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Rare Disease Clinical Development White Paper Reg

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Rare Disease Clinical Development: Novel Approaches to Overcoming Operational Challenges

This six (6) part white paper dives into the challenges that biopharmaceutical sponsors face and presents a variety of solutions capable of streamlining the process to speed the delivery of rare disease treatments to the market.

  1. Unique Challenges in the Rare Disease Space
  2. The Landscape for Clinical Research is Changing, and Patients are at the Center
  3. New Methodologies and Technologies Suited to Rare Disease Trials
  4. Reducing the Patient Burden
  5. Rare Disease Clinical Trials in the Era of COVID-19
  6. Summary

Rare diseases remain perplexing to medical science due to their complexity, diversity, and low prevalence. The pharmaceutical industry is increasing its focus on developing novel therapies using new technologies and methodologies capable of changing the game in rare disease clinical development—starting with biomarker discovery all the way through to regulatory submission. Obstacles that slow the path to approval and add considerably to drug development costs make getting the development plan right from the beginning of the trial critical. Solutions range from analytical platforms that can identify patients, to tools and trial designs that reduce the burden on patients, to methods for increasing patient engagement and sophisticated approaches for managing and analyzing data. Building a strategy that applies all of these these solutions to rare disease clinical drug development enables underserved populations to benefit from these investments and to find the cures they need sooner.

Rare Disease Clinical Development: Novel Approaches to Overcoming Operational Challenges