Clinical Researcher—November 2020 (Volume 34, Issue 9)
RECRUITMENT & RETENTION
Thirty million Americans live with a range of 7,000 rare or orphan diseases, and while many affected patients have struggled with reduced care, isolation, and adherence issues for most of their lives, the issue of self-isolation during the COVID-19 pandemic has added to their quality-of-life challenges. These individuals now face elevated stressors due to being immunosuppressed, conscious of infection, and hypersensitive to viral threats. Orphan diseases include disorders such as Huntington’s disease, amyotrophic lateral sclerosis, Cushing syndrome, Alpha-1 antitrypsin deficiency, chronic immune thrombocytopenic purpura, and muscular dystrophy.
Despite restrictions, patients continue to require a high level of therapy adherence support at a time when some pharmacies have reduced their hours. This impacts communication with physicians and other advocates at a time when patients’ contact with the “outside world” has already been severely disrupted due to the need for social distancing.
Many sponsors of drug research also report that patient enrollment in clinical trials has been compromised, prompting contract research organizations and their partners to seek technology solutions that overcome the impact of the pandemic and other challenges. Tailoring information technology hardware and software solutions based upon clients’ data-related needs can help to resolve these issues and improve patient engagement across multiple dimensions of research and development—from clinical trials to commercialization and compliance.
At this intersection, a patient-first approach that focuses on and customizes services for small patient populations is of high value. These targeted programs and services deliver specialized expertise beyond the scope of capabilities provided by traditional, legacy care models that are simply built for scale. As a result, applying this focused approach with a lens on orphan diseases can better address the patient-centric experiences, outcomes, and requirements of a clinical trial.
How Patient-First Impacts Clinical Trials
COVID-19 is proving to be a catalyst for improved clinical trial solutions, with study sponsors seeking to get patients into clinical trials more rapidly as a means of advancing research, especially for the new coronavirus. Generally speaking, clinical trials for other conditions struggle to continue during the COVID-19 pandemic, making it more important than ever to identify innovative ways to keep trials on track and moving forward.
During this kind of healthcare crisis, using tools that enable home-based clinical services, direct-to-patient support, and remote monitoring has become essential to keeping clinical trials running smoothly in an environment of unprecedented complications. In fact, in March 2020, the U.S. Food and Drug Administration published guidance on managing clinical trials during COVID-19, including carrying out assessments via phone or virtual visits and offering additional safety monitoring for clinical trial participants who can no longer access an investigational product or site.
As the clinical trials market continues to grow—it is projected to reach $65.2 billion by 2025 —the pandemic represents just one challenge that patients, providers, and drug manufacturers face in the rare and orphan market. These challenges include managing the high cost of clinical trials and encouraging ongoing patient recruitment and retention in them.
As a preferred care partner for manufacturers, the patient-first methodology provides cost-effective programs and a streamlined approach that includes such financial advantages as a flat fee for services, a single source distribution (which offers more control over the patient experience), and national access. Products are properly and promptly distributed, and patient services are designed to ensure compliance and quick, accurate reimbursement processing.
For example, one pharmacy distribution and patient management organization for the treatment of orphan and rare disorders was involved in two clinical trials at the outset of the pandemic. This participation helped patients receive investigational products without going to the doctor’s office with the help of telehealth to oversee the process. As a result, the trials had significantly more patients involved, despite the national lockdown, and the organization was able to ensure that patients and staff remained safe while participating in the studies.
Other Benefits of the Patient-First Strategy
With a patient-first approach, wholesale distributors, specialty pharmacies, and hub service providers are connected under a “One Stop Shop” program instead of operating independently. The continuity across the entire patient journey, beginning with clinical trials, strengthens communication, yields rich data for more informed decision making, and improves the overall patient experience.
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. This approach benefits the sponsors of drug research by bringing continuity of care that is critically missing from the legacy care model, with a net result: patients have a better experience, which is, after all, a critical component of a product’s value.
In the rare disease space, most drug manufacturers have worked with multiple specialty pharmacy partners and an internal or external product hub, but this unique patient-first approach offers key advantages:
- Single source—When a manufacturer uses several specialty pharmacies and a patient in clinical trials switches to the commercial drug, the payer will almost always have a distribution preference inside its network, which can force patients to change pharmacies — and potentially disrupt care. The single-pharmacy, patient-focused model finds the right drug for the patient. When there’s only one place you can get this drug, it will be covered by insurance.
- Competitive edge—Patients with rare and orphan disorders and their physicians must overcome massive hurdles in achieving any level of consistent care. When a new therapy is available on the market, switching to a new drug can lead to physical, emotional, clinical, and economic challenges. These drugs literally can cost hundreds of thousands of dollars per patient per year, and insurance companies pay close attention to the value received for every dollar spent, prompting doctors and patients to think twice before considering a new therapy. The patient-first methodology specializes in helping patients and providers overcome this reluctance when changing drugs would improve outcomes. This provides a competitive edge in addressing clinical and insurance challenges.
- 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’re focused more on monthly profits, which can lead them to focus on how much time hub service employees spend on the phone. These incentives prevent pharmacies from being truly patient-focused and able to deliver the best possible care.
Rare and orphan disease patients require a high level of support and benefit from high-touch service. A care team, including a program manager, care coordinator, pharmacist, nurse, and specialists, is 100% dedicated to the disease state, patient community, and therapy. This is a critical differentiator from other specialty pharmacies and hub service providers, which tend to push technology solutions that fail to address human needs and variability.
Key Benefit for Sponsors
Sponsors of every size can leverage this continuity of care for a streamlined, “One Stop Shop” approach to optimize the patient journey. Furthermore, the patient-first care model has been proven with a number of programs over the past decade with improved patient outcomes, and with compliance, patient retention, patient utilization, and satisfaction rates of 90% or more for orphan and rare disease therapies (this is compared to industry standards of about 70% to 80% for specialty drugs and 50% for non-specialty drugs).
Donovan Quill is President and CEO of Optime Care in Saint Louis, Mo.