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  • Health Affairs Blog, Ellen Sigal

How Do We Protect Patients From False Promises In Right-To-Try Laws?

My sister Gale had exhausted every option. Metastatic cancer raged through her body, defeating all conventional treatments. She faced a final decision: succumb to the disease, or wage one last battle with an experimental bone marrow transplant known to kill 20 percent of patients. Gale chose to fight, opting to use the unproven therapy at a time when institutional review boards and scientific peer review regulated experimental therapies rather than the Food and Drug Administration (FDA). Now, three decades later, the FDA has an expanded access policy, also known as “compassionate use,” that seeks to ensure the quality of unproven therapies used by dying patients. In Gale’s case, the side effects of the treatment were swift and violent. Within two days, she was dead. Clinical trials have since demonstrated that the therapy had limited efficacy and a greater risk of lethality than reported at the time.

Legislation before Congress seeks to grant all terminally ill patients the “Right to Try” experimental therapies once approved alternatives have failed. Although the FDA authorizes 99.5 percent of compassionate use requests, advocates of Right to Try claim the process is too slow. The FDA has streamlined the current process so that requests are reviewed within 24 hours; filling out an application now takes less than an hour. And yet, Right-to-Try laws have been approved by more than 30 statehouses. Unfortunately, the current federal legislation provides almost no protections for patients.

First, provisions for informed consent are essential. Most early-phase drugs are dangerous and ultimately proven ineffective, with upwards of 90 percent never brought to market... Provisions insuring informed consent would guarantee that patients requesting expanded access can judge the magnitude of their decision.

Second, the limits of Right to Try must be clear. Even if patients receive the right to request an experimental therapy — under the current paradigm or a new Right-to-Try regime — the drug company developing the therapy is under no obligation to provide it. There are legitimate reasons for this, including supply shortages, a lack of financial incentives, and concerns that negative “compassionate use” outcomes could be used by the FDA to delay or deny approval (which is protected against by the Right-to-Try legislation). Development roadblocks would stop therapies from reaching patients. However, the FDA cannot simply ignore expanded access outcomes. Patients petitioning for expanded access deserve accurate information about whether the potential benefits of an experimental treatment outweigh the risks. What are the side effects? What are the chances of success? This highly personal calculus is impossible if drug companies do not monitor and report side effects.

A key component is transparency... In its current form, however, Right to Try does nothing for patients other than allow them to request a drug they may never receive and that may be more likely to hurt them than to help them. Unless Right to Try is significantly revised, this law will be a threat to patient health and well-being.

Read the full article here.

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