Why Goldilocks could provide the answer to changing the way FDA regulates stem cells

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Panel on FDA regulation at World Stem Cell Summit

One of the hottest topics of the past year in regenerative medicine has been the discussion about the need for regulatory reform at the Food and Drug Administration (FDA) so it’s no surprise that topic was the subject of the first main panel discussion at the 2016 World Stem Cell Summit in West Palm Beach, Florida.

The panel, titled ‘FDA Oversight in Regenerative Medicine: What are the Options to Accelerating Translation’, kicked off with Celia Witten, Deputy Director of the Center for Biologics Evaluation and Research at the FDA. She laid out all the new steps that the agency is implementing to try and be more responsive to the needs of researchers and patients.

Perils facing pioneers

Martin McGlynn, the former CEO of StemCells Inc. was up next and he wasted little time listing the companies that had once been considered pioneers in the field only to fail for a variety of reasons. He said one of the big problems is that translational efforts, moving from a good idea to a clinical trial, take too long, saying 15 – 20 years is not unusual and that Big Pharma and strategic investors won’t invest until they see strong Phase 2 study results.

“We need to do great science and design and conduct great clinical trials to advance this field but we also have to come up with a sustainable business model to make this happen.”

A good start

He called the 21st Century Cures Act, which the US Senate approved yesterday, a good start but says many of the challenges won’t be helped by some of the new provisions:

“Many sponsors and companies don’t make it out of open label early studies, so the existence of an accelerated pathway or some of the other enabling tools included in the act will come too late for these groups.”

McGlynn warned that if we don’t take further steps, we risk falling behind the rest of the world where companies are buying up struggling US ventures:

“Many non-USA companies in Japan and China and Australia are quicker to recognize the value of many of the products and approaches that struggle here in the US.”

Too much, too little, just right

Marc Scheineson was the final speaker. He heads the food and drug law practice at Washington, DC law firm Alston & Bird and is a former Associate Commissioner for Legislative Affairs at the FDA. He began his presentation with what he said are the scariest words in the English language: “I‘m a lawyer from Washington D.C. and I’m here to help you.”

Scheineson says part of the problem is that the FDA was created long before cell therapy was possible and so it is struggling to fit its more traditional drug approval framework around stem cell therapies. As a result, this has led to completely separate regulatory processes for the transplantation of human organs and blood vessels, or for the use of whole blood or blood components.

He says it’s like the fable of Goldilocks and the Three Bears. Some of the regulation is too hard- resulting in a lengthy regulatory process that takes years to complete and costs billions of dollars – and some of the regulation is too soft allowing clinics to open up around the US offering unproven therapies. He says we need a Goldilocks approach that blends the two into regulations that are just right.

Time to take a second step

Scheineson agreed with McGlynn that the 21st Century Cures Act is a good start but it’s not enough.  He says it still relies heavily on the use of traditional criteria to regulate stem cells, and also leaves much of the interpretation of the Act to the discretion of the FDA.

“It’s a first step, an experiment to see if we can break the logjam and see if we can move things to an affordable BLA (The Biologics License Application is needed to be able to market a product once it’s approved by the FDA). But make no mistake, a cell therapy revolution is underway and I believe the FDA should seize the opportunity to promote innovation and not defensively protect the “status quo”.

 

 

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