The Daily Rise: Tuesday, May 10

FDA Blocks Last Chance for Patients

“What can you do when the drug you need is not FDA approved?” That’s the question Forbes contributor Ken Kam is asking on behalf of patients.

Kam recalls his struggle to obtain a potentially life-saving treatment for his 76-year-old mother.

“After all FDA approved drugs had failed, her doctors delivered the bad news that there was nothing left to try,” he shares of his experience. “They made it sound like I was completely out of options. They were wrong. But exploring these options requires some assertiveness, some tenacity, and even some begging.”

Among the options that Kam explored was the FDA’s “Single Patient Expanded Access Submission,” a compassionate use exemption that allows for one patient clinical trial. Yet, bureacratic obstacles make this last option nearly impossible to secure in the necessary time to help patients.

Unfortunately, there’s an incentive for companies to withhold the treatment because “their chances for approval could be hurt if my mother suffered an adverse reaction.”

“I do not know if Dificid would have saved her,” Kam shares. “But I do not think that preventing her from trying it should be the law of the land.”

Sadly, this experience is far too common. Just ask patients with Duchenne Muscular Dystrophy, who were recently voted down by the FDA advisory committee from a review of Sarepta’s Eteplirsen.

Health Care Vulnerable to Privacy Breaches

Patients are one of the most vulnerable groups to privacy breaches, according to a new report from the Brookings Institution.

As more of our personal medical information is shared electronically, there’s an increased incentive for hackers to target the health care sector.

Key findings from the report:

  • The government encouraged health care providers “to adopt electronic health records without being ready to adequately invest in security technologies.”
  • Health care organizations have failed to properly invest in digital security and patient privacy.
  • Human error is the leading cause of most security breaches.

“With the push toward more integrated care, medical data are now being shared with many different types of entities in which many employees have access to patient records,” the report states, according to Morning Consult. “Extended access to medical records increases the potential for privacy breaches.”

The Non-Linear Path of Innovation

Julie Rosenthal, an ophthalmologist at the University of Michigan Kellogg Eye Center, explains “How A Cancer Drug Has Saved People From Going Blind.”

“I feel lucky to be practicing today and to be able to offer my patients such a game-changing treatment,” she writes in a piece published at NPR. “(S)ome brilliant minds applied principles of cancer therapy to come up with a treatment that could actually improve vision in people with wet AMD. They realized that the two diseases had an important trait in common — they both involved the growth of new blood vessels, whether in tumor cells or in the retina.”

That patients with a different disease are benefiting from cancer research confirms the value of innovation. Advances in one area help deliver treatments in another. Research in one field treating one disease can spur innovation in another field fighting another disease.

“At five years, about half of patients still had vision good enough to meet most states’ driving requirements, 20/40 or better, and 10 percent had what we consider normal: 20/20 vision,” Rosenthal writes of new research published earlier this month in the journal Ophthalmology. 

The path of innovation is non-linear. We can’t quantify a treatment into a simple value framework.

ICER Backpedals

Dr. Surabhi Dangi-Garimella investigates a new report released by the Institute for Clinical and Economic Review that seems to indicate the group is backpedalling on its confidence in value frameworks.

The report confirmed that multiple myeloma treatments are clinically promising, but refused to identify meaningful findings on the “cost effectiveness” of the treatment.

“The relatively small patient population for these treatments, our estimate of the potential aggregate costs of treatment with new regimens does not reach the level at which we estimate it would raise a notable concern for affordability across the entire health system,” the report concludes.

Patients Rising will in attendance at the ICER Midwest CEPAC meeting in St. Louis on May 26th. We look forward to bringing you more as they prepare to develop their final report on the matter expected in late June.

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