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By Donovan Quill, President and CEO, Optime Care
The COVID-19 pandemic has raised a number of challenges around conducting clinical trials for specialty drug manufacturers due to self-isolation, site closures, travel limitations and interruptions to the supply chain. This has made it difficult to meet protocol-specified procedures, including administration, adherence and laboratory/diagnostic testing.
For these reasons, a patient-first approach to specialty Rx for orphan and rare disorders has become an optimal approach, offering tools to enable in-home clinical services, direct-to-patient support and remote monitoring for keeping clinical trials on track. Patient-first strategies can shorten the time from the clinical trial to commercialization, enhancing patient access to the medication and capturing 90% of patients during the transition period within 30 days.
This is significant given that about 33% of all drugs in active R&D pipelines are now in the rare disease category. Inherent to these specialized populations, there are multiple scientific and operational challenges to sponsors and clinical trial ecosystem participants, including the adoption of new strategies, operating models and processes, according to a reportfrom Tufts Center for the Study of Drug Development.
The current COVID-19 pandemic also presents additional issues for patients, providers and drug manufacturers in the rare and orphan disease market, spanning the high cost of clinical trials to patient recruitment, enrollment and retention.
Closer Look at COVID-19 Challenges
During the current pandemic, patients with orphan and ultra-orphan diseases have encountered challenges in accessing much-needed therapy adherence support. This means that 30 million Americans with a range of 7,000 rare or orphan diseases have been at greater risk while self-protecting — including Huntington’s disease, amyotrophic lateral sclerosis (ALS), Cushing syndrome, Alpha-1 antitrypsin (AAT), chronic immune thrombocytopenic purpura (chronic ITP) and muscular dystrophy (MD).
Many of these patients have struggled with reduced care, isolation and treatment adherence issues for most of their lives, but now face elevated stressors because they are immunosuppressed, conscious of infection and hypersensitive to viral threats. Adding to the challenge, some pharmacies have reduced their hours. This impacts communication with physicians and local pharmacists, which has already been severely disrupted due to curtailed hours and the need for social distancing.
Finding a Patient Management Partner
While traditional models are built for scale, stakeholders are now seeking a more individualized approach that puts patients first: a focus on customized services for small patient populations that delivers personalized expertise to overcome the limitations of legacy care models and provide cost-effective programs.
This type of model translates into more streamlined care delivery that offers financial advantages, with assurance that products are properly and promptly distributed, and patient services are designed to ensure compliance and quick, accurate reimbursement processing.
Consider the case of a specialty Rx patient management partner that was involved in two clinical trials that began just as the pandemic started. Their integrated telehealth feature enabled patients to get the products delivered without going to the doctor’s office. As a result, the trials had significantly more patients involved, despite the national lockdown.
This level of innovation can also build awareness and introduce education programs that aid manufacturers’ understanding of patient groups. As a result, manufacturers know they have the support to develop a drug and a comprehensive program based upon specific needs. This adds a layer of patient advocacy, helps physicians better understand the patient journey, helps to create a patient registry and gives manufacturers an opportunity to obtain patient-reported outcomes outside of the clinical trial protocol.
Critical Role of Pharmacists
As an integral part of the care team, pharmacists serve as lifelines for patients with rare and orphan disorders by helping to prevent lapses in continuity of care. By partnering with a patient-first organization that integrates specialty pharmacy services, product distribution and patient management into a dedicated program, pharmacists with specialized training in rare disease management enable patients to receive the intense care, outreach and dependable support they require to ensure positive outcomes.
This model also adds a much-needed layer of support for the patient’s family and caregivers, allowing them to becomemore engaged and take ownership in the care process, which leads to a stronger partnership and better patient care.