Background

The US Food and Drug Administration’s (FDA) Accelerated Approval (AA) program enables expedited access to drugs for patients with serious unmet medical needs. Medicines granted accelerated approval must adhere to the same statutory standards for approval as medicines receiving a traditional FDA approval, including the requirement to demonstrate substantial evidence of effectiveness based on adequate and well controlled investigations. The AA pathway allows the FDA to approve drugs based on surrogate endpoints that are reasonably likely to predict clinical benefit or on intermediate clinical endpoints reasonably likely to predict an effect on irreversible morbidity or mortality. Manufacturers must conduct confirmatory trials after accelerated approval has been granted. If the confirmatory trial shows that the drug provides a clinical benefit, then the FDA grants traditional approval for the drug. If the confirmatory trial does not demonstrate this, the FDA may remove the drug from the market.

The AA pathway aims to improve patient access and health status for people with unmet medical needs. At the same time, racial and ethnic minority populations often have disparities in access to prescription drugs, particularly for novel treatments and technologies. In a new analysis, Avalere sought to determine if there was a correlation between the number of recently approved AA drugs and utilization by different subgroups of Medicare beneficiaries.

Approach

Avalere selected a sample of AA drugs that were approved between 2012 and 2021 with indications for iron overload, blood cancers, and other cancers using the FDA’s Center for Drug Evaluation and Research’s (CDER) Accelerated Approval list. Clinical experts extrapolated each product’s therapeutic area from the “Accelerated Approval Indication” text.

Using Medicare fee-for-service (FFS) claims, Avalere determined the number of beneficiaries using each AA drug and compared the demographics of those beneficiaries to those of the overall Medicare population. Avalere examined utilization of products approved via the AA pathway in Medicare FFS claims and Prescription Drug Event data for years 2012–2021. The analysis then considered utilization data for these selected AA drugs by dual eligibility status (i.e., individuals receiving Medicare and Medicaid benefits), rural or urban status, beneficiaries’ race/ethnicity, and their original reason for Medicare entitlement.

Results

Drugs Approved Under AA Process: Avalere estimated the total number of drugs approved under AA that Medicare beneficiaries accessed by therapeutic area. The number of AA drugs in each of the three therapeutic areas increased from 2012 to 2021 across all therapeutic areas studied, as shown in Figure 1.

Figure 1. Number of AA Drugs Across Three Therapeutic Areas

Access to AA Drugs for Dual-Eligible Beneficiaries: Duals account for roughly one in five Medicare beneficiaries during the period studied. The analysis found that a proportional share of beneficiaries filling prescriptions for blood cancer and other cancer AA products in 2021 were duals, at 18% and 19%, respectively (Figure 2). However, a disproportionate share (69%) of those who received an AA drug for iron overload were duals.

Figure 2: Percent of Dual-Eligible Medicare Beneficiaries Using AA Drugs Across Therapeutic Areas

Use of AA Drugs in Rural vs. Urban Settings: The analysis also found that a higher proportion of Medicare beneficiaries in urban areas utilized AA products than in rural areas. Based on the available rural and urban data, utilization for AA drugs was found to be substantially higher in urban settings (72% each for blood cancers and other cancers, 75% for iron overload drugs) compared to rural settings (8% for blood cancers, 9% for other cancers, 5% for iron overload). This is consistent with the general rural/urban split of the Medicare program (e.g., more beneficiaries live in urban areas).

Use of AA Drugs Across Different Race/Ethnicity Groups: The analysis also considered utilization of AA drugs for these conditions by beneficiaries across the CMS-defined categories of race/ethnicity.

Dual-eligible beneficiaries who received AA drugs for iron overload were more often Black or Asian than other racial/ethnic groups (Figure 3). This is generally consistent with the prevalence data for beta thalassemia and sickle cell disease, two conditions commonly treated with iron overload products. For AA drugs that treat blood cancers and other cancers, both dual and non-dual Black and Asian beneficiaries utilized these medications at rates approximately proportionate to the full Medicare population, while White beneficiaries had disproportionately higher use and Hispanic beneficiaries had disproportionately lower use.

Figure 3: Racial and Ethnic Distribution of the Medicare Population and Beneficiaries Utilizing AA Drugs, 2021

Implications

The AA pathway is a means of allowing patients access to novel treatments for life threatening diseases earlier than they might otherwise receive that therapy. The number of drugs approved between 2012 and 2021 with  AA  increased the number of treatments available to groups with unmet medical needs, including historically underserved populations.

The findings of this analysis indicate that the AA pathway offers increased access to drugs for treatment of certain conditions, such as beta thalassemia and sickle cell disease, for groups of beneficiaries who are disproportionately affected by these conditions, including Black and Asian beneficiaries. Further, increased access for these populations under the AA program could lead to improved clinical outcomes for patients with unmet medical needs.

Methodology

Avalere conducted a quantitative assessment of a subset of AA drugs to evaluate utilization patterns among beneficiaries enrolled in Medicare FFS. Avalere started with the FDA CDER list of Accelerated Approvals and extrapolated each product’s therapeutic area from the “Accelerated Approval Indication” text to create the list of AA drugs for each of the selected therapeutic areas (iron overload, blood cancers, and other cancer more broadly). This encompassed 106 products in total (four for iron overload, 48 for blood cancers, and 63 for other cancers; nine products are used to treat both blood cancers and other cancers).

Beneficiaries were categorized as dually eligible if they were dually eligible in at least one month within the calendar year. To determine rural/urban status for beneficiaries in the sample, Avalere mapped beneficiary ZIP codes to Rural-Urban Commuting Area codes. Approximately 20% of beneficiaries do not have a rural/urban indicator tied to their ZIP code; those individuals are not included in the cited rural and urban data points.

The “blood cancers” category includes products that treat beneficiaries with a variety of diagnoses, including leukemia and lymphoma. The “other cancers” category includes all products that treat cancers not identified as blood cancer. Iron overload products are commonly used to treat beta thalassemia and sickle cell disease.

Funding for this research was provided by PhRMA; Avalere retained full editorial control.