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Progress in Gene Therapy Needs to Be Accelerated

By Erik Paulsen August 27, 2019

Throughout human history, transformative technological changes have faced varying degrees of opposition.

A recently published book, “Innovation and its Enemies,” points out that the Ottoman rulers forestalled the concept of printing for centuries because they believed it would diminish their role as primary sources of information. Early cellphones faced resistance in the United States as impractical tools for the rich. The automobile? Henry Ford was known to have said, “If I had asked people what they wanted, they would have said faster horses.

”When it comes to medical science, this level of hesitation costs lives. And yet, we’re now confronting arguments from those who are urging a slowdown in the approval processes for breakthrough gene therapies that offer new hope to those afflicted with devastating diseases. There are a handful of high-level commentators, including former Health and Human Services Secretary Tom Price, who say that we should pump the brakes on the progress being made in this therapeutic field, some citing potential dangers, and others warning about the costs of cutting-edge therapies.

Let’s be clear that this isn’t some Frankenstein-style rogue science experiment. For more than 20 years, scientists and institutions have been carefully studying the techniques for altering abnormal genes or replacing absent ones in order to combat genetically linked diseases ranging from cancer to Duchenne to sickle cell disease to hemophilia.

Gene therapies — often one-time treatments that cure or drastically improve patients’ quality of life — are now on the verge of transforming American health care for the better. They’re not unlike the advent of penicillin, the polio vaccine, major organ transplants and other critical advancements in medicine over the past century.

The Food and Drug Administration recognizes the importance of this progress. The agency has established new policies and infrastructures to advance gene therapy products that are both safe and effective. In fact, former FDA Commissioner Scott Gottlieb said earlier this year that the agency expects to receive more than 200 new applications for cell- and gene-based products by 2020 and to begin granting 10 to 20 product approvals annually by 2025.

The FDA has said it intends to add approximately 50 new clinical reviewers to oversee the development of these treatments and therapies. Gottlieb, in fact, has compared this innovative surge to the development of antibody drugs in the 1990s, which created the platform for so many of today’s medical treatments.

Stated simply, it’s hard to comprehend the rationale for those who want to hold up a stop sign and delay lifelines for those who are truly suffering and potentially facing premature death. Any safety concerns regarding these breakthrough therapies are being addressed by the longstanding policy guardrails and resources that the FDA has had in place to ensure safety and effectiveness. At a time when we see irrational fears raised from some quarters about vaccines, we don’t need a separate chorus generating public doubts about new treatments that can help us gain traction against diseases that have plagued humankind for generations.

As for the cost concerns, these warrant a broad and forward-thinking conversation about health care payment policy rather than an effort to slow scientific progress. Cutting-edge gene therapies are going to be expensive, possibly at a level that status quo health insurance models can’t accommodate (frankly, payers never envisioned them).

However, it’s worth exploring the creation of regulatory reforms that would better enable the gene and cell therapy innovators to work with both health plans and the government in constructing value-based payment models that would tie reimbursement to the level of treatment effectiveness over an extended period of time. Above all, these new breakthroughs put the cost of treatment alongside the lifelong cost of the burden of the disease from the outset.

There are answers that can and must be found on the payment side of the equation to align with the exciting therapies coming down the pipeline on the scientific side. History has shown us that whenever there are new innovations that change the landscape, there are always naysayers, doubters, defenders of the status quo and those who are quick to shout halt. In this case, it is critical that those negative voices be overshadowed by the voice of reason and an aspirational vision for the future of patient health.

Science is affording us the ability to conquer disease and save untold numbers of lives, particularly loved ones among us who suffer from debilitating rare diseases. We need to move faster toward this future, not shy away from it.


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