Wednesday, March 4, 2015

Group-Level Expanded Access to Unapproved Medicine: The realistic solution for patients

Guest Blog Author: Jess Rabourn*, Managing Director, ALS – Emergency Treatment Fund

Since 1962, Federal law prohibits open sales of any new pharmaceutical product until that product goes through years of clinical research and is granted approval by the FDA.  

Yet unapproved new drugs are provided to thousands of patients daily.  They’re not openly sold, but instead made available by drug companies to study safety and effectiveness in humans.    FDA allows such managed uses of unapproved drugs through authorized Investigational New Drug (IND) exemptions, which are filed by drug companies, physicians, or other program sponsors.

INDs are filed for clinical trials that are intended to gather data and to support marketing approval.  Additionally, INDs for Expanded Access may be filed when seriously ill patients lack access to traditional clinical trials and there is no other effective treatment available.  Expanded Access programs were used extensively in the late 1980s and early 1990s to provide exploratory treatment to persons living with AIDS. As new antiretroviral drugs were studied in research trials they were simultaneously made available in much larger Expanded Access programs for the many patients who were too sick to take part in the research trials.

Between 2002 and 2012, a new class of lifesaving TK-1 inhibitors was made available to tens of thousands of cancer patients through several Expanded Access programs.

FDA understands the need for treatment options and has a track record of liberally authorizing Expanded Access through group-level “Intermediate Size INDs” and larger group “Treatment INDs”.  A third kind of Expanded Access IND exists for exceptional single-patient cases, but this channel is inappropriate for most diseases, provides no useful data, and is almost never agreed to by the drug companies.  True Expanded Access programs enroll on a group level just like regular clinical trials and require no patient-level FDA filings.

Despite the easy regulatory pathway, drug companies struggle to justify the costs and burdens of providing their product to patients before commercialization.    THIS factor, more than any other, is what keeps dying patients from accessing safe exploratory medicines.  As a consequence, there aren’t nearly as many access programs as there are eligible drugs in deadly diseases.

To address this gap, a nonprofit company called ALS Emergency Treatment Fund (ALS-ETF) was formed in 2012.  ALS-ETF was built to sponsor Expanded Access programs as an independent third party, for ALL patients who otherwise have no access to investigational medicine.  Through FDA-authorized cost recovery, ALS-ETF can engage healthcare payers, patients, and charitable assistance funds to ensure enrollment is both scalable and fair.  Participating clinics work in partnership with the platform –not as service providers- allowing ALS-ETF to manage large, centrally-coordinated Expanded Access programs at little or no cost to drug companies.  Well-designed Expanded Access programs can generate vast amounts of patient response data, which is especially valuable in poorly understood diseases.

This responsible solution works under the laws that already exist and has been enthusiastically received by FDA, drug companies, and research organizations in multiple disease areas.

* Mr. Rabourn spent the first 15 years of his career in institutional asset management and was awarded the Chartered Financial Analyst designation in 2004. He held a senior position at a New York hedge fund until 2008, when his dad, Frank Rabourn, showed the first symptoms of ALS. From that point onward, he has made it his full time career and personal mission to change the treatment landscape for current and future rare disease sufferers.  Mr. Rabourn is involved in multiple drug development ventures and, in 2012, joined three ALS families to launch ALS Emergency Treatment Fund, a professional service provider and a leading authority on Expanded Access programs.