The Evolution of Specialty Medicine

In recent years, specialty pharmacy has branched out from representing a small subset of pharmacies to become its own industry. Specialty pharmacy originated nearly 50 years ago and was an area of medicine that was designed to serve patients who needed specific therapies for various conditions.

Originally, these therapies mainly consisted of infusions to treat diseases such as hemophilia, cancer, and malnutrition.¹ An industry that originally dispensed medications to treat a small number of conditions has now transformed into a conglomerate that serves a population of patients with more than 6800 rare diseases that are treated by more 450 specialty medications.^2,3

As more specialty drugs enter the market, specialty pharmacy can be considered one of the fastest growing medical industries to date. With the groundbreaking advancements that we see every day in the specialty focus, it is important to recognize the uniqueness of these medications and their respective populations.

These medications are considered to be specialty based on criteria set forth by the FDA and the manufacturers. Specialty medications are typically prescribed to a person with a complex or chronic medical condition, defined as physical, behavioral, or developmental.

Many of these conditions have no known cure, are progressive, and are debilitating or fatal if left untreated. Due to the uniqueness of these medications and the diseases that they treat, they require additional patient education, adherence monitoring, and support beyond traditional dispensing that is seen throughout regular pharmacies.

Most of these medications have high out-of-pocket costs and patients often require payment assistance programs for the opportunity to afford the medication. Unlike non-specialty medications, specialty drugs carry unique storage or shipment requirements.⁴

If these requirements are not met, it can lead to a rapid loss of stability that leaves the drug unsafe or ineffective, resulting in an expensive loss of product. In considering all the aforementioned, it is impressive that such a small industry continues to grow at such a rapid pace.

One of the major focuses in specialty pharmacy is treatment of the HIV epidemic. With approximately 1.1 million people in the United States living with HIV today, there is a specific need for this type of care.⁵ Treatments for HIV have been attracting attention since the beginnings of the specialty market.

In 1996, Merck launched the drug indinavir (Crixivan), which served as the market’s third protease inhibitor for the treatment of HIV. The significance behind this launch was that the drug was approved by the FDA just 42 days after receiving the New Drug Application, which set a new record.

This drug was to be dispensed under exclusive contracting through Stadtlanders, an independent specialty pharmacy based in Pittsburgh, Pennsylvania.⁶ This contract set a precedent for how various specialty drugs are dispensed through a limited distribution network.

To date, there are approximately 50 HIV medications that can be used as part of a treatment algorithm.⁷ The high-touch communication and care provided to patients that the specialty industry has performed has led to even greater outcomes with constant advancements to specialty medications.

As previously stated, the FDA has approved more than 450 specialty medications for these rare disorders. Many patients with rare diseases require multiple medications and complex treatment algorithms to treat their condition, many of whom have attempted multiple lines of therapy before finding any success.

Since these medications must be readily available for the small population that requires them, specialty programs constantly work in tandem to get their medications approved as fast as possible. Specialty pharmacies can customize a treatment plan so that each patient is being treated appropriately.

Within each specialty pharmacy, certain logistics are used to ensure the compliance of the patient on the medication and the compliance of the specialty pharmacy to the manufacturer they are contracted with. A few examples of these logistics are cadences and frequency of patient follow-up, continuous provider contact, phase IV FDA surveillance reporting, and constant evaluation by clinical staff to drive and maximize favorable outcomes.⁸

Specialty pharmacy will continue to progress as new rare diseases are constantly being discovered. With more than 900 accredited specialty pharmacies located within the United States, we can confidently say that specialty has made its way into a top tier practice.⁹

Specialty pharmacies have also maintained a strong role in oncology care, HIV certification programs, and the handling of hazardous medications. As many of these expansion programs continue to require more personnel to provide optimal care, pharmacists find themselves serving as an extension of a primary care provider to patients who need specific care. As specialty pharmacies continue to provide more care, pharmacists will constantly be upgrading their skills to provide for the industry’s ever-growing needs.

Advancements within this field are inevitable and are required in order to continue serving these specific patient populations. One current focus within specialty pharmacy is the use of pharmacogenomics to provide precision care for oncology patients.¹⁰

Knowing a patient’s specific metabolic enzyme activity allows physicians and pharmacists to pinpoint a medication that can be most effective within its pharmacokinetic profile. Being able to target specific tumor genes and receptor sites can also bridge major gaps in engineering a medication that is suited for a patient’s specific diagnosis.

These landmark practices are going to be the reason why specialty pharmacy continues to advance and be more profound than any other industry in medicine. The history of specialty pharmacy will continue to write itself in this generation and has already produced achievements that produce life-changing outcomes for the population it serves.

About the Authors

Jarrett Genovese will graduate from Duquesne University’s School of Pharmacy in the Spring of 2021.

Jonathan Ogurchak, PharmD, CSP, is the co-founder and CEO of STACK, a pharmacy compliance management software, and serves as preceptor for a virtual Advanced Pharmacy Practice Experiential Rotation for specialty pharmacy, during which this article was composed.

References

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• National Human Genome Research Institute. Rare Diseases FAQ. Genome.gov. https://www.genome.gov/FAQ/Rare-Diseases. Published January 10, 2020. Accessed June 25, 2020.
• Cooperman T. Trends in FDA approval of Specialty Drugs 1990 through 2017. RJ Health. https://rjhealth.com/2017/12/15/trends-fda-approval-specialty-drugs-1990-q3-2017/. Published June 4, 2018. Accessed June 25, 2020.
• Fosemckay. What is a specialty drug? PCMA. https://www.pcmanet.org/pcma-cardstack/what-is-a-specialty-drug/. Published 2020. Accessed June 25, 2020.
• Centers for Disease Control and Prevention. U.S. Statistics. HIV.gov. https://www.hiv.gov/hiv-basics/overview/data-and-trends/statistics. Published April 17, 2020. Accessed June 25, 2020.
• Letter TP. Merck's Crixivan Is Third Protease Inhibitor Cleared. TPL. https://www.thepharmaletter.com/article/merck-s-crixivan-is-third-protease-inhibitor-cleared. Published March 25, 1996. Accessed June 25, 2020.
• Food and Drug Administration. FDA-Approved HIV Medicines Understanding HIV/AIDS. National Institutes of Health. https://aidsinfo.nih.gov/understanding-hiv-aids/fact-sheets/21/58/fda-approved-hiv-medicines. Published January 30, 2020. Accessed June 25, 2020.
• Food and Drug Administration. FDA-Approved HIV Medicines Understanding HIV/AIDS. National Institutes of Health. https://aidsinfo.nih.gov/understanding-hiv-aids/fact-sheets/21/58/fda-approved-hiv-medicines. Published January 30, 2020. Accessed June 25, 2020.
• Vanscoy G. Stratification and Differentiations of Specialty Pharmacies. Pharmacy Times. https://www.pharmacytimes.com/news/stratification-and-differentiations-of-specialty-pharmacies. Published March 10, 2020. Accessed June 25, 2020.
• Bigler RL. Pharmacogenomics for Precision Specialty Pharmacy Care. Pharmacy Times. https://www.pharmacytimes.com/publications/specialty-pharmacy-times/2019/October-2019/pharmacogenomics-for-precision-specialty-pharmacy-care. Published October 18, 2019. Accessed June 25, 2020.