Walker S, Sculpher M, Claxton K, Palmer S. Coverage with evidence development, only in research, risk sharing, or patient access scheme? A framework for coverage decisions. Value Health. 2012;15:570–9.

Article  PubMed  Google Scholar 

Martelli N, van den Brink H, Borget I. New French coverage with evidence development for innovative medical devices: improvements and unresolved issues. Value Health. 2016;19:17–9.

Article  PubMed  Google Scholar 

Hutton J, Trueman P, Henshall C. Coverage with evidence development: an examination of conceptual and policy issues. Int J Technol Assess Health Care. 2007;23:425–32.

Article  PubMed  Google Scholar 

Reckers-Droog V, Federici C, Brouwer W, Drummond M. Challenges with coverage with evidence development schemes for medical devices: a systematic review. Health Policy Technol. 2020;9:146–56.

Article  Google Scholar 

Levin L, Goeree R, Levine M, Krahn M, Easty T, Brown A, et al. Coverage with evidence development: the Ontario experience. Int J Technol Assess Health Care. 2011;27:159–68.

Article  PubMed  Google Scholar 

Lu CY, Lupton C, Rakowsky S, Babar ZUD, Ross-Degnan D, Wagner AK. Patient access schemes in Asia-pacific markets: current experience and future potential. J Pharm Policy Pract. 2015. https://doi.org/10.1186/s40545-014-0019-x.

Article  PubMed  PubMed Central  Google Scholar 

Zampirolli Dias C, Godman B, Peres Gargano L, Santos Azevedo P, Morgado Garcia M, Souza Cazarim M, et al. Integrative review of managed entry agreements: chances and limitations. Pharmacoeconomics. 2020;38:1165–85.

Article  PubMed  Google Scholar 

Walker S, Sculpher M, Claxton K, Palmer S. Coverage with evidence development, only in research, risk sharing or patient access scheme? | Centre for Health Economics—The University of York [Research paper 77]. 2012. https://pure.york.ac.uk/portal/en/publications/coverage-with-evidence-development-only-in-research-risk-sharing-or-patient-access-scheme(47787ac9-d258-42e0-89a0-dc19b52939d2).html. Accessed 2020 Jul 14.

NICE. Types of technology appraisal recommendation—National Institute for Health and Care Excellence. https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guidance/types-of-recommendation. Accessed 22 Jun 2020.

NHS. Cancer drug fund list. What has changed in the CDF? National Health System England. https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guidance/cancer-drugs-fund. Accessed 2020 Jun 22.

Trueman P, Grainger DL, Downs KE. Coverage with evidence development: applications and issues. Int J Technol Assess Health Care. 2010;26:79–85.

Article  PubMed  Google Scholar 

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. https://doi.org/10.1186/s12913-018-3162-2.

Article  PubMed  PubMed Central  Google Scholar 

Kang J, Cairns J. “Don’t think twice, it’s all right”: Using additional data to reduce uncertainty regarding oncologic drugs provided through managed access agreements in England. PharmacoEconomics Open. 2022;7:77–91.

Article  PubMed  PubMed Central  Google Scholar 

Kang J, Cairns J. Exploring uncertainty and use of real-world data in the national institute for health and care excellence single technology appraisals of targeted cancer therapy. BMC Cancer. 2022. https://doi.org/10.1186/s12885-022-10350-8.

Article  PubMed  PubMed Central  Google Scholar 

Hall PS, Edlin R, Kharroubi S, Gregory W, McCabe C. Expected net present value of sample information. Med Decis Mak. 2012;32:E11-21.

Article  Google Scholar 

Charlton V, Lomas J, Mitchell P. NICE’s new methods: putting innovation first, but at what cost? BMJ. 2022. https://doi.org/10.1136/bmj-2022-071974.

Article  PubMed  Google Scholar 

Davis C, Naci H, Gurpinar E, Poplavska E, Pinto A, Aggarwal A. Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009–13. BMJ. 2017;359: j4530. https://doi.org/10.1136/bmj.j4530.

Article  PubMed  PubMed Central  Google Scholar 

NICE. Technology appraisal data: appraisal recommendations—National Institute for Health and Care Excellence. https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guidance/data/appraisal-recommendations. Accessed 8 Jan 2021.

NICE. Guidance programme: highly specialized technologies—National Institute for Health and Care Excellence. https://www.nice.org.uk/guidance/published?ndt=Guidance&ngt=Highly%20specialized%20technologies%20guidance&ps=9999. Accessed 30 Oct 2023

Higgins JPT, Altman DG, Gotzsche PC, Juni P, Moher D, Oxman AD, et al. The cochrane collaborations tool for assessing risk of bias in randomised trials. BMJ. 2011;343:d5928–38.

Article  PubMed  PubMed Central  Google Scholar 

Sterne JA, Hernán MA, Reeves BC, Savović J, Berkman ND, Viswanathan M, et al. ROBINS-I: a tool for assessing risk of bias in non-randomised studies of interventions. BMJ. 2016. https://doi.org/10.1136/bmj.i4919.

Article  PubMed  PubMed Central  Google Scholar 

Cunningham D, Zalcberg JR, Rath U, Oliver I, van Cutsem E, Svensson C, et al. Final results of a randomised trial comparing “tomudex” (raltitrexed) with 5-fluorouracil plus leucovorin in advanced colorectal cancer. Ann Oncol. 1996;7:961–5.

Article  CAS  PubMed  Google Scholar 

Cocconi G, Cunningham D, Van Cutsem E, Francois E, Gustavsson B, van Hazel G, et al. Open, randomized, multicenter trial of raltitrexed versus fluorouracil plus high-dose leucovorin in patients with advanced colorectal cancer. Tomudex colorectal cancer study group. J Clin Oncol. 1998;16:2943–52.

Article  CAS  PubMed  Google Scholar 

Maughan T, James R, Kerr D, Ledermann J, McArdle C, Seymour M, et al. Comparison of survival, palliation, and quality of life with three chemotherapy regimens in metastatic colorectal cancer: a multicentre randomised trial. Lancet. 2002;359:1555–63.

Article  CAS  PubMed  Google Scholar 

Nuki G, Bresnihan B, Bear MB. Long-term safety and maintenance of clinical improvement following treatment with anakinra (recombinant human interleukin-1 receptor antagonist) in patients with rheumatoid arthritis: extension phase of a randomized, double-blind, placebo-controlled trial. Arthritis Rheum. 2002;46:2838–46.

Article  CAS  PubMed  Google Scholar 

Cohen SB, Woolley JM, Chan W. Interleukin 1 receptor antagonist anakinra improves functional status in patients with rheumatoid arthritis. J Rheumatol. 2003;30:225–31.

CAS  PubMed  Google Scholar 

Genovese MC, Cohen S, Moreland L, Lium D, Robbins S, Newmark R, et al. Combination therapy with etanercept and anakinra in the treatment of patients with rheumatoid arthritis who have been treated unsuccessfully with methotrexate. Arthritis Rheum. 2004;50:1412–9.

Article  CAS  PubMed  Google Scholar 

Bresnihan B, Newmark R, Robbins S, Genant HK. Effects of anakinra monotherapy on joint damage in patients with rheumatoid arthritis. Extension of a 24-week randomized, placebo-controlled trial. J Rheumatol. 2004;31:1103–11.

CAS  PubMed  Google Scholar 

Popov I, Carrato A, Sobrero A, Vincent M, Kerr D, Labianca R, et al. Raltitrexed (Tomudex) versus standard leucovorin-modulated bolus 5-fluorouracil: results from the randomised phase III Pan-European Trial in Adjuvant Colon Cancer 01 (PETACC-1). Eur J Cancer. 2008;44:2204–11.

Article  CAS  PubMed  Google Scholar 

Feliu J, Castañón C, Salud A, Mel JR, Escudero P, Pelegrín A, et al. Phase II randomised trial of raltitrexed-oxaliplatin vs raltitrexed-irinotecan as first-line treatment in advanced colorectal cancer. Br J Cancer. 2005;93:1230–5.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Feliu J, Salud A, Escudero P, López-Gómez L, Pericay C, Castañón C, et al. Irinotecan plus raltitrexed as first-line treatment in advanced colorectal cancer: a phase II study. Br J Cancer. 2004;90:1502–7.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Cortinovis D, Bajetta E, Di Bartolomeo M, Dognini G, Beretta E, Ferrario E, et al. Raltitrexed plus oxaliplatin in the treatment of metastatic colorectal cancer. Tumori. 2004;90:186–91.

Article  CAS  PubMed  Google Scholar 

Laudani A, Gebbia V, Leonardi V, Savio G, Borsellino N, Cusimano MP, et al. Activity and toxicity of oxaliplatin plus raltitrexed in 5-fluorouracil refractory metastatic colorectal adeno-carcinoma. Anticancer Res. 2004;24:1139–42.

CAS  PubMed  Google Scholar 

Santini D, Massacesi C, DAngelillo RM, Marcucci F, Campisi C, Vincenzi B, et al. Raltitrexed plus weekly oxaliplatin as first-line chemotherapy in metastatic colorectal cancer: a multicenter non-randomized phase II study. Med Oncol. 2004;21:59–66.

Article  CAS  PubMed