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Examples of Performance-based Agreements and Recent HTA Outcomes for Novel Therapies

September 22, 2022 by in Actu-Real Blog

Risk-sharing agreements (RSAs), especially performance-based, are only growing in popularity among payers, health technology assessment (HTA) agencies, and manufacturers, as they provide common grounds to make new medicines (even more for high-cost ones) available to patients that otherwise wouldn’t be at the time of product launch when limited real-world data are available. HTA outcomes help payers and manufacturers enter outcome-based RSAs to overcome uncertainties at the time of launch.

Examples of some performance-based schemes/models over the years: 1, 2, 3, 4

Institute of clinical and economic review (ICER) in the US, National Institute for Health and Care Excellence (NICE) in the UK, and similar such HTA bodies across the world are known for the analysis and recommendations that they make for various drugs. Their recommendations are now being used by a vast majority of public and private payers to guide their decisions and policies. Additionally, governments are also looking to find solutions to make such high-cost therapies accessible, recent example is the signing of the Inflation Reduction Act 2022 into law in the US, that would allow Medicare to negotiate directly with pharma companies for some high-cost drugs.5

Recent Examples of Assessment Outcomes for High-cost Drugs made by HTA Bodies:

Assessment Body Drug Name and Indication Key Outcomes
Institute for clinical and economic review (ICER) (June 2022) 6 Betibeglogene autotemcel (beti-cel) - gene therapy for beta-thalassemia
  • Superior overall to current standard of care
  • Cost-effectiveness modelling finds treatment to meet accepted value thresholds at anticipated price of $2.1 million
  • 80% payback option for patients who do not achieve and maintain transfusion independence over a five-year period
HTA: Joint assessment between Ireland, Netherlands and Belgium (April 2021 and Oct 2021) 7,8 Onasemnogene abeparvovec (Zolgensma) - gene therapy for spinal muscular atrophy
  • In April 2021, the joint recommendation was not to consider Zolgensma for reimbursement unless cost effectiveness could be improved relative to existing treatments (primarily Spinraza)
  • In Oct 2021, the 3 countries jointly approved reimbursement following confidential price negotiations with marketing authorization holder
ICER (Apr/May 2019) 9,10 Spinraza and Zolgensma - treatment for spinal muscular atrophy
  • Council unanimously voted that the clinical evidence is sufficient to show a net health benefit for Spinraza and Zolgensma in Type I SMA, and for Spinraza in the later-onset and pre-symptomatic populations
  • Estimated incremental cost-effectiveness of Spinraza was $709,000 per QALY gained from a health care sector perspective and $687,000 from a modified societal perspective, far exceeding usual cost-effectiveness thresholds
  • Estimated incremental cost-effectiveness from a health care sector perspective in patients with symptomatic Type I SMA was $243,000 per QALY gained
NICE HTA Oct 2019 11 Luxturna – gene therapy for RPE65-mediated inherited retinal dystrophy (IRD)
  • Luxturna can be considered an appropriate use of NHS resources for highly specialized technologies. It therefore recommended voretigene neparvovec as an option for treating IRDs caused by RPE65 gene mutations.
  • Based on the committee's preferred assumptions, the ICER was between £114,956 per QALY gained using the company's EQ‑5D utility values and £155,750 per QALY gained using utility values based on the total population from Rentz et al. (2014) using the committee's preferred discount rate of 3.5%.
  • The company has a commercial arrangement, this makes voretigene neparvovec available to the NHS with a discount (exact discount is confidential)

In conclusion, though there have been various performance-based RSAs in play mostly from early 2000’s, there seem to be shortcomings with most of the current models or solutions provided, one where all stakeholders agree and benefit from. With most countries facing budget constraints and with more novel high-cost therapies coming into the market, there is definitely a need to find novel solutions of RSAs, one that would be acceptable to all to ensure novel therapies rightfully reach their patients.

 

References

  1. iatkiewicz, T.J., Traulsen, J.M. & Holm-Larsen, T. Risk-Sharing Agreements in the EU: A Systematic Review of Major Trends. PharmacoEconomics Open 2, 109–123 (2018). https://doi.org/10.1007/s41669-017-0044-1
  2. Appraisal and Funding of Cancer Drugs from July 2016 (including the new Cancer Drugs Fund). https://www.england.nhs.uk/wp-content/uploads/2013/04/cdf-sop.pdf 
  3. Adamski, Jakub & Godman, Brian & Ofierska-Sujkowska, Gabriella & Osińska, Bogusława & Herholz, Harald & Wendykowska, Kamila & Laius, Ott & Jan, Saira & Sermet, Catherine & Corinne, Zara & Kalaba, Marija & Gustafsson, Roland & Garuoliene, Kristina & Haycox, Alan & Garattini, Silvio & Gustafsson, Lars. (2010). Risk sharing arrangements for pharmaceuticals: potential considerations and recommendations for European payers. BMC Health Service Research. 10:153. 10.1186/1472-6963-10-153.
  4. Cigna makes performance-based drug pricing deal with Novartis. https://www.fiercehealthcare.com/payer/cigna-makes-performance-based-drug-pricing-deal-novartis. Accessed in Sep 2022.
  5. Landmark U.S. healthcare bill sets stage for lower Medicare prescription drug costs. https://www.reuters.com/world/us/landmark-us-healthcare-bill-sets-stage-lower-medicare-prescription-drug-costs-2022-08-09/. Accessed in Sep 2022.
  6. Betibeglogene Autotemcel for Beta Thalassemia: Effectiveness and Value. Final evidence report (ICER). https://icer.org/wp-content/uploads/2021/11/ICER_Beta-Thalassemia_Final-Report_071922.pdf 
  7. Onasemnogene abeparvovec (Zolgensma®). HTA ID: 20021. National Centre for Pharmacoeconomics, Ireland. http://www.ncpe.ie/drugs/onasemnogene-abeparvovec-zolgensma-hta-id-20021/. Accessed in Sep 2022
  8. Managed Access Protocol – Onasemnogene abeparvovec (Zolgensma®). https://www.hse.ie/eng/about/who/cspd/ncps/medicines-management/managed-access-protocols/onasemnogene-abeparvovec-zolgensma/onasemnogene-abeparvovec-zolgensma.pdf
  9. Spinraza® and Zolgensma® for Spinal Muscular Atrophy: Effectiveness and Value. Final evidence report (ICER). https://icer.org/wp-content/uploads/2020/10/ICER_SMA_Final_Evidence_Report_110220.pdf 
  10. Starner CI, Gleason PP. Spinal muscular atrophy therapies: ICER grounds the price to value conversation in facts. J Manag Care Spec Ph 2019;25:1306–8. 10.18553/jmcp.2019.25.12.1306.
  11. Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations. NICE Report 2019. https://www.nice.org.uk/guidance/hst11/chapter/4-Consideration-of-the-evidence 
About Ambika Subramanian

15 years' clinical research experience, including 13 years in medical writing; led multiple global medical writing projects; Authoring experience spans across regulatory writing, safety writing, and medical communication; experience of setting up robust medical writing processes.

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