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Patient-First Approach Ensures People With Rare Disorders Receive Better Care and Outcomes


Improving the patient experience for those with orphan diseases through a patient-first approach.

Thirty million Americans live with a range of 7,000 rare or orphan diseases, and while many patients have struggled with reduced care, isolation, and adherence challenges for most of their lives, the issue of self-isolation during the pandemic has impacted quality of life. These individuals now face elevated stressors resulting from immunosuppression, concern about potential infection, and hypersensitivity to viral threats. Despite restrictions, patients continue to require a high level of therapy adherence support at a time when some pharmacies have reduced their hours. This can impede communication with physicians and other advocates, which has already been severely disrupted due to the need for social distancing.

This article examines how a patient-first approach sharply focused on orphan diseases can enable pharmaceutical companies, pharmacists, and other members of the care team to better address compliance and adherence to treatment in order to impact the patient experience and improve outcomes. This model focuses on small patient populations and adapts services to include targeted programs and services that deliver specialized expertise. The skill set and level of care required goes well beyond the scope of capabilities provided by traditional, legacy care organizations that are simply built for scale.

Advantages of patient-first strategy

With a patient-first approach, specialty pharmacy, physicians, patients, and care coordinators connect effortlessly instead of operating independently. The continuity across the entire patient journey strengthens communication, yields rich data for more informed decision making, and improves the overall patient experience. This dedicated clinical team seamlessly ensures there are no treatment gaps for the patient, which is critical from clinical trial through commercial launch and throughout the lifecycle of the medication.

A patient-first focus also addresses all variables around collecting data, while maintaining frequent communication with patients and their families to ensure compliance and positive outcomes. Rare and orphan disease patients require a high level of support and have much to gain from high-touch service. A care team, including care coordinators, pharmacists, nurses, and other specialists, is 100% dedicated to the disease state, patient community, and therapy. This is a critical differentiator that improves upon the standard specialty pharmacies and hub service providers, which rely too heavily on technology solutions that fail to address human needs and variability.

Pharmacy pricing model

A core difference between the legacy and patient-first model is how pharmacies are compensated. With the legacy care model, pharmacies earn a margin on the products sold as they strive to keep millions of patients compliant with maintenance medications. When the focus is on the product instead of the patient, the pharmacy’s incentives can get misaligned: They are more focused upon monthly profits, which can lead them to calculate and emphasize how much time employees spend on the phone. These incentives prevent pharmacies from being truly patient-focused and able to deliver the best possible care.

Pharmacists and telehealth

During the pandemic, telehealth has played—and is likely to continue to play—an important role by enabling pharmacists to remain a constant part of the care team during disruptions in the continuity of care. In fact, a recent analysis found that telehealth visits increased 50% in March 2020, and a recent poll determined that more than one in eight Americans engaged in a video consult with a healthcare professional in recent months.

Effective pharmacy telehealth is particularly important for addressing the unique healthcare coordination needs of patients with a rare or orphan disease 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 including adherence tools specific to patients’ needs. This gives patients a better chance at thriving, despite disruption and uncertainty brought on by the pandemic.

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.

Customized care coordination and telehealth solutions based upon 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 coronavirus. Additionally, a 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 side effects and capture real world evidence around the therapy, disorder, and patient’s well-being.

Choosing the right patient management partner

When seeking to identify a specialty pharmacy and patient management organization that creates a partnership for personalized care with a specialty focus, look for a partner that offers a suite of comprehensive services tailored to maximize the therapeutic opportunities for the treatment of rare and orphan disorders. A patient-first approach that provides a trusted path for patients and all those involved in the treatment journey is a key metric. This adds a much-needed layer of support to 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 rely upon 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.

What’s more, the specialty partner should have the expertise to navigate the insurance landscape and prior authorization process, as needed, and know how to monitor and encourage compliance. It’s also important to find a partner with dual accreditation from the Utilization Review Accreditation Commission (URAC) for compliance with specialty pharmacy and the Accreditation Commission for Health Care (ACHC) for specialty pharmacy services. This demonstrates 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.

Donovan Quill, president and CEO, Optime Care

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