Evaluating the societal value of pharmaceutical treatments for rare diseases

Do we need to look at the protocols for rare diseases differently?

In 2020, a health economics study to measure the societal value of pharmaceutical treatments for rare diseases and inform policymakers about the unmet needs of the rare disease community in the United States (US), was commissioned by Chiesi Global Rare Diseases.

You can download the study, ‘The Burden of Rare Diseases: An Economic Evaluation’ here.

The findings of the report highlight that providing access to rare disease treatments generates substantial value for society because it potentially lowers the associated economic burden on patients and caregivers.

However, the report also reveals how the use of the Quality Adjusted Life Year (QALY) metric in federal programs is actively perpetrating societal inequities impacting rare disease patients and caregivers.

QALY-based assessments focus upon a new treatment’s impact on gain in lifespan, but do not reflect health gains reported by patients with rare diseases that have few if any treatment options available; nor do they incorporate what patients value in a treatment such as less frequent dosing or reduction in infusion schedules.

A recent Rare Disease Company Coalition (RDCC) article, “How QALY Discriminates Against People Living with Rare Diseases” explores these issues at length, and can be viewed here.

It is clear that in the US (and here in Australia) the system favours those with “more treatable” conditions and those with greater potential for health, in terms of functioning and/or longevity.

Given that less than 5-10% of known rare diseases have a treatment, we need to shift the paradigm and find new solutions to overcome current barriers to rare disease therapies.

As Amanda Malakoff, Executive Director of the RDCC, mentioned in her June update to members, “Even the most innovative and effective rare disease treatments are only as valuable as they are accessible.”

At Asteri, we couldn’t agree more, and fully support the RDCC’s key recommendations (to US federal and state policymakers, regulators, payors and other stakeholders) to streamline patient access, including:

1. Accelerated Reviews

2. Facilitate Medical Access

3. Ensure Protections in Employee Health Plans

4. Protect Accelerated Approval Therapy Coverage

5. Streamline Value-Based Payment Methods

6. Ensure Medical Necessity Determinations

We can’t lose sight of the fact that we need to continue to innovate our system, ensure it is fit for purpose and rethink approval parameters for new rare disease therapies.

Currently, the TGA may requires up to 24 months safety data for a disease of orphan designation, despite the fact that most people with Rare Diseases are severely progressed (or expire) within 2 years of being diagnosed.

This is just one example, but it clearly illustrates how the way the regulator and the payer assess and fund innovative, new therapies needs to evolve.

We need to challenge the status quo. There’s too great a cost to our society if we continue the way we are.

To learn more about why Asteri supports the Rare Disease Company Coalition, click here.