References
- Marsden G, Towse A, Pearson SD, et al. Gene therapy: understanding the science, assessing the evidence, and paying for value. ICER; 2017. Available from: https://www.ohe.org/publications/gene-therapy-understanding-science-assessing-evidence-and-paying-value
- ICER. Spinraza® and Zolgensma® for Spinal Muscular Atrophy: effectiveness and Value. Final Evidence Report; 2019. Available from: https://icer-review.org/material/sma-final-evidence-report/
- NICE. Talimogene laherparepvec for treating unresectable metastatic melanoma. Technology appraisal guidance [TA410]; 2016. Available from: https://www.nice.org.uk/guidance/ta410
- NICE. Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency.Highly specialised technologies guidance [HST7]; 2018. [cited 2020 Oct 20]. Available from: https://www.nice.org.uk/guidance/hst7
- CADTH. Tisagenlecleucel (Kymriah) for Pediatric Acute Lymphoblastic Leukemia and Diffuse Large B-Cell Lymphoma; 2019. Available from: https://www.cadth.ca/tisagenlecleucel-kymriah-pediatric-acute-lymphoblastic-leukemia-and-diffuse-large-b-cell-lymphoma
- CADTH. Tisagenlecleucel for Acute Lymphoblastic Leukemia: economic Review Report; 2019 [cited 2020 Nov 27]. Available from: https://cadth.ca/sites/default/files/pdf/car-t/op0538-tisagenlecleucel-economic-report-pALL-jan2019.pdf
- ICER. Chimeric Antigen Receptor T-Cell Therapy for B-Cell Cancers: effectiveness and Value. Final Evidence Report; 2018 [cited 2020 Oct 20]. Available from: https://icer-review.org/material/car-t-therapies-evidence-report/
- NICE. Tisagenlecleucel for treating relapsed or refractory B-cell acute lymphoblastic leukaemia in people aged up to 25 years. Technology appraisal guidance [TA554]; 2018 [cited 2020 Oct 20]. Available from: https://www.nice.org.uk/guidance/ta554
- NICE. Tisagenlecleucel for treating relapsed or refractory diffuse large B-cell lymphoma after 2 or more systemic therapies. Technology appraisal guidance [TA567]; 2019 [cited 2020 Oct 20]. Available from: https://www.nice.org.uk/guidance/ta567
- SMC. tisagenlecleucel (Kymriah) - SMC2200; 2019 [cited 2020 Oct 20]. Available from: https://www.scottishmedicines.org.uk/medicines-advice/tisagenlecleucel-kymriah-resubmission-smc2200/
- SMC. voretigene neparvovec (Luxturna) - SMC2129; 2019 [cited 2020 Oct 20]. Available from: https://www.scottishmedicines.org.uk/medicines-advice/tisagenlecleucel-kymriah-fullsubmission-smc2129/
- CADTH. Axicabtagene Ciloleucel for adults with relapsed or refractory large B-cell lymphoma; 2019 [cited 2020 Oct 20]. Available from: https://www.cadth.ca/axicabtagene-ciloleucel-adults-relapsed-or-refractory-large-b-cell-lymphoma
- NICE. Axicabtagene ciloleucel for treating diffuse large B-cell lymphoma and primary mediastinal large B-cell lymphoma after 2 or more systemic therapies. Technology appraisal guidance [TA559]; 2019. Available from: https://www.nice.org.uk/guidance/ta559
- SMC. axicabtagene ciloleucel (Yescarta®) - SMC2189; 2019 [cited 2020 Oct 20]. Available from: https://www.scottishmedicines.org.uk/medicines-advice/axicabtagene-ciloleucel-yescarta-resubmission-smc2189/
- ICER. Voretigene Neparvovec for Biallelic RPE65-Mediated retinal disease: effectiveness and value. Final Evidence Report; 2018 [cited 2020 Oct 20]. Available from: https://icer-review.org/material/voretigene-evidence-report/
- NICE. Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations. Highly specialised technologies guidance [HST11]; 2019 [cited 2020 Oct 20]. Available from: https://www.nice.org.uk/guidance/hst11
- SMC. voretigene neparvovec (Luxturna) - SMC2228; 2019 [cited 2020 Oct 20]. Available from: https://www.scottishmedicines.org.uk/medicines-advice/voretigene-neparvovec-luxturna-uoia-smc2228/
- HAS. LUXTURNA (voretigène néparvovec); 2019 [cited 2020 Oct 20]. Available from: https://www.has-sante.fr/jcms/c_2964759/fr/luxturna
- NICE. Appraising life-extending, end of life treatments; 2009 [cited 2020 Oct 20]. Available from: https://www.nice.org.uk/guidance/GID-TAG387/documents/appraising-life-extending-end-of-life-treatments-paper2
- SMC. Assessment of medicines for end of life care and very rare conditions (orphan and ultra-orphan medicines) in Scotland; 2013 [cited 2020 Oct 20]. Available from: ssessment of medicines for end of life care and very rare conditions (orphan and ultra-orphan medicines) in Scotland
- Gardner J ICER draws new gene therapy pricing framework; 2019.
- ICER. Modifications to the ICER value assessment framework for treatments for ultra-rare diseases; 2020 [cited 2020 Oct 20]. Available from: https://34eyj51jerf417itp82ufdoe-wpengine.netdna-ssl.com/wp-content/uploads/2020/10/ICER_URD_Framework_Adapt_013120.pdf
- CADTH. CADTH to Evaluate CAR T-Cell Therapies; 2018 [cited 2020 Nov 27]. Available from: https://www.cadth.ca/news/cadth-evaluate-car-t-cell-therapies-0
- ICER. 2020-2023 value assessment framework; 2020 [cited 2020 Oct 20]. Available from: https://icer.org/wp-content/uploads/2020/10/ICER_2020_2023_VAF_102220.pdf
- Drummond MF, Neumann PJ, Sullivan SD, et al. Analytic considerations in applying a general economic evaluation reference case to gene therapy. Value Health. 2019;22(6):661–18. eng
- Jönsson B, Hampson G, Michaels J, et al. Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare. Eur J Health Econ. 2019;20(3):427–438. eng
- ICER. Value assessment methods and pricing recommendations for potential cures; 2019 [cited 2020 Oct 20]. Available from: https://icer.org/wp-content/uploads/2020/10/ICER_SST_Report_Response_to_Comments_111219.pdf