Therapeutic Opportunities for Patients With Orphan, Ultra-Orphan Disorders During COVID-19 Pandemic and Beyond


When telehealth is part of a larger personalized care plan tied specifically to a particular specialty drug, pharmacists can be better positioned to empower their patients.

Patients with orphan and ultra-orphan diseases rely heavily on a support system for adherence to therapy, which has been particularly challenging during the coronavirus 2019 (COVID-19) pandemic. A single rare or orphan disease affects fewer than 200,000 Americans, according to the Orphan Drug Act of 1983.

However, there are nearly 7000 rare diseases affecting 30 million Americans, including Huntington disease, amyotrophic lateral sclerosis, Cushing syndrome, Alpha-1 antitrypsin, chronic immune thrombocytopenic purpura, and muscular dystrophy.

Patients with rare and orphan disorders have grappled with reduced care, isolation, and adherence risks throughout their lives, which leads to an extraordinary impact upon individual outcomes. During the COVID-19 pandemic, these same patients now face heightened stress because many of them are immunosuppressed, conscious of infection, and wary of getting sicker than they already are. As a result, they are hypersensitive to viral threats, more isolated than ever, and struggling to self-protect.

Furthermore, some pharmacies may be operating under limited hours, which affects communication with physicians and patients. Likewise, routine interaction with providers has been disrupted because of the demands of social distancing.

Pharmacies help tremendously by remaining a constant part of the care team during potential disruptions in the continuity of care. Telepharmacy, the delivery of pharmacy care via telecommunication to patients in locations where they may not have direct contact with a pharmacist, has played a large role during these challenging times.

In fact, a recent analysis found that telehealth visits increased 50% in March 2020, and a recent poll determined that more than 1 in 8 Americans engaged in a video consult with a health care professional in recent months. Effective telehealth and telepharmacy are particularly important to address the unique health care coordination needs of patients, especially those who have been diagnosed with a rare or ultra-rare condition and, more importantly, the newly diagnosed patient.

When telehealth is part of a larger personalized care plan tied specifically to a particular specialty drug, pharmacists can be better positioned to empower their patients. Pharmacists do this by providing the right plan to the right patient and include adherence tools specific to that patient’s needs. This gives the patient a better chance at thriving during these unprecedented times.

By partnering with a patient-first organization that integrates specialty pharmacy services, product distribution, and patient management into a dedicated program, pharmacists enable patients to receive the intense care, outreach, and consistent support they require to ensure positive outcomes.

Patient-First Care Management

Customized care coordination and telehealth solutions based on a patient-first care model provides a proactive, process-driven program that can educate the patient on potential risks, which mitigates the transmission of possible infections, such as COVID-19. Additionally, this patient-first care model fosters discussion between the patient and pharmacist that is essential to understanding the patient’s needs, providing focus on the drug’s impact, and monitoring their overall health.

By incorporating assessments and predetermined touch points each month, the care team is able to stay on top of adverse effects (AEs) and capture real world evidence around the therapy, disorder, and patient’s well-being.

A single source partner offers the continuity of care that is critically missing from legacy care models. The net result is that patients have a better and more consistent experience.

This experience has been proven to yield stronger compliance and overall outcomes. Clinical care teams reach out to patients via videoconference or phone call to determine their level of understanding, health literacy, and status around a particular disorder, helping to mitigate potential AEs, offer encouragement, and provide a level of emotional support to patients and caregivers.

A telehealth option can be fully integrated for pharmacies to provide care coordination based on each patient’s specific needs, including, but not limited to, medication counseling, education on disease states, and expectations for each drug. These customized care plans are designed to better serve patients, caregivers, providers, and payers, helping overcome key challenges around the complexity of rare, orphan, and ultra-orphan disorders.

Finding the Right Patient Management Partner

When looking for a specialty pharmacy and patient management organization that creates a partnership for personalized care with a specific focus on disorders affecting smaller patient communities, ask:

1) Do they offer a suite of comprehensive services tailored to maximize the therapeutic opportunities for the treatment of these disorders?

2) Do they have a patient first approach that provides the trusted path for patients and all those involved in the treatment journey?

A patient-first model adds a much-needed layer of support for the patient’s family and caregivers, enabling them to become more engaged and take ownership, which leads to a stronger partnership and better patient care.

The telehealth solution should be designed to streamline patient enrollment, maximize interaction with patients for adherence and compliance, and provide continuity of care to avoid lapses in therapy. It should reflect dedicated team members who are well versed in every aspect of the patient’s drug and have the expertise to answer every question and concern from patients, pharmacists, physicians, providers, and payers. They should have the expertise to navigate the insurance landscape and prior authorization process, as needed, and know how to monitor and encourage compliance.

Partners with dual accreditation from URAC for compliance with specialty pharmacy and the Accreditation Commission for Health Care for specialty pharmacy services have a demonstrated commitment to providing quality care and services to these patient populations. Ultimately, the care management solution should meet the needs of everyone involved in the patient’s journey, from specialty drug manufacturers to pharmacists, caregivers and physicians—all delivered from one central point of contact.

About the Author

Donovan Quill is president and CEO of Optime Care.

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