Demand Builds for Access to Experimental Drugs

Andrew D. Smith
Published: Tuesday, Jan 16, 2018
Dr Bruce E. Johnson
Bruce E. Johnson, MD
Right-to-try (RTT) legislation, which allows dying patients to take investigational medications without approval from the FDA, is on a roll. Legislatures in 39 states have approved such laws in recent years, as has the US Senate, and bills are pending in all other states1 and the US House of Representatives (FIGURE).


Figure. Status of Right to Try Legislation on State Level

The concept is far less popular, however, among academic researchers, learned societies, professional regulators, and other members of the medical establishment. Some objectors believe that RTT laws endanger patients. Others think the laws could hinder the FDA’s ability to weigh the harms and benefits of experimental drugs. Still others think that RTT laws are pointless bromides that leave the real obstacles to patient access fully intact.

There is, however, one aspect of RTT that generally wins the establishment’s support: the underlying goal. Organizations such as the American Society of Clinical Oncology (ASCO) and the American Cancer Society want to make it easier for people who have run out of approved treatments to receive promising new drugs that just might deliver a miracle.2,3 They don’t say, however, that RTT laws provide the best means of maximizing patient participation, benefit, and safety.

“The question isn’t whether patients should have better access to investigational agents. It’s whether right-to-try laws are the best way to improve access— and we don’t think that they are,” said Bruce E. Johnson, MD, president of ASCO, who also heads clinical research efforts at Dana-Farber Cancer Institute in Boston, Massachusetts.

Researchers outside the FDA have no real way to evaluate the efficacy of the current system for granting terminal patients access to investigational medicines. The agency has procedures in place through its expanded access program, sometimes called “compassionate use,” for 3 categories4: individual patients; intermediate-size populations, defined as smaller than the typical cohort for an investigational new drug (IND) of 20 to 80 patients5 or for a treatment protocol; and widespread use through a treatment IND or protocol. The FDA does not disclose exactly how many patients participate in the program each year, and it does not publicize any information about disease response or adverse events.

The FDA reports that it receives about 1400 requests a year, about half of them for individual patients and half for groups. The agency also has reported that it approves more than 99% of all applications.6 Unfortunately, the FDA does not disclose the size of each group, so there’s no way to know if it grants expanded access to 4500 patients a year or several times that number.

Either way, the total number is certainly tiny compared with the number of Americans who die as a result of diseases each year. Cancer, the second most common cause of death in the United States after heart disease, is expected to kill more than 600,000 people in 2017, accounting for nearly 1 of every 4 deaths.7 Although the expanded access program is open to patients with any serious medical condition, the oncology field is particularly affected.

It is, of course, impossible to compare experimental drug usage across nations when there are no concrete American figures, but compassionate use programs in several other countries seem to have far higher participation rates. France, for example, has just over a fifth as many residents as the United States, but it provided 6000 dying patients with access to investigational medications in 2013.8 This suggests to some that the number of Americans who would want experimental treatments may greatly exceed the number who get them under the current system. Others explain the discrepancy, in part at least, by asserting that the United States now approves breakthrough treatments faster than many nations and thus has less need of compassionate use.

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