By Jeni Hayes, PharmD, BCPS
Vizient Senior Clinical Manager, Pharmacy Outcomes
Specialty pharmaceuticals have all but exploded into the modern healthcare scene over the past decade, with over 300% growth in specialty pharmacies between 2015 and 2023. With one-third of that growth coming from hospital or health system-owned specialty pharmacies, awareness of specialty drug approvals and the pipeline is increasingly important for pharmacy leaders.
Likewise, the specialty drug pipeline and approvals by the U.S. Food and Drug Administration (FDA) reflect the shift of pharmaceutical therapies from small molecules to increasingly complex and sophisticated biologic and specialty drugs. In 2022, 37 new molecular entities were approved by the FDA through four distinct pathways: priority review, breakthrough therapy, accelerated approval and fast track status. Each pathway expedites the approval process in different ways and can sometimes overlap.
Below, we dive into the most novel, innovative and impactful of the 37 therapies approved by the FDA in 2022 as well as their approval pathways.
Accelerated Approval
The FDA Accelerated Approval Program is one approval option for new drugs intended to treat serious conditions that offer meaningful advantages over currently approved therapies. Through this program, drugs may be approved more quickly by using a surrogate endpoint, that is a marker that can be studied for a shorter treatment duration and is reasonably likely to predict clinical benefit. Drug companies are able to get a new medication to patients considerably faster than traditional approval methods and conduct additional post-marketing studies to ensure safety and efficacy of the new therapy.
In 2022, six of the 37 new drugs (16%) were approved under the accelerated approval program. All six of these therapies are specialty drugs indicated for the treatment of different cancer types, including lung cancer [adagrasib (Krazati)]; bile duct cancer [futibatinib (Lytgobi)]; recurrent ovarian cancer [mirvetuximab soravtansine-gynx (Elahere)]; follicular lymphoma, the most common type of low-grade non-Hodgkin lymphoma [mosunetuzumab-axgb [Lunsumio)]; myelofibrosis, an uncommon type of bone marrow cancer [pacritinib citrate (Vonjo)]; and multiple myeloma, a cancer that forms in plasma cells [teclistamab-cqyv (Tecvayli)]. Tecvayli is a treatment for relapsed/refractory melanoma and joins the class of chimeric antigen receptor (CAR) T-cell therapies for patients with advanced leukemia or lymphoma who previously had no other treatment options. Given that five-year survival rates for these advanced blood cancers is around 25-50%, any new therapies represent a promising new option to extend survival.
Breakthrough Therapy
The FDA Breakthrough Therapy designation aims to expedite the development and review of drugs intended to treat a serious condition where preliminary evidence suggests it may demonstrate substantial improvement over existing therapies for clinically significant endpoints. Unlike accelerated approval, the approval of these drugs demonstrates an effect on morbidity, mortality or serious consequences of a diseases rather than a surrogate endpoint.
Last year, the Center for Drug Evaluation and Research (CDER) at the FDA designated 13 of the 37 novel drugs (35%) as breakthrough therapies. Three of these breakthrough therapy approvals were for the treatment of dermatologic conditions, including atopic dermatitis [abrocitinib (Cibinqo)], generalized pustular psoriasis flares [spesolimab-sbzo (Spevigo)] and metastatic uveal melanoma, a cancer that spreads through the blood and forms a tumor in a different part of the body [tebentafusp-tebn (Kimmtrak)]. Cibinqo joins injectable specialty medication dupilumab (Dupixent) for the treatment of atopic dermatitis, a condition with a global atopic dermatitis market size of almost $13.5 billion and is expected to grow to $28.7 billion by 2030, based on the large segment of the population this disease state impacts.
First-in-Class
A first-in-class therapy represents a new way to treat a certain disease state that is totally different from existing therapies currently on the market. Last year, CDER identified that 20 of the 37 new drug approvals (54%) were for first-in-class therapies. First-in-class drugs have represented the majority of new drug approvals in both 2022 (54%) and 2021 (54%).
Overall, 13 of these 20 first-in-class approvals were for specialty medications treating various disease states, including hypertropic cardiomyopathy, which can lead to heart failure, [mavacamten (Camzyos)]; hemolytic anemia, a disease that destroys red blood cells at a speed faster than they are made [mitapivat sulfate (Pyrukynd)]; HIV [lenacapavir (Sunlenca)]; as well as many of the dermatology and oncology new drug approvals listed above.
As manufacturers continue to develop medications for rare diseases, specialized conditions and relapsed or refractory cancer types, the specialty drug pipeline will continue to be important for health systems, patients and insurers alike. This pipeline is expected to maintain a continued growth rate of 8% annually through 2025.
Vizient provides a variety of resources related to specialty pharmacy as well as new drug approvals:
About the author:
Jeni Hayes is the Senior Clinical Manager for Pharmacy Outcomes within the Center for Pharmacy Practice Excellence at Vizient, a health care performance improvement company in Irving, Texas. In this role, she develops actionable clinical insights and forecasting resources for members and suppliers. Hayes earned her PharmD degree from MUSC Health in Charleston, SC and her Master of Science in Pharmacoeconomics from the University of Florida. In addition, she completed a PGY1/PGY2 Health System Pharmacy Administration residency at MUSC Health and is a Board-Certified Pharmacotherapy Specialist. Prior to joining the Vizient team, Hayes spent 13 years at MUSC Health in roles related to Outcomes Management, Residency Leadership, and most recently as the Clinical Manager for the Specialty Pharmacy Program.