Snake Oil or Super Drug: How Have Regulators and Payers Perceived New Medicines that Target Asymptomatic Conditions?
Abstract
Context: Assess how relevant precedent within the European Medicines Agency (EMA) (a key regulator) and the National Institute for Health and Care Excellence (NICE) (a key health technology assessment (HTA) body) can provide insights into how such novel medicines targeting asymptomatic conditions can be brought to market from both a regulatory and payer perspective.
Methods: All phase 2 or phase 3 clinical trials with keywords such as asymptomatic, pre-symptomatic, subclinical, premanifest, or preclinical keywords were downloaded from clinicaltrials.gov. Trials were cleaned to identify those for novel medicines with a primary focus on treating asymptomatic conditions. The EMA and NICE report for such drugs were downloaded online in December 2021 and analyzed to understand how they assessed the evidence.
Results: Two hundred seventy-eight clinical trials were identified. Of those, only 8 had relevant matching reports from the EMA or NICE, 7 received a positive review from the EMA, and 4 received a positive recommendation from the NICE. Those who received positive recommendations showed or were assumed to show statistically significant survival benefits given the short life expectancy of untreated patients.
Conclusions: There is no precedent within the EMA or NICE for approving or funding new medicines for asymptomatic patients where survival benefits cannot be established. Additional research is warranted to understand how regulatory and payer agencies can prepare for future generations of innovative medicines.
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2. Bell J. European regulators say no to Aduhelm, but Biogen is pushing back. BioPharma Dive. December 17, 2021. Accessed January 2, 2022. https://www.proquest.com/trade-journals/european-regulators-say-no-aduhelm-biogen-is/docview/2613415784/se-2?accountid=9630
3. Di Meco A, Vassar R. Early detection and personalized medicine: Future strategies against Alzheimer's disease. Prog Mol Biol Transl Sci. 2021;177:157-173. doi:10.1016/bs.pmbts.2020.10.002
4. Bouvy JC, Jonsson P, O'Rourke D, et al. Regulatory and Health Technology Assessment Considerations for Disease-Modifying Drugs in Alzheimer's Disease. CNS Drugs. 2018;32(12):1085-1090. doi:10.1007/s40263-018-0581-x
5. Staab T, Isbary G, Amelung VE, Ruof J. Inconsistent approaches of the G-BA regarding acceptance of primary study endpoints as being relevant to patients - an analysis of three disease areas: oncological, metabolic, and infectious diseases. BMC Health Serv Res. 2016;16(1):651. Published 2016 Nov 14. doi:10.1186/s12913-016-1902-8
6. Angrist M, Yang A, Kantor B, Chiba-Falek O. Good problems to have? Policy and societal implications of a disease-modifying therapy for presymptomatic late-onset Alzheimer's disease. Life Sci Soc Policy. 2020;16(1):11. Published 2020 Oct 12. doi:10.1186/s40504-020-00106-2
7. Feldman L. Managing the cost of diagnosis. Manag Care. 2009;18(5):43-45.
8. Newman-Toker DE, McDonald KM, Meltzer DO. How much diagnostic safety can we afford, and how should we decide? A health economics perspective. BMJ Qual Saf. 2013;22 Suppl 2(Suppl 2):ii11-ii20. doi:10.1136/bmjqs-2012-001616
9. Teerawattananon Y, Kingkaew P, Koopitakkajorn T, et al. Development of a Health Screening Package Under the Universal Health Coverage: The Role of Health Technology Assessment. Health Econ. 2016;25 Suppl 1(Suppl Suppl 1):162-178. doi:10.1002/hec.3301
10. Huang YM, Pecanac KE, Shiyanbola OO. "Why Am I Not Taking Medications?" Barriers and Facilitators of Diabetes Medication Adherence Across Different Health Literacy Levels. Qual Health Res. 2020;30(14):2331-2342. doi:10.1177/1049732320945296
11. Morrell L, Buchanan J, Rees S, Barker RW, Wordsworth S. What Aspects of Illness Influence Public Preferences for Healthcare Priority Setting? A Discrete Choice Experiment in the UK. Pharmacoeconomics. 2021;39(12):1443-1454. doi:10.1007/s40273-021-01067-w
12. European Medicines Agency. Zytiga: EPAR - Product Information. 2020. Accessed January 2, 2022. https://www.ema.europa.eu/en/documents/product-information/zytiga-epar-product-information_en.pdf
13. NICE. Technology Appraisal Guidance: Abiraterone for treating metastatic hormone-relapsed prostate cancer before chemotherapy is indicated. 2016. Accessed December 27, 2020. https://www.nice.org.uk/guidance/ta387/resources/abiraterone-for-treating-metastatic-hormonerelapsed-prostate-cancer-before-chemotherapy-is-indicated-pdf-82602854745541
14. European Medicines Agency. Pyramax: Product Information. 2017. Accessed December 27, 2022. https://www.ema.europa.eu/en/documents/outside-eu-product-information/pyramax-product-information_en.pdf
15. European Medicines Agency. Pyramax: Assessment Report. Committee for Medicinal Products for Human Use (CHMP). 2021. Accessed January 2, 2022. https://www.ema.europa.eu/en/documents/outside-eu-assessment-report/pyramax-public-assessment-report_en.pdf
16. European Medicines Agency. Ronapreve: EPAR - Product Information. 2021. Accessed December 27, 2022. https://www.ema.europa.eu/en/documents/product-information/ronapreve-epar-product-information_en.pdf
17. European Medicines Agency. Ronapreve: EPAR - Public Assessment Report. Committee for Medicinal Products for Human Use (CHMP). 2021. Accessed January 2, 2022. https://www.ema.europa.eu/en/documents/assessment-report/ronapreve-epar-public-assessment-report_en.pdf
18. NICE. COVID-19 rapid guideline: managing COVID-19. 2021. Accessed January 2, 2022. https://www.nice.org.uk/guidance/ng191
19. European Medicines Agency. Spinraza: EPAR – Product Information. 2021. Accessed December 27, 2022. https://www.ema.europa.eu/en/documents/product-information/spinraza-epar-product-information_en.pdf
20. NICE. Nusinersen for treating spinal muscular atrophy: Technology appraisal guidance. 2019. Accessed December 27, 2022. https://www.nice.org.uk/guidance/ta588
21. European Medicines Agency. Zolgensma: EPAR - Product Information. 2021. Accessed January 2, 2022. https://www.ema.europa.eu/en/documents/product-information/zolgensma-epar-product-information_en.pdf
22. NICE. Onasemnogene abeparvovec for treating spinal muscular atrophy: Highly specialized technologies guidance. 2021. Accessed January 2, 2022. https://www.nice.org.uk/guidance/hst15
23. European Medicines Agency. Xofigo: EPAR - Product Information. 2020. Accessed January 2, 2022. https://www.ema.europa.eu/en/documents/product-information/xofigo-epar-product-information_en.pdf
24. European Medicines Agency. Evrysdi: EPAR - Product Information. 2021. Accessed January 2, 2022. https://www.ema.europa.eu/en/documents/product-information/evrysdi-epar-product-information_en.pdf
25. NICE. Risdiplam for treating spinal muscular atrophy: Technology Appraisal Guidance. 2021. Accessed January 2, 2022. https://www.nice.org.uk/guidance/ta755
26. European Medicines Agency. MabThera: EPAR - Product Information. 2021. Accessed January 2, 2022. https://www.ema.europa.eu/en/documents/product-information/mabthera-epar-product-information_en.pdf
27. NICE. Rituximab for the first-line treatment of stage III-IV follicular lymphoma: Technology appraisal guidance. 2012. Accessed January 2, 2022. https://www.nice.org.uk/guidance/ta243
28. NIH. Search results: 103005 Studies found for: Phase 2, 3. Accessed January 9, 2022. https://clinicaltrials.gov/ct2/results?cond=&term=&type=&rslt=&age_v=&gndr=&intr=&titles=&outc=&spons=&lead=&id=&cntry=&state=&city=&dist=&locn=&phase=1&phase=2&rsub=&strd_s=&strd_e=&prcd_s=&prcd_e=&sfpd_s=&sfpd_e=&rfpd_s=&rfpd_e=&lupd_s=&lupd_e=&sort=
29. European Medicines Agency. Repatha: EPAR - Product Information. 2022. Accessed January 2, 2022. https://www.ema.europa.eu/en/documents/product-information/repatha-epar-product-information_en.pdf
30. Biogen. Biogen Announces Reduced Price for ADUHELM® to Improve Access for Patients with Early Alzheimer’s Disease. Investor Relations. December 20, 2021. Accessed January 2, 2022. https://investors.biogen.com/news-releases/news-release-details/biogen-announces-reduced-price-aduhelmr-improve-access-patients
31. Ramaswamy A, Mahabala C, Basavaiah SH, Jain A, Chouhan RRS. Asymptomatic malaria carriers and their characterization in hotpops of malaria at Mangalore. Trop Parasitol. 2020;10(1):24-28. doi:10.4103/tp.TP_71_18
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| Issue | Vol 7, No 3 (2023) | |
| Section | Articles | |
| DOI | https://doi.org/10.18502/htaa.v7i3.14208 | |
| Keywords | ||
| Prevention HTA Regulatory Endpoints | ||
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How to Cite
1.
Needham B. Snake Oil or Super Drug: How Have Regulators and Payers Perceived New Medicines that Target Asymptomatic Conditions?. Health Tech Ass Act. 2023;7(3).
