Patient-First Strategy vs. “Pharma Strategy Expertise” for Building Clinical Research Success in Rare and Orphan Disease Treatment

Clinical Researcher—July 2021 (Volume 35, Issue 5)


Donvan Quill


Rare diseases affect 1% or less of the global population, with the geographic spread and small number of those impacted making the cost of research and development (R&D) prohibitive and leaving patients without treatments. Of the 7,000 known rare diseases, 95% thus far do not have a single drug treatment approved by the U.S. Food and Drug Administration (FDA).

Historically, rare diseases have not attracted significant pharmaceutical investment; today, however, that is changing. In fact, large pharmaceutical companies have begun to focus on rare diseases, drawn by government incentives and the growing likelihood that treatments for what are often life-threatening or severely debilitating diseases will be successful.

About 33% of all drugs in active R&D pipelines are now included in the rare disease category, presenting scientific and operational challenges to sponsors and clinical trial ecosystem participants, as well as spurring the adoption of new strategies, operating models, and processes.

Clinical research professionals seeking to build a go-to market strategy, however, may feel overwhelmed about where to start and may be tempted to revert to a so-called “pharma strategy expert.” This term is misleading, given that it’s impossible to provide expertise across the wide range of therapies for rare and orphan conditions. Each patient is unique in terms of the treatment, points of care, physicians, and level of caregiving they require.

Self-appointed rare condition experts may be virtuosos in big pharma or skillful in commercialization strategies with a particular condition, but too often they fall far short of expectations. They offer an ever-increasingly complicated process that fails to be cost effective. What’s more, cookie-cutter pharma strategies have no place in today’s complex and ever-evolving healthcare environment. What’s needed is a patient-first approach that relies on a team of experts who bring a specific understanding to each patient’s condition to provide effective therapy and care management.

The Downside of Rare Disease Experts

Rare disease “experts” begin with inherent assumptions about care delivery and optimization to map the patient journey. They attempt to break down a strategy into multiple phases, such as precommercial planning and distribution. All of this can be justified with outmoded approaches to care and may sound rational. The problem is that each new phase of the process contains hidden costs and growing complexity with the creation of layers between the clinician and patient that often fail to improve patient outcomes.

In fact, this approach can become so complicated that pharma execs must pay for additional management staff to oversee the process and inform the clinician about next steps in patient services. This not only adds costs and unnecessary layers between the patient and clinician, but also obscures data and outcomes.

When optimizing care for rare and orphan disease patients, the best place to begin is at the end: the patients who require therapy. It’s important for stakeholders to learn their individual needs and expectations. They must also understand that compassion—and not managerial layers—is vital for patient quality of life and improved outcomes.

With a patient-first strategy, pharmaceutical manufacturers and their clinical research professionals can build a commercialization team that is open, curious, and empathetic. 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.

A Patient-First Strategy Mitigates Clinical Trial Disruption

A patient-first approach provides comprehensive, best-in-class services tailored to maximize therapeutic opportunities for people in the rare disease community, including counseling, guidance, and education based upon patient and caregiver needs.

The benefits of partnering with a specialty pharmacy and patient management organization that takes a patient-first approach have been put in bold relief during the COVID-19 pandemic. The best of these organizations use tools to enable in-home clinical services, direct-to-patient support, and remote monitoring for keeping clinical trials on track. This approach can significantly shorten the time from the clinical trial to commercial drug access.

The pandemic represents additional issues facing patients, researchers, providers, and drug manufacturers in the rare and orphan disease market. These challenges include the high cost of clinical trials and patient recruitment. While traditional models are built for scale, a patient-first approach focuses on and customizes services for small patient populations, delivering expertise to overcome the limitations of legacy care models and providing cost-effective programs. This streamlined approach includes financial advantages, assurance that products are properly and promptly distributed, and patient services designed to ensure compliance and quick, accurate reimbursement processing.

Further, integrated telehealth features have enabled patients to get the products delivered without going to the doctor’s office. As a result, the trials conducted during the pandemic had significantly more patients involved, despite the national lockdown. This approach helps to build awareness and introduce education programs that aid understanding of patient groups, and clinical research professionals and manufacturers know they have the support to develop a drug and a comprehensive program based upon specific needs.

High Level Support for Specialty Patients

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. Dedicated clinical teams are better able to seamlessly eliminate treatment gaps for the patient. This strategy also addresses all variables around collecting data, while maintaining frequent communication with patients and their families to ensure compliance and positive outcomes.

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

Finding the Right Patient Management Partner

When identifying 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 the trusted path for patients and all those involved in the treatment journey. This adds much-needed 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.

Telehealth Considerations

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

Effective 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.

As part of a larger personalized care plan, and 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.

Customized care coordination and telehealth solutions add another layer to a proactive, process-driven program, educating the patient on potential risks. This fosters discussion between the patient and providers that is essential 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 stay on top of side effects and capture real-world evidence around the therapy, the disorder, and the person’s well-being.

Closing Thoughts

On top of everything else already mentioned, the most effective specialty partner should demonstrate expertise in navigating 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.

Ideally, the care management solution should meet the needs of everyone involved in the patient’s journey, from clinical research professionals and specialty drug manufacturers to pharmacists, caregivers, and physicians.

Donovan Quill is President and CEO of Optime Care.