Therapeutic Areas: Rare diseases

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Infectious diseases incl. Covid-19
Rare diseases
Cromos Pharma has in-depth experience in providing services for clinical research related to rare and orphan diseases

Rare diseases are defined as conditions that affect fewer than 200,000 people in the United States or fewer than 1 in 2,000 people in the European Union. Due to the small patient populations, there are unique challenges associated with conducting clinical trials for rare diseases, which require specialized expertise. Having extensive international capabilities and thus access to vast patient populations, gives Cromos Pharma a significant edge over many of its competitors.

We offer a wide range of services to support clinical research in rare disease indications. These include:

  • Protocol design: we can help design clinical trials that take into account the unique features of rare diseases, such as the small patient populations, heterogeneity of the disease, and lack of available natural history data. We can help to develop study designs that are statistically sound and appropriate for regulatory approval.
  • Patient recruitment: One of the biggest challenges in conducting clinical trials for rare diseases is patient recruitment. Cromos Pharma has experience in identifying and engaging with patient advocacy groups, as well as establishing relationships with clinical investigators and research sites in the US and ex-US that specialize in rare diseases. We are experts at devising referral programs that help outlying clinicians identify and refer eligible prescreened patients to our clinical trial sites.
  • Clinical trial management: Cromos Pharma provides project management and oversight for the most complex and challenging clinical trials, ensuring that they are conducted in compliance with regulatory requirements and that data is collected and analyzed effectively.
  • Data management and analysis: we can help manage and analyze complex datasets, including developing case report forms (CRFs) and databases that are tailored to the specific needs of the rare disease being studied.
  • Regulatory affairs: Cromos Pharma provides expertise in navigating the regulatory landscape for rare diseases, including preparing and submitting regulatory dossiers and interacting with regulatory agencies.
  • Medical writing: we provide medical writing services for clinical trial documents, such as protocols, informed consents, study reports, and regulatory submissions.
  • In addition to these services, we have specialized expertise in many rare disease therapeutic areas. As such, Cromos Pharma has experience in conducting clinical trials for genetic disorders, rare cancers, and autoimmune diseases.

We pride ourselves on our ability to provide a valuable service for pharmaceutical and biotech companies that are developing treatments for rare diseases. By leveraging our specialized expertise and experience, Cromos Pharma can help to accelerate the development of treatments for these challenging conditions.

Our rare/orphan disease experience includes:
    • Bulimia nervosa
    • Congenital afibrinogenemia
    • Growth hormone deficiency (GHD)
    • Glioblastoma
    • Hemophilia A
    • Hemophilia B
    • Hereditary angioedema
    • Niemann-Pick disease Type C
    • Meniere’s disease
    • Retinitis pigmentosa
    • Sjogren-Larsson syndrome
    • X-linked adrenomyeloneuropathy
    • Von Willebrand’s Disease
Our rare/orphan disease experience includes:
Clinical Sites
our Success story
A Phase III Placebo-Controlled Ascending Dose trial in Adult Growth Hormone Deficiency (GHD) Patients
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Why Cromos Pharma
  • We are the only CRO to offer “No Patients, No Payment” risk sharing program, where the sponsor pays only if we deliver on our milestones.
  • We have the scientific and clinical expertise to support your clinical studies, from early to late phase, from single- to multi-country execution strategies.
  • By leveraging our international expertise, we deliver unparalleled results through diligent feasibility analysis, rapid startup, and accelerated recruitment, while utilizing proactive risk management strategies and maintaining the highest data quality.
  • Our team has vast expertise in organizing scientific advisory board meetings of key opinion leaders, clinicians, and industry stakeholders.



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