Drugs extending my life, but Boston institute disagrees

I'm fighting ALS -- and the Institute for Clinical and Economic Review

WHEN I WAS diagnosed 6 years ago with amyotrophic lateral sclerosis—ALS or Lou Gehrig’s disease as most people know it—it was a shock to my system, both figuratively and literally. No one wants to learn they have an incurable disease.

For people living with ALS, our best hope is that the life sciences field will innovate and find, if not cures, then medicines that can delay symptoms and extend life.

When I was first diagnosed, I went on two medicines. And about a year ago, based on the results of a trial, and under the care of my neurologist, I started a third. I’m convinced this cocktail of treatments is slowing the progression of the disease, buying me time with my three wonderful adult children who help me with my mobility which is limited and has affected my lower body.

Access to these medicines is critical. That’s why it’s both disappointing and alarming that a quasi-governmental organization seems to have the power to determine whether a person’s condition is worth treating, by using a discriminatory math tool that doesn’t take into consideration the patient’s lived experience. I’m talking about the Institute for Clinical and Economic Review (ICER) here in Boston.

The tool ICER uses is called Quality Adjusted Life Years (QALY). Basically, it boils a treatment’s worth down to whether the patient’s life is worth the cost of the drug. Sound galling? It is.

Again, I am absolutely convinced my treatment is slowing my disease progression. That translates into more time speaking clearly, more time able to eat normal food, and, most importantly, more time with family and friends. It is literally extending my life.

I recently testified at an ICER meeting regarding the medicines I’m on. ICER’s final report regarding one of my medicines found that the results from one trial aren’t enough, despite approval by the Federal Drug Administration (FDA). To add insult to injury, ICER made its decision by comparing treatment of a rare, rapidly progressing terminal illness to other livable diseases with more treatment options.

It’s simply not a valid comparison and the repercussions are drastic. Already the insurer CIGNA has said it will stop covering that one treatment.

It’s a gut punch. I’m on Medicare which covers most of the cost.  But others aren’t so lucky. ICER’s review doesn’t account for how a patient perceives his or her quality of life with the treatment and whether they feel it’s working.

At the end of every day, I’m a guy fighting ALS with every tool available to me to stay alive and functioning as long as I can. In terms of approved treatments, that current toolbox is severely limited, and my hope is that the pipeline will soon produce more.

But another consequence of ICER’s QALY evaluation metric is that it disincentivizes pharmaceutical companies from investing in new research.

The National Council on Disability has said the QALY scale is discriminatory. And the federal government has barred Medicare from using it.

Another Boston-based organization called No Patient Left Behind proposes another alternative assessment methodology called Generalized Cost-Effectiveness Analysis, which asks a broader set of questions that more fully capture the value of a medicine. Two key examples are:

What will the savings be over the entire life span of the drug when a drug goes generic?

Will a drug ease the burden on caregivers with evidence of reduced costs in total care services?

One thing I have learned is that fighting ALS itself is only half the battle. Whether it’s private insurance, Medicare, the FDA, or federal and state government health policy makers, ALS is fought on multiple fronts. It’s a constant struggle and sometimes feels like I’m slowly being buried alive.

We all want to reduce the cost of prescription drugs. A holistic approach is needed by all stakeholders. The individual evaluation by ICER, on a drug-by-drug basis, has an unproven track record of success to date. Costs have increased dramatically and there is little to no evidence their process has had an impact on reducing costs.

Meet the Author
The goal is to make ALS a livable disease. Let us use every tool we have and encourage the development of new ones.

Steve Kowalski is a Boston resident living with ALS and an ALS advocate.