Обзор зарубежных подходов к оценке и финансированию агностических лекарственных препаратов для лечения злокачественных новообразований
Загрузок: 162
Просмотров: 196
pdf

Ключевые слова

агностические лекарственные препараты
злокачественные новообразования
зарубежный опыт
клинические рекомендации
регистрация лекарственных препаратов
оценка технологий здравоохранения

Как цитировать

Омельяновский, В. В., Никитина, А. В., Рыжова, О. Р., Мухортова, П. А., & Струнина, Ю. В. (2023). Обзор зарубежных подходов к оценке и финансированию агностических лекарственных препаратов для лечения злокачественных новообразований. Вопросы онкологии, 69(5), 805–814. https://doi.org/10.37469/0507-3758-2023-69-5-805-814

Аннотация

В связи с растущей заболеваемостью и распространенностью злокачественных новообразований (ЗНО) на территории РФ появление новых медицинских технологий является важной составляющей борьбы с данными заболеваниями. Одной из новых разработок для лечения таких заболеваний являются агностические препараты, уникальность которых связана с механизмом действия, направленным на лечение ЗНО независимо от локализации опухоли при наличии определенного биомаркера. В настоящее время существует 3 агностических лекарственных препарата (ЛП) — энтректиниб, ларотректиниб, достарлимаб. Подавляющая часть клинических исследований агностических ЛП является когортными несравнительными, что обуславливает ряд проблем при рассмотрении данных ЛП к регистрации и возмещению как в РФ, так и в зарубежных странах. Несмотря на данное ограничение, рассматриваемые ЛП зарегистрированы или чаще условно зарегистрированы в зарубежных странах (США, Канада, страны Европейского Союза, Австралия, Япония). Агностические ЛП включены в зарубежные клинические рекомендации (КР) и финансируются за счет бюджетных средств в ряде зарубежных стран, несмотря на ограниченные данные по клинической эффективности. Данная тенденция обусловлена необходимостью удовлетворить медицинские потребности в терапии заболеваний, являющихся жизнеугрожающими или приводящими к фатальным последствиям для пациента.

https://doi.org/10.37469/0507-3758-2023-69-5-805-814
Загрузок: 162
Просмотров: 196
pdf

Библиографические ссылки

Под ред. А.Д. Каприна, В.В. Старинского, А.О. Шахзадовой Злокачественные новообразования в России в 2021 году (заболеваемость и смертность). М.: МНИОИ им. П.А. Герцена − филиал ФГБУ «НМИЦ радиологии» Минздрава России. 2022:252 [Kaprin AD, Starinsky VV, Shakhzadova AO, editors. Malignant neoplasms in Russia in 2021 (morbidity and mortality). Moscow:P.A. Herzen MNIOI - branch of FGBU «NMRC Radiology» of the Ministry of Health of Russia. 2022:252 (In Russ.)].

Prescribing information ROZLYTREK (entrectinib) capsules, for oral use [Internet]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/212725s000lbl.pdf (accessed: 01.08.2022).

Государственный реестр лекарственных средств: Витракви® [Internet]. Available from: https://grls.rosminzdrav.ru/Grls_View_v2.aspx?routingGuid=f44bb994-1a9c-4c49-ae30-80ca9bdc21b4 (accessed: 01.08.2022).

Prescribing information JEMPERLI (dostarlimab-gxly) injection, for intravenous use [Internet]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/761174s002lbl.pdf (accessed: 01.08.2022).

Gronchi A, Miah AB, Dei Tos, et al. Soft tissue and visceral sarcomas: ESMO-EURACAN-GENTURIS Clinical Practice Guidelines for diagnosis, treatment and follow-up☆. Annals of Oncology. 2021;32(11):1348-65. http://dx.doi.org/10.1016/j.annonc.2021.07.006.

Casali PG, Blay JY, Abecassis N, et al. Gastrointestinal stromal tumours: ESMO-EURACAN-GENTURIS Clinical Practice Guidelines for diagnosis, treatment and follow-up. Annals of Oncology. 2022;33(1):20-33. http://dx.doi.org/10.1016/j.annonc.2021.09.005.

Filetti S, Durante C, Hartl DM, et al. ESMO Clinical Practice Guideline update on the use of systemic therapy in advanced thyroid cancer. Annals of Oncology. 2022;33(7):674-84. http://dx.doi.org/10.1016/j.annonc.2022.04.009.

Hendriks LE, Kerr KM, Menis J, et al. Oncogene-addicted metastatic non-small-cell lung cancer: ESMO Clinical Practice Guideline for diagnosis, treatment and follow-up. Annals of Oncology [Internet]. 2023;34(4):339-57. http://dx.doi.org/10.1016/j.annonc.2022.12.009. Available from: https://www.esmo.org/guidelines/guidelines-by-topic/lung-and-chest-tumours/clinical-practice-living-guidelines-metastatic-non-small-cell-lung-cance (accessed: 01.08.2022).

Gajjar A, Mahajan A, Abdelbaki M, et al. Pediatric central nervous system cancers, Version 2.2023, NCCN Clinical Practice Guidelines in Oncology [Internet]. J Natl Compr Canc Netw. 2022;20(12):1339-1362. Available from: https://www.nccn.org/professionals/physician_gls/pdf/ped_cns.pdf (accessed: 01.08.2022).

von Mehren M, Kane JM, Riedel RF, et al. NCCN Guidelines® Insights: Gastrointestinal Stromal Tumors, Version 1.2022 [Internet]. J Natl Compr Canc Netw. 2022;20(11):1204-14. http://dx.doi.org/10.6004/jnccn.2022.0058. Available from: https://www.nccn.org/professionals/physician_gls/pdf/gist.pdf (accessed: 01.08.2022).

Swetter SM, Johnson D, Thompson JA, et al. NCCN Guidelines® Insights: Melanoma: Cutaneous, Version 2.2023. J Natl Compr Canc Netw [Internet]. 2022;231. Available from: https://www.nccn.org/professionals/physician_gls/pdf/cutaneous_melanoma.pdf (accessed: 01.08.2022).

Ettinger DS, Stevenson MM, Ahn D, et al. NCCN Guidelines® Insights: Occult Primary (Cancer of Unknown Primary [CUP]). Version 3.2023 [Internet]. 2022;76. Available from: https://www.nccn.org/professionals/physician_gls/pdf/occult.pdf (accessed: 01.08.2022).

Kumar ShK, Callander NS, Adekola K, et al. Breast Cancer, Version 4.2022, NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Canc Netw. 2021;107.

Ajani JA, D'Amico TA, Bentrem DJ, et al. Esophageal and Esophagogastric Junction Cancers, Version 3.2023, NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Canc Netw. 2023;172 [Internet]. Available from: https://www.nccn.org/professionals/physician_gls/pdf/esophageal.pdf (accessed: 01.08.2022).

Pfister DG, Spencer Sh, Adkins D, et al. Head and Neck Cancers, Version 2.2023, NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Canc Netw. 2023;250 [Internet]. Available from: https://www.nccn.org/professionals/physician_gls/pdf/head-and-neck.pdf (accessed: 01.08.2022).

Benson AB, Venook AP, Al-Hawary MM, et al. Small bowel adenocarcinoma, Version 1.2022, NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Canc Netw. 2023;54 [Internet]. Available from: https://www.nccn.org/professionals/physician_gls/pdf/small_bowel.pdf (accessed: 01.08.2022).

Abu-Rustum NR, Yashar CM, Arend R, et al. Uterine Neoplasms, Version 3.2021, NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Canc Netw. 2021;19(8):888-895 [Internet]. https://doi.org/10.6004/jnccn.2021.0038. Available from: https://www.nccn.org/professionals/physician_gls/pdf/uterine.pdf (accessed: 01.08.2022).

Ajani JA, D'Amico TA, Bentrem DJ, et al. Gastric Cancer, Version 2.2022, NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Canc Netw. 2022;20(2):167-192 [Internet]. https://doi.org/10.6004/jnccn.2022.0008. Available from: https://www.nccn.org/professionals/physician_gls/pdf/gastric.pdf (accessed: 01.08.2022).

Go RS, Jacobsen E, Baiocchi R, et al. Histiocytic Neoplasms, Version 2.2022, NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Canc Netw. 2022 [Internet]. Available from: https://www.nccn.org/professionals/physician_gls/pdf/histiocytic_neoplasms.pdf (accessed: 01.08.2022).

Ettinger DS, Wood DE, Aisner DL, et al. Non-Small Cell Lung Cancer, Version 3.2022. NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Canc Netw. 2022 [Internet]. Available from: https://www.nccn.org/professionals/physician_gls/pdf/nscl.pdf (accessed: 01.08.2022).

Benson AB, Venook AP, Al-Hawary, et al. Rectal Cancer, Version 1.2022, NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Canc Netw. 2022 [Internet]. https://doi.org/10.6004/jnccn.2022.0051. Available from: https://www.nccn.org/professionals/physician_gls/pdf/rectal.pdf (accessed: 01.08.2022).

Benson AB, Venook AP, Al-Hawary, et al. Colon Cancer, Version 1.2022, NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Canc Netw. 2022 [Internet]. Available from: https://www.nccn.org/professionals/physician_gls/pdf/colon.pdf (accessed: 01.08.2022).

Garcia-Foncillas J, Bokemeyer C, Italiano A, et al. Indirect treatment comparison of Larotrectinib versus Entrectinib in treating patients with TRK gene fusion cancers. Cancers (Basel). 2022;14(7):1793. https://doi.org/10.3390/cancers14071793.

Pollack M, Keating K, Wissinger E, et al. Transforming approaches to treating TRK fusion cancer: historical comparison of larotrectinib and histology-specific therapies. Curr Med Res Opin. 2021;37(1):59-70. https://doi.org/10.1080/03007995.2020.1847057.

Chu P, Antoniou M, Bhutani MK, et al. Matching-adjusted indirect comparison: entrectinib versus crizotinib in ROS1 fusion-positive non-small cell lung cancer. J Comp Eff Res. 2020;9(12):861-876. https://doi.org/10.2217/cer-2020-0063.

Doebele RC, Perez L, Trinh H, et al. Comparative effectiveness analysis between entrectinib clinical trial and crizotinib real-world data in ROS1+ NSCLC. J Comp Eff Res. 2021;10(17):1271-1282. https://doi.org/10.2217/cer-2021-0131.

Krebs MG, Blay JY, Le Tourneau C, et al. Intrapatient comparisons of efficacy in a single-arm trial of entrectinib in tumour-agnostic indications. ESMO Open. 2021;6(2):100072. https://doi.org/10.1016/j.esmoop.2021.100072.

Oaknin A, Gilbert L, Tinker AV, et al. Safety and antitumor activity of dostarlimab in patients with advanced or recurrent DNA mismatch repair deficient/microsatellite instability-high (dMMR/MSI-H) or proficient/stable (MMRp/MSS) endometrial cancer: interim results from GARNET-a phase I, single-arm study. J Immunother Cancer. 2022;10(1):e003777. https://doi.org/10.1136/jitc-2021-003777.

Patnaik A, Weiss GJ, Rasco DW, et al. Safety, antitumor activity, and pharmacokinetics of dostarlimab, an anti-PD-1, in patients with advanced solid tumors: a dose-escalation phase 1 trial. Cancer Chemother Pharmacol. 2022;89(1):93-103. https://doi.org/10.1007/s00280-021-04358-3.

Drilon A, Tan DSW, Lassen UN, et al. Efficacy and safety of Larotrectinib in patients with tropomyosin receptor kinase fusion-positive lung cancers. JCO Precis Oncol. 2022;6:e2100418. https://doi.org/10.1200/PO.21.00418.

Hong DS, Bauer TM, Lee JJ, et al. Larotrectinib in adult patients with solid tumours: a multi-centre, open-label, phase I dose-escalation study. Ann Oncol. 2019;30(2):325-331. https://doi.org/10.1093/annonc/mdy539.

Laetsch TW, DuBois SG, Mascarenhas L, et al. Larotrectinib for paediatric solid tumours harbouring NTRK gene fusions: phase 1 results from a multicentre, open-label, phase 1/2 study. Lancet Oncol. 2018;19(5):705-714. https://doi.org/10.1016/S1470-2045(18)30119-0.

Drilon A, Laetsch TW, Kummar S, et al. Efficacy of Larotrectinib in TRK fusion-positive cancers in adults and children. N Engl J Med. 2018;378(8):731-739. https://doi.org/10.1056/NEJMoa1714448.

Le X, Baik C, Bauman J, et al. Larotrectinib Treatment for patients with TRK fusion-positive salivary gland cancers. Oncologist. 2022:oyac080. https://doi.org/10.1093/oncolo/oyac080.

Waguespack SG, Drilon A, Lin JJ, et al. Efficacy and safety of larotrectinib in patients with TRK fusion-positive thyroid carcinoma. Eur J Endocrinol. 2022;186(6):631-643. https://doi.org/10.1530/EJE-21-1259.

Doz F, van Tilburg CM, Geoerger B, et al. Efficacy and safety of larotrectinib in TRK fusion-positive primary central nervous system tumors. Neuro Oncol. 2022;24(6):997-1007. https://doi.org/10.1093/neuonc/noab274.

Hong DS, DuBois SG, Kummar S, et al. Larotrectinib in patients with TRK fusion-positive solid tumours: a pooled analysis of three phase 1/2 clinical trials. Lancet Oncol. 2020;21(4):531-540. https://doi.org/10.1016/S1470-2045(19)30856-3.

McDermott R, van Tilburg CM, Farago AF, et al. 1955P Survival benefits of larotrectinib in an integrated dataset of patients with TRK fusion cancer. Annals of Oncology. 2020;31:S1101-2. http://dx.doi.org/10.1016/j.annonc.2020.08.1347.

Desai AV, Robinson GW, Gauvain K, et al. Entrectinib in children and young adults with solid or primary CNS tumors harboring NTRK, ROS1, or ALK aberrations (STARTRK-NG). Neuro Oncol. 2022;24(10):1776-1789. https://doi.org/10.1093/neuonc/noac087.

Doebele RC, Drilon A, Paz-Ares L, et al. Entrectinib in patients with advanced or metastatic NTRK fusion-positive solid tumours: integrated analysis of three phase 1-2 trials. Lancet Oncol. 2020;21(2):271-282. https://doi.org/10.1016/S1470-2045(19)30691-6.

Dziadziuszko R, Krebs MG, De Braud F, et al. Updated integrated analysis of the efficacy and safety of Entrectinib in locally advanced or metastatic ROS1 fusion-positive non-small-cell lung cancer. J Clin Oncol. 2021;39(11):1253-1263. https://doi.org/10.1200/JCO.20.03025.

Drilon A, Siena S, Dziadziuszko R, et al. Entrectinib in ROS1 fusion-positive non-small-cell lung cancer: integrated analysis of three phase 1-2 trials. Lancet Oncol. 2020;21(2):261-270. https://doi.org/10.1016/S1470-2045(19)30690-4.

Drilon A, Siena S, Ou SI, et al. Safety and antitumor activity of the multitargeted pan-TRK, ROS1, and ALK inhibitor entrectinib: combined results from two phase I trials (ALKA-372-001 and STARTRK-1). Cancer Discov. 2017;7(4):400-409. https://doi.org/10.1158/2159-8290.CD-16-1237.

Demetri GD, De Braud F, Drilon A, et al. Updated integrated analysis of the efficacy and safety of Entrectinib in patients with NTRK fusion-positive solid tumors. Clin Cancer Res. 2022;28(7):1302-1312. https://doi.org/10.1158/1078-0432.CCR-21-3597.

Porta M, ed. A Dictionary of Epidemiology (6 ed). 2014 [Internet]. http://dx.doi.org/10.1093/acref/9780199976720.001.0001. Available from: https://www.oxfordreference.com/view/10.1093/acref/9780199976720.001.0001/acref-9780199976720-e-2306 (accessed: 07.09.2022).

Доступ к новым лекарственным средствам в Европе: технический доклад о политических инициативах и возможностях для сотрудничества и исследований. Всемирная организация здравоохранения. Европейское региональное бюро. 2015;180. [World Health Organization. Regional Office for Europe. Access to new medicines in Europe: technical review of policy initiatives and opportunities for collaboration and research. 2015;180. World Health Organization. Regional Office for Europe. (In Russ.)]. Available from: https://iris.who.int/bitstream/handle/10665/365424/WHO-EURO-2015-3211-42969-67837-rus.pdf?sequence=1&isAllowed=y.

Государственный реестр лекарственных средств [Internet]. [State Register of Medicines (In Russ.)]. Available from: https://grls.rosminzdrav.ru/Default.aspx (accessed: 03.04.2023).

European Medicines Agency [Internet]. Available from: https://www.ema.europa.eu/en (accessed: 03.04.2023).

U.S. Food and Drug Administration. [Internet]. Available from: https://www.fda.gov/ (accessed: 03.04.2023).

Therapeutic Goods Administration (TGA) | Australian Government Department of Health. [Internet]. Available from: https://www.tga.gov.au/ (accessed: 03.04.2023).

Government of Canada. [Internet]. Canada.ca. The official website of the Government of Canada. Available from: https://www.canada.ca/en.html (accessed: 03.04.2023).

Pharmaceuticals and Medical Devices Agency [Internet]. Available from: https://www.pmda.go.jp/english/ (accessed: 03.04.2023).

Institute for Quality and Efficiency in Health Care | IQWiG.de [Internet]. Available from: https://www.iqwig.de/en/ (accessed: 03.04.2023).

Haute Autorité de Santé - Professionnels [Internet]. Available from: https://www.has-sante.fr/ (accessed: 03.04.2023).

Canada’s Drugs and Health Technology Agency. CADTH. [Internet]. Available from: https://www.cadth.ca/ (accessed: 03.04.2023).

The National Institute for Health and Care Excellence. NICE [Internet]. NICE. Available from: https://www.nice.org.uk/ (accessed: 03.04.2023).

Tandvårds- och läkemedelsförmånsverket TLV / The Dental and Pharmaceutical Benefits Agency. [Internet]. TLV. Available from: https://www.tlv.se/in-english.html (accessed: 03.04.2023).

Danish Medicines Council [Internet]. Medicinradet. Available from: https://medicinraadet.dk/om-os/in-english (accessed: 03.04.2023).

Healthcare Improvement Scotland [Internet]. The Scottish Medicines Consortium (SMC). Available from: https://www.scottishmedicines.org.uk/ (accessed: 03.04.2023).

Мельникова Л.С., Федяев Д.В., Герасимова К.В. Инновационные методы лекарственного обеспечения: соглашения о разделении рисков. Злокачественные опухоли. 2016;(4 спецвыпуск 1):99-102 [Melnikova LS, Fedyaev DV, Gerasimova KV. Innovative methods of drug provision: risk-sharing agreements. Malignant tumors. 2016;4(special issue 1):99-102 (In Russ.)].

Dostarlimab for previously treated advanced or recurrent endometrial cancer with high microsatellite instability or mismatch repair deficiency. Technology appraisal guidance [TA779]. [Internet]. National Institute for Health and Care Excellence NICE. 2022. Available from: https://www.nice.org.uk/guidance/ta779/chapter/1-Recommendations (accessed: 03.04.2023).

Larotrectinib for treating NTRK fusion-positive solid tumours. Technology appraisal guidance [TA630] [Internet]. National Institute for Health and Care Excellence NICE. 2020. Available from: https://www.nice.org.uk/guidance/ta630 (accessed: 03.04.2023).

Larotrectinib. [Internet]. CADTH. 2020 [updated 2021]. Available from: https://www.cadth.ca/larotrectinib (accessed: 03.04.2023).

Entrectinib for treating NTRK fusion-positive solid tumours. Technology appraisal guidance [TA644] [Internet]. National Institute for Health and Care Excellence NICE. 2020. Available from: https://www.nice.org.uk/guidance/ta644 (accessed: 03.04.2023).

Entrectinib (Rozlytrek). [Internet]. Scottish Medicines Consortium. 2021. Available from: https://www.scottishmedicines.org.uk/medicines-advice/entrectinib-rozlytrek-full-smc2295/ (accessed: 03.04.2023).

Entrectinib for treating ROS1-positive advanced non-small-cell lung cancer. Technology appraisal guidance [TA643] [Internet]. National Institute for Health and Care Excellence NICE. 2020. Available from: https://www.nice.org.uk/guidance/ta643/chapter/1-Recommendations (accessed: 03.04.2023).

Entrectinib (Rozlytrek) for ROS1-positive Non-Small Cell Lung Cancer CADTH. 2020 [updated 2021]. Available from: https://www.cadth.ca/entrectinib-rozlytrek-ros1-positive-non-small-cell-lung-cancer (accessed: 03.04.2023).

Entrectinib (Rozlytrek) Scottish Medicines Consortium. 2021. Available from: https://www.scottishmedicines.org.uk/medicines-advice/entrectinib-rozlytrek-full-smc2294/ (accessed: 03.04.2023).

Лицензия Creative Commons

Это произведение доступно по лицензии Creative Commons «Attribution-NonCommercial-NoDerivatives» («Атрибуция — Некоммерческое использование — Без производных произведений») 4.0 Всемирная.

© АННМО «Вопросы онкологии», Copyright (c) 2023