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s patients clamor for greater access to experimental medicines, a survey released Tuesday finds that just 19 percent of 100 drug makers publicly post policies about their programs for obtaining these drugs, which are known as compassionate use. Moreover, only one of those companies posted information about specific procedures for making requests, and this company did not list any contact information.

More specifically 52 percent of the 25 companies with a market capitalization of greater than $10 billion analyzed by consulting firm Avalere Health disclosed policies on their web sites. By contrast, 14 percent of medium-sized companies — those with market caps between $1.5 billion and $10 billion — posted details and only 4 percent of smaller companies, with market caps of less than $1.5 billion, did the same.

The findings underscore arguments by a growing number of patient advocacy groups and lawmakers that the process for gaining access to experimental medicines is difficult, a complaint that has generated criticism of the US Food and Drug Administration and sparked social media shaming campaigns of some companies. The issue has, in turn, prompted federal and state legislation to alter the process.

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Currently, under federal law, if terminally ill patients are not eligible to obtain an experimental drug through a clinical trial, they can apply to the FDA for its expanded access program, which is also known as compassionate use. The agency actually approves the vast majority of these requests — over 99 percent of nearly 1,900 applications last year.

Although drug makers must agree to make their medicines available, patient groups and their supporters maintain the FDA program is arbitrary and cumbersome, and so have focused much of their criticism at the FDA. As a result, 33 states have passed so-called Right to Try Laws that are designed to circumvent the FDA. Federal legislation is also pending. In response, the FDA has streamlined its applications.

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But other say the frustration directed at the FDA is misplaced. That’s because drug makers are actually the final gatekeepers and may deny requests in order to meet strict criteria needed to win FDA approval for a medicine. An unexpected patient reaction, for example, might jeopardize the chance that a clinical trial will succeed. Sometimes, a company may lack sufficient supplies of their drug.

As a result, the Avalere findings should be seen as a reason to pressure the pharmaceutical industry to do more to disclose policies toward compassionate use, even if a company’s official position is not to agree to make medicines available, according to Art Caplan, who heads the division of bioethics at the New York University Langone Medical Center.

“This ought to make it clear that the FDA role is utterly secondary and corporate decision-making is the driver and primary actor in giving access to investigational products,” he said. “The FDA doesn’t get involved until a company has made a decision about granting access. And if a company doesn’t have a policy on its web site, it’s a huge obstacle to patients and doctors more than anything else.”

However, as Avalere Vice President Brenda Huneycutt noted, Congress is also considering another piece of legislation that would more broadly address health care products. The House, in fact, passed what is known as the 21st Century Cures legislation, which would require drug makers to disclose various details about a compassionate use policy. The Senate is still working on comparable legislation.

The House bill would require companies to provide procedures and contact information for making requests, the general criteria the drug maker will consider or use to approve requests, and the length of time a company anticipates will be needed to acknowledge a request has been received.

Avalere found, however, that most drug makers currently are not doing a good job of meeting those requirements right now. For instance, just three of the 19 companies that have posted their policies included information about the length of time needed before compassionate use requests are likely to be acknowledged.

“Patients need to understand what potential medicines in clinical development are available to them and be able to request access easily and fairly,” Huneycutt said in a statement. “Accessible compassionate use policies posted on company websites reduces the burden on patients to obtain this information.”

[UPDATE: We received a note from the Goldwater Institute, a conservative think tank that has written many of the Right to Try laws. “While we encourage companies to make their compassionate use policies public, we believe that the most important first step to creating a transparent compassionate use system is for the FDA to make the decision process for deciding who gets access to potentially life-saving treatments and who does not,” said Naomi Lopez Bauman, the health care policy director, which she added has tried to get the FDA to make this information public.

“The FDA should clearly state in policy how adverse events under compassionate use will affect the ongoing clinical trial process for companies. While the FDA claims that this is a rare occurrence, the reality is that the FDA’s failure to issue clear policy leaves companies in a precarious position of jeopardizing their investments. If companies had clarity from the FDA about how offering drugs under the compassionate use program would impact ongoing trials, they’d be more likely to make their policies public and, possibly, to participate.”]

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  • It is impossible for a drug company to craft a one size fits all policy for compassionate use. The protocol depends on many things, including size of patient pool and availability of drug, since many times supplies are limited and by definition therapy must be rationed based on demand. I ran a compassionate use program for NYHA Class 3 and 4 CHF for a novel vasodilator in the 1990’s. The drug was being discontinued so supplies were not abundant. We had a specific protocol for that drug alone. Each new compassionate use drug requires its own set of inclusion criteria based on the above criteria. Even if there were to be a general policy companies would be reluctant to publish it lest they be besieged with phone calls, which happen soon enough anyway once doctors know of its availability.

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