Patient Advocates Need to Oppose Anti-Patient Policies

Being a patient advocate is an uphill battle, and those of us in this community are often fighting because of personal struggles or loss. Advocating for patients with rare diseases is even harder. There are fewer of us to make noise and certainly no pink wristband-clad football players helping to raise awareness, which is crucial to developing cures and expanding access to treatments.

Thirty million Americans suffer from a rare disease – one in ten of our friends, co-workers and family members. I know this all too well because my husband is one of them. In July 2010, my husband was diagnosed with Mast Cell Activation Disease, a rare disorder caused by too many mast cells in the immune system. My children were diagnosed soon after. The symptoms are many, painful and devastating.

No cure exists, but major developments over the course of the last two decades have allowed patients to live with the disease. This has proven most effective when children receive treatments soon after diagnosis. With a disease so rare, treatments are horrifically expensive. Some of this is covered by insurance, but as insurers and others in the health care industry increasingly seek to avoid paying for costly treatments, patients and their families are left to foot the bill.

Anyone who’s ever dealt with an insurance company knows how complicated recouping the costs of treatment can be, from the most basic checkups to lengthy hospital stays. Prescription drug coverage, though, might take the cake. It seems like every time I try and fill a prescription for my husband or kids, the forms get longer and the process trickier. And that’s after the prescription is approved! Insurance companies seem to be working harder and harder to keep their own costs down by shirking their responsibility to pay for drug coverage, even as my family’s premiums are more expensive than ever.

Prior authorization may be one of the most frustrating policies we’ve dealt with. It’s a requirement that “your physician obtain approval from your health insurance plan to prescribe a specific medication for you,” according to one article from Consumer Affairs, and is a “technique for minimizing costs.” It’s also enough to pull your hair out because, as the article notes, “Without this PA, your insurance plan may not pay for your medication. Even then, the insurance company – not the doctor – has the final say in the matter. And they can turn you down.”

Insurance companies and pharmacy benefits managers (PBMs), which negotiate prices and set drug coverage plans for tens of millions of Americans, are requiring prior authorizations more and more. To justify this, they’re increasingly turning to reports from a Boston-based group called the Institute for Clinical and Economic Review (ICER). These reports analyze the cost of new drugs. Really, though, they provide public cover for insurance companies to require prior authorizations or implement other restrictive policies for accessing expensive treatments.

In May, ICER claimed that new drugs for a rare blood cancer, called multiple myeloma, are too expensive. The drugs are designed for patients who have not responded to previous treatments, but insurers can now claim that ICER’s report justifies reducing patient coverage. That’s how insurers and PBMs have reacted to past reports.

On July 15, ICER will have a public meeting in Portland, Maine, on a report about drugs for certain rare liver diseases. ICER has already indicated that it views the drugs to be too expensive.

ICER’s review system should raise alarm bells among the patient community. It uses a metric common in the United Kingdom called Quality-Adjusted Life Years (QALYs), which often results in drug rationing. Jennifer Hinkel, a cancer survivor and health economist, described QALYs in a column titled “Having Cancer Doesn’t Make Me Worth Less” this way: “In the most basic terms: if you get sick, you become a fraction, and your time no longer has the same value as a fully healthy person’s time.”

Advocates for patients should join her in calling for insurance companies, PBMs and others to publicly reject ICERs reports. We need more innovation and awareness to find new treatments and cures. We don’t need excuses.