For individuals affected by rare diseases, early diagnosis and access to treatment through clinical trials often provide the best opportunity to slow disease progression and improve quality of life. As an industry dedicated to accelerating the development of new treatments to address unmet medical needs, it is important for us to ensure that every individual affected by a rare disease is given the opportunity to participate in clinical trials that could benefit them and their families.
The diagnostic and treatment journey is unique for each of the more than 300 million people in the world affected by about 7,000 different rare diseases. Identifying and recruiting patients in specific indications with a low disease prevalence requires a comprehensive and proactive approach that can include multiple components, such as:
- Precision medicine: Translational medicine experts take pharmacokinetic (PK), pharmacodynamic (PD), biomarker, and genomic data to help inform clinical development strategies, guide drug development decisions, and help answer critical questions throughout the drug development process to predict and optimize outcomes for patients.
- Data-driven insights: These are critical in helping companies visualize disease prevalence around the world, focus their search within the right geographic regions, and identify treating physicians.
- Engagement with medical specialists: Data can pinpoint where to start researching and begin reaching out to treating physicians, key opinion leaders (KOLs), and specialist networks. These experts can offer insight into the best strategies for recruitment.
- Relationships with patient and advocacy groups: Cultivating ongoing partnerships with rare disease advocacy groups, organizations that maintain patient registries, and patient and caregiver support networks helps establish trust and improve patient care and experience from the earliest stages of drug development through treatment and beyond.
- Patient-centric trials: Research has shown that recruiting patients for rare disease trials using a patient-centric approach can reduce the time it takes to recruit for these trials by up to 40% compared to non-patient-centric trials.¹ As rare diseases often affect the entire family, it is important to reduce the unique burdens a family faces in living with that disease and participating in a clinical trial. Consider trial designs (e.g., virtual trials, adaptive designs, crossover trials) that can simplify the study without compromising the quality of clinical data collected.
- Centralized patient interaction: It is common for the biopharmaceutical industry to use a number of different technologies, processes, and vendors to accomplish specific functions in a clinical trial. Centralizing patient-related interactions and services from informed consent to travel arrangements, telemedicine, technology support, and more helps improve the clinical trial experience for patients and their caregivers as well as sites.
Research and development investment in rare disease treatments continues to grow and challenges remain in the global identification and recruitment of eligible patients for specific indications with a low disease prevalence. Leveraging precision medicine, data-driven solutions, and collaboration across the rare disease community are instrumental when developing a customized strategy for patient identification and recruitment. Through this approach, the biopharmaceutical industry will be poised to ensure that every individual affected by a rare disease is given the opportunity to participate in clinical trials and that no patient will be left behind.
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¹ The Innovation Imperative: The Future of Drug Development, Part I: Research Methods and Findings, The Economist Intelligence Unit Ltd., ©2018. All rights reserved.