Seated woman making notes on paper, with laptop open; value of rare disease therapies concept

Do Payers Have the Data They Need to Understand the Value of Rare Disease Therapies?

Prices for rare disease therapies continue to feel upward pressure from several sources. Consequently, payers increasingly expect evidence of the therapeutic value of rare disease therapies and their efficacy, expressed in data-driven ways.

In the digital world, creating and capturing data is easier than ever — but analyzing it for necessary insight and evidence still requires work. With the right data, however, providers, pharmacies and other parties involved in rare disease patient care can provide payers the information they need to understand the true value of various therapies.

Payer Demands for Data-Backed Decisions

Value continues to be a major concern for payers when it comes to rare disease treatments. Although pharmaceutical companies have stepped up their work in researching and creating treatments for rare diseases, the costs of doing so remain high.

On average, orphan drugs need four additional years for development. They also reach approval at half the overall success rate, writes Christiane Truelove in Pharma Live.

Fewer than ten percent of known rare diseases have a corresponding approved treatment, says Truelove. When new treatments for rare diseases do appear, payers take note — both of the treatment itself and of the need for evidence to support its cost.

Calls to use value-related data to address orphan drug costs are increasing. In an April 2022 white paper for the Institute for Clinical and Economic Review (ICER), authors Caroline Pearson, Lindsey Schapiro and Steven D. Pearson recommend new and adapted contract structures as a way to improve affordability.

Whether or not the specific examples offered in the ICER white paper are adopted, payers are showing interest in using data to inform and drive payment decisions. Efforts to create value-based contracts, in which manufacturers are paid based on outcomes, are already underway. These contracts offer one way for payers to leverage data for better understanding of value and better decision-making.

In the U.S., federal regulations are also driving payers’ interest in data-driven, value-based decisions. The Value-Based Purchasing (VBP) Final Rule, finalized in December 2020, allows manufacturers to report more granular value-related pricing data to the Centers for Medicare & Medicaid Services (CMS). Payers can evaluate best prices with more information about treatment outcomes under VBP arrangements.

For manufacturers, pharmacies and others in the rare patient treatment chain, understanding payers’ interest in data can support better decision-making when it comes to data collection and analysis. With better data in hand, these participants can persuade payers to follow the paths that result in the best likely outcomes for patients.

Close-up of two professionals shaking hands; value of rare disease therapies concept

The Data Payers Need and Want

Currently, payer data demands vary at the payer level and at the individual contract or policy level. In a 2022 Orphanet Journal of Rare Diseases article, Nikoletta M. Margaritas and fellow researchers examined 65 health insurance policies by 17 U.S. private insurers, looking for the criteria each policy used to approve the use of four disease-modifying therapies for rare neuromuscular diseases.

“No therapy was covered the same way by all insurers,” the authors write. Sixty of the 65 reviewed policies restricted coverage more tightly than the FDA-approved indications for the four studied therapies. Fifty-six of the 65 policies “applied clinical criteria beyond the FDA label indication.” The plans also differed in how severe patients’ symptoms were required to be and which providers they would accept prescriptions from.

Currently, meeting these varied expectations places a significant burden on the healthcare system — as well as being a burden on patients. In the Margaritas et al. study, for example, 37 of the 65 studied policies require a neurologist to prescribe the disease-modifying therapy, and 22 policies require the neurologist to have expertise in the patient’s specific rare neuromuscular disease. Tracking down these specialists poses a burden for some patients, one that may drive them away from using a particular therapy.

Improved data analysis and sharing can help reduce these burdens. Payers will receive more information in a format that is easier to access. They can focus on the data they demand and ignore information they don’t need.

Standardized, interoperable data on efficacy and patient outcomes may help to standardize payers’ expectations as well. With a single set of comprehensive information spanning hundreds or thousands of patients, payers have easy access to key data.

Three professionals in discussion; value of rare disease therapies concept

Organizing Data for Better Payer Decisions

Some participants in rare disease therapy distribution communicate directly with payers. Others may communicate through other parties. Patients, for instance, often rely on their providers’ offices for communication with insurance payers. Patients also rely on industry-sponsored patient support programs to help them coordinate care, writes Nicholas Basta, the founder of Pharmaceutical Commerce.

Patient support groups provide a number of benefits for patients — benefits that remain “underutilized,” says Basta. Support groups can offer a source of data, but connecting payers to this information requires effort.

One way to ensure that payers, patients and other participants collaborate on data demands is by relying on a single platform to bring various parties together. A single digital environment helps to standardize and streamline data. It enhances communication and connection. It allows payers to easily access the data they need, so they can make evidence-based decisions that reflect value and optimize outcomes.

The cost of rare diseases and their treatment remains high — about ten times higher than common conditions in per patient per year terms, according to Pedro Andreu and fellow researchers. “Investment in diagnostic tools, newborn screening, and development of new therapies is justified,” they write, noting that better analysis of cost data can provide “a full picture” of the costs of rare diseases and the value of treating patients.

Rare diseases are still being identified, and rare disease treatments continue to be developed. Payers’ demand for data is likely to remain a constant in any payer relationship for decades to come.

With the right digital tools, other participants in rare disease patient treatment can collect, analyze and share the data payers demand, including the value of rare disease therapies. Better data sharing can lead to better cooperation with payers and a smoother path toward effective patient care.

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