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Despite Pharma Ties, Some Patient Groups Protest High Drug Prices

Xconomy National — 

For 20 years, Deborah Long (pictured) has been relying on powerful drugs to keep her multiple sclerosis in check. Her out-of-pocket costs were pretty reasonable until two years ago, she says, when she had to switch insurance companies. Her new insurer made her switch to a different drug, Copaxone. When she tried to fill the prescription, the Walgreen’s pharmacist told her the monthly copay would be a whopping $2,500—well out of reach for Long, 65, a former administrative assistant in Chicopee, MA, who no longer works and is on disability.

She didn’t fill the prescription. After going without medicine for four months, Long finally found an assistance program run by a nonprofit that is covering her copays for now. She must re-apply every year, with no guarantees. Fighting for access to her medication has been stressful and exhausting, says Long. “I want someone to fight and give me a break, so I don’t have to keep fighting for it,” she says.

Even though the high price of drugs has been a major concern across the American economic and political landscape for years, only a few patient advocacy groups have recently started taking action on drug costs. It’s a significant move for these organizations. Most are partly funded by the pharmaceutical industry, making it difficult for them to speak out on this issue. And they have traditionally focused more on funding basic research and drug development, as well as advocating for regulatory approval of new treatments.

The R&D funding and advocacy work are paying off; many new drugs have come to market in recent years for a range of diseases. But patient groups are realizing the new drugs are often too expensive, and competition from the new products isn’t necessarily lowering the price of older drugs.

Take multiple sclerosis (MS), for example: there are now more than a dozen drugs available, 10 of which the FDA approved just in the last decade. Prices for almost all these drugs, including warhorses like Biogen’s Avonex and Bayer’s Betaseron approved in the 1990s, have continued to escalate at rates much higher than inflation (see chart below of MS drug prices).

The message from patients about this mounting “financial toxicity” is getting through. “One of the most frequent questions I was getting was, ‘How come this medication I’ve been on is so much more expensive?’” says Bari Talente, executive vice president for advocacy at the National Multiple Sclerosis Society (NMSS).

Rising List Prices of MS Drugs

In 2016, after hearing from MS patients who were skipping doses and not paying other bills because of their expensive drugs, the NMSS became the first patient group to take a public position on high drug prices: “Medications and the process for getting them must be affordable, simple and transparent.” The society also issued recommendations to drug makers, insurers and policymakers for easing the financial burden on patients.

The Leukemia and Lymphoma Society and the National Health Council followed with their own recommendations in 2017 for curbing healthcare costs for patients. Other groups have opted for different strategies: lobbying for legislation, having private discussions with insurance and drug companies, and engaging with groups that assess the value of drugs such as the Institute for Clinical and Economic Review (ICER).

It’s still too early to assess the impact of these actions, says Kim McCleary, managing director of FasterCures, a biomedical R&D think tank. For one thing, talking to insurance companies is relatively new territory for patient groups. “It’s just another whole different series of competencies to have to master,” says McCleary, whose organization has been trying to facilitate conversations between patient groups and insurers.

Most patient organizations, though, have remained quiet on rising drug costs. Some say it’s not part of their mission. They want to stick to funding R&D and helping support patients by steering them to clinical trials, for example. “We can’t take a position on drug pricing,” says Lynn O’Connor Vos, president and CEO of the Muscular Dystrophy Association, which takes funding from drug makers but relies mostly on non-life science companies including Harley Davidson and Citgo. “It’s not our place.”

Whatever their stated reasons for remaining neutral or silent, most advocates won’t speak out for fear of losing funding from the drug industry, says David Mitchell, a multiple myeloma patient who founded the nonprofit Patients for Affordable Drugs last year to push for lower drug prices. “On the issue of drug prices, they won’t speak,” says Mitchell. A 2017 report from the New England Journal of Medicine concluded that more than 80 percent of the largest US-based patient advocacy groups took money from biopharma and device companies. And a survey of patient groups published in JAMA Internal Medicine last year revealed that 12 percent receive more than half of their funding from industry. (Mitchell’s group is funded by private foundations and his own money and does not take money from pharma or healthcare companies.)

The few groups that have come out publicly against high healthcare costs felt it was imperative. “To advocate and be the voice of patients, there wasn’t really a choice but to talk about the financial struggles of patients,” says Gwen Nichols, chief medical officer of the Leukemia and Lymphoma Society (LLS) (the LLS says it is not influenced by its pharma funders).


Tackling high drug costs is complicated. Patient groups first started by reaching out to insurance companies, to try to help them make more patient-centric coverage decisions. They soon realized how difficult it was to navigate the vast patchwork of different private insurers. The task was especially tough for smaller patient groups, says McCleary. “There wasn’t an easy interface,” says McCleary. “There’s no centralized entry point like with the FDA. It was a huge issue of scale.”

About four years ago, patient groups found an easier way into the problem. That was when ICER started doing value assessments of new drugs. Not long after, the American Society of Clinical Oncology (ASCO) came out with a tool to help doctors and patients assess the value of various cancer treatments. As these “value frameworks” started rolling out, patient groups recognized their influence over insurance coverage, and that they needed to talk to the people behind them. ICER and ASCO provided patient groups with “a point of focus,” says McCleary.

One group that reached out to ICER was the National Psoriasis Foundation (NPF). (Like most patient groups, the NPF receives funding from several drug companies.) The two organizations worked together in 2016 as ICER evaluated eight different psoriasis drugs; seven were FDA-approved at the time. The foundation had patients and doctors talk with ICER staff about the challenges of both having and treating the disease.

ICER’s draft report concluded that only one of the eight drugs under review was cost-effective. But after public input and feedback from the NPF, ICER adjusted some key drug price data in its models. It also heard from the NPF about how psoriasis can lead to other health problems and lower quality of life, and that some of the draft report’s assumptions didn’t capture the real-world uses of certain drugs.

After all this input, ICER’s final report came to a very different conclusion at the end of 2016: all eight drugs were of good value.

ICER made other recommendations favorable to patients, too. For example, it urged limiting or eliminating the insurers’ practice of step therapy—which requires patients to first try cheaper drugs, then “step up” to the one prescribed by their doctor only if the cheaper ones failed to work.

“We were pleased to see that individual patient perspectives and our overarching perspectives were a factor in [ICER’s] decision-making process,” says National Psoriasis Foundation chief operating officer Leah Howard.

ICER says it includes input from patients and patient groups in all of its drug reviews. “When we include information that’s important to patients, then we’re doing a better job of determining what high-value treatments look like,” says Sarah Emond, ICER’s executive vice president and chief operating officer.

Howard of the NPF says her group has been citing ICER’s report in its discussions with payers, and she hopes ICER’s recommendations will soon have an impact on coverage decisions on the drugs in the report.

The NPF hasn’t taken a public position on high drug prices, but Howard says in recent years her group has ramped up its policy work and lobbying at the state and federal level. The foundation has supported state legislation to curb step therapy, limit out-of-pocket expenses, and boost access to biosimilar drugs. (See articles from the New York Times and Stat on actions taken by states on drug pricing)


In 2015, the National Multiple Sclerosis Society conducted a survey of nearly 9,000 people with MS and was struck by the results. Nearly 40 percent said they had trouble affording their medications. Patients reported they had cut back on other necessities, failed to pay bills, or skipped doses of their drugs.

After the survey, the NMSS released its recommendations to improve drug access, despite feedback from a couple of donor pharma companies to be careful about what it said publicly on the topic (as first reported by the New York Times). (Five percent of the NMSS’s funding comes from pharma companies).

The NMSS’s recommendations include limiting price increases and out-of-pocket costs and lowering prices of drugs that have seen large price hikes. Trying to move on these recommendations “has been a major focus of ours,” says Talente.

The society’s efforts are starting to pay off. After more than a year of talks with Roche, the international drug maker decided to price an MS drug—Ocrevus, which the FDA approved last year—nearly 20 percent below the average of other MS “disease-modifying therapies.” The company did not increase the list price, $65,000 per year, for 2018.

And Sanofi Genzyme, which makes two approved MS drugs and was also in discussions with the NMSS, pledged last year to be more transparent and provide a clear rationale for its pricing decisions. It also promised to keep price increases at or below the rate of medical inflation. (Both Roche and Sanofi provide funding to the NMSS.)


The NMSS has had to figure out how to address the pressing needs of patients while also acknowledging its funding from pharma. Talente says the key has been open lines of communication with drug companies. “There are some places where we align and other places, like around prices, where we generally don’t,” says Talente. “But we’ve had some really productive conversations around prices.”

The Leukemia and Lymphoma Society, which relies on drug companies for 18 percent of its funding, took a similar approach with its drug access recommendations. The LLS decided not to blame any one of its “partners” in the drug pricing debate. “To point fingers at any one of our partners was not our intent,” says LLS chief medical officer Nichols.

Instead, it targeted its recommendations broadly. For example, the LLS called on cancer patient organizations to be more vocal about patients’ financial struggles, drug companies to adopt value-based payment models, and pharmacy benefit managers (PBMs; they run prescription drug programs and negotiate drug prices) to pass along to patients more savings from negotiated drug rebates. (See this Wall Street Journal article about UnitedHealthcare’s recent announcement to share some of its drug discounts with customers.)

Mitchell of Patients for Affordable Drugs is skeptical of LLS’s approach. He says he’s grateful to the LLS for supporting research that led to the drugs that have kept him alive (LLS is focused on all blood cancers, including multiple myeloma.). But he says its recommendations are not focused enough to make real change. “They’re not targeting the root cause,” says Mitchell—the high prices set by drug companies, and their tactics to keep generic competition off the market. Mitchell also calls for greater price transparency from both drug companies and PBMs. (In recent speeches, Health and Human Services Secretary Alex Azar and FDA Commissioner Scott Gottlieb also emphasized the need for greater price transparency. And some states have passed legislation on this.)

Mitchell’s group is focused on building a community of patients, training them to be advocates, and lobbying for bills such as CREATES, which is aimed at boosting generics competition. He has also formed a new group to support political candidates who are pushing for lower drug prices.

Mitchell and his group are also talking with ICER about the price of Novartis’s CAR-T leukemia therapy ($475,000). In a recent draft report, ICER called the cell therapy cost-effective. But Mitchell and others published an article arguing that the price should be $160,000.

John Rother, CEO of the National Coalition on Health Care, a nonprofit funded by insurers and healthcare provider and consumer groups, says drug pricing recommendations from patient advocacy groups are welcome, but to really have an impact, these groups need to “support new legislation in Congress that can be more effective in restraining drug prices.”

Whichever approach patient groups take, McCleary of FasterCures says there is at least “a growing sense of responsibility to represent the patient community in this dialogue.” And Rother hopes that this will translate into more action, because patients have a lot of credibility and can sway public opinion. “Patients have the most at stake,” says Rother.