Patients should fear partnership between the FDA and anti-research ‘watchdog’

By Kenneth E. Thorpe

FDA regulators have approved more than 600 new medicines since the turn of the century. And more treatments are on the way. Scientists are currently developing more than 7,000 experimental drugs.

Unfortunately, one self-styled healthcare “watchdog” could significantly slow this research. The non-profit Institute for Clinical and Economic Review (ICER) routinely analyzes the cost-effectiveness of new drugs. But ICER uses a flawed methodology that often finds cutting-edge, lifesaving medicines aren’t worth the price.

ICER wants the FDA to rely on those cost-benefit analyses when deciding whether to approve experimental drugs. And now, agency officials say they’re “receptive” to reviewing ICER’s data.

Any collaboration between our nation’s premier health safety regulator and ICER — an organization which seeks to put a price on human lives — would prove problematic for patients.

The healthcare watchdog claims to have patients’ best interests at heart, but its methodology suggests otherwise. ICER relies on an outdated, discriminatory benchmark known as a QALY or “quality-adjusted life year.”

This metric evaluates treatments’ cost-efficiency. If a medicine delivers one QALY, that means it offers one additional year of perfect health. ICER recommends that drugs not cost more than $175,000 for each QALY they deliver. And if a medicine fails to meet this criterion, ICER advises that insurers or government agencies not cover the treatment.

Already, governments in foreign countries flat-out deny certain treatments to patients based on QALY assessments. Consider the United Kingdom. Around one in six British cancer patients can’t access the drugs that their doctors recommend — all because British health regulators say the medicines aren’t sufficiently cost-effective. British regulators also recently blocked sales of a new cystic fibrosis treatment.

QALY assessments are bad enough for regular patients, but they’re even worse for Americans suffering from rare diseases.

Here’s why. It takes $2.6 billion and up to 15 years to create a single new drug. Drug manufacturers normally recoup those costs by selling enormous quantities of medicines to large groups of people at relatively low prices. For example, about 35 million Americans take statins to treat high cholesterol. In many cases, each pill costs mere pennies.

But when medications are only purchased by a small handful of rare disease patients, companies have to charge higher prices to recoup their costs. Consequently, rare disease drugs are often quite expensive — and thus score badly on value assessments. ICER evaluated five rare disease drugs between December 2014 and 2018 and judged four of them “low value.”

ICER insists it wants to help patients and the FDA make more-informed decisions. The group has proposed hosting debriefing sessions with patient groups at the end of each cost-effectiveness review. Then, ICER and the patients would co-write a letter to the FDA with tips for gathering more effective data in future clinical trials.

But ICER has spent years devaluing the lives of people living with chronic illnesses and disabilities. Patients would be wise to think twice before trusting the organization to have their best interests at heart.

Kenneth E. Thorpe is a professor of health policy at Emory University and chairman of the Partnership to Fight Chronic Disease.