Publications by category
Journal articles
O'Connor E, Nikram E, Grangeon L, Danno D, Houlden H, Matharu M (2022). The clinical characteristics of familial cluster headache.
Cephalalgia,
42(8), 715-721.
Abstract:
The clinical characteristics of familial cluster headache.
BACKGROUND: a positive family history predisposes to the development of cluster headache. The distinct characteristics of familial cluster headache have yet to be confirmed, however, evidence suggests a younger age of onset and higher proportion of females in this subgroup. OBJECTIVES: to assess the rate and mode of inheritance of familial cluster headache in a tertiary referral centre for headache. To describe the clinical features of familial cluster headache. METHODS: a retrospective study conducted between 2007 and 2017. Cluster headache was confirmed in probands and affected relatives. Differences in demographics, clinical characteristics, and response-to-treatment in familial cluster headache were delineated through multivariate analysis using a control cohort of 597 patients with sporadic cluster headache. RESULTS: Familial cluster headache was confirmed in 48 (7.44%) patients and predominantly reflected an autosomal dominant mode of inheritance with reduced penetrance. Familial cases were more likely to report nasal blockage (OR 4.06, 95% CI; 2.600-6.494, p
Abstract.
Author URL.
Farmer C, Bullement A, Packman D, Long L, Robinson S, Nikram E, Hatswell AJ, Melendez-Torres GJ, Crathorne L (2020). Voretigene Neparvovec for Treating Inherited Retinal Dystrophies Caused by RPE65 Gene Mutations: an Evidence Review Group Perspective of a NICE Highly Specialised Technology Appraisal.
Pharmacoeconomics,
38(12), 1309-1318.
Abstract:
Voretigene Neparvovec for Treating Inherited Retinal Dystrophies Caused by RPE65 Gene Mutations: an Evidence Review Group Perspective of a NICE Highly Specialised Technology Appraisal.
The UK National Institute for Health and Care Excellence (NICE) considered evidence for voretigene neparvovec (VN; Luxturna®) for the treatment of RPE65-mediated inherited retinal dystrophies (IRD) within its highly specialised technology programme. This paper summarises the evidence provided by the company; the appraisal of the evidence by the Peninsula Technology Appraisal Group, who were commissioned to act as the independent evidence review group (ERG); and the development of the NICE guidance by the appraisal committee. The evidence presented by the company highlighted the significant lifelong burden of IRD for patients and carers. Evidence to support the effectiveness of VN was lacking, but the available evidence showed a modest, sustained improvement across a variety of vision-related outcomes. While patients would remain visually impaired, the committee considered that VN would prevent further deterioration in vision. The modelling approach used by the company had a number of limitations and relied heavily upon a large volume of clinical expert input to produce cost-effectiveness estimates with large uncertainty around long-term effectiveness. The ERG's main concerns revolved around these long-term outcomes and the plausibility of utility values. The NICE committee were convinced that the clinical benefits of VN were important and an appropriate use of national health service resources within a specialised service. The committee concluded that a high unmet need existed in patients with RPE65-mediated IRD and that VN represents a step change in the management of this condition.
Abstract.
Author URL.
(2020). Voretigene neparvovec good value in inherited retinal dystrophies. PharmacoEconomics & Outcomes News, 847(1), 35-35.
(2019). NICE recommends benralizumab for severe eosinophilic asthma. PharmacoEconomics & Outcomes News, 819(1), 37-37.
Conferences
Tikhonova I, Long L, Ocean N, Barnish M, Robinson S, Nikram E, Bello S, Dodman S, Hoyle M (2019). BENRALIZUMAB FOR TREATING SEVERE EOSINOPHILIC ASTHMA: NICE SINGLE TECHNOLOGY APPRAISAL.
Author URL.
Reports
Bello S, Griffin E, Farmer C, Nikram E, Robinson S, Packman D, Salmon A, Cossburn M, Melendez-Torres GJ, Crathorne L, et al (2019). Fremanezumab for preventing migraine: a Single Technology Appraisal. NICE.
Farmer C, Bullement A, Packman D, Long L, Robinson S, Nikram E, Bello S, Dodman S, Rezaei Hemami M, Churchill A, et al (2019). Voretigene neparvovec for inherited retinal dystrophies (RPE65 mutations) [ID1054]: a Highly Specialised Technology Evaluation. NICE.
Tikhonova I, Long L, Ocean N, Barnish M, Robinson S, Nikram E, Bello S, Dodman S, Halpin D, Hoyle M, et al (2018). Benralizumab for treating severe asthma: a Single Technology Appraisal.
Griffin E, Farmer C, Packman D, Nikram E, Matthews J, Barnish M, Briscoe S, Dorey N, Dangoor A, Mujica Mota R, et al (2018). Pembrolizumab with pemetrexed and platinum chemotherapy for untreated metastatic non-squamous non-small-cell lung cancer [ID1173]: a Single Technology Appraisal. NICE.
Publications by year
2022
O'Connor E, Nikram E, Grangeon L, Danno D, Houlden H, Matharu M (2022). The clinical characteristics of familial cluster headache.
Cephalalgia,
42(8), 715-721.
Abstract:
The clinical characteristics of familial cluster headache.
BACKGROUND: a positive family history predisposes to the development of cluster headache. The distinct characteristics of familial cluster headache have yet to be confirmed, however, evidence suggests a younger age of onset and higher proportion of females in this subgroup. OBJECTIVES: to assess the rate and mode of inheritance of familial cluster headache in a tertiary referral centre for headache. To describe the clinical features of familial cluster headache. METHODS: a retrospective study conducted between 2007 and 2017. Cluster headache was confirmed in probands and affected relatives. Differences in demographics, clinical characteristics, and response-to-treatment in familial cluster headache were delineated through multivariate analysis using a control cohort of 597 patients with sporadic cluster headache. RESULTS: Familial cluster headache was confirmed in 48 (7.44%) patients and predominantly reflected an autosomal dominant mode of inheritance with reduced penetrance. Familial cases were more likely to report nasal blockage (OR 4.06, 95% CI; 2.600-6.494, p
Abstract.
Author URL.
2020
Nikram E, Balkenborg D (2020). A Generalized Hotelling-Downs model with Asymmetric Candidates.
Farmer C, Bullement A, Packman D, Long L, Robinson S, Nikram E, Hatswell AJ, Melendez-Torres GJ, Crathorne L (2020). Voretigene Neparvovec for Treating Inherited Retinal Dystrophies Caused by RPE65 Gene Mutations: an Evidence Review Group Perspective of a NICE Highly Specialised Technology Appraisal.
Pharmacoeconomics,
38(12), 1309-1318.
Abstract:
Voretigene Neparvovec for Treating Inherited Retinal Dystrophies Caused by RPE65 Gene Mutations: an Evidence Review Group Perspective of a NICE Highly Specialised Technology Appraisal.
The UK National Institute for Health and Care Excellence (NICE) considered evidence for voretigene neparvovec (VN; Luxturna®) for the treatment of RPE65-mediated inherited retinal dystrophies (IRD) within its highly specialised technology programme. This paper summarises the evidence provided by the company; the appraisal of the evidence by the Peninsula Technology Appraisal Group, who were commissioned to act as the independent evidence review group (ERG); and the development of the NICE guidance by the appraisal committee. The evidence presented by the company highlighted the significant lifelong burden of IRD for patients and carers. Evidence to support the effectiveness of VN was lacking, but the available evidence showed a modest, sustained improvement across a variety of vision-related outcomes. While patients would remain visually impaired, the committee considered that VN would prevent further deterioration in vision. The modelling approach used by the company had a number of limitations and relied heavily upon a large volume of clinical expert input to produce cost-effectiveness estimates with large uncertainty around long-term effectiveness. The ERG's main concerns revolved around these long-term outcomes and the plausibility of utility values. The NICE committee were convinced that the clinical benefits of VN were important and an appropriate use of national health service resources within a specialised service. The committee concluded that a high unmet need existed in patients with RPE65-mediated IRD and that VN represents a step change in the management of this condition.
Abstract.
Author URL.
(2020). Voretigene neparvovec good value in inherited retinal dystrophies. PharmacoEconomics & Outcomes News, 847(1), 35-35.
2019
Tikhonova I, Long L, Ocean N, Barnish M, Robinson S, Nikram E, Bello S, Dodman S, Hoyle M (2019). BENRALIZUMAB FOR TREATING SEVERE EOSINOPHILIC ASTHMA: NICE SINGLE TECHNOLOGY APPRAISAL.
Author URL.
Bello S, Griffin E, Farmer C, Nikram E, Robinson S, Packman D, Salmon A, Cossburn M, Melendez-Torres GJ, Crathorne L, et al (2019). Fremanezumab for preventing migraine: a Single Technology Appraisal. NICE.
(2019). NICE recommends benralizumab for severe eosinophilic asthma. PharmacoEconomics & Outcomes News, 819(1), 37-37.
Farmer C, Bullement A, Packman D, Long L, Robinson S, Nikram E, Bello S, Dodman S, Rezaei Hemami M, Churchill A, et al (2019). Voretigene neparvovec for inherited retinal dystrophies (RPE65 mutations) [ID1054]: a Highly Specialised Technology Evaluation. NICE.
2018
Tikhonova I, Long L, Ocean N, Barnish M, Robinson S, Nikram E, Bello S, Dodman S, Halpin D, Hoyle M, et al (2018). Benralizumab for treating severe asthma: a Single Technology Appraisal.
Griffin E, Farmer C, Packman D, Nikram E, Matthews J, Barnish M, Briscoe S, Dorey N, Dangoor A, Mujica Mota R, et al (2018). Pembrolizumab with pemetrexed and platinum chemotherapy for untreated metastatic non-squamous non-small-cell lung cancer [ID1173]: a Single Technology Appraisal. NICE.
