Regulatory Consultancy

PSR offers regulatory support in the Orphan Drug niche to our clients for a wide range of therapeutics, including new chemical entities, biologics, gene therapies and advanced therapies (ATMPs). Our regulatory team is seasoned in the rare disease space and has regular interactions with authorities such as the CHMP, the Committee for Orphan Medicinal Products (COMP) and the PDCO.

PSR typically handles orphan drug designations using an integrated approach which includes review of the available data package, implementing strategic components (including medical plausibility & prevalence sections), in order to smoothen the next steps of the development process (protocol assistance, drug development plan and actual execution of the clinical studies), and performing all the relevant administrative activities (including pre-submission meetings if applicable), submitting the application and responding to questions / attending regulatory body meetings.

PSR has been involved in over 40 orphan drug applications and we can subsequently facilitate obtaining scientific advice and protocol assistance at EMA and FDA level. We can also help our clients identify indications for their product that potentially qualify for an orphan drug status.


Many, and specifically Orphan drug projects are discussed before kick-off and/or during execution at ad-hoc scientific advisory board meetings.

Our Regulatory Consultancy service includes:

  • EMA & FDA Orphan Drug Designation Applications (drafting, submission and meeting attendance)
  • Regulatory/Development Strategy Consulting
  • National or Central Scientific Advice Meeting Preparation and Attendance (SAWP)
  • Protocol Assistance Meeting Preparation and Attendance
  • Core Document Preparation and Approval- Electronic Submissions
  • Investigational Medicines Product Dossier (IMPD) Preparation
  • Clinical Trial Applications
  • Paediatric Investigational Plans
  • Pre-IND, IND Application and IND Maintenance