Effective patient-first approach in rare disease relies on true sales—not channel stocking


How the development of more quantitative and qualitative data around the patient can drive better-informed care decisions—and help avoid potential overstock scenarios that can deplete earnings

Channel stocking is a common practice in which pharmaceutical executives opt to sell a sizable unit amount of a drug up-front to a wholesaler or specialty pharmacy without a rare and orphan disease patient attached to the sale. These are not true sales, however, because the product has not gone to a patient. What’s more, filling the channel negatively impacts long-term revenues and decreases the visibility of the therapy’s impact, which is non-beneficial for the patient.

To avoid this, it’s critical to take a patient-first approach to better identify the patients who need the drug and avoid risking a level of overstock that depletes earnings. Channel stocking not only makes it more challenging to determine how to report revenues, but also carries the chance that the pharmacy may ask for a credit or discount for storage. Depending on payer reimbursement, this scenario could leave manufacturers upside down on the product.

Stocking the channel puts distance between the drugmaker and patients, leading to lost data and a lost opportunity to improve care and support for specialty patients and their families. It’s also important to understand that, for rare disease patients, availability is not the biggest challenge because month-after-month the growth of the population is too small to deplete stock.

Taking a patient-first approach enables the development of more quantitative and qualitative data around the patient so that informed decisions can be made, better patient care plans created and outcomes improved.

Understanding the rare/orphan patient population

The 30 million Americans who live with a range of 7,000 rare or orphan diseases require a high level of therapy adherence support, with individuals having higher medical needs, often missing work, retiring early and relying on caregiver assistance. This represents a substantial economic burden for the patient, the unpaid family caregivers and the health system, as well as additional medical costs for managing the disease.

The estimated total economic burden of 379 rare diseases with a prevalence of 15.5 million people in 2019 was $966 billion, including a direct medical cost of $418 billion and an additional $548 billion in indirect and non-medical cost. Direct medical cost represents more than one-third of the total burden (43%), followed by absenteeism losses (15%) and loss due to presenteeism (25%). Overall, labor market productivity losses due to rare diseases, including absenteeism, presenteeism and earnings losses from forced retirement, represent roughly the same proportion of the total burden as direct medical costs (44%). Non-medical costs represent about 12% of the total burden.

Commercial payers face the largest share of medical cost, while employers pay for significant productivity losses associated with absenteeism and presenteeism, including productivity losses of $135 billion from adults with rare disease whose disease progression and diagnoses require time away from the workplace and $152 billion from their caregivers.

Fortunately, a patient-first care model can enable every stakeholder impacted by rare disease to more effectively address compliance and adherence to mitigate costs and improve outcomes. Patient-first strategies offer targeted programs and services that deliver specialized expertise that transcends the scope of capabilities provided by traditional, legacy care organizations, which are often designed exclusively for scale.

Meeting specialty patient needs

The higher level of care continuity delivered by a patient-first approach strengthens communication, yields rich data for more informed decision-making and improves the overall patient experience. Clinical teams are well positioned to seamlessly eliminate treatment gaps for the patient. This strategy also addresses all variables around collecting data and maintains frequent communication with patients and their families to ensure compliance and positive outcomes.

A patient-first care team, including care coordinators, pharmacists, nurses and other specialists, focuses on the disease state, patient community and therapy. This is important for overcoming the limitations of the standard specialty pharmacy and hub service provider, which too often rely on technology solutions that can potentially fail to address human needs and variability.

Optimal patient-first partner

When finding a specialty pharmacy and patient management organization that creates a partnership for personalized care, 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 can provide a reliable path for patients and all those involved in the treatment journey. This additional support for the patient’s family and caregivers enables them to become more engaged and take ownership, leading to a stronger partnership and better patient care.


Make sure the partner’s telehealth solution is 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 have expertise in every aspect of the patient’s drug and can address every question and concern from patients, pharmacists, physicians, providers and payers.

Optimized telehealth addresses the unique healthcare coordination needs of patients with a rare or orphan disease and, more importantly, the newly diagnosed patient.

As part of a larger patient-first plan tied specifically to a particular specialty drug, telehealth enables pharmacists to empower their patients to thrive, even during times of disruption and uncertainty brought on by the Covid-19 pandemic and other unforeseen emergencies.

Tailored care coordination and telehealth solutions add a layer to a proactive, process-driven program, educating the patient on potential risks. This enhances discussion between the patient and providers that is crucial to understanding the patient’s needs, providing focus on the drug’s impact and monitoring overall health. By incorporating assessments and predetermined touch points each month, the care team is able to monitor side effects and capture real-world evidence around the therapy, disorder and person’s well-being.

Specialty partners that demonstrate expertise in navigating the insurance landscape and prior authorization process, as needed, and know how to monitor and encourage compliance, are important. It’s also useful 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 right care management support can meet the needs of everyone involved in the patient’s journey, from specialty drug manufacturers to pharmacists, caregivers and physicians.

About the author

Donovan Quill is President and CEO of Optime Care.

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