{"id":604743,"date":"2024-06-03T08:22:58","date_gmt":"2024-06-03T12:22:58","guid":{"rendered":"https:\/\/platohealth.ai\/elevating-your-rare-neurological-study-in-any-competitive-environment\/"},"modified":"2024-06-03T14:12:58","modified_gmt":"2024-06-03T18:12:58","slug":"elevating-your-rare-neurological-study-in-any-competitive-environment","status":"publish","type":"post","link":"https:\/\/platohealth.ai\/elevating-your-rare-neurological-study-in-any-competitive-environment\/","title":{"rendered":"Elevating Your Rare Neurological Study in Any Competitive Environment","gt_translate_keys":[{"key":"rendered","format":"text"}]},"content":{"rendered":"
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Authors: Amy Raymond<\/strong>, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease & Cellular and Genetic Medicines; Lisle Kingery<\/strong>, Therapeutic Strategy Lead, Neuroscience; Nathan Chadwick<\/strong>, Senior Director, Therapeutic Strategy Lead, Rare Disease<\/p>\n

Groundbreaking research in rare neurological disorders demands a pioneering approach, carefully mapped out based on the indication, research development stage, and the extent of global awareness of the indication. Practical differences exist in ideal study considerations across high- and low-competition environments, and the intricacies of participant availability underscore the necessity of standing out to those who qualify for your trial. Positioning your study at the forefront requires a patient-forward mindset. This philosophy distinguishes your research and includes a comprehensive strategy designed to navigate the complexities inherent to rare neurological research.<\/p>\n

At Worldwide, our experience across all the subspecialties of rare neurology has allowed us to detect and leverage best practices to ensure a trial prevails, even in a highly competitive environment. Below, we delve into strategies for study success in various scenarios.<\/p>\n

Key Considerations Differentiating High vs. Low Competition Rare Neurological Disorders<\/strong><\/p>\n

Drug development increasingly recognizes patients as stakeholders in the process, ones whose input can optimize trial timelines and help accelerate later commercialization. Our experience confirms that incorporating the patient voice<\/a>\u202finto study planning creates trials with fewer study recruitment and retention barriers. For each patient community who shares a rare disease, there is an interplay of drug development activity and that community\u2019s readily accessible input to industry. Development teams with some of the earliest trials for a given indication face the least competition for these rare disease patients and potentially the least pre-existing information on patient perceptions of the meaningfulness of study endpoints if the community has not yet coalesced to create and broadcast priorities. Additionally, steps to maximize the value of clinical assessment data and methods to increase global access are vital considerations. Specific approaches for these, however, vary depending on the competitive landscape. For instance, in high-competition indications, it is all the more important to ensure the trial is easy for the patient and reduces the burden as much as possible. This is in addition to leveraging support from advocacy and other recruitment tactics, such as concierge, to help the trial stand out from competitors and create a higher value proposition.<\/p>\n

Optimize the Value Proposition<\/strong><\/p>\n

For indications with high competition, potential participants will have many studies to consider and will likely choose the study that offers the highest value proposition. These communities are also more likely to have highly structured advocacy organizations, which may provide a qualitative ranking of current clinical trial opportunities scored by the criteria their community deemed important. Many patient advocacy organizations create lists of trial options and sometimes even give each a patient-centricity score to help patients evaluate their options according to criteria important to their community.<\/p>\n

In both high and low-competition indications, patient centricity addresses the most significant sources of friction for study recruitment and retention, which differ depending on the competitive environment. For high-competition spaces, this means enhancing value propositions above competing trials. However, with lower competition environments, trials often must work with less infrastructure of researchers experienced in the indication, fewer regulatory precedents, and challenges meeting patient-defined needs. A lack of patient-defined needs further emphasizes working with patients in designing the trial and helping to guide the design and operationalization of the trial by taking time and asking carefully considered questions as early as possible.<\/p>\n

Gather & Implement Patient Input Before, During, & After Your Trial<\/strong><\/p>\n

Patients offer valuable insights from their experiences living with their condition every day. Ensuring their voice drives your approach can position your study to have better recruitment outcomes in all rare neurological disorders, especially in crowded indications. In high-competition spaces, such as Duchenne Muscular Dystrophy, Huntington\u2019s Disease, or Amyotrophic Lateral Sclerosis, incorporating insights regarding a patient community\u2019s needs and wants are controllable factors that can tilt the value proposition in favor of your trial. These communities have typically had the opportunity to define what successful treatment means for them and which symptoms they find the most troublesome or debilitating; reviewing and incorporating their needs is a simple way to make it truly fit-for-purpose, thereby increasing recruitment. Some methods to collect the patient\u2019s perspective before and during the trial include:<\/p>\n