Suggested remit: To appraise the clinical and cost effectiveness of leniolisib within its marketing authorisation for untreated activated phosphoinositide 3-kinase delta syndrome in people 12 years and over.
Status In progress
Technology type Medicine
Decision Selected
Reason for decision Anticipate the topic will be of importance to patients, carers, professionals, commissioners and the health of the public to ensure clinical benefit is realised, inequalities in use addressed, and help them make the best use of NHS resources
Process HST Standard
ID number 6130

Provisional Schedule

Committee meeting 10 October 2024
Expected publication 18 December 2024

Project Team

Project lead Celia Mayers

Email enquiries

External Assessment Group Newcastle NIHR TAR Team, Newcastle University


Companies sponsors Pharming Group N.V (leniolisib)
Others Cambridge University Hospitals, NHS Foundation Trust
  Department of Health and Social Care
  NHS England
  Royal Papworth Hospital, NHS Foundation Trust
Patient carer groups Anthony Nolan
  Gene People
  Genetic Alliance UK
  Immunodeficiency UK
  South Asian Health Foundation
  Specialised Healthcare Alliance
Professional groups Association of Genetic Nurses & Counsellors
  British Geriatrics Society
  British Society for Gene and Cell Therapy
  British Society for Genetic Medicine
  British Society for Immunology
  British Society of Blood and Marrow Transplantation and Cellular Therapy
  Neonatal and Paediatric Pharmacists Group
  NHS Blood and Transplant
  Royal College of General Practitioners
  Royal College of Nursing
  Royal College of Paediatrics & Child Health
  Royal College of Pathologists
  Royal College of Physicians
  Royal Society of Medicine
  United Kingdom Primary Immunodeficiency Network
  UK Clinical Pharmacy Association
Associated public health groups Public Health Wales
  UK Health Security Agency
Comparator companies None
General commentators All Wales Therapeutics and Toxicology Centre
  Allied Health Professionals Federation
  Board of Community Health Councils in Wales
  British National Formulary
  Care Quality Commission
  Cell and Gene Therapy Catapult
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Medicines and Healthcare products Regulatory Agency
  National Association of Primary Care
  National Pharmacy Association
  National Services Division
  NHS Confederation
  Scottish Medicines Consortium
  Welsh Government
  Welsh Health Specialised Services Committee
Relevant research groups Cochrane Cystic Fibrosis & Genetic Disorders Group
  Cochrane UK
  Genomics England
  MRC Clinical Trials Unit
  National Institute for Health Research


Key events during the development of the guidance:

Date Update
11 January 2024 Please note that following on from advice received from the company this evaluation has been rescheduled to align with latest regulatory expectations. Therefore, we now anticipate that the evaluation will recommence during late May 2024 when the deadline for submissions is expected.
19 September 2023 Topic selection
19 September 2023 Topic selection. Following a challenge from the company, the topic routing was re-considered by the Topic Selection Oversight Panel (TSOP) in July 2023. The panel concluded that the topic be routed to the Highly Specialised Technologies (HST) work programme. Please see project documents for further details.
31 August 2023 Invitation to participate
06 June 2023 The topic was discussed at the Topic Selection Oversight Panel (TSOP) in March 2023. The panel concluded that the topic was suitable for a Technology Appraisal (TA) evaluation. The company have challenged the routing decision and the timelines for the evaluation are to be confirmed.
12 January 2023 (14:00) Scoping workshop
08 November 2022 - 06 December 2022 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
13 October 2022 In progress
16 May 2022 Awaiting development. Status change linked to Topic Selection Decision being set to Selected

For further information on how we select topics for development, please see our page about topic selection