Sebelipase alfa for treating lysosomal acid lipase deficiency that is not Wolman disease (terminated appraisal)

Appeal

Alexion Pharma UK : Appeal letter (PDF 1.52 MB)

Alexion Pharma UK : Written submission (PDF 462 KB)

Alexion Pharma UK : Scrutiny letter (MSWord 59 KB)

Alexion Pharma UK : Response to scrutiny letter (PDF 315 KB)

Alexion Pharma UK : Final scrutiny letter (MSWord 58 KB)

Birmingham Children’s Hospital NHS Foundation Trust : Appeal letter (PDF 386 KB)

Birmingham Children’s Hospital NHS Foundation Trust : Scrutiny letter (MSWord 58 KB)

Birmingham Children’s Hospital NHS Foundation Trust : Response to scrutiny letter (PDF 102 KB)

Birmingham Children’s Hospital NHS Foundation Trust : Final scrutiny letter (MSWord 57 KB)

MPS Society : Appeal letter (PDF 312 KB)

MPS Society : Scrutiny letter (MSWord 58 KB)

MPS Society : Response to scrutiny letter (PDF 136 KB)

MPS Society : Final scrutiny letter (MSWord 56 KB)

Salford Royal Hospital NHS Foundation Trust : Appeal letter (MSWord 213 KB)

Salford Royal Hospital NHS Foundation Trust : Scrutiny letter (MSWord 57 KB)

Salford Royal Hospital NHS Foundation Trust : Response to scrutiny letter (Unknown 244 KB)

Salford Royal Hospital NHS Foundation Trust : Final scrutiny letter (MSWord 56 KB)

The British Inherited Metabolic Disease Group (BIMDG) : Appeal letter (MSWord 78 KB)

The British Inherited Metabolic Disease Group (BIMDG) : Scrutiny letter (MSWord 57 KB)

The British Inherited Metabolic Disease Group (BIMDG) : Response to scrutiny letter (Unknown 257 KB)

The British Inherited Metabolic Disease Group (BIMDG) : Final scrutiny letter (MSWord 55 KB)

Appeal decision (PDF 463 KB)

Final evaluation determination

Final evaluation determination document (PDF 557 KB)

Committee papers (PDF 2.52 MB)

Committee papers Additional Papers (PDF 406 KB)

Evaluation consultation: 2

Evaluation consultation document (PDF 435 KB)

Committee papers (PDF 2.55 MB)

Evaluation consultation: 1

Evaluation consultation document (PDF 417 KB)

Committee papers (PDF 8.26 MB)

Lysosomal acid lipase deficiency - sebelipase alfa: draft scope for consultation (pre-referral) - January 2015 (PDF 104 KB)

Lysosomal acid lipase deficiency - sebelipase alfa: provisional matrix (pre-referral) - January 2015 (PDF 97 KB)