FDA's "Compassionate Use" Gets Simpler: What Will This Mean For Drug Manufacturers?

By Anna Rose Welch, Editorial & Community Director, Advancing RNA

Since the beginning of the year, there have been a number of stories crossing my desk focusing on patients’ “Right to Try” experimental drugs.  But I’m also curious what this will mean for drug manufacturers who will no doubt be faced with more of these requests in the future.

Just last week, the FDA announced changes to its “compassionate use” policy that will make filing an application an easier and quicker experience for the patient. The FDA has had its “expanded access program” in place since 1987, but patients have often complained that it takes too much time to see a turnaround on their application. In fact, according to Regulatory Affairs Professional Society (RAPS), Form 1571, which required 26 different types of information and seven attachments, could take physicians 100 hours to complete.   

However, the FDA has now replaced Form 1571 with Form FDA 3926 that calls for only eight types of information and one attachment — all of which the doctor should be able to complete within 45 minutes. (They weren’t kidding about cutting it down.)   

But the long hours in front of the form weren’t keeping patients/physicians from applying. Last year, the FDA saw a staggering increase in the number of “Right to Try” requests. According to RAPS, the regulatory agency saw a 92 percent increase in requests — the largest jump in apps since 2010 — and it approved 1,873 emergency access requests. The agency only rejected 9 applications total in 2014.

According to the New York Times, “Right to Try” laws have emerged in various states in the nation, including Colorado, Michigan, Missouri, Louisiana, and Arizona, and there are more expected to come in 2015. Kansas, Tennessee, Texas, and Wyoming have already filed these bills, and there are around 10 others that have announced their plans to do so as well.

But the whole process can be dangerous territory for drug companies. Bloomberg Businessweek recently published a piece on the former CEO of biotech Chimerix, Ken Moch, who was faced with the tough decision of whether to grant access to an experimental drug for a young boy suffering from kidney cancer. The family, after being denied access twice, turned to social media. A popular Facebook page, a trending hashtag, as well as thousands of emails, phone calls, and several death threats to CEO Moch later, Chimerix worked out a plan with the FDA to grant early access.

While in the end, the young boy was given the treatment, the increasing amount of compassionate use applications will put smaller biotechs and companies that have no product yet on the market in a tricky situation. While granting access to those requesting the drug is putting the patient first and fulfilling the industry’s goal, giving out the experimental drug for free, as well as diverting resources to help patients that aren’t enrolled in the formal trial can both lead to higher costs and delay future FDA approval. Not to mention, should the patient react poorly to the drug, the FDA could halt the trial. (In the case of Chimerix, the situation also led to the dismissal of the company’s CEO, Bloomerg reported.)  

Social media holds great promise for gathering data and engaging patients. However, the prominence of patients’ voices in these channels is another potential complication to keep in mind as the compassionate use process becomes more popular. BIO CEO, James Greenwood, told Bloomberg, “When you add social media and start tweeting all over the place, then reason flies out of the window. Social media creates opinion storms, we all know that.”  

Considering the large number of applications the FDA received last year, it behooves companies to have some kind of plan in place to ensure they’ve outlined how compassionate use will be approached, and how they will manage “crisis” situations. BIO has already begun to help member companies prepare, Bloomberg says, while PhRMA has released “Principles on Expanded Access to Investigational Drugs,” a set of “commitments” members must consider when deciding whether or not to grant access to a drug.

PhRMA also says that companies should establish methods, whether these be online or telephone-based sources, to educate and ensure open communication between companies and healthcare providers about expanded access programs.