Clinigen: Navigating risks and benefits of early access to novel drugs

October 3, 2014 ctheodoropulos

The more pharma and biotech companies focus on novel molecules for unmet medical needs and rare diseases, the greater the demand for early access to these promising drugs, well ahead of market authorization.

Patients are driving this movement, said Mark Corbett, Senior VP for Clinigen Global Access Programs. “They have greater access to information. There are very strong patient organizations, particularly in rare diseases. These groups are actively working with their physicians, pharma and biotech companies to gain early access to novel drugs because they no longer have alternatives, they are out of options. And they can create a huge, very public pressure on the companies to make drugs available earlier.”

Mark Corbett, Clinigen Global Access Programs

Mark Corbett, Clinigen Global Access Programs

Corbett offers the example of seven-year-old cancer survivor Josh Hardy, who was denied access to a novel medication. His parents launched a very proactive campaign on Facebook and Twitter, which was picked up in March, 2014 by The Boston Globe and The New York Times.  Under pressure, the company backtracked and opened a clinical trial for just this one patient, he said. It is reported that after receiving the drug Josh had a miraculous recovery.

“Early access is a widely used mechanism, yet it is an area that is relatively unknown and sometimes misunderstood among people within pharma,” said Corbett.

At BIO-Europe® 2014 in Frankfurt, Corbett will join a panel discussion on Monday afternoon entitled, “Mind the GAP? Meeting an emerging demand for access to medicines in late-stage clinical trials, prior to commercial launch.” The pharma perspective will be represented by Markus Kosch, Lead Oncology, Europe, Africa and Middle East, Pfizer; and biotech by Luc Dochez, Chief Business Officer, Prosensa. Steve Bates, CEO of the UK’s Bioindustry Association, will moderate the panel.

In an interview with partneringNEWS Mark Corbett teased out some of the issues the panel will be discussing in depth, starting with the risks and benefits of an early access program.

The first principle for discussion, he said, is understanding that early access to a drug is outside clinical development of that drug.

“Early access is not a clinical trial and does not replace the clinical trial process. Instead it is a program that utilizes a different set of regulatory mechanisms that sit alongside the clinical development program and should be considered as part of an overall strategy.”

The primary benefit of creating early access to a novel entity is that the pharma company is seen as doing the right thing by providing patients with access to a drug that may essentially be their last hope.

Another compelling, commercial benefit, he said, is that “if you have a wider groups of physicians and patients with access to your drug prior to commercial launch, at the point when you go to launch there will be benefits in terms of the number of people who have experience with the drug beyond those who participated in the clinical trial.”

Another benefit that could be  attractive to smaller companies without any income, said Corbett, is that in some countries a company can charge for the drug prior to authorization and gain some revenue through making the drug available early. This can potentially cover the cost of making the drug available through an early access program.

As for the risks of early access programs, Corbett commented on a series of potential situations that need to be considered.

“The biggest fear for companies in considering early access is  that something going wrong,” he said. “What happens if the drug  is used with inappropriate patients, or if it is used in appropriate patients in an inappropriate manner? What happens if its use creates adverse events? This is where we work through education, patient information, training of physicians, patient selection and making sure the early availability is very controlled and monitored to mitigate the risk of anything untoward occurring.”

Another risk is that the drug does not progress in development, yet having made the drug available early there is now a group of patients who are getting a benefit from the drug. What is the exit strategy? At what point will a company stop providing the drug? This is part of the planning that companies need to think about so they go into an early access program with their eyes open, he said.

A third risk to be considered is what happens if no one wants to use the drug.  If a company has a novel entity and makes it available early, what do they do if there are no requests for it? This can represent a risk in terms of a company evaluation.

In the past seven years Corbett and his colleagues at Clinigen Global Access Programs (Clinigen GAP) have worked on almost 150 programs to make pre-license products available for patients on a compassionate use or named patient basis. It is a complicated, highly specialized area that does not fit neatly within traditional divisions at pharma companies; sometimes the responsibility falls in a clinical area, sometimes with the medical group, and at least once, Corbett said, it fell to the supply chain group.

Among the top 20 pharma companies, he said all but two have managed an early access program, either outsourcing the responsibility or managing it internally.

Clinigen GAP is a leading provider for outsourcing services for early access that Corbett said “involves everything from planning, to setting up a program, to physically getting the drug to the right patients in a compliant manner and reporting results back to the pharma client.”

He added, “Often a pharma company doesn’t really want to do this itself as the priority is clinical development and bringing the drug to market. Because it is often done on a patent by patient basis, it can be extremely distracting, whereas we specialize in this and are set up to be responsive.”


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