What Exactly Is a Specialty Medication?
By Myles Starr and Gina Shaw
Health-system specialty pharmacies are known for supplying expensive and complex medications. However, the exact definition of a specialty medicine varies between HSSPs, and even between staff at the same pharmacy.
Data presented in a poster (39-T) at the ASHP Pharmacy Futures 2025 meeting, in Charlotte, North Carolina, indicate that it is possible for stakeholders from a group of HSSPs to agree upon the definition of a specialty medication with the intent to streamline formulary determinations and patient management programs.
“Health systems often collaborate and share best practices about patient care; when we have inconsistent definitions, it makes it harder to share knowledge because we may be talking about fundamentally different things,” lead study author Karen Thomas, PharmD, PhD, of the University of Illinois Chicago, explained in an interview with Specialty Pharmacy Continuum. “Having a consistent definition that works for [HSSPs] will make it easier to communicate and share knowledge.”
After meeting in a working group that used a modified Delphi process, representatives of several HSSPs agreed that the following nine criteria can be used to label a medication as specialty:
1. average wholesale price of more than $24,000 annually;
2. requires a Risk Evaluation and Mitigation Strategy (REMS) program with Elements to Assure Safe Use;
3. limited drug distribution;
4. high-risk adverse event profile, that is, a boxed warning or risk for one or more serious side effects that have at least a 10% chance of occurring;
5. nonadherence, gaps in treatment, or abrupt discontinuation of the medication that’s likely to result in hospitalization, irreversible disease progression, treatment resistance, and/or potential acute adverse events if the medication is not tapered;
6. requires baseline laboratory tests or other screening prior to initiation;
7. requires lab monitoring or imaging at least every three months;
8. complex administration and dosing; and
9. specialized therapeutic classifications.
To arrive at this consensus, potential elements were proposed at an open committee and put through three rounds of asynchronous surveys. Survey participants were asked two questions regarding each possible element:
• Do you recommend this element be considered in defining a specialty medication?
• How important is it (low/med/high)?
If an element met a 75% threshold for consensus, it was included in the list of elements to be further defined. A total of 17 elements met the threshold. In a subsequent open meeting, objective definitions for each element were proposed and some elements combined leading to the final list of nine elements.
To validate the classification system, members of each HSSP who participated in the study were asked to check the specialty designation of 27 medications using the agreed-upon elements. The researchers then used Fleiss’ kappa score to analyze how often respondents gave the same answer for the same drug. A score of 1 means that all people agreed on the same answer all the time.
The elements that were most often agreed upon were:
• requires REMS with Elements to Assure Safe Use (0.87);
• high cost (0.70); and
• specialized therapeutic classifications (0.61).
The elements with the least agreement were:
• significant impact on disease control with nonadherence (0.27); and
• the need for frequent ongoing monitoring (0.36).
Despite the lack of perfect agreement on all criteria, there was strong agreement (0.81) observed in determination of specialty status for medications between institutions.
“Our next step will be to revise these elements, and hopefully be able to put this tool forth as a potential way for institutions to evaluate medications for specialty classification,” Dr. Thomas said. “The idea is to have a baseline for that conversation that is rooted in thoughtful science.”
Dr. Thomas reported no relevant financial disclosures.
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