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(Blog) FDA’s Project Facilitate is a Step in the Right Direction

May 17, 2019

Six is Lydia’s age in years. Six is also a number of months she lived after being diagnosed with DIPG.

Ten and younger is the age children are most often diagnosed with DIPG — diffuse intrinsic pontine glioma, a brain cancer.

Zero is the number of cures for DIPG.

Thirty one is the number of states that declared May 17 DIPG awareness day.

Lydia lived in one of these states. We met her parents in person yesterday, 51 days after her passing, having previously worked with them on trying to get a compassionate use drug for Lydia.

Three is the number of times her oncologist applied to the company that manufactured the investigational drug, and three is the number of times Lydia was denied the drug.

Her parents knew that DIPG has no cure. They understood from day one that all they could hope for was a bit longer time with Lydia. A longer time while she can keep her eyes open. Time until kindergarten graduation. Time for one more smile.

Lydia’s oncologist believed the drug he was seeking might help Lydia to live longer. In his letter to the drug company after the denial, he wrote: “I will continue to support them, especially Lydia during this process and will advise them the best I know how, but I cannot and will not tell them to stop continuing to fight.”

Patients and families should not have to fight for access to compassionate use drugs.  Access also should not require pleading, begging, threatening, or using of political or media connections.  Despite many positive changes in the past few years to bring clarity, equality, and transparency to the process, applying for compassionate use remains unpredictable.

“Unpredictable” was the word used most often by the participants at yesterday FDA’s Oncology Center of Excellence public workshop on its planned pilot program, Project Facilitate, to describe their experience with compassionate use applications. FDA plans to create a call center for physicians seeking compassionate use (single patient IND) for their patients and will provide guidance, assistance, and follow up with companies on compassionate use requests.

FDA will request data on the numbers of individual patient requests from companies. This is valuable as we do not know how many requests companies deny every year. The only available data is on the numbers of requests that companies grant and forward to FDA. Knowing the scale of the problem is the first step to solving it.

Project Facilitate has a potential to significantly improve access to drugs. In part, because FDA plans to inquire why companies deny a drug in each case.

In Lydia’s case, the company stated that as a result of feedback from FDA, it does not provide the drug under single patient INDs. The company opened an expanded access program for multiple patients (intermediate-size protocol) – which had its own eligibility criteria, and Lydia did not qualify. Does it make sense – there is an expanded access program but no expanded access can be granted to a single patient? These patients – usually the sickest – are left behind.

Project Facilitate will help dispel the myth that FDA prohibits companies from providing compassionate use drugs on a single patient IND basis.

The sad irony of Lydia’s case is that the drug company that denied her the DIPG drug received funding last year from a charity organization to expand its expanded access program. That charity organization was created by parents whose daughter, like Lydia, died from DIPG.

We applaud FDA for Project Facilitate and hope it will make compassionate use access more fair and predictable. FDA should work with companies that offer expanded access programs to review and justify eligibility criteria for those programs and inquire what can be done for patients who fall through the cracks.

* The name has been changed to protect the family’s privacy.

 

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