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“Biotechnology innovation is advancing at an unprecedented pace, delivering new treatments and cures that offer long-awaited hope to patients and their families,” said Karin Hoelzer, Senior Director of Patient Advocacy at the Biotechnology Innovation Organization (BIO), during BIO’s March Coffee Chat.
“Yet, affordability and timely access remain a persistent challenge, both in the United States and abroad,” she continued. “Recent efforts by policymakers to implement arbitrary drug price controls are exacerbating these barriers while, paradoxically, increasing out-of-pocket costs for many patients.“
Hurting patient access through misguided policies
Nearly four years after the Inflation Reduction Act’s (IRA) “price negotiations” were passed into law, one thing has become increasingly clear: for many patients, the law has done little to reduce out-of-pocket costs, while driving up utilization management by health insurers and raising concerns about the future of biotech innovation.
While the law’s $2,100 cap on Medicare beneficiaries’ annual out-of-pocket costs and the ability to spread these costs throughout the plan year provides crucial protections for some, many beneficiaries face additional cost-shifting from their health plan. Mounting evidence shows that, on average, patients who do not reach the cap may actually be paying more—in some cases more than double the average expected out-of-pocket cost—due to increases in deductibles and shifts from co-pays to co-insurance.
Medicare Access for Patients’ (MAPRx) recent report, “Inflation Reduction Act: Access Barriers Undermine Affordability,” certainly supports this. The report finds that, as the program enters 2026, “early evidence suggests that the benefits of the out-of-pocket cap are increasingly being offset by changes in plan behavior. Beneficiaries are facing higher premiums, fewer plan choices, and more aggressive formulary management. These developments risk shifting financial and administrative burden back onto beneficiaries in ways that were not the focus of the IRA’s affordability reforms.”
Meanwhile, proposals such as Most Favored Nation (MFN) type policies—which would tie U.S. drug prices to those in other countries—could exacerbate these trends and further limit access by importing foreign pricing systems that often use health economics metrics that discriminate against people with chronic conditions and limit the availability of new therapies.
These tensions were a central focus of the March Biotechnology Innovation Organization (BIO) Patient Advocacy Coffee Chat, Advocacy in Action: The Hope of Biotechnology and the Threat of Misguided Policies such as MFN-Type Drug Pricing Policies. Panelists explored how drug pricing policies, in the U.S. and globally, are narrowing patient access under the guise of improved affordability.
‘Affordability’ – but for whom?
To frame the day’s conversation, Patrick Wildman, Senior Vice President, Advocacy & Government Relations at the Lupus Foundation of America and a key leader in the MAPRx Coalition, reminded the audience “We have to look at affordability and access in tandem.”
“When we’re looking at the different policy issues out there, we want to see if there’s a balance when it comes to affordability and access,” he said.
He continued to lay out four key questions patient advocates ask to evaluate policy proposals:
- “First, we want to understand: Who is actually benefiting from these policies? Is it truly improving affordability for patients, or is it for the system or for other stakeholders?
- Does affordability come at a cost? Are we creating barriers to care? Is it leading to increased utilization management, increased prior authorization, more step therapy, or limitations on formularies? Is it leading to cost shifting? Are we seeing higher deductibles? Are we seeing higher premiums or other shifts in cost sharing?
- What do all these policies mean for the availability of care? Will these policies narrow available networks and access to specialists for patients, or ensure their availability?
- And what does it mean for the future of care? How are these policies impacting the development of new treatments?”
Evaluating each policy holistically, Wildman and other patient advocates strongly oppose MFN-type policies such as those recently outlined by the Centers for Medicare & Medicaid Services (CMS) that would tie U.S. reimbursement to foreign countries.
In particular, MAPRx advocates have opposed the Guarding U.S. Medicare Against Rising Drug Costs (GUARD) model, which aims to align U.S. prices with international ones.
As MAPRx explains in their comments to the CMS and Department of Health and Human Services (HHS), instead of lowering costs, the model is projected to actually increase beneficiary out-of-pocket spending; it creates strong incentives for plans to restrict access through various utilization management tools; and threatens access for orphan and protected class drugs, among other issues.
These “solutions” fail to take into account the access issues that patients face abroad, and instead risk importing them to the U.S.
Challenging Access Environments Abroad
Will Greene, board member at the Foundation for Prader-Willi Research, recounted his experiences as the parent of a rare disease patient, living and working in Singapore.
“Even though I worked for a global biopharmaceutical company with a multi-billion-dollar rare disease portfolio, congenital diseases were carved out of my employer-provided health insurance plan while I worked in Singapore,” he said. “So, I was essentially on my own financially. After more than $100,000 dollars in out-of-pocket expenses, I said, Okay, enough.”
Greene and his family moved back to the U.S. and started over in the Bay Area. “Our experiences here in the U.S. have been much, much better than they were in Asia Pacific,” Greene said.
His experience will not come as a surprise to many patient advocates. While the public discussion of drug affordability and access often assumes that healthcare systems abroad translate into better access, the reality is far more complex—and in many cases, far more restrictive for patients, particularly for those with rare genetic diseases.
Access can vary dramatically abroad—a point made even clearer when looking at Europe’s complex drug approval and reimbursement systems.
“In Europe, drug approval is centralized, but access is not,” explained Myriam Rai, PhD, Director of Global Relations & Initiatives at Friedreich’s Ataxia Research Alliance (FARA). “So a therapy may receive European-level approval, but whether a patient can actually receive it really depends entirely on the country where the person lives. This is a fundamental fragmentation that creates a big discrepancy from country to country.”
Abroad, economic metrics that devalue people with chronic diseases are widely used
As Rai noted, countries across Europe also differ in how they define and assess the value of a drug. “Some prioritize the clinical benefit, and others will focus more on cost effectiveness,” she said. “In both cases, unfortunately, proving clinical benefit and how meaningful it is for the quality of life is often really challenging. Additionally, most of the time, unfortunately, the voice of the patient is not being considered early enough.”
For patients, these dynamics create a compounding effect: inconsistent national standards, rigid cost-effectiveness thresholds, and the use of quality-adjusted life year (QALY) metrics. In many countries abroad, cost-effectiveness frameworks rely on QALY metrics which systematically and inherently devalue treatments for people with chronic diseases, disabilities, or older adults.
And for patients waiting to access transformative new therapies, time is of the essence. “You have the other big piece: the pricing negotiation,” Rai said. “While it will take maybe a year and a half to get reimbursement in some efficient countries in Europe, it will take much longer in other countries—and some may never reach reimbursement at all.”
And, contrary to the U.S., there is no pathway to pay out-of-pocket for therapies.
“Comparatively, in our closed European system, if it’s not greenlit to be reimbursed, you can’t even access the drug—even if you had philanthropy or could raise funds that enabled you to cover it,” Rai explained. “You would need to travel to the U.S. to get it, which is a vicious circle, and which is not fair.”
Improving access in the U.S. by simplifying the system
Just like in Europe, when patients get caught up in the overly complex U.S. healthcare system, access barriers can mean life or death, as Sarah Jones, MPA, MS, Community Engagement at the Eosinophilic & Rare Disease Cooperative (ERDC) explained.
Her organization recently conducted a survey of over 200 patients with rare diseases (which will be published later this year), and their findings were stark. The vast majority of patients interviewed had health insurance coverage but still struggled with both affordability and access. ERDC found many people filing for bankruptcy or maxing out their credit cards just to cover the exorbitant out-of-pocket costs for their medications. Others were rationing their medication, delaying treatment and experiencing active disease as a result.
As Jones explained, many chronic disease patients, herself included, rely on a large number of therapies to manage their conditions and all the associated symptoms. And, she and her organization are concerned that things are not trending in the right direction, with policies such as IRA and MFN actually increasing out-of-pocket costs across the board.
“You look at things like the foreign price controls that are lurking out there, and you think, that’s only going to complicate things, and currently there is no mechanism in the proposed legislation to pass the savings on to patients” she said. “I keep thinking, we have got to get the word out there. You hear something like these foreign price controls, and the general community thinks that’s a great idea because that’s the talking point.”
