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Regulatory Focus™ > News Articles > FDA’s Nonprofit Arm Plots Out New Compassionate Use Navigator

FDA’s Nonprofit Arm Plots Out New Compassionate Use Navigator

Posted 07 April 2016 | By Zachary Brennan 

FDA’s Nonprofit Arm Plots Out New Compassionate Use Navigator

Patients and physicians desperate for access to a potentially life-saving investigational drug will be able to take some solace in the fact that the US Food and Drug Administration’s (FDA) nonprofit, known as the Reagan-Udall Foundation, is currently working on an online tool to help aid the process of obtaining what are known as "compassionate use" or "expanded access" treatments.

The small foundation, which is currently operating with six employees, has drafted a plan for Expanded Access Navigator, which is expected to break down some of the barriers and technicalities associated with helping patients and physicians seek compassionate use of a drug in a clinical trial or one that is not currently marketed.

Nancy Beck, Ph.D., Acting Deputy Director of the Reagan-Udall Foundation, told Focus in an interview on Thursday that the Navigator is a priority of FDA’s and that it will offer a thorough explanation of what compassionate use and expanded access mean, what forms need to be completed and resources to contact to seek access to the investigational treatments.

Right to Try

The need for such a navigator comes as more than 20 states since 2014 have introduced “Right to Try” bills that aim to help terminally ill patients or those with no treatment options gain access to potential drugs that are still undergoing clinical trials. But what’s unclear about these new laws is whether they are actually helping patients gain access to investigational therapies.

The Goldwater Institute, which has pushed for the passage of such legislation at the state level as a way around what they believe to be an abundance of red tape at FDA, has said in recent weeks that it’s aware of physicians and patients accessing investigational drugs because of Right to Try legislation, though none of the physicians or patients are willing to come forth with details.

And often times, requests for early access to a developing drug are denied because the patients don’t meet certain criteria established by the company running the trial.

The difficulty for many of these patients who have either run out of treatment options (or didn’t have any to begin with) isn’t getting FDA to sign-off on the use of the investigational drug (FDA signs off on more than 99% of the expanded access requests it receives) but in getting the companies running the clinical trials to allow new patients to gain access to the experimental treatments while trials are ongoing or even before a trial begins.

What’s tricky is that companies with expensive therapies in development have to make concessions for such new patient requests, which can potentially disrupt a trial, as has happened in the past.

And one of the reasons why FDA’s so willing to sign off on almost every expanded access request that it receives is because all of the nitty-gritty work with getting a company developing an investigational drug or biologic to sign off on a patient’s compassionate use has already been finished by the time an expanded access application has been submitted to FDA, Beck told us.

New Navigator

But what this navigator will do is help provide physicians (as they are the ones that must make the expanded access request) with a more complete picture of how to make a single patient Investigational New Drug (IND) request; the role of FDA, manufacturers and Institutional Review Boards in the process; the principles of expanded access eligibility criteria and risk-based decision-making; the challenges of translating early research findings into clinical outcomes; and perhaps most importantly, a directory of information on FDA and manufacturers’ policies, procedures and points of contact.

Beck said Reagan-Udall is still ironing out the specifics of the navigator and whether it might contain a database of potential drugs as many larger pharmaceutical and biotech companies already have a unique website or call center to help patients understand how expanded access works.

The Reagan-Udall Foundation will hold a public workshop at FDA’s White Oak Campus on 16 May to offer any patient groups or other potential participants the ability to weigh in on the development of the navigator.

The development of such a navigator comes as FDA has worked to dramatically simplify the compassionate use process, which can be difficult, particularly for a rare disease treatment.

RUF Public Workshop- Developing an Expanded Access Navigator for Individual Patient Access to Investigational Drugs

Expanded Access Navigator Draft Proposal


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