| 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
- If you have any queries please email HST@nice.org.uk
| 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 | |
| Royal Papworth Hospital, NHS Foundation Trust | |
| Patient carer groups | Anthony Nolan |
| Beacon | |
| Contact | |
| 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 |
Timeline
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