Orphan Drugs Defined

Orphan drugs are medicinal products intended for diagnosis, prevention, or treatment of rare diseases or disorders. It is defined as a condition that affects fewer than 200,000 people in the US (5 per 10,000 in Europe) or the prevalence is greater but the drug manufacturer is not expected to recover the costs of developing and marketing the treatment.


Building a complete orphan dossier based on data from so few patients is challenging. Coté has the necessary specialist proficiency, experience, and a strong working relationship with the regulatory authorities to provide solutions to client’s regulatory challenges.


  • Strategic Oversight: Product development strategy, assessment of current regulatory environment, optimization of orphan drug development strategy
    • Epidemiology analysis
    • Therapeutic area assessment
  • Orphan Drug Designations: FDA (US) and EMA (EU)
    • Construction and submission of the application
    • Management – Annual reports and amendments
  • Meeting Request and Briefing Package: FDA and EMA Meetings
    • Provide submission-ready documents
    • Strategically construct meeting questions to obtain desired meeting outcomes
    • Attend meetings with or on behalf of the client
  • Breakthrough Therapy Designation: Preparation and filing
  • Priority Review Voucher: Rare Pediatric Diseases and Tropica l Diseases
  • INDs and NDAs: Preparation, submission, and on-goi ng support
  • Thought Leadership: White papers, general advice, informational videos, and webinars



  • Unparalleled experience in the regulatory development of orphan drugs.
  • Highly educated team with more than fifty years of combined experience in regulatory and scientific consulting.

Result Oriented

  •  Successfully optimized the regulatory outcomes for over 230 clients globally by obtaining favorable and concrete opinions from the review divisions regarding clinical development programs.
  •  Work alongside a global network of biotechnology and pharmaceutical companies, patient advocacy groups, key opinion leaders, venture capitalists, healthcare professionals, scientists, policy makers, and regulators.
  •  Work independently or bolster your in-house regulatory capacity to help you overcome FDA challenges to meet corporate objectives.