References
- Marabelle A, Le DT, Ascierto PA, et al. Efficacy of pembrolizumab in patients with noncolorectal high microsatellite instability/mismatch repair-deficient cancer: results from the phase II KEYNOTE-158 study. J Clin Oncol. 2019;38(1):1–10. doi: 10.1200/jco.19.02105.
- Thibodeau SN, Bren G, Schaid D. Microsatellite instability in cancer of the proximal colon. Science. 1993;260(5109):816–819. doi: 10.1126/science.8484122.
- Cancer Genome Atlas Network. Comprehensive molecular characterization of human colon and rectal cancer. Nature. 2012;487(7407):330–337. doi: 10.1038/nature11252.
- Eso Y, Shimizu T, Takeda H, et al. Microsatellite instability and immune checkpoint inhibitors: toward precision medicine against gastrointestinal and hepatobiliary cancers. J Gastroenterol. 2020;55(1):15–26. doi: 10.1007/s00535-019-01620-7.
- Lorenzi M, Amonkar M, Zhang J, et al. Epidemiology of microsatellite instability high (MSI-H) and deficient mismatch repair (dMMR) in solid tumors: a structured literature review. J Oncol. 2020;2020:1–17. doi: 10.1155/2020/1807929.
- Le DT, Durham JN, Smith KN, et al. Mismatch repair deficiency predicts response of solid tumors to PD-1 blockade. Science (New York, NY). 2017;357(6349):409–413.
- Venderbosch S, Nagtegaal ID, Maughan TS, et al. Mismatch repair status and BRAF mutation status in metastatic colorectal cancer patients: a pooled analysis of the CAIRO, CAIRO2, COIN and FOCUS studies. Clin Cancer Res. 2014;20(20):5322–5330. doi: 10.1158/1078-0432.CCR-14-0332.
- Li K, Luo H, Huang L, et al. Microsatellite instability: a review of what the oncologist should know. Cancer Cell Int. 2020;20(1):16. doi: 10.1186/s12935-019-1091-8.
- Oaknin A, Bosse TJ, Creutzberg CL, et al. Endometrial cancer: ESMO clinical practice guideline for diagnosis, treatment and follow-up. Ann Oncol. 2022;33(9):860–877. doi: 10.1016/j.annonc.2022.05.009.
- Lordick F, Carneiro F, Cascinu S, et al. Gastric cancer: ESMO clinical practice guideline for diagnosis, treatment and follow-up. Ann Oncol. 2022;33(10):1005–1020. doi: 10.1016/j.annonc.2022.07.004.
- Benson AB, Venook AP, Al-Hawary MM, et al. Small bowel adenocarcinoma, version 1.2020, NCCN clinical practice guidelines in oncology. J Natl Compr Canc Netw. 2019;17(9):1109–1133. doi: 10.6004/jnccn.2019.0043.
- Vogel A, Bridgewater J, Edeline J, et al. Biliary tract cancer: ESMO clinical practice guideline for diagnosis, treatment and follow-up⋆. Ann Oncol. 2022;34(2):127–140. doi: 10.1016/j.annonc.2022.10.506.
- Benson AB, Venook AP, Al-Hawary MM, et al. Colon cancer, version 2.2021, NCCN clinical practice guidelines in oncology. J Natl Compr Canc Netw. 2021;19(3):329–359. doi: 10.6004/jnccn.2021.0012.
- U.S. Food and Drug Administration. FDA grants accelerated approval to pembrolizumab for first tissue/site agnostic indication. 2017 [2023 Jul 21]. Available from: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-pembrolizumab-first-tissuesite-agnostic-indication
- Le DT, Kim TW, Van Cutsem E, et al. Phase II open-label study of pembrolizumab in treatment-refractory, microsatellite instability-high/mismatch repair-deficient metastatic colorectal cancer: KEYNOTE-164. J Clin Oncol. 2019;38(1):11–19. doi: 10.1200/jco.19.02107.
- Eisenhauer E, Therasse P, Bogaerts J, et al. New response evaluation criteria in solid tumours: revised RECIST guideline (version 1.1). Eur J Cancer. 2009;45(2):228–247. doi: 10.1016/j.ejca.2008.10.026.
- National Institute for Health and Care Excellence (NICE). Entrectinib for treating NTRK fusion-positive solid tumours. Technology appraisal guidance [TA644]; 2020 [2023 Jun 26]. Available from: https://www.nice.org.uk/guidance/ta644
- National Institute for Health and Care Excellence (NICE). Larotrectinib for treating NTRK fusion-positive advanced solid tumours. Technology appraisal guidance [TA630]; 2020 [2023 Jun 26]. Available from: https://www.nice.org.uk/guidance/ta630
- Orfanos P, Wong W, Bennett I. PCN94 challenges and an approach to performing cost-effectiveness analyses based on single arm basket trials for pan-tumor indications. Value Health. 2019;22:S73. doi: 10.1016/j.jval.2019.04.218.
- Briggs A, Beth W, Jennifer GG, et al. Comparison of alternative methods to assess the cost-effectiveness of tumor-agnostic therapies: a triangulation approach using larotrectinib as a case study. Value Health. 2022;25(6):1002–1009. doi: 10.1016/j.jval.2021.11.1354.
- Murphy P, Glynn D, Dias S, et al. Modelling approaches for histology-independent cancer drugs to inform NICE appraisals. Health Technol Assess. 2021;25(76):1–228. doi: 10.3310/hta25760.
- National Institute for Health and Care Excellence (NICE). The NICE methods of health technology evaluation: the case for change. 2020 [2023 Jun 28]. Available from: https://www.nice.org.uk/Media/Default/About/what-we-do/our-programmes/nice-guidance/chte-methods-consultation/NICE-methods-of-health-technology-evaluation-case-for-change.docx
- Aguiar-Ibáñez R, Hardern C, van Hees F, et al. Cost-effectiveness of pembrolizumab for the first-line treatment of patients with unresectable or metastatic MSI-H/dMMR colorectal cancer in the United States. J Med Econ. 2022;25(1):469–480. doi: 10.1080/13696998.2022.2043634.
- Thurgar E, Gouldson M, Matthijsse S, et al. Cost-effectiveness of pembrolizumab compared with chemotherapy in the US for women with previously treated deficient mismatch repair or high microsatellite instability unresectable or metastatic endometrial cancer. J Med Econ. 2021;24(1):675–688. doi: 10.1080/13696998.2021.1917140.
- National Institute for Health and Care Excellence (NICE). NICE health technology evaluations: the manual. 2022 [2023 Jun 26]. Available from: https://www.nice.org.uk/process/pmg9/chapter/the-reference-case
- Cancer Research UK. Bowel cancer incidence by sex and UK region. 2018 [2022 Nov 22]. Available from: https://www.cancerresearchuk.org/health-professional/cancer-statistics/statistics-by-cancer-type/bowel-cancer/incidence#heading-Zero
- Cancer Research UK. Uterine cancer incidence by sex and UK region. 2018 [2022 Nov 22]. Available from: https://www.cancerresearchuk.org/health-professional/cancer-statistics/statistics-by-cancer-type/uterine-cancer/incidence#heading-Three
- Cancer Research UK. Stomach cancer incidence. 2018 [2022 Nov 22]. Available from: https://www.cancerresearchuk.org/health-professional/cancer-statistics/statistics-by-cancer-type/stomach-cancer#heading-Zero
- Cancer Research UK. Small intestine cancer incidence. 2018 [2022 Nov 22]. Available from: https://www.cancerresearchuk.org/health-professional/cancer-statistics/statistics-by-cancer-type/small-intestine-cancer#heading-Zero
- National Institute for Health and Care Excellence (NICE). Pemigatinib for treating relapsed or refractory advanced cholangiocarcinoma with FGFR2 fusion or rearrangement. 2021 [2022 Jan 19]. Available from: https://www.nice.org.uk/guidance/ta722
- MSD. Microsatellite instability-high advisory board meeting. Data on file. 2022.
- MSD BV. Keytruda (pembrolizumab). Summary of product characteristics. 2020 [2023 Jun 26]. Available from: https://www.ema.europa.eu/en/documents/product-information/keytruda-epar-product-information_en.pdf
- Guyot P, Ades AE, Ouwens MJNM, et al. Enhanced secondary analysis of survival data: reconstructing the data from published Kaplan-Meier survival curves. BMC Med Res Methodol. 2012;12(1):9. doi: 10.1186/1471-2288-12-9.
- Combescure C, Foucher Y, Jackson D. Meta-analysis of single-arm survival studies: a distribution-free approach for estimating summary survival curves with random effects. Stat Med. 2014;33(15):2521–2537. doi: 10.1002/sim.6111.
- Hernández Alava M, Pudney S, Wailoo A. Estimating EQ-5D by age and sex for the UK. NICE DSU Report; 2022 [2023 Jul 20]. Available from: https://www.sheffield.ac.uk/sites/default/files/2022-02/DSU%20Age%20based%20utility%20-%20Final%20for%20website.pdf
- Grothey A, Cutsem EV, Sobrero A, et al. Regorafenib monotherapy for previously treated metastatic colorectal cancer (CORRECT): an international, multicentre, randomised, placebo-controlled, phase 3 trial. Lancet. 2013;381(9863):303–312. doi: 10.1016/S0140-6736(12)61900-X.
- Monthly Index of Medical Specialities (MIMS). MIMS. [cited 2022 Oct 20]. Available from. https://www.mims.co.uk/
- Department of Health and Social Care. Drugs and pharmaceutical electronic market information tool (eMIT). 2021. Available from: https://www.gov.uk/government/publications/drugs-and-pharmaceutical-electronic-market-information-emit
- National Health Service. 2020/21 National Cost Collection Data Publication. 2022 [2023 Jun 28]. Available from: https://www.england.nhs.uk/publication/2020-21-national-cost-collection-data-publication/
- Unit Costs of Health and Social Care 2019, PSSRU. 2019. Available from: https://www.pssru.ac.uk/pub/uc/uc2019/sources-of-information.pdf
- National Institute for Health and Care Excellence (NICE). Diagnostics guidance [DG27]: molecular testing strategies for Lynch syndrome in people with colorectal cancer. 2017 [2023 Mar 22]. Available from: https://www.nice.org.uk/guidance/dg27/informationforpublic
- Fujiyoshi K, Yamamoto G, Takenoya T, et al. Metastatic pattern of stage IV colorectal cancer with high-frequency microsatellite instability as a prognostic factor. Anticancer Res. 2017;37(1):239–247. doi: 10.21873/anticanres.11313.
- Le DT, Uram JN, Wang H, et al. PD-1 blockade in tumors with mismatch-repair deficiency. N Engl J Med. 2015;372(26):2509–2520. doi: 10.1056/NEJMoa1500596.
- Round J, Jones L, Morris S. Estimating the cost of caring for people with cancer at the end of life: a modelling study. Palliat Med. 2015;29(10):899–907. doi: 10.1177/0269216315595203.
- National Institute for Health and Care Excellence (NICE). TA488: regorafenib for previously treated unresectable or metastatic gastrointestinal stromal tumours. 2017 [2021 Jan 12]. Available from: https://www.nice.org.uk/guidance/ta488/
- Schneider P, McNamara S, Love-Koh J, Doran T, Gutacker N. QALY Shortfall Calculator. 2021 [2023 Jun 26]. Available from: https://shiny.york.ac.uk/shortfall/
- Bellone M, Zaniolo O, Martinelli E, et al. PCN147 entrectinib in NTRK tumor agnostic indication compared to different standard of care in various tumors types: a cost-effectiveness analysis in Italian patients. Value Health. 2019;22:S464. doi: 10.1016/j.jval.2019.09.343.
- Michels RE, Arteaga CH, Peters ML, et al. Economic evaluation of a tumour-agnostic therapy: Dutch economic value of larotrectinib in TRK fusion-positive cancers. Appl Health Econ Health Policy. 2022;20(5):717–729. doi: 10.1007/s40258-022-00740-1.
- Spiegelhalter DJ, Abrams KR, Myles J. Bayesian approaches to clinical trials and health-care evaluation. New York (NY): Wiley; 2004.
- Thall PF, Wathen JK, Bekele BN, et al. Hierarchical Bayesian approaches to phase II trials in diseases with multiple subtypes. Stat Med. 2003;22(5):763–780. doi: 10.1002/sim.1399.