Key factors to consider for rare disease/orphan drug development

Key factors to consider for rare disease/orphan drug development

Rare disease clinical trials pose challenges above and beyond those encountered in trials involving more common conditions including: very few patients being available for clinical trials, those who are available are often geographically dispersed, lack of knowledge and data about a particular rare disease, the amount and quality of data generated by small-scale trials and the need for multi-site, multi-country trials requiring regulatory expertise in a number of regions. Cromos Pharma’s Head of Start-Up, Mikhail Kamkha, MD, MRQA outlines some of the specific considerations necessary to improve the chances for success in rare disease drug development:

Keys to success for rare disease/orphan drug development

Be where the patients are and expert study planning

  • Assessment of epidemiology/treatment availability (insurance or governmental program coverage)/centralization of subjects and subject data (i.e. Registers)/competitive studies
  • KOLs experience and professional long-term relationships
  • Input from KOLs to the study protocol to speed-up enrollment and compliance
  • Review of sources\Registers to initially determine possible study population
  • To develop study-related country-specific strategy in a close collaboration with sites
  • To choose only sites which showed initial quality
  • Continuous risk management
  • Focus on quality from the beginning to end

Partnering with a CRO with a strong track record in innovative trial design, exceptional multi-country patient recruitment and proven regulatory expertise is vital.

Cromos Pharma is an international CRO with 16 years of experience in managing all aspects of clinical trials across all phases in a wide range of therapeutic areas. Our team has managed several rare disease clinical trials and can provide your next study with the support and expertise to help ensure success.

Find out more:





To arrange an introductory meeting and find out how our experience can benefit your next clinical project.