Widely regarded as a powerhouse among patient advocacy groups, the Cystic Fibrosis Foundation (CFF) “is a bit unique from some of our peers in that we are by default the medical specialty society, so we’re convening for evidence-based guidelines” at the upper levels while working on behalf of individual patients on the front lines, Mary Dwight, the senior vice president for policy and patient assistance programs with CFF, told BioWorld Today. “We’re able to come in not only [wearing] the patient hat, but with the clinician hat of the experts who treat the disease.”

It’s important, Dwight said, to crusade for public-policy changes but also help with “granular, day-to-day, on the ground” matters that confront patients in their struggle with big bills and how to pay them. “We think about it holistically, in terms of a conversation [about] what’s required for CF” to be treated to an extent that matters and in a way that doesn’t financially bankrupt patients in a “health care system [that] overall is changing pretty rapidly,” she said. “We really see our job as advancing not only research but innovative policies that make sure people with CF have access to high-quality specialty care.” Though drugs represent “a major component,” ancillary costs are involved (as with other diseases), and those add more strain to the drug burden. In CF, patients find they need doctors to assist them in looking after general respiratory well-being, nutrition, physical therapy and mental-health counseling. “Our job is to put all those pieces together comprehensively for the payers, the specialty pharmacies, the pharmacy benefit managers [to say,] ‘This is what CF looks like, this is what we as the CF community see as good CF care. It’s not just one drug,’” she said.

And, as with other illnesses, a worrisome hurdle is getting payers to provide what’s needed. “What’s the reality of our interactions with the insurance companies? Most of the time we’re coming in with information, and I think by and large the insurers – and we engage with Medicaid agencies, Medicare and a lot of the private plans – are open to what we bring,” Dwight said. Along with the same kind of clinical evidence that’s used to inform recommendations about guidelines, CF reps arrive with “information from our CF patient registry, which tells the story of who would benefit from a particular therapy and what the outcomes of that have looked like over time,” she said. Such a package “really makes for productive conversations,” she added. “I think the challenge a lot of times with insurance-benefit design for something like CF, which is a rare disease, is that we kind of get lumped in with larger efforts to contain costs.” Typically pancreas-insufficient CF patients, for example, may find themselves taking, along with the average of eight to 15 drugs for their condition, 30 to 40 pancreatic enzymes per day in order to digest food. But the pills also are prescribed for cancer patients, who “would never take that volume,” and the insurers’ criteria may be tailored to fit oncology rather than people with CF, she said. “We’re able to get the CF expert on gastrointestinal issues and nutrition on the phone with the medical director to have that conversation so the formulary can be adapted.”

Not every patient, though, has the weight of CFF to rely on. Pharma company programs offering copay assistance represent an “important piece,” even as advocates deal with the matter “systemically, [starting] the conversation around health care and insurance benefits, and what constitutes the right level of coverage” for patients in general and specifically, Dwight said. “If I’m going to meet with a nutritionist today, is that included in my visit to my CF care center or is that a separate copayment? Those are factors that people with CF have to consider when they’re choosing an insurance plan, and when they’re making an appointment with their CF care team.”

making your own case

Although severe across the board, the pricing pinch may be especially clear in rare diseases. Cynthia Buness, an advocate with the Washington-based National Patient Advocate Foundation, said she sees merit on both sides of the payer vs. patient argument. “Insurance carriers and providers want to try a cheaper alternative first, and so if something’s not proven and it’s an expensive treatment, I understand why a carrier is not going to want to pay for it,” she said. In that scenario, lacking an outfit like the CFF backing her up, a patient who’s in that situation “has to be able to make a case as to why this treatment is going to work for them,” she said, a tricky proposition in any case but especially so as the Affordable Care Act continues to undergo refinements.

Buness focuses on primary sclerosing cholangitis (PSC), a chronic disease that damages the bile ducts and leads to liver failure in 10 to 15 years. Her daughter is afflicted. No accepted therapy for PSC exists, but vancomycin has been shown to help some patients. “It may be that there’s a subcategory of these patients who have a separate type of disease,” she said. “When it does work for patients, it works phenomenally. They have reversal of their symptoms, and it does not appear that the disease progresses.”

The problem is that scant data mean patients not only have a hard time making the case, but may not always be sure the case is worth making. “It’s kind of an experiment on a patient-by-patient basis,” she said. Generic medicine seems to work differently from branded ones, but nobody knows why. “Some insurance companies cover it and some don’t, [so patients] have to try to pay for it on their own and the drug, if you take the pill form, is very expensive,” she said. A cheaper liquid form of vancomycin exists, “assuming that you could even get your doctor to prescribe it, because most doctors refuse,” given that no clinical trials have proved its worth in PSC. Yet “companies are not willing to fund a study [to prove its value], and they’re not willing, typically, to donate the drug,” she said.

Buness is pursuing the possibility of trials. One, in pediatric patients, could be funded by the Patient-Centered Outcomes Research Institute (PCORI). For another, in adults, she’s seeking private money. Meanwhile, she has located a pharma firm willing to donate liquid vancomycin. “We’re very blessed,” she said, adding that the company “has been very gracious and interested in this work. We wouldn’t be able to pursue any of our studies” otherwise. In support of potential trials, individual case reports are being published, and the Mayo Clinic is putting together a series. “Once we get some minimal data out there,” the financial wherewithal might come for larger experiments. “You’ve got a catch-22 for patients right now,” she said.

Buness lucked out in another way. Her insurance carrier, which she asked not be named, paid for vancomycin, albeit only when she was done persuading the doctors. “After the first three months of treatment, I got a call from [the insurance company],” she recalled. “We take monthly blood tests, and I showed how [vancomycin] affected her liver enzymes. When you’ve shown that kind of causative effect, it’s hard for them to turn around and say, ‘OK, we’re going to make her go back to being sick and progressing to liver failure.’ One of the routes that patients have is to get a trial period on the medication, and then they have to be very diligent about proving there’s a positive effect.”

But procuring enough of the drug to individually test is hardly simple because of the same conundrum. At first, she said, “I couldn’t get the doctors in Phoenix to prescribe it,” so she gathered information from adult research in PSC at Mayo, and found that a researcher at Stanford University had published a small study (not a clinical trial) in 11 to 12 children. “I went to our doctor in Phoenix and said, ‘I’d like to try this, nothing else has worked for her,’” she said. The answer was no, and Buness ultimately had to travel to California for the drug. Within a few days on vancomycin, her daughter was improving, gained weight and soon “was basically normal.” Buness said she expects to hear in a month or so whether PCORI will fund a full study.

‘What the industry is about’

While lobbyists in Washington try to navigate – and influence – changing policies, Erin Singleton, chief of mission delivery for the Hampton, Va.-based Patient Advocate Foundation, works solely in the trenches with patients. Flexibility is the key here, as relief may come via many routes. “Sometimes, affordability issues can be presented because you didn’t know that you were using a pharmacy that’s not part of network,” she said. “Maybe you can get a mail-order so you can get a reduction in out-of-pocket costs, if you knew that was an option you could tap into,” as many patients do not. The free-drug and discount programs set up by some pharma companies can help those who are altogether without insurance, but of course “not every drug has a program. And there are income guidelines, sometimes disclosed and sometimes not,” she said, though most of the programs that do exist “have gotten to the point where you can call them and run through some eligibility guidelines. We do that a ton with patients we serve here.”

Frustrated and helpless, patients may give up too easily. “All of it is urgent, all of it’s very important for the course of treatment for the person, but if they don’t know that there are potential solutions, I would hate to see they are making decisions” not to pursue options, she said. Charitable copay programs may be sorted through, and “sometimes, we can be creative here at the organization and find resources that might be very specific to a disease, or an area of the state,” she said.

Jeremy Levin, CEO of Ovid Therapeutics Inc., of New York, has been an outspoken activist in the storm over drug costs. “My position is this is more about structural issues that bring pricing into being, the incentives that drive pricing, and the parties that are involved,” he said, noting that the present state of affairs is “not a crisis [that happened just] now. This is something that has built for more than 30 years, and it’s grown specifically because it was able to grow.”

Insurance companies, pharmacy benefit managers and sometimes hospital intermediaries have added to drug expenses; pharma companies represent only the “obvious target,” he said. The trouble arrives when “any one member of that chain of supply to the patient suddenly says it needs to increase profits,” he said. In any other transaction, “you hand over cash, and you pay full value, and you know exactly what you buy. When you buy a drug, you do not know what you’re actually buying and who you’re paying [your money] to.”

Solutions? In generics, Levin said, innovation in manufacturing will have more significant effects on pricing, because it will allow for better margins, but the “only three that matter” in generics are Mylan Inc., Teva Pharmaceutical Industries Ltd. and Sandoz Inc. “The flip side to this is that, in the branded area, you have a much more difficult task, because of the plethora of companies and of new products,” he said.

“You need to have all parties who are providing medicines to patients understand that they have a primary obligation to the patient, and if they are successful in executing on that, they will be profitable,” Levin said. “They need to work together to establish that obligation, and those who are not trying to do this, who are simply trying to exploit the patient should be identified and, in many ways, they should be called out.”

Discounts and coupons from pharma firms represent “an important gesture,” he said, but they fall far short of the respectful partnership that should rightly be maintained between the provider of a drug and the patient whose life will be changed by it. (See The ‘tragedy’ of discount coupons, below.)

Levin praised Johnson & Johnson’s published corporate credo which, in its first paragraph, brings up affordable pricing. “This is not a question of moral [obligation],” he said. “It’s a question of good business. It’s a question of what the industry is about.”

The ‘tragedy’ of discount coupons

By Anette Breindl
Senior Science Editor

In one sense, discount coupons have some similarities to the “tragedy of the commons,” where overutilization of common resources leads to their depletion. From the point of view of the companies giving the drug assistance, one goal is to steer patients toward high-cost brand-name medications. But that depletes the overall pot of funds that is available to pay for health care.

But health policy researcher Peter Ubel, of Duke University, told BioWorld Today that the situation is more complicated for health care.

The classic example of the tragedy of the commons, and the one that was first used to describe the problem in the 19th century, is that of sheep grazing on common land. For each individual farmer, it makes sense to put as many sheep as possible on the land in the short run. But in the long run, such behavior wrecks the commons.

Money for health care, Ubel said, is “not as obviously scarce a resource . . . We don’t have a fixed amount of money we can put into the health care system.”

At some point, the overall price of health care becomes unsustainable. But because no individual patient, and no individual company, has the overall price of health care as their primary concern, at the level of the individual prescription “it feels like we can be giving everybody everything. That’s what’s really tricky.”

In a recent editorial in the Annals of Internal Medicine, Ubel and his colleague Peter Bach, of Memorial Sloan Kettering Cancer Center, argued that as it is currently practiced, copay assistance leads to higher prices overall by steering patients toward more expensive medicines.

They also make the high cost of medication more palatable to the individual who is filling a prescription, and that, Ubel said, prolongs the strain on the system, because “public pressure on prices is going to be a big lever to drive prices down.”

Ubel and Bach do not advocate getting rid of copay assistance altogether. “If we did away with [it], it would hurt patients,” Ubel said.

But the programs are nevertheless the opposite of a free lunch, he said: “It passes the costs on to other people.”