Daily Press (Sunday)

Drug board plan could undermine rare disease treatments

- By Michael Eging Michael Eging is the executive director of the Rare Access Action Project, based in Purcellvil­le. He has been a long time advocate for developing policy solutions for challenges with rare disease patient access to health care.

While we applaud efforts by advocates across the country to lower health care costs for all patients, we are alarmed that Virginia is considerin­g creating prescripti­on drug advisory boards (PDABs). The Rare Access Action Project (RAAP), based in Northern Virginia, has serious concerns with PDABs and their inability to lower patients’ out-of-pocket costs.

By nature, rare disease therapies benefit comparativ­ely few patients. PDABs in other states are blunt instrument­s that have focused on therapies for smaller population­s. Last year, the initial PDAB list of drugs in Colorado disproport­ionately included rare therapies, most used by fewer than 100 Coloradans. In Virginia, we anticipate that the PDAB designed by Senate Bill

274 and House Bill 570 will also disproport­ionately focus on rare therapies. However, there are unintended consequenc­es to patients who simply cannot afford to have their current treatments interrupte­d.

Only 5% of rare diseases have

a treatment approved by the

Food and Drug Administra­tion and, for one-third of individual­s with a rare disease, it can take between one and five years to receive a proper diagnosis. Half of all patients diagnosed with a rare disease are children, and as many as 3 in 10 children with a rare disease will not live to see their 5th birthday. For the few fortunate to have a treatment, patients face many barriers to these orphan therapies across our

health care system in addition facing a patient journey filled with misdiagnos­is and lack of treatment options.

Consider the experience we already have with price setting challenges, on which proponents of PDAB claim they have modeled this form of price control.

The Inflation Reduction Act, passed in 2022, puts the federal government into a price settling role. Even though a limited exemption for rare therapies was included in the bill, the implementa­tion is problemati­c because it eroded the incentives of the seminal Orphan Drug Act. Since the bill’s passage, we have seen the closure of rare programs, investment resources fleeing to other therapeuti­c areas, and market uncertaint­y caused by federal policymaki­ng.

PDAB proponents have shown no evidence that attacking rare therapies will lower copays, encourage lower premiums, create transparen­cy with associated health care costs, lower drug markups through the health care system, or not damage access for rare patients currently with insurance.

Further, PDAB proponents don’t appear to understand the impact of copay accumulato­r programs, a feature of insurance plans whereby manufactur­er or other third-party assistance payments do not count toward the patient’s deductible and out of pocket maximum. This practice allows plans to “double dip” non-profit foundation­s and other charitable programs, while keeping all patients on the hook for copays throughout the year if they use such assistance. Virginia banned these programs a few years ago which made a significan­t impact for consumers. Yet there is more we can do today to have an immediate impact for rare patients.

So, the question becomes, can a PDAB really bring savings to patients and provide those savings to patients in a transparen­t fashion? Remember, we have seen PDABs created in other states using the same flawed methods and focus. And those experiment­al efforts cannot protect patient access for those with existing coverage or provide patients with meaningful assistance.

Ignoring the complexiti­es of rare therapies could create a long-term negative impact on a vulnerable community where one size fits all policymaki­ng doesn’t meet the needs of rare and ultrarare communitie­s. Virginia can do better.

 ?? PAUL RATJE/THE NEW YORK TIMES ?? Prescripti­on drugs line the shelves at a pharmacy in Albuquerqu­e, New Mexico, in 2021.
PAUL RATJE/THE NEW YORK TIMES Prescripti­on drugs line the shelves at a pharmacy in Albuquerqu­e, New Mexico, in 2021.

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