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: 2 06 February 2025

Project Team

Project lead Leena Issa

Email enquiries

External Assessment Group Newcastle NIHR TAR Team, Newcastle University

Stakeholders

Companies sponsors Pharming Group N.V (leniolisib)
Others Cambridge University Hospitals, NHS Foundation Trust
  Department of Health and Social Care
  NHS England
Patient carer groups Gene People
  Immunodeficiency UK
Professional groups British Society for Immunology
  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 Government
  Welsh Health Specialised Services Committee
Relevant research groups National Institute for Health Research

Timeline

Key events during the development of the guidance:

Date Update
31 October 2024 - 12 December 2024 Evaluation consultation
10 October 2024 Committee meeting
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