Rare and Orphan Diseases

Icon, clinical trials in central & easter europe

An orphan or rare disease is a rare medical condition that affects a small percentage of the population. It is increasingly recognized that while each rare disease may affect a small number of individuals, the collective burden of rare or orphan diseases is a major global healthcare challenge. The World Economic Forum estimates that 475 million people worldwide are affected by a rare disease.


This figure, however, cannot begin to account for the household members, employers, friends, and families who are also profoundly impacted by the rare disease patient’s diagnosis, prognosis, and care (which is definitionally, often debilitating and with no known treatment or cure). 


Additional challenges facing rare disease clinical trials

Drug development for rare diseases involves significant challenges above and beyond those encountered in large trials for more common diseases. Some of these challenges include a lack of widespread clinical knowledge about rare diseases, uncertainty in relation to regulatory practice, and a lack of consensus on endpoints for clinical studies. In addition, rare disease clinical trials often need to access geographically dispersed patients and develop research-naïve sites.  


When facing these additional hurdles partnering with a clinical research organization with a strong track record in innovative trial design, exceptional multi-country patient recruitment, and proven regulatory expertise is vital.


Integrated expertise and support

That’s where Cromos Pharma comes in. Since our foundation in 2004, we have built significant experience in orphan drug research, and offer our global services to successfully overcome many of the common obstacles in this field. Our dedicated team is comprised of medical and regulatory experts with practical experience and deep knowledge of this challenging area.


Our unique approach to orphan and rare diseases

Our key differentiator in expediting rare disease development is achieved through the implementation of a unique strategy on a case-by-case basis to optimize the execution of an orphan drug study. For example, at the stage of trial planning, our team applies the lessons learned from oncology and pediatric studies in rare disease programs, combined with the Key Opinion Leaders’ (KOL) feedback, to provide the best advice on implementing adaptive trial designs.

The distinctive methodology used to achieve the appropriate site selection allows us to perform realistic feasibility analyses to deliver the required patients. In this analysis, Cromos Pharma’s Feasibility Team performs comprehensive diligence on patient eligibility in addition to reviewing other key aspects of the project, such as Investigators’ experience, equipment accessibility, competing trials, varying standards of care, and regulatory aspects of particular countries.

Rare disease drug development requires extra attention, that’s why our study team provides the Investigators with additional training and support throughout the study duration.


Cromos Pharma’s expertise in rare disease drug research

Cromos Pharma has contributed to the analysis, design, management and/or conduct of 20+ studies in rare diseases, covering Phases II to IV, including but not limited to:

  • Autism spectrum disorders
  • Bulimia nervosa
  • Congenital afibrinogenemia
  • Glioblastoma
  • Growth hormone deficiency (GHD)
  • Haemophilia A
  • Haemophilia B
  • Hereditary angioedema
  • Ménière’s disease 
  • Niemann-Pick disease Type C
  • Retinitis pigmentosa
  • Sjögren-Larsson syndrome
  • Von Willebrand’s Disease
  • X-linked adrenomyeloneuropathy

Cromos Pharma has over 18 years of experience in managing all aspects of clinical trials across a range of therapeutic areas. With operations in the US, Central and Eastern Europe we combine global expertise with profound local market knowledge to deliver end to end solutions supporting our pharma and biotech clients.