Making the FDA More Patient-Friendly

June 16th, 2010 at 2:03 pm | 2 Comments |

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In recent weeks, case the news in the war against cancer has been good.

Two new drugs have shown solid results in the treatment of the deadliest cancers. Bristol Myers’s drug Ipilimumab extends the lives of patients with advanced melanoma skin cancer. Pfizer’s drug Crizotinib shrinks fatal tumors in lung cancer.

My friend and colleague Dr. Scott Gottlieb writes lucidly about the heavy lifting to get these drugs to market. Ipilimumab, see for example, cure taps basic lab work into the workings of the body’s immunity from the 1980s. In a sense, the newest cancer drug has been three decades in the making.

Of course, time will tell whether these new drugs are, in fact, breakthroughs.

But one thing is clear: medicine has had incredible breakthroughs in recent decades.  Childhood leukemia is now treatable, not a death sentence; polio has been confined to the history books; death by cardiovascular disease has dropped by two-thirds since 1950.

Many of these advances are due in whole or in part to pharmaceutical and biotech innovation.

In an important new paper, University of Chicago Professor Tomas Philipson and Stanford physician Eric Sun mull the regulatory hurdles faced by drug companies. Cost of Caution: The Impact on Patients of Delayed Drug Approvals, published by the Manhattan Institute (where I have an affiliation), begins by noting the cost of bringing a drug to market: “studies conducted at Tufts have estimated that the cost of developing a single successful drug increased from $413 million (in 2003 dollars) three decades ago to roughly $939 million today…” And, yes, there is also the incredible time delay.

Philipson and Sun look at various attempts to reduce the time delay, including the user fees created by the Prescription Drug User Fee Act of 1992.

Though criticism of the FDA isn’t new, Philipson and Sun take an unusual tact: they focus on three medications (for HIV, breast cancer, and non-Hodgkin’s lymphoma) and calculate the social benefit to the patients of these drugs – and then the benefits had these meds come to market earlier. The results aren’t surprising: caution at the FDA impacts patient health, undermining our ability to treat people earlier.

The paper concludes with some suggestions for reforms at the FDA:

  • Allowing the payment of stipends to clinical-trial volunteers. Facilitation of recruitment by such measures would help clear the present bottlenecks, while having a minimal effect on the safety of participants.
  • Adopting technologies such as biomarkers, which offer the potential to accelerate clinical trials. These would have special value in the case of diseases such as multiple sclerosis that progress slowly to clinical endpoints such as disability. To ensure no reduction in safety, the FDA could require trials employing biomarkers to meet a higher evidentiary standard.
  • Strengthening the Prescription Drug User Fee Act (PDUFA). After an initial improvement in FDA processing rates resulting from the hiring of additional staff, review times stagnated. A new influx of reviewers could shorten development times further.
  • Establishing an ombudsman. Such an office could review the effects of FDA regulations and practices on the speed of drug development.
  • Strengthening the financial position and authority of the Reagan-Udall Foundation. That independent body is charged with setting up projects and programs that assist the FDA in modernizing the drug-development process while continuing to serve its obligations to the public.

With so much focus on the larger issue of healthcare, reforming the FDA hasn’t received much attention inside or outside the administration. Philipson and Sun remind us of the importance of this issue and offer a reasonable prescription.

The full paper, well worth reading, can be found here.

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2 Comments so far ↓

  • TerryF98

    Yep, let the drug companies release any old crap for us to consume, what could go wrong with that!!!

    I don’t believe the rubbish that passes for a story here. Far better to be safe than sorry. Remember Thalidomide

  • Rabiner

    Damn the FDA, the morning after pill should of been approved ages ago!