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Patient advocates from across the country gathered in Washington, D.C., on October 23-24 for the BIO Patient and Health Advocacy Summit (PHAS). This event served as a vital platform for advocates to share their experiences, network with each other and with key stakeholders in the regulatory and industry landscapes, and address pressing issues affecting patients today, such as barriers to access, strategies for effective communication with the FDA, and the transformative role of AI in drug development.
Here are a few of the highlights and takeaways from the event.
Bio.News interviewed Michele Oshman of the Biotechnology Innovation Organization (BIO) and Sunitha Malepati of the CACNA1A Foundation at the 2024 Patient & Health Advocacy Summit in Washington, D.C.
Patient advocates fight barriers to access
There are still a number of barriers that patients face when seeking treatment and care. Whether it is delays or denials of coverage by healthcare insurance companies or poorly designed/misused legislation, such barriers can be devastating for patients and caregivers.
Over the two days, advocates highlighted several critical barriers that continue to hinder patient access to essential treatments, ranging from alternative funding programs and prescription drug affordability boards to the misuse of 340B.
Alternative funding programs
One such barrier: alternative funding programs. These complex programs essentially offload a health insurance plan’s responsibility to cover certain specialty drugs. This may result in patients’ access to specialty drugs essential for their health being delayed or denied.
“Alternative funding programs are mechanisms designed by third-party vendors, and they are marketed to self-funded plans,” said Kim Czubaruk, Associate Vice President of Policy at Cancer Care. (Self-funded plans are plans in which the employer takes on most or all of the financial cost of the benefit claims.) “They market to self-funded plans as having more flexibility, and the goal of these alternative funding programs is to have the employer plan not have to cover or pay for specialty drugs.”
Billed as a cost-saving measure by employer insurance plans, these programs not only ultimately don’t save employer insurance plans all that much money because the third-party vendors still charge hefty administrative fees; but they also put patient lives at risk and perpetuate an insurance system that values profits for the middlemen more than doctors or patients.
“They take two paths,” Czubaruk continued. “One that can exclude specialty drugs or the second path is automatically denied prior authorization. The end is the same. The purpose for that is to make the patient practically be uninsured or under-insured.”
Coverage for accelerated approval drugs
Drugs that have gone through the Food and Drug Administration’s accelerated approval pathway are, at times, being ‘second-guessed’ by the Centers for Medicare and Medicaid Services and some employer group healthcare plans, leading to access barriers for patients who need them.
As Melanie Lendnal, Senior Vice President of Policy & Advocacy at the ALS Association, explained, in the case of one potentially curative gene-therapy ALS drug, the small pool of patients for whom it is intended are being denied their new lease on life. She shared the story of how the ALS Association had to fight for one patient to get access.
“Shelby, 22 years old, lives in Texas, diagnosed with ALS at 21 years old,” began Lendnal. The ALS Association had to fight for her access at the federal and state levels, including with state insurance commissioners. “When none of that worked in the external appeal process, we had to use the fourth branch of government: the media.”
It wasn’t until social pressure became so overwhelming that Shelby got access to her medication.
“It is absolutely inexcusable that there could be a treatment that is actually showing that it might be a cure for a particular type of ALS, yet people are blocked from getting access to it and are only able to access it when we are able to stand the alarm at such a high level that it forces the industry that’s not where we should be,” Lendnal concluded.
Bio.News interviewed Melanie Lendnal of the ALS Association and Pam Traxel of ACS CAN at the 2024 Patient and Health Advocacy Summit in Washington, D.C.
Step therapy
Many patients are familiar with step therapy—the process of having to take a cheaper (possibly less effective or even ineffective) drug before being moved to a more expensive (and possibly more effective) option. While this can be a mere annoyance to some patients, this can be deadly for other patients whose conditions advance and whose health deteriorates while they take ineffective therapies. And patients may have to go through the same step therapy protocol again and again—for instance, each time their health insurance carrier changes.
Charles Husser, Federal Affairs Manager at the Arthritis Foundation, shared the story of Matthew, a patient who was diagnosed with juvenile arthritis at the age of nine. While he was able to treat his arthritis as he grew, when he finally switched to his own insurance plan as an adult, he faced a step therapy process.
“They required him to go through multiple steps, multiple medications before he could access that treatment,” Husser explained. “Arthritis is a very progressive condition, and so any delays to care can cause irreversible progression in the disease. Sometimes it can lead to difficulty walking, eventually leading to a wheelchair. And so we work very hard to ensure that those delays are not anywhere possible.”
Work is being done to try to combat step therapy at the state level, yet more work is needed at the federal level.
“There have been close to 40 states that have passed step therapy legislation, and that is so incredible,” said Husser, who works with a coalition of organizations on this issue. “We know working in many of these other issue areas that it can be very difficult to get legislation passed at the state level, and so that’s super helpful for us at the federal level.”
Prescription drug affordability boards
Prescription drug affordability boards (PDABs) are no mystery to the Bio.News audience, nor the patient advocacy organizations working to educate states on why these boards are so misguided.
Billed as a cost-saving measure for patients, PDABs can opt to set an upper payment limit (UPL) for a set of drugs every year. As advocates explain, PDABs completely misunderstand the cost barriers in the insurance system and more often lead to patients being unable to access a needed drug or therapy.
“It’s pitched to save patients money,” explained Jen Laws, President & CEO of the Community Access National Network (CANN), “and even some states and legislators are a little bit more honest to say, We’re trying to save ourselves money. The mechanism of a UPL doesn’t actually do that.”
Additionally, no two PDABs are the same. Maryland’s PDAB is different from Colorado’s PDAB, which is different from Oregon’s PDAB, and so on. This means that each state advocacy group needs to be able to navigate a unique set of issues—making the work of national groups that much more cumbersome.
“These different states have different processes, different ways patients can be engaged, for example, different ways they choose their drug lists,” noted Tiffany Westrich-Robertson, Chief Executive Officer and Co-Founder at AiArthritis. “It is a free-for-all. It also makes it very complex and confusing, especially for patients who want to get involved.”
Bio.News interviewed Sarah Jones of the Eosinophilic & Rare Disease Cooperative and Tiffany Westrich-Robertson of AiArthritis at the 2024 Patient & Health Advocacy Summit in Washington, D.C.
340B
Another issue: the misuse of the 340B program, the second largest government healthcare program next to Medicaid.
The 340B program requires drug manufacturers to provide outpatient drugs to eligible health care organizations and covered entities at significantly reduced prices. However, similar to the tactics of pharmacy benefit managers in the insurance space, hospitals abusing the 340B program are not passing the savings on to underserved or at-risk patient populations as the program intended.
“The biggest issue with 340B, especially at the intersection of Medicaid, is duplicate discounts and the lack of transparency,” explained Laws, “and how that’s driving hospital consolidation as much as anything else. I often hear from that industry that 340B dollars are their dollars—no! They’re our dollars. They’re committed to us. The intent is to stretch those federal resources in such a way that serves patient access to care.”
The power of telling patient stories to the FDA
Bio.News interviewed BIO CEO John F. Crowley and Paul Hastings of Nkarta Therapeutics at the 2024 Patient & Health Advocacy Summit in Washington, D.C.
The conference covered a number of other topics and offered storytelling workshops to advocates in the interest of giving them the tools they need to do their important work.
Patient stories were, of course, the lifeblood of the conference, with stories being shared on the stage, as well as one-on-one during networking sessions. These stories are so key to events such as PHAS, because telling patient stories leads to change.
As explained by BIO’s President & CEO John F. Crowley, along with Dr. Patrizia Cavazzoni and Dr. Peter Marks of the FDA, patient stories can change the mindsets of federal regulators for the better.
“When I ran Amicus therapeutics, we had a policy for at least the last seven years that we would always bring in patients, even to seemingly routine meetings with regulators,” recalled Crowley. This policy opened the doors to innovation at the company more than once.
The potential of AI
The potential of AI was also discussed at the summit. Presenters noted that not only can AI help tackle profound research and drug development problems, but, when used prudently, it can also help relieve administrative burden among providers and lead to better patient care and improved health outcomes.
However, there is still a great deal of work to be done when it comes to breaking down both research and AI development silos, as well as building trust among patients and transparency as to when and how AI is in use.
