Rare Pharmacy Emerges From Specialty, Part 3

Publication
Article
Pharmacy Practice in Focus: OncologyNovember 2020
Volume 2
Issue 5

The series concludes looking at the proposition that patients with rare and devastating disorders require and deserve a higher acuity of care and service that only an accredited rare pharmacy can deliver.

Part 1 of this 3-part series explored the details of how changes in US federal policy and advances in scientific research have led to the dawn of medical breakthroughs for rare disorders. Part 2 of the series examined how rare pharmacy has emerged distinctly from specialty pharmacy to address the unique needs of patients, biopharma, providers, and payers. The series concludes looking at the proposition that patients with rare and devastating disorders require and deserve a higher acuity of care and service that only an accredited rare pharmacy can deliver.

Rare Pharmacy Solutions

Located at the center of the medication use process, a rare disease—focused pharmacy can play a leading role in providing optimized care for patients while tackling challenges, easing burdens, and addressing all stakeholder needs. With a specialized, multifunctional team that coordinates care, a rare disease–focused pharmacy, or more succinctly, a rare pharmacy, can contribute to improved quality of life for people living with chronic and devastating diseases. A rare pharmacy can simplify the complex process of getting orphan medicines from innovators to the people who need them most by removing barriers to access therapy and patient care.

A rare pharmacy must possess clinical expertise, robust disease-state management programs, mechanisms to optimize compliance, adverse drug effect counseling, reporting capabilities, prior authorization support, and co-pay card support, and focused educational capabilities for patients, physicians, and payers. Data management, customization, and sharing capabilities are also critical, as many orphan medications are approved based on a single clinical trial in a small patient population and require ongoing data analysis.

It is fundamental for a specialty pharmacy to provide patients with a more specialized experience than would be expected from a traditional pharmacy. A pure rare pharmacy accurately and precisely delivers consistent quality care to patients with rare and devastating diseases without the distraction of the diverse responsibilities encumbered with larger populations (traditional specialty pharmacy). In the case of rare pharmacy, the patient experience needs to be specialized to the level of individualization, and high-frequency patient engagement must be the norm. Ongoing, personalized communication is crucial to this experience, as patients and their families deal with the burden of a rare and devastating disease state. The support network for such diseases, though available, are not as readily accessible as those for conditions with more ubiquitous presences, and a rare pharmacy must be the conduit for this support. Interactions with the patient—such as discussions regarding medication adverse effects (AEs), the impact of the disease, nutrition, and addressing financial barriers through coordinated co-pay assistance—can, when conducted properly, reduce stress and make days more manageable for people dealing with these debilitating diseases. In many cases, these therapeutic agents will not cure their diseases; however, when part of a carefully managed plan, they will still provide patients with a priceless improvement in quality of life.

What Makes a Rare Pharmacy Different Than a Specialty Pharmacy?

Investment in the Patient Journey

Rare pharmacies are adapting to the emerging orphan drug market and providing new solutions to address the challenges facing the health care system. The chasm separating specialty and rare pharmacy ultimately stems from a rare pharmacy’s need to comprehend a patient’s entire journey, which begins well in advance of medication dispensing. A rare pharmacy must establish individualized relationships with patients and understand the entire journey, including onset of a patient’s first symptom, difficulties of diagnosis, and day-to-day challenges. Because patient populations are so small and available data are much more limited, each experience can add depth to a patient’s profile and, if understood and used correctly, can allow a rare pharmacy to deliver more effective care. By understanding the depth and variety of possible patient journeys for a specific rare disease state, a rare pharmacy can establish a framework and develop and execute a standard of care.

Furthermore, a rare pharmacy recognizes that no two patient journeys are the same. In collaboration with industry partners, rare pharmacies customize solutions built around a product and personalize the support services delivered to the patients based on their individual needs. As a standard, each rare program is tailored to maximize the benefits of an individual therapy and then adapted to the symptoms, lifestyle, and preferences of each patient. Every patient should feel that they have the undivided attention of their rare pharmacy care team and that each team member is invested in their health.

Directed Clinical Program Development and Patient Support

Patients living with a rare and devastating condition often require additional support services and a pharmacy that can go beyond drug dispensing. The clinical and patient engagement programs extend to understanding the patient journey and what they have experienced to get to this point in treatment. The program design must integrate clinical information with an understanding of that patient’s health beliefs and what motivates them. This design requires new thinking and an informatics system that can respond to queues throughout the care process. The rare pharmacy’s ability to leverage specialized teams and technology to onboard new medications and ensure patients receive uncompromised care will differentiate them from other specialty pharmacies. Dedicated efforts during predrug launch, implementation, and postlaunch ensure successful execution.

Single Point of Contact and Targeted Education

To appropriately care for patients with rare diseases, specialized, cross-functional, disease state—focused teams must be the model. These teams should be comprised of, but not be limited to, program managers, pharmacists, nurses, clinical advisors, patient care representatives, insurance specialists, and data analytics professionals. Every member of these teams must serve as a patient advocate, assisting patients as they navigate their treatments and progress through their journey. Team size, composition, and the roles/responsibilities should be customized based on the specific needs and characteristics of the patient population being served.

As part of the customized program development, a rare pharmacy must implement a robust training program on the disease state, patient journey, and therapeutic options. Product and AE management training and operational protocols are essential for teams to provide optimal patient care.

Deliberate and Consistent Communication

It is imperative that patients are informed about, and engaged in, every step of their prescription journey. Initial communications and introductions to pharmacy services are critical parts of developing the relationship between a pharmacy and its patients. These conversations allow patients to articulate their specific goals and the pharmacy team to curate an understanding of their treatment histories, providing targeted counseling and outlining medication expectations. In instances of complex dosing or titration schedules, patients must be strategically contacted to review new dosing, ensure appropriate administration, aid in AE management, and discuss adherence.

Adverse Drug Event Reporting and Management

Due to the small number of patients with any one rare disease, novel therapeutic agents are approved and enter the market based on results from limited clinical trial data. Many of these medications are approved on an accelerated track, and the true clinical risks and benefits are still being vetted and established. Postmarket surveillance is crucial to ensure patient safety in the rare disease market. Specialty pharmacies are very familiar with AE reporting, but rare pharmacies need to be innovative to enhance the standard process and give insights into real-world therapy outcomes on a patient-by-patient basis.

Rare pharmacies need to have comprehensive reporting structures and communication platforms that allow for direct interfacing and real-time updating. Based on the individual product needs, reporting can be conducted in various ways to keep manufacturers updated and to remain compliant with FDA requirements. The analysis of these data is essential, as it allows the pharmacy to provide updated educational programs to patients and health care providers, resulting in increased safety. In addition to the data collection platform, the use of dedicated pharmacists who are experts in specific diseases and treatments allows for consistent reporting of adverse effects and uniform medication education.

Ability to Alleviate Financial Obstacles

If a patient does not receive a prescribed medication, there can be no positive impact on the patient’s disease state and health outcome. Obtaining the necessary medications is the only way to alleviate their symptoms, but high costs and complicated prior authorization (PA) processes often delay the process.

Rare pharmacy must involve personnel who understand and work through financial barriers and payer hurdles to obtain therapy for the patient. This may include PAs and appeals process support. Understanding payer policies and the information required for approval can reduce rejections and the time to get a patient on therapy.

Even when payer coverage criteria are cleared, the patient’s financial responsibility may cause them to walk away from receiving the needed medication. The rare pharmacy must have processes in place to direct patients in need to either co-pay assistance programs or foundation support.

A dedicated insurance resolution specialist is essential to guaranteeing that all options, including third-party payment assistance programs, foundation support, and free goods or bridge programs, are investigated for each patient. All stakeholders recognize the important role a rare medication has in a patient’s life and appreciate that cost alone should not be a barrier to seeking the only treatment that may be available.

Triaging Referrals and Hub Capabilities

Quickly and effectively managing referrals is vital to improving patient outcomes. Prescriptions may be obtained through hubs, electronic prescribing platforms, phone, or fax. While referral processing is standardized across the industry, a rare pharmacy can differentiate itself by ensuring effective communication with the health care team from the start of the process. Resources need to be dedicated to understanding the payer landscape, and previous experience with complex coverage criteria is essential. Prioritizing interprofessional collaboration with the entire health care team allows for a seamless patient journey and optimal patient health outcomes.

Rare pharmacies should also have capabilities to serve as a hub, as is often the case when a manufacturer has chosen to distribute its rare therapy through an exclusive pharmacy arrangement. By serving as both the central point for referrals and the pharmacy, the rare pharmacy can reduce the time to process referrals and give a complete picture to the manufacturer. Additionally, this may reduce confusion for the patients and prescribers, as only one entity is calling and coordinating the prescription journey. The rare pharmacy would operate the spectrum of services utilizing specialized teams, strong communication platforms, and high-touch patient and health care provider engagement.

Efficient Prior Authorization Support

Due to their unusually high costs, new-to-market novel and targeted rare therapies typically have payer requirements that must be met prior to coverage. A survey of payers reported that 97% of orphan drugs require PA, 55% are placed on a specialty tier, and 27% required the addition of step edits. In addition, 25% of payers may require an increase in cost sharing with patients.1,2

A rare pharmacy uses expert clinicians and auxiliary internal processes to request and obtain the required clinical information mandated by the payer. The rare pharmacy’s high level of disease knowledge, medication expertise, and health care provider integration allow for proactive systematic collection of information that aids in the processing of PAs. This expertise ensures that PAs and appeals reach their determination points as complete and as quickly as possible.

Expert Fulfilment, Distribution, and Supply Chain Support

The ability to coordinate mail-order shipping, temperature-sensitive supply chain requirements, and inventory management is essential to rare pharmacies. Due to the high costs of therapies and unique circumstances of associated rare disease states, fulfilment and distribution errors could be financially damaging to a company and prevent a patient from obtaining life-saving medications according to their care plan.

Exclusive distribution networks allow for a consistent patient experience, which is ideal for small patient populations. Rare medications are not suited to the high-volume, standardized patient management approaches seen in other areas of pharmacy, including traditional specialty pharmacy. Exclusive distribution networks offer benefits for patients with rare diseases because the distributing pharmacy has the expertise to provide extensive monitoring and customized therapy support.

Exclusive distribution networks allow patient management to be tailored to specific patient types and impacted health care providers. In addition, these partnerships can adhere to strict shipping, tracking, and storage requirements and handle complicated logistics scenarios, such as cold chain management.

Ultimately, a rare pharmacy will use myriad resources to ensure the timely delivery of a safe and effective product.

Systematic and Customizable Data Reporting Capabilities

Reporting data to manufacturer partners is a standard practice in the specialty pharmacy industry. Rare pharmacy differentiates itself by providing data-reporting elements that are fully customizable based on each medication’s distinct needs to capture all facets of the patient journey. Additionally, data are reported to manufacturers and payers at agreed-upon frequencies based on individual partnerships. The small populations associated with rare medication dispensing demand highly specialized data reporting and analytical capabilities so that data can be reviewed on a patient-by-patient basis to identify trends and insights. It is also critical for a rare pharmacy to have the capability to track patients who transition from a clinical trial to the expanded-access environment.

In addition to data reporting, a rare pharmacy monitors key performance indicators and utilizes dashboard technology to benchmark data, track progress, and ensure operational excellence at the therapy level. Similar to the disease state—specific team structure, data analytics teams focus on individual drug products and customize their processes and outputs accordingly. Focused analysis allows for continuous process improvement; real-time metrics and data-driven program changes result in enhanced patient experiences.

Respected Accreditations and Specialized Distinctions

Manufacturers, providers, and payers agree that specialty pharmacies must have high standards and strict requirements to ensure excellent patient care. One mechanism of ensuring care quality at specialty pharmacies is the accreditation process. In a recent payer survey, 50% said they require their specialty pharmacies to have at least 2 accreditations, and all payers require URAC accreditation specifically.3 Specialty pharmacies most commonly hold 2 accreditations: URAC and the Accreditation Commission for Health Care (ACHC).3,4

With the emergence of rare pharmacies, industry stakeholders have recognized the importance of creating accreditations associated specifically with rare pharmacy that require even higher standards and stricter requirements than specialty pharmacy accreditations. In 2019, the establishment of the ACHC Distinction in Rare Diseases and Orphan Drugs differentiated rare disease—focused pharmacies from traditional specialty pharmacies.5

This year, URAC also introduced a rare disease—focused designation. This offers existing URAC-accredited specialty pharmacies the opportunity to further distinguish themselves from other specialty pharmacies to demonstrate competence in rare pharmacy.

Conclusion

The rare drug industry continues to grow exponentially. Orphan drug sales are projected to account for more than one-third of research-and-development pipeline sales by 2024.6 With this expected growth, specialty pharmacies need to focus and develop enhanced service capabilities to become better suited to provide care to patients with rare diseases. Advances and innovations in drug development and genetics have already transitioned rare pharmacy into a new era of precision medications that present new and more complex challenges. Payers, manufacturers, and specialty pharmacies are combining their efforts to establish a model for payment, distribution, and administration. The emergence of value-based models and care-coordination strategies will promote the future of precision-therapy advancements.7 Rare pharmacies are positioned to be a leading force in the next era of patient care.

It is exceedingly important that specialty pharmacy continues to grow and adapt to the changing health care environment. The medications and patients in the rare and orphan category bring unique challenges to pharmacies, and the industry needs to be prepared.

Patients with rare diseases still face considerable obstacles to care, including management of their complex medication therapy, drug access, and high cost of treatment. Rare pharmacies are in a pivotal position to provide reliable, high-touch care with flexibility and nimbleness that can make a difference in the lives of patients, caregivers, and every member of the health care system.

GORDON J. VANSCOY, PHARMD, MBA, is Chairman and CEO of PANTHERx Rare and associate dean for business innovation at the University of Pittsburgh School of Pharmacy in Pennsylvania.PAMELA KOERNER, PHARMD, is an associate professor in the School of Pharmacy, and director of the Giant Eagle Center for Pharmacy Practice, Duquesne University, Pittsburgh, Pennsylvania.RICHARD FARIS, PHD, RPH, is senior vice president and head of pharmacy at PANTHERx Rare.The authors would like to acknowledge Brittany Albert, PharmD, and Marissa Puc, PharmD, for their assistance in the research and writing of this white paper.

REFERENCES

  • Vanscoy J, Gebhard D, Ogurchak J, et al. Specialty pharmacy today: improving the lives of patients with rare diseases through orphan drug management. Pharmacy Times®. September 2017. Accessed April 13, 2020. https://www.pharmacytimes.com/publications/specialty-pharmacy-times/2017/september-2017/specialty-pharmacy-today-improving-the-lives-of-patients-with-rare-diseases-through-orphan-drug-management
  • Grogg AL. Payer trends impacting the orphan drug market. Amerisource Bergen. April 18, 2017. Accessed October 12, 2020. https://docplayer.net/50090131-Payer-trends-impacting-the-orphan-drug-market.html
  • CSI Specialty Group. 2019 state of specialty pharmacy report. Accessed April 13, 2020. https://academynet.com/sites/default/files/csi2019spreport.pdf
  • Fein AJ. Exclusive: a record number of specialty pharmacies now have accreditation. Drug Channels. April 10 2018. Accessed October 12, 2020. https://www.drugchannels.net/2018/04/exclusive-record-number-of-specialty.html
  • ACHC Launches First Distinction in Rare Diseases and Orphan Drugs. News release. Accreditation Commission for Health Care. October 1, 2019. Accessed April 15, 2020. https://www.achc.org/pdf/Distinction_Rare_Diseases_Orphan_Drugs_PR.pdf
  • Evaluate. EvaluatePharma orphan drug report 2019. April 25, 2019. Accessed April 13, 2020. https://www.evaluate.com/thought-leadership/pharma/evaluatepharma-orphan-drug-report-2019
  • CVS Health. Gene therapy: keeping costs from negating its unprecedented potential. 2020. Accessed October 14, 2020. https://payorsolutions.cvshealth.com/sites/default/files/cvs-health-payor-solutions-gene-therapy-keeping-costs-from-negating-its-unprecedented-potential-white-paper-january-2020.pdf

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