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Elevating Your Rare Neurological Study in Any Competitive Environment

Authors: Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease & Cellular and Genetic Medicines; Lisle Kingery, Therapeutic Strategy Lead, Neuroscience; Nathan Chadwick, Senior Director, Therapeutic Strategy Lead, Rare Disease

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.

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.

Key Considerations Differentiating High vs. Low Competition Rare Neurological Disorders

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 into 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’s 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.

Optimize the Value Proposition

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.

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.

Gather & Implement Patient Input Before, During, & After Your Trial

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’s Disease, or Amyotrophic Lateral Sclerosis, incorporating insights regarding a patient community’s 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’s perspective before and during the trial include:

  • Connecting directly with disease-specific or disease-supporting patient advocacy organizations in potential countries for study implementation to collect or confirm this community’s priority list and ensure the trial has shared overlap.
  • For trials in the US, read and watch FDA Patient-Focused Drug Development meetings, as these are a rich source of intentionally gathered community perspectives. In the EU (EMA), you can access the Patient Experience Data (PED) workshops and the non-profit organization Patient Focused Medicines Development, where a wealth of resources is available to tap into.
  • Perform desktop research by exploring public-facing websites run by the relevant patient advocacy organizations to learn whether structured metrics guide how trial opportunities are ranked and according to what attributes.
  • Convene program-specific Patient Advisory Boards, scheduling engagement at intervals that allow board members to review your plans in development thoroughly. Meet on a recurring, iterative basis throughout trial planning, centralizing communication through the advisory board chair, who can collect the board’s sentiments.
  • Hold focus groups with members of the patient community outside of the advocacy group leaders to gather insights that will inform the trial’s operational plan.

In lower competition indications, such as leukoencephalopathy with vanishing white matter or GM2 gangliosidosis, development teams that partner with the community to define the treatment goals are best positioned to measure outcomes that most matter to these patients. When there is little competition for rare disease patients, it’s often because little or no development has occurred in that indication.

We can and must include the patient’s voice despite challenges. Researchers can work with the communities to guide them in answering important questions and help drive a unified community sentiment to inform optimal study design and recruitment. While this requires additional creativity and thoughtfulness for documentation, it is possible.

We can further tap the patient’s voice through structured exit interviews, patient-reported outcomes focused on what matters most to the patient, health-related quality of life comparisons between pre- and post-trial, and long-term follow-ups on therapeutic effects. By including these measures in your study announcement, you convey compassion, empathy, and an active interest in your patient experiences beyond the face-value trial data. By actively partnering with the treatment community, you increase your likelihood of optimized study recruitment and retention. These tools are also valuable in supporting the FDA, EMA, and other regulatory bodies in addressing the results and setting your therapeutic up for more payor interest (i.e., insurance company reimbursement decisions and treatment coverage selections).

Maximize the Meaning of Your Assessments

The need for clinical assessments that rely on subjective scoring is a pervasive challenge in developing neurological treatments. When there are few patients, as in rare diseases, the smaller trials have even less tolerance for noise in the assessment data. This challenge is often further compounded by the expected small magnitude of change over the trial course in slow-progressing diseases.

At Worldwide, our Clinical Assessment Technologies (CAT) team of extensively trained assessment experts maximizes the value of each data point by ensuring suitability, reliability, and accuracy. Our CAT team provides comprehensive assessment services and training to reduce variability between raters and reduce placebo response. Moreover, our integrated approach streamlines the start-up process, reducing timelines by consolidating scale acquisition, translations, and training program creation, avoiding the need for additional external vendors.

Embrace Globalization to Reduce Site Competition

Sites competing in close physical proximity to your rare neurological trial increase the recruitment challenge, especially for high-patient competition spaces. Instead of focusing exclusively on sites that have conducted the maximum number of trials in the indication, consider branching out to sites with strong trial experience that are also likely to see these patients, even in countries with limited historical rare indication-specific trials. Additionally, utilizing countries where the availability of medicines treating the condition is low makes a clinical trial de facto the standard of care – a much more exciting prospect for patients and physicians. Regulatory requirements may differ between nations, but at Worldwide, we support sponsors with global trials and offer expert support by engaging with all regional regulatory agencies. We also support cross-border enrollment and mitigate the complexities and challenges of enrolling patients in other countries, which benefits greatly from our local teams’ cultural awareness and clinical expertise.

Additionally, you can take a data-driven approach to site selection by considering the following:

  • Previous enrollment successes or failures at potential sites
  • Patient location and selection of a central location for optimal recruitment
  • Diverse site profiles (e.g., community hospitals versus academic centers)

Choose Excellence Without Compromise

At Worldwide, we deliver patient-focused enrollment strategies for indications in high- and low-competition environments, and we’re passionate about pioneering breakthroughs in rare neurological disorders. We bring customized strategies to the table that differentiate your study to meet your recruitment needs, all while making trials more accessible to patients. We’re always exploring innovative approaches to drive better results for our sponsors and patients. Worldwide Clinical Trials has conducted 500+ neuroscience clinical projects, with more than 50 projects specific to rare neurological disorders. Whether you’re tackling challenges in an oversaturated market or venturing into novel indications, contact us today to discuss your study and see how we can support your enrollment needs.