ICER says rare disease patients are too expensive to treat. Here’s why ICER is wrong.

Rare disease patients face challenges at every step of their patient journey.

One insurance-backed care-cutting think tank wants to erect another barrier between patients living with rare diseases and the treatments they need and deserve.

In its latest white paper, “Assessing the Effectiveness and Value of Drugs for Rare Conditions,” ICER does little to present compelling evidence-based data to justify a regime of price controls, less to justify a retro consideration of the concept of orphan drugs in the scope of medical treatment, and practically nothing to inspire confidence for patients and the millions of caregivers helping them.

This ICER value framework for the assessment of treatments for ultra-rare conditions will manifestly not help patients in new ways, especially those in ICER’s upcoming assessments: CAR-T Therapies, Tardive Dyskinesia and Voretigene Neparvovec.

Whenever we evaluate an ICER report – and we do read all of them cover to cover – we are always left asking: Why are we evaluating a treatment that lacks the empirical data and mainstream methodology needed and necessary to advance a meaningful recommendation for payers, manufacturers and patients?

ICER doesn’t appear to be assessing medicines, so much as chasing FDA approved drugs for assessment, one after another, hounding the medical industry with roadblocks already overcome and questions already asked and answered.
ICER Rare Disease Report: Assessing the Value of Drugs for Rare Conditions

The Orphan Drug Act was a breakthrough idea in 1983 – a different and age and less-advanced age. Further, we would argue that with 95% of rare disease patients waiting for treatment – it’s needed now more than ever. We have engaged actively with the rare disease community and the one through line for these patients is what it costs them every year — not having a treatment. Living with and caring for someone with a rare disease is an emotional burden on families and to your point, a cost burden for society at large.

For the patients who need The Orphan Drug Act, it’s certainly not moving fast enough. They needed it yesterday, and the only hope they have lies in its existence. That’s why Patients Rising recently joined with the Global Liver InstituteFibrolamellar Cancer Foundation, and Melanoma Research Foundation in highlighting the flaws with the ICER Rare Disease Report.

Here are four flaws with the ICER Rare Disease Report highlighted by our coalition representing the rare disease community, including patients with melanoma, rare liver diseases, liver-mediated diseases, and liver cancers.

1. ICER invents new categories and arbitrary designations.

The ICER Rare Disease report establishes a novel category of disease: the “ultra-rare” diseases. This category is arbitrary and undermines the Orphan Drug Act of 1983 which has proven to be a successful driver of innovative, life-changing and life-saving treatments for patients in the United States.

Under ICER’s proposed amendment, patients who have a disease that affects between 10,000 and 200,000 individuals will be effectively lumped into the “common diseases” category, and the complexities of clinical trial recruitment, study design, evidence generation, and relevant elements to value calculation involving rare diseases that fall in that category will be essentially ignored.

2. QALYs misrepresent value of patients with disabilities, chronic diseases

QALYs are a flawed method of determining value. Quality-adjusted life years assumes a greater value for individuals with a higher baseline health status. Individuals with disabilities or chronic conditions are disadvantaged anytime QALYs are used.

By embracing QALYs, ICER’s determinations conflict with federal policy. By law, “the Secretary [for Health and Human Services] shall not utilize such an adjusted life year (or similar measure) as a threshold to determine coverage, reimbursement, or incentive programs” in the Medicare program.

As so well articulated in the Partnership to Improve Patient Care’s white paper, “Measuring Value in Medicine: Uses and Misuses of the QALY,” the disconnect in using an academic tool to influence real-life policy based on a presumed ability to quantify the quality of individual patients’ lives can be seen in how QALYs are measured and calculated.  The seminal Second National Panel on Cost Effectiveness notes that the “quality and usefulness of QALYs depends on the quality and validity of the utility scores used to calculate them.”

Most concerning of all, ICER won’t vote on ultra-rare treatments that exceed $175,000 cost per QALY threshold. In essence, ICER is preemptively declaring a price – without debate or input from patients and caregivers. It is imperative that, in assessing the value of any treatment, especially for rare diseases, the patient and caregiver experience be heard.

3. ICER ignores the substantial costs associated with the absence of a treatment.

ICER ignores the substantial costs incurred when an effective treatment doesn’t exist. The right treatment can reduce our overall health care spending by eliminating the need for large teams of specialists, endless tests and a patchwork of mitigating therapies.

Patient Kelly Lease, who is living with Stiff Person Syndrome, has spent years visiting different hospitals and specialists. There’s no treatment for her rare neurological disease, which means she must constantly undergo more tests, experiment with new medical studies and modify her treatment plan as it loses effectiveness. If a treatment existed, she would no longer need a large team of doctors, caregivers and specialists. She would no longer need new medical tests. And she could also increase her productivity — with more hours at a higher-paying job.

The right treatments for rare diseases can reduce health care spending on medical expenses that only mitigate symptoms and manage pain.

4. ICER Rare Disease Report discourages innovation for patients with rare diseases

The ICER Rare Disease Report discounts the practical challenges of clinical research and development in all rare diseases. Categorical price caps will greatly reduce investment in therapies for rare diseases, a landscape that only has 625 FDA approved orphan drug approved treatments for 7,000 conditions.

Adoption of these proposals could have a profound negative impact on patients with both so-called “ultra-rare” and rare conditions such as, autoimmune hepatitis, primary sclerosing cholangitis primary biliary cholangitis (PBC) and fibrolamellar cancer, rare hepato-biliary diseases which require more, not fewer, incentives for research as they have no cures.

What’s your take on the ICER Rare Disease Report?

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