In a nod to patients clamoring for greater access to experimental medicines, the 21st Century Cures legislation would require companies to publicly disclose their policies for making such drugs available.
The language in the bill, which is designed to jump start medical innovation, appeared after complaints that many drug makers make it difficult for patients and their physicians to sort out the process for gaining access to medicines that are not yet approved for use by the US Food and Drug Administration.
A recent survey found that only 19 of 100 drug makers publicly post policies about their programs for obtaining experimental medicines. Moreover, only one of those companies posted information about specific procedures for making requests, and this company did not list any contact information. And many of the largest companies do a poor job disclosing their policies, according to the survey by the Avalere Health consulting firm.
The legislation would require drug makers to provide procedures and contact information for making requests, the general criteria a company will consider or use to approve requests, and the length of time anticipated to acknowledge a request has been received. Companies must also post a link or reference to a clinical trial record containing information about expanded access to any of its drugs (see section 3032).
However, the consulting firm found that most drug makers currently are not doing a good job of meeting those proposed requirements at the moment. 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.
The provision in the bill is “a big deal,” said Arthur Caplan, who heads the division of bioethics at the New York University Langone Medical Center and is working with Johnson & Johnson to develop a compassionate use program. “Patients and doctors complain they do not know who to approach with requests, what policies companies have and when they can expect to get an answer.”
“I have argued for a year and a half that requiring posting – even if it is to say a company won’t do any compassionate use or expanded access or only if the cost of a drug or device is covered – is a big step forward for patients,” he explained. “At least they know what is going on and then can appeal it or challenge it or move on to something else.”
The survey found that, of 25 companies with a market capitalization of greater than $10 billion, only 52 percent 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.
As we noted at the time that the survey was released, the findings underscored arguments by patient advocacy groups and lawmakers that the process for gaining access to experimental medicines is unwieldy, which has also generated criticism of the FDA and sparked social media shaming campaigns of some companies. The issue has, in turn, prompted federal and state legislation to alter the process.
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 approves the vast majority of these requests — over 99 percent of nearly 1,900 applications last year.
But while 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 Right to Try laws designed to circumvent the FDA. Federal legislation was also introduced. In response, the FDA has streamlined its applications.
Yet the frustration directed at the FDA appears off base, because drug makers are the ultimate gatekeepers and may deny requests in order to meet strict criteria needed to win FDA approval for a medicine. For instance, an unexpected patient reaction may jeopardize the chance that a clinical trial will succeed or a company may lack sufficient supplies of their drug.
The Goldwater Institute, a conservative think tank that has written many Right to Try laws, maintained that FDA policy needs to be revamped. In particular, the think tank argued that the agency should “clearly state” how any adverse events that occur during a compassionate use program will affect ongoing clinical trials.
The failure to issue such a policy leaves companies in a “precarious position of jeopardizing their investments,” Naomi Lopez Bauman, the health care policy director at the think tank, wrote us. She suggested that companies would be more willing to make policies public and, possibly, participate in expanded access programs if the FDA offered more clarity.