Trends in FDA approval of Specialty Drugs 1990 through 2017


There are several publications on the Food and Drug Administration’s (FDA) activities over time, but specific information on specialty drug approvals and trends since 1990 are difficult to find. Our overall purpose for this blog is to review the history of approvals and trends since 1990 for specialty medically covered drug.  Initially, we will provide an overview of drug approvals regardless of the potential benefit of coverage, but will subsequently focus on drugs covered only under the medical benefit.

The specific definition of what is and is not specialty is really in the eyes of the beholder and their underlying motives in the way they choose to define specialty, although there are some overlapping consistencies from one organization to the next.  For the purposes of this analysis, we defined a specialty drug per the current definition from the Academy of Managed Care Pharmacy. To be tagged as a specialty drug it had to meet more than 1 of the following criteria:

  1. Drugs produced through biotechnology processes
  2. Traditionally utilized to treat rare and/or complex conditions
  3. Require expertise in handling
  4. Patient education and/or monitoring may be required
  5. High cost

In addition, we flagged each product as having either a high probability of being covered under the medical or pharmacy benefit.  This decision was primarily determined based on the combination of dosage form and route of administration. Actual benefit of coverage may vary depending on the specific health plan or pharmacy benefit management company.

Specialty Drug Approvals

Since 1990, the FDA has approved 789 products and 49 percent of these drugs would potentially receive coverage under the medical benefit (Figure 1).  Focusing primarily on specialty drugs, there has been an overall trend towards increased approvals of specialty drugs (Figure 2). Although the distribution of benefit coverage is variable year over year, the overall trend has been increasing towards greater potential coverage under the medical benefit with 61 percent in 2016 and 62 percent through the third quarter of 2017. On average since 2000, 57 percent of approved specialty drugs would be covered under the medical benefit (Figure 3).

Figure 1: Drug approvals 1990 through 2017 and potential insurance benefit of coverage

Figure 2: Specialty drug approvals 1990 through 2017 and potential insurance benefit of coverage

Figure 3: Specialty drug approval by benefit of coverage percentages

Specialty Drug FDA Review Designation

A comparison of specialty versus non-specialty drugs under the medical benefit that were approved since 2000, supports the current thought that specialty drugs are aligned with usage within conditions where there is a special need. Looking at non-specialty drugs, 76 percent of approvals were for standard drug applications, whereas 77 percent of specialty products were approved through a prioritized review process (Figure 4 and 5).

Figure 4: FDA approval pathways for medically covered non-specialty drugs

Figure 5: FDA approval pathways for medically covered specialty drugs

Review of Medically Covered Specialty Products

Evaluation of the 228 medically covered specialty drugs that have been approved since 1990 shows that there is a greater volume of drugs within a handful of specific therapeutic classes including: oncology, hematological, immunology, central nervous system, and genetic disorders (Figure 6). In general, the volume of drug approvals within these top classes has been steadily increasing since 2000 (Figure 7). This trend should be expected to continue with 309 products in the pipeline for the top 5 classes in either pre-registration or phase III (Figure 8 and 9).

Figure 6: Therapeutic Classes for FDA approved Medically Covered Specialty Drugs (1990 through 2017)

Figure 7: FDA drug approval for the top 5 therapeutic classes 2000 through 2017

Figure 8: Number of pipeline products in pre-registration and phase III for top 5 classes

Figure 9: Number of pipeline products in pre-registration and phase III for top 5 classes


Our analysis supports prior studies indicating a trend towards 50% of specialty drug approvals covered under the medical versus pharmacy benefit.  Although the actual percentage distribution is variable over time, the average since 1990 is in alignment with the 50 percent value.  Finally, our evaluation validated a trend towards increased approvals of specialty drugs reviewed through a non-standard application process.

In the coming year, the RJ Clinical Insights and Analytics team will be working on deeper analysis within specific therapeutic classes to provide insights into historical and future drug trends, which will assist our clients in planning for preferred therapeutic regimens or determine optimal coverage policies for their members.  As part of this process, we would like to solicit recommendations from our customers to help guide the prioritization of drug class reviews.

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