№ lp_2_3_20065
File format: docx Character count: 13417 File size: 84 KB
Formal appeal letter submitted by a patient organisation to a national health authority challenging a negative appraisal decision on a highly specialised therapy and disputing the evaluation of clinical benefits and carer quality-of-life evidence.
Date: 19 March 2024
Organisation: Niemann-Pick UK (NPUK)
Address: Suite 2, Vermont House, Concord, Washington NE37 2SQ
Addressee: Dr Mark Chakravarty, Lead Non-executive Director for Appeals
Institution: National Institute for Health and Care Excellence
Subject: Appeal of Final Draft Guidance decision
Technology Appraised: Olipudase alfa
Indication: Acid sphingomyelinase deficiency (Niemann-Pick disease) type AB and type B
Reference ID: ID3913
Grounds for Appeal: Failure to act fairly; Unreasonable recommendation in light of evidence
Document Type: Formal appeal letter
Geographical Scope: United Kingdom
Cited Studies: Raebel et al., Orphanet Journal of Rare Diseases (2024); Fraser et al.
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Appeal Hearing Decision on Olipudase Alfa for Treating Niemann-Pick Disease Types AB and B [ID3913], 24 May 2024
Year: 2024
Date of hearing: 24 May 2024
Country: England
Document type: Appeal hearing decision summary
Subject: Olipudase alfa for treating Niemann-Pick disease types AB and B (ASMD)
Institution: National Institute for Health and Care Excellence
Appeal body: Highly Specialised Technologies Appeal Panel
Appellant: Niemann-Pick UK
Legal adviser: DAC Beachcroft LLP
Grounds of appeal: Ground 1(a) – failure to act fairly; Ground 2 – unreasonableness in light of the evidence
Technology appraisal identifier: ID3913
Therapeutic area: Acid sphingomyelinase deficiency (ASMD)
Target population: People with ASMD types AB and B in England
Appeal Hearing Decision on Olipudase Alfa for Treating Niemann-Pick Disease Types AB and B [ID3913], 24 May 2024
Year: 2024
Date of hearing: 24 May 2024
Country: England
Document type: Appeal hearing decision summary
Subject: Olipudase alfa for treating Niemann-Pick disease types AB and B (ASMD)
Institution: National Institute for Health and Care Excellence
Appeal body: Highly Specialised Technologies Appeal Panel
Appellant: Niemann-Pick UK
Legal adviser: DAC Beachcroft LLP
Grounds of appeal: Ground 1(a) – failure to act fairly; Ground 2 – unreasonableness in light of the evidence
Technology appraisal identifier: ID3913
Therapeutic area: Acid sphingomyelinase deficiency (ASMD)
Target population: People with ASMD types AB and B in England
Topics: Sphingomyelinase Olipudase Niemann-Pick