In the U.S., it is estimated that 25-30 million people, almost 10% of the population, have rare diseases. While there are approximately 7,000 rare diseases, unfortunately, more than 90% of rare diseases are without an FDA-approved treatment.

Since the majority of rare diseases are life-threatening and 80% of rare diseases are genetic in origin, the need for new medicines is paramount. While unprecedented scientific innovation has led to over 600 orphan drugs being approved by the FDA since the passage of the Orphan Drug Act of 1983, there are currently over 560 medicines in clinical development for the treatment of rare diseases. So when a new drug treatment for a rare disease is approved, it is critical that the right specialty pharmacy is selected. What criteria should you consider? While there are many considerations that are important, ultimately the patient experience with your specific drug should be one of the highest priorities. Two of the most important aspects of the patient's experience with your specific drug are the clinical services and operational services of your selected specialty pharmacy.

Specialty pharmacies offer a range of customizable clinical and operational services that enhance the safety, quality, and affordability of care for patients using specialty drugs. Here are some of the critical elements to consider when evaluating the clinical and operational services of a specialty pharmacy for your next drug launch.

Specialty Pharmacy Clinical Services

Healthcare provider access: Provides around the clock access to specially trained pharmacists, nurses, and clinicians who offer patients guidance and insight on disease states, as well as the use and management of specialty drugs.

Physician consultations: Consults directly with physicians to address patient side effects, adverse drug reactions, non-compliance, and other patient concerns.

Care management: Performs disease and drug-specific patient care management services that meet the unique needs of each patient and incorporate multiple safeguards when dispensing and delivering the drug to ensure patient safety

Clinical outcome measures: Collects data and tracks outcomes for specific patients

Patient adherence programs: Manages patient adherence and persistency of drug regimens

Patient care quality measures: Integrates defined quality measures into all aspects of patient care, providing staff with ongoing training, monitoring, and remediation when necessary

REMS programs: Manages care for manufacturer Risk Evaluation and Mitigation Strategies (REMS) program requirements, including REMS reporting, Phase IV trials, the dispensing of FDA trial drugs under strict protocols, and related clinical and cognitive counseling

Specialty Pharmacy Operational Services

Supply chain management: Adheres to rigorous storage, shipping, and handling standards to meet product label shipping requirements, such as temperature control and the timely delivery of products in optimal conditions. In addition, when time is of the essence, the ability to expedite a shipment as needed, to ensure a patient is able to receive their treatment, is paramount. When the staff is alerted by the site of care of the need to expedite an order, filling of the order is accelerated and a shipment method to get the shipment to the site of care in the expedited time frame occurs. This may include the use of courier or next day first delivery services.

Quality control measures: Uses enhanced quality control, electronic imaging, drug utilization reviews, and final quality assurance checks when dispensing medications

Care coordination: Offers coordinating services with other healthcare providers, including those providing skilled nursing services, custodial care, infusion administration, and direct-to-physician distribution

Insurance navigation: Expedites access to therapy by working directly with insurers and navigating their benefits, utilization management, and prior authorization processes across both medical and pharmacy benefit designs. Having strong medical benefit coverage expertise is especially necessary when dealing with rare diseases.

Patient assistance: Facilitates eligible patients’ enrollment in patient assistance programs and access to charitable resources. In addition, provides extended payment arrangements for patients in need of support for these costly therapies.

Plan optimization: Aligns economic incentives across medical and pharmacy benefits while helping patients navigate the complexity of sometimes-siloed benefit structures. Designed to bridge the benefit investigation process between pharmacy and medical benefit to allow a faster and more efficient onboarding process for the patient, every program developed is customized to a manufacturer’s unique needs and business strategy, including workflow, data alignment, and customizations specific to each drug program.

Launch readiness: Rare specialty pharmacy develops an implementation plan for each new product that launches. Each implementation plan typically starts at least 90 days prior to the anticipated launch or operational readiness date. As part of the implementation plan, the rare specialty pharmacy begins communications with the manufacturer, their HUB, data aggregator, prescribers, health plans/PBMs and other partners to drive key launch initiatives to a readiness state. This includes weekly calls with the manufacturer and other partners to provide status updates, refine processes as needed, and complete collaborative tasks. In addition, the rare specialty pharmacy will begin to have conversations with payors about potential new pipeline products and how the plan expects them to be covered. This would include working in advance with the payors to have the product staged and ready to add to existing payor contracts once the product is FDA approved.

Manufacturer reporting: This includes tracking key metrics to ensure adequate staffing and resources are assigned to the program, and overall outcomes are achieved. Having an extensive data dictionary allows for the creation of custom reports that have successfully met many complex program needs. Providing custom, robust reporting that includes transactional data related to de-identified patient, prescriber, payor and reimbursement, dispense, and custom data points and delivery data at a frequency of daily, weekly, monthly, quarterly, and annually is crucial for rare disease management. It is paramount to participate in Quarterly Business Reviews (QBRs) to identify trends across the business and recommend and implement approved continued program improvement measures.

In our recent guide, Rare Diseases, Gene, and Cell Therapies: Specialty Pharmacy Considerations, we cover what is most important when selecting a specialty pharmacy to partner with for your next drug launch. Download your digital copy of the rare disease guide. If you have immediate questions, you can also connect with an Orsini representative at any time.