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Study: Health Plans Impose Access Restrictions on Orphan Drugs


This Tufts study has found that plans that restrict orphan drugs do so by narrowing the patient population who can receive them.

Health plans impose restrictions on orphan drugs in about 30% of their coverage decisions, according to a recent study in the journal PharmacoEconomics. These restrictions often go beyond the product’s label, narrowing the patient population who is eligible to receive therapy, lead author James D. Chambers, Ph.D., M.Pharm., said in an interview with Formulary Watch. Chambers is associate professor, Tufts Medical Center Institute for Clinical Research and Health Policy Studies, The Center for the Evaluation of Value and Risk in Health.

James D. Chambers, Ph.D.

James D. Chambers, Ph.D.

These decisions, he said, have the impact of restricting access to orphan drugs. “The common restrictions are those based on patient subgroups, meaning that they're restricting access based on different clinical criteria than the trials,” he said.

Chambers is currently conducting a study to get insight into the clinical restrictions used by plans vs. the clinical criteria used in trials to approve a therapy.

But health plans, he said, appear to be sensitive to cost-effectiveness. “We find that some things are associated with restricted coverage. Across studies, the higher the budget impact the product may have, the more likely health plans would restrict it. The better value for money, the more likely the drug will be covered by health plans.”

In the current study, Chambers and his colleagues assessed commercial health plan coverage decisions for orphan drugs, as well as cost-effectiveness estimates from the Tufts Medical Center Cost-Effectiveness Analysis Registry and the Institute for Clinical and Economic Review’s value assessments.

They found that plans restricted drug coverage in 29.7% of decisions. Plans were more likely to restrict drugs with incremental cost-effectiveness ratios about $50,000 per quality-adjusted life-year (QALY) compared with drugs that had incremental cost-effectiveness ratios of less than $50,000 per QALY. Investigators pointed out, however that the cost-effectiveness data was only available for a minority of the drugs in the sample.

“Seventy percent of the time, the health plans are covering for the same patient population as the FDA approved the product,” Chamber said. “It is encouraging that plans are giving more generous access to products that are more cost-effective and offer greater value for money.”

Investigators also found that plans covered cancer treatments and those without therapeutic alternatives more generously. Plans covered drugs more generously the longer they were on the market. Plans more often imposed restrictions on FDA expedited drugs.

“Accelerated approval is often based on a surrogate endpoint, not a hard clinical end point,” Chambers said. “Health plans are a little bit reticent to cover the product generously. It could be that they are less comfortable with approving the product based on the surrogate end point. There is also the evidence showing the products that the FDA expedites are more likely to be assigned a black box safety warning or even removed from the market. Health plans are just more cautious with those products.”

Chambers said another ongoing study is assessing plans’ coverage of expedited drug approvals. This study is looking at four FDA expedited review programs: breakthrough therapy, priority review, accelerated approval and fast track. “These programs have different criteria for eligibility and have different benefits. For instance, accelerated approval is based on the surrogate end point, not a hard clinical end point,” he said.

Chambers pointed out that the current study didn’t take into account patient cost-sharing, which can be an important factor in access.

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