Hostname: page-component-cd9895bd7-fscjk Total loading time: 0 Render date: 2024-12-25T05:31:21.719Z Has data issue: false hasContentIssue false
  • English
  • Français

Medicine characteristics affecting the time to guidance publication by National Institute for Health and Care Excellence in the single technology appraisal process

Published online by Cambridge University Press:  22 December 2021

Shunsuke Takada Open the ORCID record for Shunsuke Takada [Opens in a new window]  and
Mamoru Narukawa
Shunsuke Takada*
Affiliation:
Department of Clinical Medicine (Pharmaceutical Medicine), Graduate School of Pharmaceutical Sciences, Kitasato University, 5-9-1 Shirokane, Minato-ku, Tokyo 108-8641, Japan Daiichi Sankyo Co., Ltd., Tokyo, Japan
Mamoru Narukawa
Affiliation:
Department of Clinical Medicine (Pharmaceutical Medicine), Graduate School of Pharmaceutical Sciences, Kitasato University, 5-9-1 Shirokane, Minato-ku, Tokyo 108-8641, Japan
*
Author for correspondence: Shunsuke Takada, E-mail: [email protected]
Get access Rights & Permissions [Opens in a new window]

Abstract

Objective

In England, the time gap between marketing authorization (MA) and guidance publication by National Institute for Health and Care Excellence (NICE) can limit patients’ access to new medicines. In this study, our aim was to identify medicine characteristics associated with the long time gap between MA and guidance publication and explore the influencing factors.

Methods

We identified 116 single technology appraisals from 2016 to 2020 using publicly available data, and extracted information on the year of appraisal completion, application type, experiences of similar appraisals, orphan medicinal products (OMPs), cancer medicines, and accelerated assessment. Multiple regression analyses were performed to analyze the associations between the medicine characteristics and key time periods related to health technology assessment and MA processes.

Results

OMPs were associated with a long period between MA and guidance publication. Specifically, OMPs and cancer medicines were associated with slow guidance publication after the final scope (FS) development. However, there was no association between OMPs and the period between validation of MA application and FS development. Non-double-blinded randomized clinical trials and the use of comparators not specified in the FS were associated with slow guidance publication after the FS development.

Conclusions

Our results demonstrate that OMPs are associated with a longer period between MA and guidance publication by the NICE than non-OMPs; this may be attributed to the slow guidance publication after the FS development. These findings indicate the necessity to shorten the appraisal process for OMPs.

Keywords

Health technology assessmentNational Institute for Health and Care ExcellenceAccess to medicineOrphan medicinal product
Type
Assessment
Information
International Journal of Technology Assessment in Health Care , Volume 38 , Issue 1 , 2022 , e11
Copyright
Copyright © The Author(s), 2021. Published by Cambridge University Press

Access options

Get access to the full version of this content by using one of the access options below. (Log in options will check for institutional or personal access. Content may require purchase if you do not have access.)

References

National Institute for Health and Care Excellence [Internet] Single technology appraisal timeline. 2018 [cited 2021 Oct 28]. Available from: https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/technology-appraisal-guidance/process/sta-timeline.Google Scholar
Stoykova, B, Drummond, M, Barbieri, M, Kleijnen, J. The lag between effectiveness and cost-effectiveness evidence of new drugs. Implications for decision-making in health care. Eur J Health Econ. 2003;4:313–18.CrossRefGoogle ScholarPubMed
Mayor, S. NICE to issue faster guidance on use of drugs by NHS. BMJ. 2005;331:1101.CrossRefGoogle Scholar
Haycox, A. Does ‘NICE blight’ exist, and if so, why? Pharmacoeconomics. 2008;26:987–9.CrossRefGoogle ScholarPubMed
Kaltenthaler, E, Tappenden, P, Booth, A, Akehurst, R. Comparing methods for full versus single technology appraisal: A case study of docetaxel and paclitaxel for early breast cancer. Health Policy. 2008;87:389–400.CrossRefGoogle ScholarPubMed
National Institute for Health and Care Excellence [Internet] Technology appraisal recommendations. 2021 [cited 2021 Oct 28]. Available from: https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guidance/data/appraisal-recommendations.Google Scholar
Kaltenthaler, E, Papaioannou, D, Boland, A, Dickson, R. The national institute for health and clinical excellence single technology appraisal process: Lessons from the first 4 years. Value Health. 2011;14:1158–65.CrossRefGoogle ScholarPubMed
National Institute for Health and Care Excellence [Internet] Guide to the processes of technology appraisal. 2018 [cited 2021 Oct 28]. Available from: https://www.nice.org.uk/process/pmg19/chapter/foreword.Google Scholar
Casson, SG, Ruiz, FJ, Miners, A. How long has NICE taken to produce technology appraisal guidance? A retrospective study to estimate predictors of time to guidance. BMJ Open. 2013;3:e001870.CrossRefGoogle Scholar
Ford, JA, Waugh, N, Sharma, P, Sculpher, M, Walker, A. NICE guidance: A comparative study of the introduction of the single technology appraisal process and comparison with guidance from Scottish Medicines Consortium. BMJ Open. 2012;2:e000671.CrossRefGoogle ScholarPubMed
Barham, L. Single technology appraisals by NICE: Are they delivering faster guidance to the NHS? Pharmacoeconomics. 2008;26:1037–43.CrossRefGoogle ScholarPubMed
Department of Health and Social Care, UK [Internet] Voluntary scheme for branded medicines pricing and access. 2018 [cited 2021 Oct 28]. Available from: https://www.gov.uk/government/publications/voluntary-scheme-for-branded-medicines-pricing-and-access.Google Scholar
Connolly, E, O'Donnell, H, Lamrock, F, Tilson, L, Barry, M. Health technology assessment of drugs in Ireland: An analysis of timelines. Pharmacoecon Open. 2020;4:287–96.CrossRefGoogle ScholarPubMed
Sabry-Grant, C, Malottki, K, Diamantopoulos, A. The cancer drugs fund in practice and under the new framework. Pharmacoeconomics. 2019;37:953–62.CrossRefGoogle ScholarPubMed
Morrell, L, Wordsworth, S, Schuh, A, Middleton, MR, Rees, S, Barker, RW. Will the reformed cancer drugs fund address the most common types of uncertainty? An analysis of NICE cancer drug appraisals. BMC Health Serv Res. 2018;18:393.CrossRefGoogle ScholarPubMed
National Institute for Health and Care Excellence [Internet] Conditions and diseases. 2021 [cited 2021 Oct 28]. Available from: https://www.nice.org.uk/guidance/conditions-and-diseases.Google Scholar
National Institute for Health and Care Excellence [Internet] Ibrutinib for previously treated chronic lymphocytic leukaemia and untreated chronic lymphocytic leukaemia with 17p deletion or TP53 mutation. Technology appraisal guidance [TA429]. 2017 [cited 2021 Oct 28]. Available from: https://www.nice.org.uk/guidance/ta429.Google Scholar
Hettle, R, Corbett, M, Hinde, S, Hodgson, R, Jones-Diette, J, Woolacott, N, et al. The assessment and appraisal of regenerative medicines and cell therapy products: An exploration of methods for review, economic evaluation and appraisal. Health Technol Assess. 2017;21:1–204.CrossRefGoogle Scholar
Aballéa, S, Thokagevistk, K, Velikanova, R, Simoens, S, Annemans, L, Antonanzas, F, et al. Health economic evaluation of gene replacement therapies: Methodological issues and recommendations. J Mark Access Health Policy. 2020;8:1822666.CrossRefGoogle ScholarPubMed
Walton, M, Sharif, S, Simmonds, M, Claxton, L, Hodgson, R. Tisagenlecleucel for the treatment of relapsed or refractory B-cell acute lymphoblastic leukaemia in people aged up to 25 years: An evidence review group perspective of a NICE single technology appraisal. Pharmacoeconomics. 2019;37:1209–17.CrossRefGoogle ScholarPubMed
Kahan, BC, Cro, S, Doré, CJ, Bratton, DJ, Rehal, S, Maskell, NA, et al. Reducing bias in open-label trials where blinded outcome assessment is not feasible: Strategies from two randomised trials. Trials. 2014;15.CrossRefGoogle Scholar
Cox, E, Wade, R, Peron, M, Dietz, KC, Eastwood, A, Palmer, S, et al. The clinical and cost effectiveness of inotuzumab ozogamicin for the treatment of adult relapsed or refractory B-cell acute lymphoblastic leukaemia: An evidence review group evaluation of a NICE single technology appraisal. Pharmacoeconomics. 2019;37:1081–91.CrossRefGoogle ScholarPubMed
Thorlund, K, Zafari, Z, Druyts, E, Mills, EJ, Sadatsafavi, M. The impact of incorporating Bayesian network meta-analysis in cost-effectiveness analysis - A case study of pharmacotherapies for moderate to severe COPD. Cost Eff Resour Alloc. 2014;12:8.CrossRefGoogle ScholarPubMed
Rinciog, C, Watkins, M, Chang, S, Maher, TM, LeReun, C, Esser, D, et al. A cost-effectiveness analysis of nintedanib in idiopathic pulmonary fibrosis in the UK. Pharmacoeconomics. 2017;35:479–91.CrossRefGoogle ScholarPubMed
Ramaekers, BLT, Wolff, RF, Pouwels, X, Oosterhoff, M, Van Giessen, A, Worthy, G, et al. Ixekizumab for treating moderate-to-severe plaque psoriasis: An evidence review group perspective of a NICE single technology appraisal. Pharmacoeconomics. 2018;36:917–27.CrossRefGoogle ScholarPubMed
Supplementary material: File

Takada and Narukawa supplementary material

Takada and Narukawa supplementary material

Download Takada and Narukawa supplementary material(File)
File 119.3 KB