Real World Evidence as part of a Managed Access Program

The lack of treatment for COVID-19 , which resulted in a decision by the FDA to allow expanded access and compassionate use  for remdesivir  and Emergency Use Authorization  (EUA) for chloroquine, has brought renewed attention to these methods of getting treatment to patients with life-threatening illnesses when there is no approved drug.

The FDA’s response to remdesivir was groundbreaking because it included orphan drug status, a designation that provides seven years of market exclusivity and has been traditionally reserved for drugs that treat rare diseases.  After critics, including Senator Bernie Sanders, objected, Gilead rescinded just the orphan portion of their request. 

Gilead is currently transitioning from emergency access for individual compassionate use requests to expanded access programs in countries around the world in order to “manage the volume of requests, expedite the delivery of the drug for patients, accelerate the gathering of data outside of clinical trials and, ultimately, should the data be positive, be in a position to more quickly apply for potential regulatory approvals that can enable broader access to remdesivir.”

While Gilead races to ramp up production, data is already being published in advance of clinical trials.  An April 10, 2020 article in the New England Journal of Medicine reported clinical improvement in 36 of 53 patients (68%) hospitalized for severe Covid-10 and treated with compassionate-use remdesivir.

While this is potentially great news, these are extraordinary times and some medicines may be tried that are not helpful. Careful consideration should always be made about whether it actually makes sense for a therapy to be offered in a compassionate use setting taking into account safety data, unmet need, supply and other factors.  

In rare disease, urgent need for treatment is the rule, not the exception. Of the 7000 rare diseases, 95% have no treatment.  Seventy-percent of genetic diseases start in childhood, and often they are degenerative and life-limiting. Faced with the prospect of rapidly declining health and possibly death, patients and caregivers are understandably frustrated with the slow pace of drug development and seek a method of gaining access to treatment as quickly as possible. 

Drug developers often think of compassionate use as something companies do for humanitarian reasons to benefit patients without fully taking into account whether they should do a program and what aspects they need to consider to make the program as beneficial and effective as possible. By carefully designing the right program for the right patients and offering support beyond therapy while collecting data along the way there are opportunities to:

  • Enable better therapy lifecycle decisions by adding insights on the drug’s effectiveness 
  • Improve global strategy, showing companies where the patients and treating physicians are
  • Support conversations with payers 
  • support faster uptake and better outcomes 

In many countries around the world, nearly 100% of rare disease patients lack access to treatment.  By working with patient groups and physicians globally, drug developers can alleviate some of this suffering, while advancing development of new treatments.

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