New Therapies and the Role of Specialty Pharmacy Providers

Specialty pharmacies service patients who require high-cost or “high-touch” medications for treating rare, chronic, or complex disease states such as cancer, multiple sclerosis, hepatitis C, rheumatoid arthritis, and rare genetic disorders. Products dispensed by specialty pharmacies include oral, injectable, and intravenous (IV) medications, ranging from those approved by the FDA up to 7 decades ago to newly approved therapies from the drug development pipeline. In disease states that carry a poor prognosis or lack suitable treatment options, such as certain types or stages of cancer, a newly approved product may bring newfound hope to a patient who previously faced mortality. No other field has generated as much excitement as cancer care in this regard.

In 2012, congressional lawmakers approved the Food and Drug Administration Safety and Innovation Act (FDASIA), which allows the FDA to use surrogate clinical end points to grant accelerated or expedited approval of products that treat serious medical conditions and fill an unmet need. Regarding oncology clinical trials, these end points must be significantly superior to currently available treatments and usually include overall response rate data rather than overall survival data. Due to a streamlined approval process, the FDA has greenlit 56 novel oncology products since January 1, 2013, 52% of which are orally available anticancer agents.

Oncology limited distribution drug (LDD) networks range in size from a single specialty pharmacy provider to more than 20. In certain circumstances, a sole entity is responsible for 100% of a product’s prescription dispensing. If a manufacturer decides to provide access to their product via an LDD network, the member specialty pharmacies become de facto experts on the drug. In a newly FDA-approved LDD product launch, specialty pharmacies have a responsibility to provide their employees, prescribing customers—physicians, nurse practitioners, and physician assistants—and patients with complete drug information. Additionally, member specialty pharmacies are obligated to provide certain data metrics to product manufacturers to help them ascertain the real-world applications of their newly approved product, as well as assess how postmarketing clinical data compare with clinical trial data.

Specialty pharmacies provide thorough clinical education regarding all newly approved medications to employees who are involved in patient care—pharmacists, pharmacy technicians, patient advocates (such as those who source funding), billing team members, procurement/inventory team members, and information technology staff. New product education is typically provided in coordination with an expert from the manufacturer, such as a medical science liaison. Fully educating all appropriate employees improves a specialty pharmacy’s operational efficiency, from having the right protocols built into the patient care system to ensuring that pharmacists possess the right knowledge and access to information so they can perform critical counseling to patients on the new drug.

To provide optimal care to patients with complex, rare, or life-threatening illnesses, specialty pharmacies must develop and maintain relationships with their prescribing customers. It is incumbent on the specialty pharmacy to not only promote awareness regarding newly approved products but also to serve as an information resource regarding a medication. Often, prescribers have no experience in treating patients receiving a new drug unless they participated in its clinical trials. Specialty pharmacies play a critical role in informing prescribers of appropriate dosing regimens, clinically relevant drug interactions, laboratory monitoring parameters, and required supportive care related to the new product. During the launch phase and through early adoption and early majority, these services are particularly critical.

As the nature of cancer treatment changes from patients receiving IV infusions in an outpatient or inpatient setting to patients administering oral oncolytics at home, the responsibilities of specialty pharmacy practices in direct patient care are growing exponentially. Traditionally, specialty pharmacies have been viewed as facilities that dispense medications and mail them to patients. Due to the complexity of care involved with patients battling cancer, specialty pharmacies, such as Onco360, have taken a new approach to assisting patients throughout their therapeutic journey. In addition to offering core services, such as prescription fulfillment and new medication counseling, specialty pharmacies seek to provide all patients with the means to afford their medications. Whether through third-party foundation assistance or manufacturer-based free-drug programs, specialty pharmacies attempt to alleviate the financial toxicities of cancer care as much as possible.

As cancer treatment transitions from the clinic to the home setting, diminishing prescribers’ oversight of their patients’ care, the role of the pharmacist as a midlevel practitioner has expanded. Onco360 aims to serve as an extension of the prescriber’s office by performing medication therapy management (MTM) for patients receiving particularly high-touch oral oncolytic agents. MTM programs focus mainly on outbound support calls to patients at scheduled intervals to help maintain compliance with cancer treatment regimens and prevent or manage adverse effects (AEs).

Specialty pharmacies engage in pharmacovigilance programs geared toward identifying adverse drug reactions and AEs related to newly approved products. Oncology products may be approved based on phase 1 or 2 clinical trial data, which usually contain smaller patient populations than phase 3 clinical trials. Specialty pharmacies, through their care of patients, fill the void by collecting and providing integral safety data that would otherwise be unavailable to manufacturers. Product manufacturers use that data for required postmarketing surveillance and pharmacovigilance programs that are instrumental in delineating new safety signals. Additionally, specialty pharmacies track other patient care utilization metrics and outcomes related to newly approved mediations, such as duration of therapy, rate of prescription discontinuation, and reasons for discontinuation. These data provide manufacturers with visibility into the real-world patient experience with their product.

In conclusion, specialty pharmacies serve as a vital component to the success of a medication newly approved by the FDA. Through interactions with prescribers, patients, and manufacturers, specialty pharmacies function as a valuable intermediary for distribution of drug information, provision of exceptional care for clinically complex patients, and engagement in postmarketing surveillance.