FDA “Breakthrough Therapies” — Patching A Broken System

“U.S. drugmakers cheer 'speed lane' for breakthrough therapies.”

FDA “Breakthrough Therapies” — Patching A Broken System

That was the headline on the Reuters news service following a Washington briefing by some influential pharmaceutical executives July 25th. They were responding to the new “breakthrough therapy” designation that Food and Drug Commissioner Margaret Hamburg, M.D. announced at the ASCO cancer conference in early June.

Yes, it’s a start. We are pleased the FDA acknowledges that critically needed new drugs must work their way through the approval process faster. But the FDA approach only goes so far. It’s like adding a car pool lane to an already clogged highway; it helps just a few privileged drivers move slightly faster, but it doesn’t fix the system. Instead, as I recently wrote in a NY Post article, “The key to success is scrapping bureaucratic science and embracing tools that can attack cancer at the speed of life.”

Let’s take a look at what the FDA is doing

Dr. Hamburg explained that developers of drugs who fall under this new umbrella will receive “more intense guidance on an efficient drug development program, beginning as early as phase 1.” In practical terms, quoting Reuters news service, “Dr. Jay Siegel, head of global regulatory affairs at Johnson & Johnson, said he expects two years to be knocked off the time it world typically take the FDA to review… the company's experimental cancer drug.”

For cancer patients, faster isn’t
nearly fast enough

Targeting a two year reduction in approval time isn’t aggressive enough and doesn’t take advantage of advances in trial design and other medical innovations. We’re ready to move past cutting two years off the approval time. We need to cut the approval time down to two years, period.

As noted on valueofinnovation.org, “The development and approval process for a new cancer drug can take up to 10 years.” So even with the new “breakthrough” designation it could still take eight years to approve a lifesaving drug. That’s too long if it’s your life that needs saving.

One graph showing average approval time and another showing percentage approved.

As the chart above indicates, FDA requirements result in an inordinate amount of time and expense needed to develop a medicine. The breakthrough designation may make a dent, but it applies to only a limited number of drugs. As of mid-July the FDA had received 67 requests for breakthrough designation. It had granted 24 and denied 18.1

The pathway to a new, streamlined system

It’s time to move beyond patching the old system. We need to apply what we know about genetics, molecular diagnostics and targeted treatments to build a new system. How do we do that? We begin with the FDA’s own situation analysis. Dr. Hamburg notes, “oncologic disease is being redefined based on our growing knowledge of molecular pathways.” And Dr. Janet Woodcock, director of the FDA's drugs division, noted the intention “to accommodate new science, particularly targeted therapies that may work in people with certain genetic mutations.”

The problem is, the current system of drug approvals is not based on these factors. Again, quoting my article about winning the war on cancer: “Cancer researchers still conduct randomized, placebo-controlled trials, RCTs. Such studies ignore the variation among tumors, assuming contrary to fact that every cancer grows in the same way. They also require thousands of patients and years to conduct. And RCTs still divide patients into one group that gets a drug with the other getting a placebo — even when a genetic link to treatment is known. So we force dying patients into studies that can’t cure them, knowing they might not get a new treatment.”

So let’s scrap the current system. Let’s move to personalized medicine and scientific knowledge to evaluate drugs quickly and effectively.

Using our understanding of genetic mutations that cause a cancer, we can match drug interventions to specific patients. Let’s test drugs in patients where we expect them to work. Using our knowledge of molecular pathways, we can follow the example of the International Myeloma Foundation’s research initiative looking for ways to rapidly evaluate drug efficacy based on response at the cellular level.

Addressing the issues associated with faster approvals

In accelerating the evaluation and approval of potential life-saving drugs, questions are raised about even the FDA’s modest plan.

One such question is, “The drug may work, but what if it’s not safe?” Fortunately, a reasonable answer has been suggested. In an article titled Get Rid of the Randomized trial; Here’s a Better Way, Dr. Eric Topol, Director of the Scripps Translational Science Institute and Editor-in-Chief of Medscape Genomic Medicine, argues for approval in phases. He posits, “Why can't we have conditional approval for a new drug… and then monitor that very carefully? Then we can grant, if the data are supported, final approval.”

Another such questions is, “Will insurance companies pay for these drugs?” This challenge is of special concern to us because even today nearly half of all cancer patients are forced to choose the treatment that is covered by their insurance, instead of being able to choose the treatment that would be most likely to save their lives. So we not only need a new system, we must require insurance programs to accept it. It’s in their own best interest: Advances in cancer treatment are saving lives and cutting health-care costs.

What are we waiting for?

Today it takes nearly nine years to develop new cancer drugs. The FDA “breakthrough” initiative could drop that to about seven years for certain select drugs, and even then Dr. Woodcock points out, “just because the review process is sped up there is no guarantee of approval.” Instead, let’s follow the HIV model, and we should be able to approve drugs critically needed for cancer patients in only two or three years. With lives on the line, there’s no time to waste. Move medical innovation forward as fast as possible. Join the movement!

By Robert M. Goldberg, PhD

August 9, 2013

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