Achievements and Opportunities in Pan-Tumor Oncology European HTA & Access (Guest blog)
- Joint HTA legislation calls for EU-level cooperation to standardise submission and review procedures for health technologies in order to enhance transparency, efficiency, and patient access to these technologies1-2.
- Europe’s Beating Cancer Plan outlines a multi-dimensional, patient-centric approach to addressing cancer, including specific “flagship initiatives” such as "Cancer Diagnostic and Treatment for All" featuring next generation sequencing (NGS), and "Cancer Inequalities Registry" to identify and resolve disparities among Member States and regions3-4.
- The #Resolution4Rare global campaign co-led by EURORDIS, RDI, and NCO Committee for Rare Diseases champions for further inclusivity, empowerment, and access for those living with rare diseases at the UN level, with resolution adoption decision pending in September 20215-7.
- The "future of drug development in rare genomic entities" and the foundation for a new "paradigm under construction".
- Clinical trial innovation through its single-group, basket trial design.
- Diagnostic technology advancement through integration of multigene sequencing alongside conventional methods.
These attributes and the clinical results achieved led to a rating of highest magnitude of clinical benefit under the Magnitude of Clinical Benefit Scale developed by the European Society of Medical Oncology (ESMO) for single-group trials10.
- Regulatory rules of what qualifies as rare disease, and in turn, which treatments are eligible for orphan drug status, a designation which may facilitate, and even expedite, HTA and access by allowing medicine appraisal under different criteria in some countries. Germany is one such example, but the TRK inhibitors approved to date, despite targeting an ultra-rare genomic alteration, nevertheless received “no evidence of additional benefit”.
- How HTA systems assess evidence hierarchy requirements to assign “additional medical benefit”, with a strict adherence to requiring large-scale RCTs, thereby precluding such an adjudication in case of single-group trials.
- How HTA systems in some countries, such as France and Italy among others, maintain a conventional tumor-by-tumor approach for evaluating effectiveness, risk/benefit, and reimbursement, instead of evolving to a unified approach for pan-tumor, single biomarker-targeted therapies. In the same vein, some HTAs separately consider adult versus paediatric populations, at times leading to differential conclusions.
1. "Health Technology Assessment: Informal deal between Council and European Parliament." European Council, Council of the European Union, 22 June 2021, https://europa.eu/!Bb9ngX. Accessed 20 July 2021.
2. "Deal on boosting member states’ cooperation in assessing health technologies." European Parliament, 22 June 2021, https://www.europarl.europa.eu/news/en/press-room/20210617IPR06472/deal-on-boosting-member-states-cooperation-in-assessing-health-technologies. Accessed 20 July 2021.
3. "Communication from the commission to the European Parliament and the Council, Europe's Beating Cancer Plan COM/2021/44 final." EUR-Lex, 3 February 2021, https://eur-lex.europa.eu/legal-content/en/TXT/?uri=COM:2021:44:FIN. Accessed 20 July 2021.
4. "EU Health Union: Europe's Beating Cancer Plan - Factsheet - Europe's Beating Cancer Plan." European Commission, 3 February 2021, https://ec.europa.eu/commission/presscorner/detail/en/fs_21_341. Accessed 20 July 2021.
5. Ostapenko, Stanislav. "#Resolution4Rare: Global campaign for the first UN Resolution on Persons Living with a Rare Disease." EURORDIS Rare Diseases Europe, 12 May 2021. https://www.eurordis.org/news/resolution4rare-global-campaign-first-un-resolution-persons-living-rare-disease. Accessed 20 July 2021.
6. "UN Resolution on Persons Living with a Rare Disease and their Families." Rare Diseases International, https://www.rarediseasesinternational.org/un-resolution-to-address-the-challenges-of-people-living-with-rare-diseases/. Accessed 20 July 2021.
7. "United Nations General Assembly Resolution on Persons Living with a Rare Disease and their Families." Rare Voices Australia, 14 May 2021, https://rarevoices.org.au/united-nations-general-assembly-resolution-on-persons-living-with-a-rare-disease-and-their-families/. Accessed 20 July 2021.
8. "EFPIA Patients W.A.I.T. Indicator 2020 Survey." EFPIA, April 2021, https://efpia.eu/media/602652/efpia-patient-wait-indicator-final-250521.pdf. Accessed 20 July 2021.
9. Amatu A, Sartore-Bianchi A, Siena S. “NTRK gene fusions as novel targets of cancer therapy across multiple tumour types.” ESMO Open 2016;1(2):e000023. DOI: https://www.esmoopen.com/article/S2059-7029(20)32579-5/fulltext.
10. André, F. "Developing Anticancer Drugs in Orphan Molecular Entities — A Paradigm under Construction." N Engl J Med 2018; 378:763-765. DOI: 10.1056/NEJMe1716821.