A David and Goliath set-up: a qualitative study of the challenges of ensuring the introduction of cost-effective new cancer medicines in Finland

References

• WHO report on cancer: setting priorities, investing wisely and providing care for all. Geneva: World Health Organization; 2020. Licence: CC BY-NC-SA 3.0 IGO.
   Google Scholar
• European Commission. Communication from the Commission to the European Parliament, the Council, the European Economic and Social Committee and the Committee of the Regions. Pharmaceutical Strategy for Europe {SWD(2020) 286 final}, COM(2020) 761 final, Brussels, 25.11.2020.
   Google Scholar
• NormanV. FDA and EU approval of drugs and devices. JACC Basic Transl Sci. 2016;1(5):399–412.
   PubMedGoogle Scholar
• DavisC, NaciH, GurpinarE, PoplavskaE, PintoA, AggarwalA. 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.
   Google Scholar
• MichelM, ToumiM. Access to orphan drugs in Europe: current and future issues. Expert Rev Pharmacoecon Outcomes Res. 2012;12:23–9. https://doi.org/10.1586/erp.11.95.
   PubMed Web of Science ®Google Scholar
• WolchokJD, Chiarion-SileniV, GonzalezR, et al. Overall survival with combined nivolumab and ipilimumab in advanced melanoma. N Engl J Med. 2017;377(14):1345–56.
   PubMed Web of Science ®Google Scholar
• AsciertoPA, McArthurGA, DrénoB, et al. Cobimetinib combined with vemurafenib in advanced BRAF V600-mutant melanoma (coBRIM): updated efficacy results from a randomised, double-blind, phase 3 trial. Lancet Oncol. 2016;17(9):1248–60.
   PubMed Web of Science ®Google Scholar
• RobertC, KaraszewskaB, SchachterJ, et al. Improved overall survival in melanoma with combined dabrafenib and trametinib. N Engl J Med. 2015;372(1):30–9.
   PubMed Web of Science ®Google Scholar
• MokTSK, WuYL, KudabaI, et al. Pembrolizumab versus chemotherapy for previously untreated, PD-L1-expressing, locally advanced or metastatic non-small cell lung cancer (KEYNOTE-042): a randomised, open-label, controlled, phase 3 trial. Lancet. 2019;393:1819–30.
   PubMed Web of Science ®Google Scholar
• HellmannMD, Paz-AresL, Bernabe CaroR, et al. Nivolumab plus ipilimumab in advanced non-small-cell lung cancer. N Engl J Med. 2019;381:2020–31.
   PubMed Web of Science ®Google Scholar
• SocinskiMA, JotteRM, CappuzzoF, et al. Atezolizumab for first-line treatment of metastatic nonsquamous NSCLC. N Engl J Med. 2018;378:2288–301.
   PubMed Web of Science ®Google Scholar
• KashokiM, HanaiziZ, YordanovaS, VeselyR, BouyguesC, LlinariesJ, KwederS. A comparison of EMA and FDA decisions for new drug marketing applications 2014–2016: concordance, discordance, and why. Clin Pharmacol Ther. 2020;107(1):195–202. https://doi.org/10.1002/cpt.1565.
   PubMed Web of Science ®Google Scholar
• GrössmannN, Del PaggioJC, WolfS, SullivanR, BoothCNM, RosianK, EmprechtingerR, WildC. Five yeas of EMA-approved systemic cancer therapies for solid tumors—a comparison of two thresholds for meaningful Clinical benefit. Eur J Cancer. 2017;82:66–71.
   PubMed Web of Science ®Google Scholar
• WHO. Technical report: pricing of cancer medicines and its impacts: a comprehensive technical report for the World Health Assembly Resolution 70.12: operative paragraph 2.9 on pricing approaches and their impacts on availability and affordability of medicines for the prevention and treatment of cancer. World Health Organization. 2018. Available from https://apps.who.int/iris/handle/10665/277190. License: CC BY-NC-SA 3.0 IGO.
   Google Scholar
• OECD. Pharmaceutical Innovation and Access to Medicines, OECD Health Policy Studies, OECD Publishing, Paris, 2018; https://doi.org/10.1787/9789264307391-en.
   Google Scholar
• OECD. Addressing challenges in access to oncology medicines. Analytical report. OECD, Paris, 2020.
   Google Scholar
• Tay-TeoK, IlbawiA, HillSR. Comparison of sales income and research and development costs for FDA-approved cancer drugs sold by originator drug companies. JAMA Netw Open. 2019;2(1): e186875. https://doi.org/10.1001/jamanetworkopen.2018.6875.
   PubMed Web of Science ®Google Scholar
• Cross country collaboration to improve access to medicines and vaccines in the WHO European region. Copenhagen: WHO 2020.
   Google Scholar
• BoomCM, AggarwalA, SullivanR. Commercial determinants of cancer medicines. Eurohealth. 2022;28(2):18–21.
   Google Scholar
• ChapmanAL, HadfieldM, ChapmanCJ. Qualitative research in healthcare: an introduction to grounded theory using thematic analysis. J R Coll Physicians of Edinb. 2015;45(3):2015. https://doi.org/10.4997/JRCPE.2015.305.
   Google Scholar
• BonoP, HiltunenK-M, KorpelainenJ, PietiläM, VanninenE. Kalliiden lääkkeiden käyttöönotosta suositus. [Recommendation on introducing expensive medicines] Näkökulma. Suom Laakaril. 2018;73(36):1936–7.
   Google Scholar
• HonkanenH, SnickerK, AhlamaaJ. Lääkkeiden riskinjakosopimukset Suomen sairaaloissa—kyselytutkimus. Suomen Lääkäril. 2019;72(49):2872–6.
   Google Scholar
• Ajosenpä�� M, Huupponen R, Turpeinen M, Rannanheimo P. Uusien sairaalalääkkeiden hallitun käyttöönoton sopimukset—maksajan näkökulma. [Managed Entry Agreements among new hospital medicines—the payer’s view] Dosis 37: 278–300, 2021.
   Google Scholar
• Pennanen T. Sairaalalääkehankinnat tehdään jatkossa yhteistyössä. [Hospital medicines will be procured together in the future] Lääkärilehti, 9.9.2022, available from https://www.laakarilehti.fi/ajassa/ajankohtaista/sairaalalaakehankinnat-tehdaan-jatkossa-yhteistyossa/. Accessed 2 August 2022.
   Google Scholar
• Ministry of Social Affairs and Health, Finland. Näkökulmia lääkehoitoon ja lääkkeiden jakeluun liittyvistä muutostarpeista. [Perspectives on the need to develop pharmaceutical care and delivery] Virkamiesmuistio. Sosiaali- ja terveysministeriön raportteja ja muistioita 2019:5, available from https://julkaisut.valtioneuvosto.fi/handle/10024/161340. Accessed 2 August 2022.
   Google Scholar
• Huff-RoussellM. The logical underpinnings and benefits of pooled pharmaceutical procurement: a pragmatic role for our public institutions. Soc Sci Med. 2012;75(9):152–8.
   Web of Science ®Google Scholar
• Perehudoff K, Mara K, ‘t Hoen E. What is the evidence on legal measures to improve the transparency of markets for medicines, vaccines and other health products (World Health Assembly resolution WHA72.8)? Copenhagen: WHO Regional Office for Europe; 2021. Available from https://apps.who.int/iris/handle/10665/342474. Accessed 2 August 2022.
   Google Scholar
• PokornyAMJ, FabbriA, BeroLA, et al. Interactions with the pharmaceutical industry and the practice, knowledge and beliefs of medical oncologists and clinical haematologists: a systematic review. Br J Cancer. 2022;126:144–61. https://doi.org/10.1038/s41416-021-01552-1.
   PubMed Web of Science ®Google Scholar
• Florio M, Pancotti C, Prochazka DA. Could public infrastructure overcome market failures. European pharmaceutical research and development. Panel for the future of science and technology. European Parliamentary Research Service. Service Foresight Unit (STOA), PE 697.197, December 2021, available from https://www.europarl.europa.eu/thinktank/en/document/EPRS_STU(2021)697197. Accessed 2 August 2022.
   Google Scholar
• HaslamA, LythgoeMP, Greenstreet AkmanE, PrasadV. Characteristics of cost-effectiveness studies for oncology drugs approved in the United States form 2015–2020. JAMA Netw open. 2021;4(11): e2135123. https://doi.org/10.1001/jamanetworkopen.2021.35123.
   Web of Science ®Google Scholar
• ParikhR, MinE, WileytoE, et al. Uptake and survival outcomes following immune checkpoint inhibitor therapy among trial-ineligible patients with advanced solid cancer. JAMA Oncol. 2021;7:1843–50. https://doi.org/10.1001/jamaoncol.2021.4971.
   PubMed Web of Science ®Google Scholar
• TorkkiP, LeskeläR-L, LinnaM, MäklinS, MecklinJP, BonoP, KatajaV, KarjalainenS. Cancer costs and outcomes in the Finnish population 2004–2014. Acta Oncol. 2018;57(2):297–303. https://doi.org/10.1080/0284186X.2017.1343495.
   PubMed Web of Science ®Google Scholar
• Koskinen H, Juntunen L, Junttila T, Kurko T. Korvausjärjestelmään hyväksytyt uudet syöpälääkkeet. Kohtaavatko saatavuus ja hyödyt? [New cancer medicines granted reimbursement. Do the accessibility and benefits meet] Suomen Lääkäril 2019;74(6):327–30.
   Google Scholar
• Finnish Statistics on Medicines 2019. Fimea and KELA, Helsinki 2020, available http://urn.fi/URN:NBN:fi-fe20201217101079. Accessed 2 August 2022.
   Google Scholar
• Fimea, KELA. aluekohtainen tilannekuva avo- ja sairaanhoidon lääkekustannuksista. [Region-specific snapshot of outpatient and inpatient pharmaceutical costs]. Press release, December 2021, available from https://laaketieto.kela.fi/docs/tilannekuva_avo-ja_laitoshoidon_laake.pdf. Accessed 2 August 2022.
   Google Scholar
• Wenzl M, Chapman S. Performance-based managed entry agreements for new medicines in OECD countries and EU member states: How they work and possible improvements going forward. OECD Health Working Papers No 115, 2019. Available from https://www.oecd.org/health/health-systems/pharma-managed-entry-agreements.htm. Accessed 2 August 2022.
   Google Scholar
• MorganSG, VoglerS, WagnerAK. Payers’ experiences with confidential pharmaceutical price discounts: a survey of public and statutory health systems in North America Europe and Australia. Health Policy. 2017;121:354–62.
   PubMed Web of Science ®Google Scholar
• GambaS, PertileP, VoglerS. The impact of managed entry agreements on pharmaceutical prices. Health Econ. 2020;29(S1):47–62.
   PubMedGoogle Scholar
• WHA (World Health Assembly) 2019. Improving the transparency of markets for medicines, vaccines, and other health products, WHA 72.8. 2019, available from https://apps.who.int/gb/ebwha/pdf_files/WHA72-REC1/A72_2019_REC1-en.pdf#page=25. Accessed 2 August 2022.
   Google Scholar
• MarselisD, HordjikL. From blockbuster to “nichebuster”: how a flawed legislation helped create new profit model for the drug industry. BMJ. 2020;370:m2983. https://doi.org/10.1136/bmj.m2983.
   PubMed Web of Science ®Google Scholar