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US streamlines gene therapy oversight to ease burden on drugmakers, researchers

The U.S. government is eliminating some of the overlapping study review processes, paperwork and safety reporting requirements for drugmakers and academic researchers that are pursuing gene therapies, with the aim of easing their regulatory burdens.

Gene therapies are drugs that modify a person's DNA to treat or cure a disease.

With the gene therapy field now well-established — three products are now approved for the U.S. market — the layers of reviews and reporting that were once thought critical to protect the American public are no longer needed, U.S. National Institutes of Health Director Francis Collins and U.S. Food and Drug Administration Commissioner Scott Gottlieb said.

The NIH has decided to step back from its role of clinical trial oversight for gene therapies and let the FDA take it from here, the two leaders said in an article published late on Aug. 15 in the New England Journal of Medicine.

No longer will gene therapy researchers and developers be required to register their clinical study protocols with the NIH and provide trial updates to the agency to undergo reviews by its Recombinant DNA Advisory Committee, or RAC. Such reviews are already being conducted by the FDA and the studies' local-level institutional biosafety committees.

"We have moved past the time where having this duplicative belt-and-suspenders approach between the NIH and the FDA really makes sense," Carrie Wolinetz, associate director for science policy at the NIH and acting chief of staff to Collins, told S&P Global Market Intelligence in an interview.

The redundant reviews by the NIH and the FDA for gene therapy studies have never been required of any other field of clinical research, Collins and Gottlieb noted in their article.

Originally, the overlaps of submissions to the two agencies were viewed as harmonized reporting that enabled the FDA to conduct regulatory oversight, while maintaining confidentiality with drug developers, and allowed the NIH to provide transparency to the public about the research, the two agency chiefs said.

But the NIH's clinicaltrials.gov website, which was made available to the public in February 2000, already provides a high level of transparency for many of the gene therapy studies, they said.

And NIH and FDA senior leaders have concluded there no longer is sufficient evidence to claim that the risks of gene therapy are entirely unique and unpredictable or that the field still requires special oversight that falls outside our existing framework for ensuring safety, Collins and Gottlieb said.

The NIH is accepting comments from the public on the action over the next 60 days, Collins noted in a statement.

An established field

Novartis AG's Kymriah, a chimeric antigen receptor T cell, or CAR-T, therapy, was the first gene therapy approved in the U.S. — gaining the FDA's nod to enter the market on Aug. 30, 2017.

Gilead Sciences Inc. followed on Oct. 18, 2017, with its own CAR-T drug Yescarta, which the company obtained through its $11.9 billion acquisition of Kite Pharma Inc.

CAR-T drugs are made by taking immune cells from a patient's blood and re-engineering them to attack cancer cells. The cells are then infused back into the person's body.

On Dec. 19, 2017, Spark Therapeutics Inc. gained approval to market its gene therapy Luxturna to treat a rare progressive genetic eye disease that causes significant visual impairment, eventually resulting in near total blindness. Luxturna works by supplying a functional copy of the RPE65 gene within the retinal pigment epithelium cells, allowing for restoration of the visual cycle.

The FDA declined to acknowledge how many marketing applications for gene therapies currently are under review at the agency — information a spokeswoman told S&P Global Market Intelligence regulators do not disclose to protect companies' confidentiality.

But Gottlieb and Collins noted that there are more than 700 active investigational new drug applications for the products at the FDA authorizing the experimental drugs to be administered to humans in studies.

Given the field's rapid evolution, "it seems reasonable to envision a day when gene therapy will be a mainstay of treatment for many diseases," the NIH and FDA leaders said.

Restoring RAC to original role

The NIH's RAC was created in 1974 out of concern about the ethical, legal and social implications of altering human genes and the emerging field of recombinant DNA technology. The advisory panel's mission was later expanded to include oversight of gene therapy protocols.

The death of an 18-year-old gene therapy study participant in 1999 brought more scrutiny to the field, including increased oversight.

But now that there is a greater understanding about the basic biology of disease, gene therapy delivery and the potential for adverse events with those products, Collins and Gottlieb said it was time to restore RAC to its original intent of focusing on scientific, safety and ethical issues associated with new and emerging biotechnologies.

Giving the FDA and the institutional biosafety committees the primary oversight role for gene therapy protocols will free up the RAC to focus on the bigger picture and examine issues like gene editing tools, such as clustered regularly interspaced short palindromic repeats, or CRISPR, the NIH's Wolinetz said.

Thoughtful process

For anyone concerned that the elimination of RAC's protocol-by-protocol reviews came too soon, Wolinetz said deliberations about that move have been underway for some time.

In 2013, the National Academy of Medicine, formerly known as Institute of Medicine, studied the situation and provided a set of recommendations to the NIH, including advising it to limit the RAC's reviews to human gene therapy protocols that raised exceptional issues or concerns.

After the NIH implemented that change in 2016, the agency determined that only three of 275 such protocols warranted RAC review.

"You want a field of medicine to mature to the point where you are seeing positive therapies for patients and you feel confident in the oversight," Wolinetz said. "We can now turn our attention to the next challenge. It's a celebration of how far we've come in gene therapy."