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Highly Specialised Technology Evaluation Givosiran For Highly Specialised Technology Evaluation Givosiran for treating acute hepatic porphyria [ID1549] Evaluation Report © National Institute for Health and Care Excellence 2021. All rights reserved. See Notice of Rights. The content in this publication is owned by multiple parties and may not be re-used without the permission of the relevant copyright owner. NATIONAL INSTITUTE FOR HEALTH AND CARE EXCELLENCE Highly Specialised Technology Evaluation Givosiran for treating acute hepatic porphyria [ID1549] Contents: The following documents are made available to consultees and commentators: The final scope and final stakeholder list are available on the NICE website. 1. Company submission from Alnylam 2. Company clarification response 3. Consultee submissions from: a. British Porphyria Association: i. Submission ii. Supporting document on patient case studies b. Global Porphyria Advocacy Coalition submission c. National Acute Porphyria Service, Cardiff and Vale UHB d. National Acute Porphyria Service, King’s College Hospital NHS Foundation Trust e. NHS England and Improvement 4. Evidence Review Group report prepared by PenTAG 5. Evidence Review Group report – factual accuracy check 6. Evidence Review Group addendum Please note that the appendices to the company’s submission and company model will be available as a separate file on NICE Docs for information only. Any information supplied to NICE which has been marked as confidential has been redacted. All personal information has also been redacted. © National Institute for Health and Care Excellence 2021. All rights reserved. See Notice of Rights. The content in this publication is owned by multiple parties and may not be re-used without the permission of the relevant copyright owner. NATIONAL INSTITUTE FOR HEALTH AND CARE EXCELLENCE Highly Specialised Technologies Evaluation Programme Givosiran for treating acute hepatic porphyria [ID1549] Version: 24 November 2020 Submitted by: xxxxxxxxxxxxxxx xxxxxxxxxxxxxxxxxxxxxxxxxxxxx Alnylam Pharmaceuticals Specification for company submission of evidence 1 of 145 Contents Contents ......................................................................................................................................................... 2 List of figures .................................................................................................................................................. 6 List of tables .................................................................................................................................................... 7 Abbreviations .................................................................................................................................................. 9 Executive Summary ...................................................................................................................................... 11 Overview of the proposed technology....................................................................................................... 11 Nature of the condition .............................................................................................................................. 11 Impact of the new technology ................................................................................................................... 12 Impact on the NHS—costs and health effects .......................................................................................... 13 Conclusions .............................................................................................................................................. 13 Section A – Decision problem ....................................................................................................................... 15 1. Statement of the decision problem ....................................................................................................... 15 2. Description of technology under assessment ....................................................................................... 15 2.1. Brand name, approved name and therapeutic class ...................................................................... 15 2.2. Principal mechanism of action of the technology ........................................................................... 16 2.3. Dosing Information of technology being evaluated ........................................................................ 16 3. Regulatory information .......................................................................................................................... 17 3.1. UK marketing authorisation status ................................................................................................. 17 3.2. Anticipated launch date in the UK .................................................................................................. 17 3.3. Regulatory approval of the technology outside the UK .................................................................. 17 3.4. Current use of technology in England ............................................................................................ 17 4. Ongoing studies .................................................................................................................................... 17 4.1. Completed and ongoing studies on the technology from which additional evidence relevant to the decision problem will be available in the next 12 months ......................................................................... 17 4.2. Summary of other planned assessments of technology in the UK ................................................. 18 5. Equality ................................................................................................................................................. 18 5.1. Equality considerations .................................................................................................................. 18 5.2. How the submission will address equality issues ........................................................................... 18 Section B – Nature of the condition .............................................................................................................. 18 6. Disease morbidity ................................................................................................................................. 18 6.1. Disease overview ........................................................................................................................... 19 6.2. Number of patients in England who will be covered by this particular therapeutic indication in the marketing authorisation each year ............................................................................................................ 26 6.3. Life expectancy of people with the disease in England .................................................................. 27 7. Impact of the disease on quality of life .................................................................................................. 27 7.1. Impact on the quality of life of patients, their families and carers ................................................... 27 7.2. Impact that the technology will have on patients, their families and carers .................................... 29 8. Extent and nature of current treatment options ..................................................................................... 30 Specification for company submission of evidence 2 of 145 8.1. Relevant NICE, NHS England or other national guidance or expert guidelines for the condition for which the technology is being used .......................................................................................................... 30 8.2. Description of the clinical pathway of care that includes the proposed use of the technology ....... 30 8.3. Issues relating to current clinical practice, including any uncertainty about best practice .............. 32 8.4. The new pathway of care incorporating the new technology that would exist following national commissioning by NHS England. .............................................................................................................. 32 8.5. How the technology is innovative in its potential to make a significant and substantial impact on health-related benefits, and how it is a ‘step-change’ in the management of AHP ................................... 32 8.6. Changes to the way current services are organised or delivered as a result of introducing the technology ................................................................................................................................................ 33 8.7. Additional tests or investigations needed for selecting or monitoring patients, or particular administration requirements, associated with using this technology that are over and above usual clinical practice ..................................................................................................................................................... 33 8.8. Additional facilities, technologies or infrastructure that need to be used alongside the technology under evaluation for the claimed benefits to be realised ........................................................................... 33 8.9. Tests, investigations, interventions, facilities or technologies that would no longer be needed with using this technology ................................................................................................................................ 33
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