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Rare Diseases

Rare Disease Clinical Trial Expertise

An Opportunity for Real Impact

For drug developers committed to creating new therapies for treating rare diseases, the opportunity to make a significant impact in the lives of patients suffering from these orphan conditions is enormous. However, the process to achieve this goal is substantially different and often much more challenging than what typically characterizes traditional drug development.

Despite the challenges, PPD helps clients navigate the complexities to accomplish success in rare disease trials.

Rare Disease Framework

PPD deploys a four-part framework to rare disease studies that ensures the application of best practices in study development. The key elements of this framework include:

  1. Innovative and executable development plans
    • Capture data using innovative approaches when needed
    • Balance the difficulties of enrolling patients and executing the study with client’s needs
    • Ensure the speed of the trial with an innovative study design to improve the patient experience
  2. Rigorous feasibility and study planning
    • Reviews with advocacy groups and investigators, physician phone interviews and methods other than survey data assessment are used to pressure-test study designs
      • Patient pathways are mapped for each indication to determine where and how to get patients into clinical trials and to understand the patients’ experiences
  3. Study execution
    • Focus on high-touch patient engagement with CRAs allocated based on need for a particular trial and assurance of ongoing physician engagement
    • Develop patient retention approaches, such as a concierge service with a single point of contact for the patient to coordinate all logistics 
    • Study data cleaned appropriately to maximize enrollment since overall patient enrollment and experience, as well as minimizing patient disqualifications, are key
  4. Proactive post-study planning
    • Develop longitudinal site relationships for entire programs, not just single trials
    • Plan for the entire landscape of a program and the needs of patients that may enroll in natural history studies after a rare disease trial 
    • Partner with sponsors at the beginning to plan and optimize the patient flow through the entire program

Customized Approach

At PPD, we develop a customized approach designed specifically to address the unique needs of rare disease trials. Each project is also assigned a dedicated project manager with the required expertise to move the study along successfully and provide you with the confidence of knowing that every detail of your trial is being monitored and managed.

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