Draft remit: To appraise the clinical and cost effectiveness of eladocagene exuparvovec within its marketing authorisation for treating aromatic L-amino acid decarboxylase deficiency.
Status In progress
Technology type Medicine
Decision Selected
Process HST
ID number 3791

Provisional Schedule

Expected publication 29 March 2023

Project Team

Project lead Gavin Kenny

Email enquiries


Companies sponsors PTC Therapeutics (eladocagene exuparvovec)
Others • Department of Health and Social Care
  • NHS England
  • Welsh Government
Patient carer groups AADC Trust
Professional groups Royal College of Physicians
Comparator companies NONE
General commentators • All Wales Therapeutics and Toxicology Centre
  • British National Formulary
  • Department of Health, Social Services and Public Safety for Northern Ireland
  • Healthcare Improvement Scotland
  • Medicines and Healthcare products Regulatory Agency
  • Scottish Medicines Consortium
  • Welsh Health Specialised Services Committee


Key events during the development of the guidance:

Date Update
07 March 2022 Invitation to participate
18 August 2021 (14:00) Scoping workshop
01 July 2021 - 29 July 2021 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
15 October 2020 In progress. topic referral

For further information on our processes and methods, please see our CHTE processes and methods manual