Affordability and Access: Why patient perspectives matter

“Context around patient-identified affordability and access challenges is really critical,” began Karin Hoelzer, DVM, PhD, Senior Director, Patient Advocacy during the Biotechnology Innovation Organization’s (BIO) August Coffee Chat, The Facts are out There! What Patients Really Want (… timely access to treatments).

“Oftentimes,” she continued, “chronic disease patients take multiple therapies at the same time to manage a number of conditions, and so really looking at the whole picture, and truly understanding the root causes of the challenges patients face—including a lot of the changes we’ve seen to insurance design, the increase in utilization management, etc.—is key to what we’re talking about today.”

And indeed, as many patient advocacy groups have found, affordability and access cannot be considered in isolation.

From harmful utilization management tactics like step therapy and prior authorization, to consequences of drug pricing provisions within the Inflation Reduction Act (IRA), to the 340B program being manipulated in ways that harm rather than help patients in need, patients are increasingly facing roadblocks accessing the care they need.

As advocates explained during the Coffee Chat, the patient’s voice and experience must guide health policy.

And there is a great deal of work being done in the patient community to understand and channel those experiences. For example, the Ensuring Access through Collaborative Health (EACH) & Patient Inclusion Council (PIC) coalition published the Patient Experience Project: Patient-Reported Affordability and Unaffordability Survey 2.0 report to better understand what affordability really means to patients. One of the survey’s key findings was that “Drug price alone does not determine affordability. Confirming findings from the pilot survey, insurance barriers, high cumulative medication costs, perspectives on how much medication should cost, and evolving life experiences were all primary drivers of reporting a drug as unaffordable.” In fact, the report found that “95% of patients who stopped taking their medication cited insurance-related challenges, not cost, as the primary cause.” Moreover, “72% who never started taking their medication cited insurance-related challenges, including denial of coverage and high out of pocket costs even with insurance.”

Similar findings were reflected in CancerCare’s Red Tape Report, which surveyed 1,201 cancer patients receiving treatment to understand the barriers that utilization management posed.

“We focused particularly on prior authorization, but our work was certainly not limited to that,” said Kim Czubaruk, JD, Vice President of Policy, CancerCare. “In our report, 85% of people treated for cancer were put through prior authorization just in the past year. And several of those had five or more prior authorizations in the past year, particularly those with employer-sponsored insurance.”

But although step therapy and other utilization management tools represent key barriers for patients, the problem goes far beyond utilization management.

Shrinking Access for Medicare Beneficiaries

The Inflation Reduction Act (IRA) is creating adverse effects on patients’ access to medications. Parts of the law, like the Part D out-of-pocket cap and Medicare Prescription Payment Plan (MPPP), are meaningful steps towards patient affordability. Other provisions, namely the Medicare Drug Price Negotiation Program (MDPNP), are restricting access for patients.

As Pat Wildman, Senior Vice President, Advocacy & Government Relations, Lupus Foundation of America explained, some IRA policies have led to accelerate narrow formularies and harmful utilization management practices, rather than putting the brakes on these trends.

“We’ve seen a lot of cost shifting [in Medicare]—moving from fixed copays to cost sharing, which increases people’s out of pocket costs,” Wildman said. “You go from a fixed copay to cost sharing, that’s a little bit harder to manage, harder to predict, and a challenge for folks, particularly when we talk about lupus.”

But it doesn’t stop there. Wildman and his team have also seen insurers narrowing formularies, i.e. covering fewer drugs in a therapeutic class or placing them on higher tiers with greater cost-sharing responsibilities.

“We’re also seeing other changes with the shrinking of the standalone prescription drug plan market, and that’s going to shrink further too,” Wildman said. “And premiums are going to become more of an issue as well with some of the changes that the administration has announced in eliminating the premium stabilization demonstration.”

“We’ve really been emphasizing in our conversations on the Hill that affordability and access aren’t just either-or issues, and you can’t just take one; you’ve got to look at both of them,” said Wildman. “We can improve affordability on the one hand, but undermine those improvements by creating access barriers.”

Patient preferences have to be a key part of the equation, too, advocates reminded the group.

For instance, the route of drug delivery is an important consideration for patients that can have tremendous impacts on the daily lives of patients and caregivers, but unfortunately such patient preferences are often overlooked in the policy debate. As the panelists noted, the Centers for Medicare and Medicaid Services (CMS) recently proposed changes to the definition of ‘Qualified Single Source Drug’ under the IRA ‘negotiation’, that would create strong disincentives to develop drug formulations that offer more patient-friendly administration routes such as injection rather than infusion. This is despite the fact that a February 2025 study published on PubMed found that patients strongly prefer injection to infusion as this administration route is less time consuming, easier to access, and has less caregiver burden, among other benefits.

This is yet another example of policy falling short of being patient-informed.

340B and its misuse

“340B is the largest program that no one’s ever heard of,” explained Wildman. Unfortunately, the program’s obscurity and lack of transparency has long played a strong role in its misuse. Advocates are now shining a light on the patient impact of this program’s misuse.

“Our goal was to humanize 340B because the direct impact on patients, and what the program’s intended to do as far as helping people who are underserved or are low income, is incredibly important,” said CancerCare’s Czubaruk. “But it has not been serving those needs as it was intended to.”

CancerCare partnered with the Pioneer Institute to conduct a study that compared the levels of charity care provided by 340B hospitals and non-340B hospitals. This report investigated whether or not 340B hospitals were providing charity care at greater levels than non-340B hospitals.

“After all,” Czubaruk noted, “they are getting the discounts. And they are non-profits so they are getting the tax benefits. So we asked, Were they providing that charity care at a higher level? And the report, in summary, says no.”

While there are some good players in the system, Czubaruk explained, far too many are not acting above board.

“We conducted lengthy interviews with a number of patients who received cancer care at 340B hospitals, and from those interviews, we captured tremendous stories and quotes of how the 340B system has not provided charity care,” she said. “These are people in real need who are on the verge of eviction month to month. Some can’t afford their food. Most of them didn’t even know, understandably, that 340B existed and what its purpose was. The hospital never told them.”

And the lack of transparency in the 340B program does not stop at charity care, a report commissioned by LFA and Arthritis Foundation found 340B margin revenue for just arthritis and lupus alone is over $2 billion.

“Where is that money going?” Wildman asked. “It’s certainly not all going to charity care or lowering patient costs.”

Defrayal and its effects on access

The final topic addressed in the Coffee Chat was defrayal, a little-known element of the healthcare affordability and access conversation that is coming more and more to the fore.

As Bio.News reported earlier this year: “Under the Affordable Care Act (ACA), all health insurance plans offered through the ACA marketplace exchange must, at a minimum, cover federally defined essential health benefits (EHBs)… States can mandate that insurers cover additional benefits beyond the EHBs in all plans offered on the ACA exchange—but, under the original law, the states are required to ‘defray’ the added costs.”

“In the very simplest terms, it is the part of the ACA that is meant to discourage states from enacting new legislation and laws around specific mandated benefits,” explained Catherine Peters, Strategic Director, State & Local Campaigns at the American Cancer Society Cancer Action Network (ACS CAN). “Certain state laws that require insurance companies to cover certain benefits could trigger defrayal.”

As advocates noted, there is still a great deal to be learned about the nuances around this issue, so investigation and communication with stakeholders is key as the situation develops.

“Defrayal dictates that the state has to pay the difference between what a plan would have cost without the new mandate versus what it would cost now with the new mandate,” Peters continued. “There’s been a lot of back and forth between administrations about the enforcement of that provision of the ACA, but with the latest Notice of Benefit and Payment Parameters rule that was just finalized in May, the administration is doubling down on requiring states to pay for any mandated benefit that has passed since 2012.”

But, Peters said, they are already starting to see movement on this issue at the state level.

“We’re already hearing from a couple of states that are considering either repealing laws or appropriating money to cover the defrayal,” she observed. “We’re trying to gather as much intel as we can from state capitals to try to figure out what we’re going to be dealing with. But there’s a lot of unknowns, and some of this is just going to be kind of learning as we go once January comes. Unfortunately, we’re not sure how far state lawmakers are going to take some of this. Are they going to actually repeal statutes that provide important coverage protections to patients or find other workarounds? We don’t know.”

It all comes back to patients

Whether it is utilization management, elements of the IRA, 340B, or issues with defrayal, patient advocates remind that one element to the healthcare conversation must always stay front and center: patient need.

“When we talk about costs; people need to look at the bigger picture,” said Wildman. “You need to look at the cost of transportation, the cost of going to get care, the cost of daycare, the cost of missed school, the cost of missed work—those are all barriers to care. When you provide different options to get care, addressing those extra barriers is a good thing. If we ignore that, we are in essence, dismissing the value to the patient.”

“This is a nonpartisan issue,” added Czubaruk. “So we need to make sure that when we go up to the Hill and share our patient stories that they are reflected in the legislation that is being presented and passed.”

As the panelists explained, don’t lose the patient story in the data. Numbers may reflect patient experience, but they cannot express the reality fully. Patient stories and one-on-one interaction do that, and it is always the job of the advocate to maintain the drumbeat of patient-first always.

“It’s not just saving money at all costs,” said Wildman. “The question that isn’t being asked is, Saving money for who? And oftentimes on the Hill, they see the dollars, the price tag, and the score, and in their minds it’s to save money for the government. It doesn’t necessarily translate to saving money for patients.”

“We’re on the defense of trying to respond to all of these actions that are actually taking away access for people to promote savings,” concluded Czubaruk. “But if savings to the government means less people are getting care, then we’ve defeated the entire purpose of innovative treatments and having a robust healthcare system in and of itself.”

If you are a patient advocate, and want to continue this conversation, consider registering for the Biotechnology Innovation Organization’s Patient Advocacy Changemakers Event (PACE): an empowering and inspiring event focused on breaking barriers to access and ensuring that innovative medicines reach the patients that need them.

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