provide the unapproved drugs and work with the FDA. | Manufacturer is not obligated to provide the drug and typically bears no liability if the drug is used under these laws. |
The key distinction lies in the role of the FDA; compassionate use operates within the FDA's regulatory framework, while right-to-try laws seek to circumvent it, asserting a perceived right to access without federal oversight.
FAQs
Who is eligible for compassionate use?
Patients are typically eligible for compassionate use if they have a serious or immediately life-threatening disease or condition, have exhausted all available comparable or satisfactory medical treatments, and are unable to participate in a clinical trial. The potential benefits must outweigh the potential risks, and providing the experimental product should not interfere with the ongoing drug development process.
How does a patient apply for compassionate use?
The process for compassionate use generally begins with the patient's treating physician. The physician must determine if the patient meets the criteria, identify a suitable investigational product, and then secure the cooperation of the manufacturer to provide the drug. Once the manufacturer agrees, the physician submits an Expanded Access request to the Food and Drug Administration for authorization.
Does insurance cover compassionate use drugs?
Typically, health insurance does not cover the cost of the investigational drug itself or the associated medical care when provided through compassionate use. This can lead to significant financial burdens for patients and their families, as they may be responsible for the full cost of the experimental treatment and related medical expenses.