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Meeting Unmet Medical Need Means Changes in Medicare

By Kenneth E. Thorpe
April 13, 2023


Over 190 million Americans live with at least one chronic disease. And their actions in the next few days may determine how Medicare does or does not address unmet medical needs for decades to come.
The agency that oversees Medicare and Medicaid – the two federal insurance programs that virtually all Americans will depend on at some point in their lives – is soliciting patients' feedback on its new drug price negotiation program until April 14.
In other words, patients have only a brief window of opportunity left to help shape the program – which last year's Inflation Reduction Act mandates – and ensure it doesn't restrict Americans' access to medicines too severely.
Under the IRA, the Center for Medicare & Medicaid Services (CMS) is able to negotiate prices for dozens of drugs. When choosing which medicines to include in the program, and in determining the "maximum fair price" for those medicines, CMS plans to consider a number of factors. One of these determinants is whether the drug meets an "unmet medical need" for "a condition for which treatment or diagnosis is not addressed adequately by available therapy."
However, there are no clear criteria for what constitutes an "unmet medical need" in the bill. CMS's choice of how narrowly – or broadly – to define that term will heavily influence Medicare reimbursements, and thus impact biotech companies' investment decisions.To understand how a narrow definition could harm patients, consider the progress that scientists and doctors have made against the scourge of HIV/AIDS.
Until the first antiretroviral drugs came to market in the mid-1990s, the disease was a virtual death sentence. Since then, treatments have gradually improved to the point that young people diagnosed with HIV today can enjoy nearly normal life-expectancies, so long as they take their medicines as prescribed.
Of course, taking those treatments precisely on schedule is easier said than done. HIV patients historically needed to consume a veritable pile of pills each day to keep their viral loads suppressed. Because of this challenging regimen and the ensuing side effects, individuals with HIV often missed life-saving doses of their medications.
But through innovative combination therapies and other advances, the pill regimen to suppress HIV became much easier. Even more recently, the FDA approved injectable HIV medications that only need to be administered every two months.
Will CMS deem such a treatment – one that to some may not be technically more effective than the existing standard of care under idealized conditions, but that is vastly easier for patients to adhere to in real-world scenarios – as meeting an "unmet need?" CMS has proposed to consider a drug as meeting an "unmet medical need" if it treats "a disease or condition in cases where very limited or no other treatment options exist." So, a treatment advance that cut pills required per day from twelve to two wouldn't fit this narrow definition and would not receive any added value.
Or what about cancer treatments? Many patients currently rely on drugs that must be infused in a doctor's office or outpatient clinic. Traveling to and from these appointments can pose a considerable burden for patients – especially those who are elderly, low-income, residents of rural areas, or without reliable transportation.
Understandably there is considerable desire among patients managing one, and often multiple chronic conditions, to have their medicines in pill form – which allows patients to have the drugs dispensed at a local pharmacy or even shipped to their doors. That added convenience improves adherence – and is especially important, given that the shortage of doctors is only worsening with time, meaning that it will become increasingly harder for patients to access care in healthcare facilities in the future. Will CMS consider these drugs as addressing an "unmet medical need?"
And of course, different patients often have different reactions to the exact same medications, based on their health status, other illnesses, genes, and lifestyles. If CMS adopts its proposed, narrow definition – and essentially determines that there is no unmet need in a particular therapeutic category, so long as one or two treatments are already on the market – it will disincentivize biotech companies from pursuing new therapies in those categories.
CMS will soon make its final decision on the "unmet need" provision. If patients want to preserve their access to a wide range of treatments, they'll need to make their voices heard by April 14th.
Kenneth E. Thorpe is chair of the Department of Health Policy and Management at the Rollins School of Public Health, Emory University. He is also chairman of the Partnership to Fight Chronic Disease.