The Rise of Specialty Medications: Hope for Patients, Hurdle for Health Care

 |  Austin Raper, Ph.D.

In part three of our history of healthcare IT (HIT) series, we explore the origins and industry response to specialty medications. Very few developments in healthcare have drastically impacted stakeholders quite like the emergence and wide-spread utilization of these high-cost, high-complexity drugs.

Beyond potential life-changing clinical benefits to patient health, specialty medications have profound implications for providers, payers, pharmacies and life science brands. We will also discuss current challenges of specialty that can limit patient access and the need for electronic innovation to improve the difficult process for all stakeholders.

Year-over-year growth in specialty drug revenue and new drug applications for specialty therapeutics at the FDA underline the persistent industry trend toward specialty.1,2  Despite accounting for less than 2 percent of all U.S. outpatient prescriptions, it is projected that nine out of ten top-selling drugs will be specialty by 2020 and specialty drugs will account for 47 percent of pharmacy revenue by 2022.1,3


In the past, specialty medications were low volume, often limited to small patient populations for such rare diseases as hemophilia, human immunodeficiency virus (HIV), multiple sclerosis (MS) and Gaucher’s disease.4

Continued research into rare (i.e. ~7,000 diseases affecting less than 200,000 people) and complex diseases (e.g. cancer, inflammatory conditions); however, has resulted in more specialty medications reaching the market.5  During the early 1990’s there were fewer than 30 specialty medications approved by the FDA.4  Today, this number has increased to over 400.6

Rare diseases collectively affect 25 – 30 million Americans and nearly 2 million Americans are diagnosed with cancer each year.5,7  Many of these patients are suffering with few and limited treatment options; however, between 2019 and 2023, it is projected that 65 percent of new drug launches will be specialty therapies, many indicated for rare diseases and cancer.2

The federal government has prioritized research to help such patient populations through FDA-approval incentives (e.g. fast track, priority review, breakthrough therapy designation, accelerated approval) offered in legislation like the Orphan Drug Act, FDA Modernization Act and FDA Safety and Innovation Act.8,9

The Complexity of Specialty Medications

Approved specialty medications and those in development can be complex – often far different than traditional small organic molecules. Many are classified as biopharmaceuticals or “biologics” and can demand novel methods for large-scale manufacturing as well as strict storage and handling instructions. An example of this is cold-chain distribution wherein the product must be strictly temperature controlled from the time it is manufactured to immediately before administration.10

For some newer immunotherapies, like chimeric-antigen receptor T-cells (CAR-T), a cancer patient’s immune cells are removed from their body and sent to a lab for genetic-modification to specifically recognize the cancer, before being reinfused back into the patient. Such complicated science is accompanied with complicated logistics in terms of patient eligibility, provider education and therapy distribution.10

An image depicting the amount of specialty medications on the market today
Biologic (Antibody)

Molecular comparison of aspirin and a specialty biologic (antibody). Relative to traditional drugs, specialty therapies are often large, complex molecules with unique considerations for distribution channel, route of administration and patient access.

Despite all of the attention surrounding specialty, the industry has yet to reach a consensus for what qualifies as specialty. According to the Centers for Medicare and Medicaid Services, generic or brand-name Part D drugs with average wholesale acquisition cost exceeding a $670 per month threshold are eligible for specialty tier placement.11

Beyond this high-cost definition, it is generally agreed that specialty medications can require unique administration (e.g. nebulizer, injections, infusions), consistent patient monitoring through lab tests or regular checkups and time-intensive up-front processes, including enrollment documentation, benefit verification (i.e. coverage determination, medical or pharmacy benefit) safety and educational components, prior authorization (PA) as well as risk evaluation and mitigation strategies (REMS).12

While this extensive process may seem excessive, such regulations are often needed to protect patients while preventing financial waste.

Unfortunately, communication and coordination of efforts among the various healthcare stakeholders to fulfill all of these steps in a timely and efficient manner is difficult, historically not done well and delays time to therapy for patients. Stark differences in distribution, enrollment and reimbursement processes for specialty medications relative to traditional drugs present unique challenges to each healthcare stakeholder.

Specialty pharmacies and specialty hubs have evolved to help navigate and mitigate the challenges impeding patient access.4  While such services attempt to help patients and providers, the overall process is still often considered inefficient and burdensome, with manual fulfillment of steps the current standard.

A centralized and electronic process for streamlined specialty access is lacking industry-wide, leading to time to therapy delays for patients and administrative burdens for pharmacies, payers and providers.

Much like the science behind specialty therapies, patient access to these medications can be extremely complicated. Moving forward, the industry must strive to make manual steps an exception to a mostly electronic specialty process.

As specialty affects the entire healthcare ecosystem, leveraging the capabilities and considering the needs of each stakeholder will deliver a comprehensive, network solution to decrease time to therapy while relieving burdened providers and stressed patients on the specialty journey.


  1. The 2018 – 2019 Economic Report on Pharmaceutical Wholesalers and Specialty Distributors, The Drug Channels Institute
  2. The Global Use of Medicine in 2019 and Outlook to 2023, IQVIA Institute
  3. The Drug Channels Institute, Future Vision: The Top 10 Drugs of 2020
  4. The Evolution of Specialty Pharmacy
  5. FAQs from Genetic and Rare Disease Information Center, U.S. Department of Health and Human Services
  6. Trends in FDA Approval of Specialty Drugs 1990 through 2017, RJ Health
  7. Cancer Statistics, National Cancer Institute
  8. Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review, U.S. Food and Drug Administration
  9. Designating an Orphan Product: Drugs and Biological Products, U.S. Food and Drug Administration
  10. The Key to Commercializing Revolutionary Gene Therapies and Other Orphan Drugs: High-Touch Services that Enhance Patient Outcomes, McKesson
  11. CMS data: CY Specialty Tier Methodology
  12. Section 03: Specialty, 2019 ePA National Adoption Scorecard, CoverMyMeds
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