Patient-First Strategy Transcends Limitations of “Pharma Strategy Expertise,” Builds Clinical Research Success and Improves Treatment for Rare/Orphan Diseases

By Donovan Quill, President and CEO, Optime Care 

In the past, rare diseases have not attracted significant pharmaceutical investment, but this is changing. Large pharmaceutical companies have begun to focus on rare diseases due to government incentives and the increased likelihood that treatments for life-threatening or severely debilitating diseases will be effective.

Approximately 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 the adoption of new strategies, operating models and processes. 

Clinical research professionals wanting to build a go-to market strategy often feel overwhelmed and may seek advice from so-called “pharma strategy expert.” Unfortunately, this term is confusing, 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 treatment, points of care, physicians and level of caregiving they require.   

Most rare disease “experts” understand big pharma or are skilled at strategizing commercialization, but too often they offer an ever-increasingly complicated process that fails to be cost-effective. 

In today’s complex environment, a patient-first approach is more effective because it relies on a team of experts who bring a specific understanding to each patient’s condition to provide effective therapy and care management. 

The Disadvantages of Rare Disease Experts  

Rare disease specialists often break down a strategy into multiple phases, such as precommercial planning and distribution, justifiying this approach with outmoded approaches to care. But each new phase of the process contains hidden costs and growing complexity that only adds layers between the clinician and patient. Each layer can force pharma execs to pay for additional management staff simply to oversee the process and inform the clinician about next steps in patient services. This approach also strips away transparency around data and outcomes. 

Instead, stakeholders must focus on learning patient’s needs and expectations and understand that compassion 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 overly focused on scale alone. 

Patient-First During Clinical Trial Disruption

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

A specialty pharmacy and patient management organization that takes a patient-first approach uses tools to enable in-home clinical services, direct-to-patient support and remote monitoring for keeping clinical trials on track. This approach shortens the time from the clinical trial to commercial drug access. In less than a month, a patient-centric program can transition over 90% of those patients. 

The COVID-19 pandemic has presented additional issues facing patients, researchers, providers and drug manufacturers in the rare and orphan disease market, including high cost of clinical trials and patient recruitment. While traditional models are built for scale, a patient-first approach customizes services for small patient populations, delivering expertise to overcome the limitations of legacy care models and providing cost-effective programs.

This includes financial advantages, assurance that products are properly and promptly distributed, and patient services designed to ensure compliance and quick, accurate reimbursement processing.

Integrated telehealth features have enabled patients to get the products delivered without going to the doctor’s office during the pandemic. As a result, the trials conducted during this time had significantly more patients involved, despite the national lockdown. As a result, clinical research professionals and manufacturers know they have the support to develop a drug and comprehensive program based upon specific needs despite disruption. 

By putting the patient first, 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. 

Choosing a Patient-First Management Partner

When seeking a specialty pharmacy and patient management partner, make sure they offer 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 other stakeholders. This adds much-needed support for the patient’s family and caregivers, enabling them to becomemore engaged and take ownership, which leads to a stronger partnership and better patient care.

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. 

The most effective specialty partner should also 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.