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Are Regulators Too Hasty in Approving Cancer Drugs?

The British Medical Journal ignited debate earlier this month when it announced that about half of cancer drugs approved by the European Medicines Agency don’t improve survival or quality of life.  The claim begs several complex question: How can regulators evaluate drugs both quickly and effectively?  And whose values should determine access to drugs for deadly and debilitating diseases?

Findings on Cancer Drugs

As the British Medical Journal reports, the European Medicines Agency approved 48 cancer drugs for 68 medical uses between 2009 and 2013.   At the time these drugs came to market, 24 of the 68 had proven to increase survival for cancer patients. Seven had demonstrated improved quality of life.

Roughly five years later, 35 demonstrated improvement in duration or quality of life.  The remaining 33 were still uncertain. “Most drugs entered the market without evidence of benefit on survival or quality of life,” the article summarizes, noting that many still lacked conclusive evidence of benefit several years later.

So, are European regulators too hasty in approving innovative drugs for cancer?

The European Medicines Agency’s Defense

Not so, the European Medicines Agency says.  In a response to the British Medical Journal, the agency explains that, “It is well known that in many situations demonstrating a clear effect on survival or [quality of life] is not feasible.”  Determining a single drug’s impact on overall survival can be difficult, the authors point out, when patients try multiple innovative treatments over time.  The authors also note that taking the added time to do a randomized controlled trial when a single-arm trial has already indicated a drug’s benefit is unethical – requiring cancer patients to wait and suffer unnecessarily.

Methodology is valuable, the European Medicines Agency affirms.  But “restricting approvals of cancer drugs only to situations where there is indisputable evidence of improvement in survival or [quality of life] will not improve the lives of cancer patients.”

Health Technology: Assessment and Access

The debate suggests that, as treatments become more targeted and more advanced, approval processes must also evolve.  So explains The Institute of Cancer Research, which notes that trials may need to become smaller themselves, assessing targeted groups of patients who are most likely to benefit.

Considering factors outside of survival matters too.  In fact, other trial endpoints may better reflect patients’ values.  Consider, for example, a patient who would prefer to improve the quality of the time he has remaining rather than simply extending its duration.

But it’s not just drug trials that must become more flexible. Health technology assessments should also evolve to accommodate innovative developments for deadly or debilitating diseases.

While the British Medical Journal reflects the challenges faced by those in the European Union, the struggle has hit home for Americans through the work of the Institute for Clinical and Economic Review. ICER, as it’s called, has become a prominent voice in the United States’ health technology assessment debate.  The group uses a value framework to establish an ideal price point for innovative therapies. As patient groups continue to point out, however, number crunching cannot always account for inherently qualitative factors – patients’ values, for instance, and the critical quality of life benefits that certain therapies provide.

Rising health care costs will continue to yield health technology assessments – some more accurate than others.  Yet policymakers must keep in mind the need to make cutting-edge drugs available quickly, especially for sick patients with few or no other treatment options.

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The Alliance for Patient Access is a national network of physicians dedicated to ensuring patient access to approved therapies and appropriate clinical care.
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