The Daily Rise: Thursday, May 26

Don’t Jeopardize My Health to Fix Health Care

Our friend Don Wright shares his concerns with the push to impose “value frameworks” in our health care decisions. Writing in the Minneapolis Star Tribune, he explains the experience of how his first diagnosis changed over time through the benefits of innovation.

“When I was first diagnosed 13 years ago, my doctor’s optimistic prognosis for me was to live three to five years. But medical progress in the intervening years has changed everything for me and tens of thousands of others,” he shares. “Thirteen years later, I’m not only alive, but well.”

Don’s quality of life doesn’t match the formulas set by health care accountants. After his diagnosis, he started running marathons. He hasn’t stopped.

“My concern is that ICER is right now developing an analysis — first for my disease, then for others — that focuses solely on drug costs. Health care companies and federal policymakers will likely use it to deny treatment they deem too expensive,” he writes.

He adds, “The problem is that no mathematical formula can measure a patient’s worth. We’re individuals, not averages. Some do better on a specific treatment, others with another option. Nor can value be based on a snapshot in time. With some treatments, as they’re used longer, they can be applied to an increasing number of indications and their value actually increases with time.”

Quote of the Day

“They will limit what our system spends on patients and the treatments they receive. With cancer and other serious illnesses that patients have, cutting their care absolutely means cutting their survival.”

— Jonathan Wilcox, co-founder and policy director of Patients Rising

Our quote of the day comes from behind the paywall at the Pink Sheets Daily, which covered our news briefing about the damaging effects of ICER’s framework.

Renewed Hope for the Duchenne Boys

The Duchenne Boys have renewed hope that they’ll soon gain access to the right treatment. Last month, an FDA panel rejected a drug by Sarepta Therapeutics that could become the first drug for Duchenne muscular dystrophy.

This week, the FDA announced that it will continue to review the treatment’s application for approval past an initial deadline of May 26.

“Not only was the worst-case scenario averted, but this extension by FDA, which appears open-ended and not the standard three-month type, buys more time for the company’s supporters and the DMD patient community to exert additional pressure to FDA,” RBC Capital Markets analyst Simos Simeonidis explained the significance of the FDA announcement.

The FDA is prohibited by law from commenting on pending applications so we’re cautiously optimistic. This treatment proved optimistic to the few boys able to enroll in the small trial, and yet it is only for 13% of Duchenne’s patients. Depending on what statistics you read, that’s less than 3,000 boys in the U.S.

“Based on all I heard, the drug definitely works, but the question was framed differently,” Dr. Bruce I. Ovbiagele, chairman of neurology at the Medical University of South Carolina and a member of the FDA advisory panel that reviewed the drug, said last month as he voted against approval for the drug.

The panel rejected the treatment due in part to the small number of patients in the clinical trial. It turns out the reason Sarepta Theraputic’s main clinical trial only involved 12 boys was that the company lacked the resources to manufacture more when it started out. Sarepta is a small biotech company with three compounds in their pipeline and no drugs on the market.

Too Much Power for Regulators Opposition to Medicare Experiment

Do federal regulators have too much power over our health care system? That’s the question raised by a recent story in the Washington Post on Medicare’s planned drug pricing experiment.

“The last thing I want to see is Centers for Medicare and Medicaid Services trying to make decisions on the relative value of drugs,” says Scott Gottlieb of the American Enterprise Institute, who previously served as a deputy FDA commissioner. “It’s a grab bag of policy prescriptions related to drug pricing.”

Patients have been voicing our concerns that the new plan will undercut our access to vital care. According to Kaiser Health News, “More than 1,300 comment letters were submitted by the deadline earlier this month.”

It’s a positive sign that so many people took time to contact an obscure government agency to provide a comment on the proposal.

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