By Jeff Anders
As an organization committed to promoting access to life-saving therapies for patients with rare diseases, the Rare Access Action Project (RAAP) recognizes the very laudable goal of making medications more affordable. However, with the recent hearings and votes on HB424/SB357, the Maryland state legislature is once more debating an expansion of the Maryland Prescription Drug Affordability Board’s (PDAB) authority that among other things would allow it to implement statewide a price control called an upper payment limit (UPL). While the proponents argue that this will address rising healthcare costs, the reality is that UPLs are more likely to have very negative unintended consequences, such as treatment delays and interruptions. Indeed, PDABs will not result in lower drug costs and greater access to treatments, but instead are a disaster waiting to happen with regard to patient access to therapies and care.
Supporters of this experiment argue that PDABs will make medications more affordable. However, these boards fail to account for the complexities of patient access to therapies, especially for treatments targeting rare and orphan diseases, where the stakes are often life or death. While supporters desperately push the talking point of lowering drug costs, PDABs in reality will bring higher patient costs and diminished access to treatments.
Only 5% of rare diseases have a treatment approved by the Food and Drug Administration (FDA) 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. Rare patients face enormous hurdles already in gaining access to these rare disease therapies across our health care system in addition to often being faced with a patient journey filled with misdiagnosis and lack of treatment options. And for some rare patients, the cost assigned to their therapies includes not just the drug, but also services related directly to their care.
Since Maryland passed the first PDAB in 2019, nationwide, no PDAB has saved any patients or states money. That is the reality. Further, prescription medications account for only a fraction of overall healthcare spending, and the list prices set by manufacturers often differ significantly from the net prices after rebates to and negotiations with plans. In a recently published study, nearly 50 percent of drug costs goes to other actors in health care, including Pharmacy Benefit Managers (PBMs) and insurance plans.
Patients in Maryland largely access their medicines through health benefits; with copays and out of pocket costs assigned by the plans. If patients cannot afford the copays and out of pocket costs, there are non-profit foundations and supplemental assistance to help them. PDABs in other states have disproportionately focused on drugs for rare patients, for which there is often only one medication and limited use. If the PDAB pushes the drug out of reach due to upper payment limits for health care providers, pharmacies, and patients, there is no other choice. This experiment leaves rare disease patients as collateral damage.
Maryland’s proposed PDAB expansion will set a dangerous precedent, positioning the government as an arbiter of medical value and access. For rare patients, this is particularly acute as many patients seek professional care across state lines, and their medicines are sourced outside the jurisdiction of the PDAB. This leaves patients and providers to pay for costs above the UPL to be set by a PDAB. States struggling to implement PDABs have already seen troubling signs of reduced access to innovative treatments, a risk Maryland’s rare disease community cannot afford to take.
PDAB expansion legislation in 2025 represents a challenge for many other patients beyond rare patients. This renewed attempt at government price controls ignores the evidence of harm such measures cause. Lawmakers must reject this terribly flawed experiment and instead prioritize reforms that will genuinely lower patient out of pocket costs without compromising access to critical treatments. For patients with rare and chronic conditions, they need assurances that critical therapies will remain available and that future innovations will continue to address unmet medical needs. They need choices for affordable insurance coverage and the opportunity to ensure that copay and charitable assistance counts toward their out of pocket costs.
The Rare Access Action Project urges lawmakers to resist the allure of experimental solutions like PDABs and instead work toward meaningful, proven reforms that address the drivers of healthcare that impact patients’ out of pocket costs. We strongly encourage our Maryland policymakers to focus on such issues that truly and directly impact patients such as PBM regulation and reform; and copay assistance for out-of-pocket costs. We stand ready to work with them to find ways to lower patient costs, and improve access to health care in Maryland..
Jeff Anders is a Maryland resident and policy advisor to the Rare Access Action Project.
