HTA News Around the World

NICE, UK

In October 2023, the UK’s National Institute for Healthcare Excellence (NICE) updated its methods and processes, introducing the proportionate approach to HTA appraisals. The proportionate approach, which was piloted in 2022-23, is a simplified approach, where parts of the appraisal process have been removed or reconfigured. This enables NICE to evaluate low-risk treatments within a shorter timeframe than conventional assessment processes, allowing for faster decisions on reimbursement. The proportionate approach has already enabled NICE to appraise six treatments in approximately half the time, benefiting 180,000 patients. [1]

The proportionate approach also permits NICE to allocate sufficient time to complex areas that require tailored support. The update covers information on several aspects of the proportionate approach, including its cost comparison process and streamlined committee decision-making. NICE is currently implementing a second phase of its proportionate approach, which will pilot further novel approaches that will simplify, remove, or reconfigure parts of the appraisals process. [1]

Scottish Medicines Consortium, Scotland

In February 2023, the Scottish Medicines Consortium (SMC) revealed that the abbreviated submission process has been proven so successful that the SMC has committed to using it in the long term. The abbreviated process was introduced during the COVID-19 pandemic to streamline decision-making on the assessment of new medicines where alternative medicines of the same therapeutic class have previously been accepted for use. [2]

The evaluation of this process revealed that it was highly beneficial in terms of saving resources and time for the SMC assessment teams, committees, the pharmaceutical industry and Patient Group Partners. Further, it allowed SMC committees to focus on new, first-in-class medicines. [2]

EMA, Europe

New HTA Regulation

EUnetHTA 21, a collaborative consortium established to develop the future EU HTA system per the new HTA Regulation, wrapped up its activities in September 2023. The responsibility for preparing the implementation of the new HTA Regulation has now transitioned to the leadership of the HTA Coordination Group. [3]

The Regulation on Health Technology Assessment (EU) 2021/228, which entered into force in January 2022 and applies as of January 2025, will govern the European cooperation between medicine regulators and HTA bodies. Under the new framework, EMA and HTA bodies will collaborate in the context of joint clinical assessments, joint scientific consultations and the identification of emerging health technologies. [3]

The regulation aims at improving the availability of innovative medicines and certain medical devices for patients in the EU. Further, it aims to ensure the efficient use of resources, to enhance the quality of HTA and to reduce duplication of efforts for national HTA authorities and the industry. [3]

During the transition period between September 2023 until January 2025, drug developers can request the involvement of HTA bodies when applying for EMA scientific advice. The outcome of the procedure will be a scientific advice letter from EMA and written recommendations from participating HTA bodies. The Agency has identified several priorities and opportunities for the next 15 months. These include defining a single evidence plan to facilitate development programmes, harmonising views on the strength of the evidence, and involving patients, clinical experts and other relevant experts in decision-making. [3]

DARWIN EU®

The Data Analysis and Real-World Interrogation Network (DARWIN EU®) has completed its first year of operation. DARWIN EU® is a platform to generate real-world evidence (RWE) to support the decision-making of EMA scientific committees and national competent authorities in EU Member States throughout regulatory processes. RWE complements the evidence generated by randomized controlled clinical trials and contributes to more data-driven regulatory decision making. [4]

A key design feature of DARWIN EU® is that the data are converted into the OMOP Common Data Model (CDM). Further, data remains local and is analysed locally by the respective data partners. This combination of features ensures personal data protection, while enabling the swift execution of large-scale, multi-database studies. [4]

The network comprises 20 public and private institutions spanning 13 European countries.  The data partners generate real-world evidence (RWE) from sources such as hospitals, primary care, health insurance, registries and biobanks, thus enabling access to data from approximately 130 million patients across Europe. [5]

As of March 2024, 14 RWD studies have been successfully completed, with eleven more currently underway. These studies encompass various areas such as drug usage, vaccine efficacy, disease patterns, and patient profiles, offering valuable insights for EU regulators to deepen their understanding of diseases, populations, and the efficacy of pharmaceutical treatments. The results from the completed studies have been shared with the relevant EMA committees and other stakeholders to inform their initiatives. Upon completion, both the protocols and outcomes of all DARWIN EU studies are made publicly accessible through the new HMA-EMA Catalogue of RWD studies. [5]

The inclusion of new data partners in both 2024 and 2025 is set to significantly boost the volume of studies. By 2024, the goal is to complete over 70 real-world data (RWD) studies, with projections indicating a doubling of this output, to surpass 140 studies annually starting from 2025. This concerted endeavour is aimed at bolstering data-driven decision-making on health technology across the EU. [5]

ICER, United States

The Institute for Clinical and Economic Review (ICER) announced the creation of a Patient Council to advise on ICER’s patient engagement strategy, outreach and process for drug reviews and broader initiatives. The Council has involved several patient representatives with a range of expertise on HTA, offering a variety of perspectives during ICER evaluations. [6]

Further, ICER and the Peterson Health Technology Institute (PHTI) collaborated on an assessment framework for digital health technologies (DHTs), which includes the conceptual model and associated methods that will guide assessments of DHTs within broader evaluative reports produced by the PHTI. [7] PHTI’s independent assessments will evaluate the performance of health technologies designed to replace or augment traditional care delivery, including digital therapeutics, chronic care management apps, remote patient monitoring and administrative technologies, many of which involve artificial intelligence. The assessment framework will prioritize products’ clinical benefits and economic impact, as well as their effects on health equity, privacy, and security. [8]

CADTH, Canada

The Canadian Agency for Drugs and Technologies in Health (CADTH) has introduced time-limited drug reimbursement recommendations, a new recommendation category that will aim to help provide earlier access to promising new treatments that target the unmet needs of people in Canada living with severe, rare, or debilitating illnesses. A time-limited recommendation is a recommendation to publicly fund a drug for a certain period of time. This recommendation is based on the condition the manufacturer will conduct ongoing clinical studies to address uncertainty in the evidence. CADTH will conduct a future reassessment of additional evidence, which will lead to a final reimbursement recommendation. [9]

Collaboration between NICE, ICER και CADTH

As part of their collaboration to create a more consistent approach to the handling of clinical data, NICE, ICER and CADTH jointly issued a statement in April 2023 to amend the management of confidential information on health technologies to improve their processes and increase transparency. [10]

Considering the need to streamline the assessment process, which is now conducted earlier in the health technology development cycle, organisations will cease to extract confidential data, which previously remained unpublished, from public documents. As a consequence, NICE and CADTH have ceased to exempt clinical data awaiting publication from public documents, while ICER has reduced the exemption period for data formally scheduled for publication from 18 to 12 months. Each international organisation will set a different policy for the management of data for which there is no specific publication plan. The agencies will continue to exclude data that is commercially sensitive, such as information on pricing and the terms of compensation agreements. [10]

Two more HTA bodies added to the international cooperation for the improvement of HTA methods and procedures

The international cooperation to improve HTA methods and procedures was established in 2021 between NICE, CADTH, the Australian Government Department of Health and Aged Care, the Healthcare Improvement Scotland, Health Technology Wales (HTW) and the All Wales Therapeutics & Toxicology Centre (AWTTC). It was recently expanded to include two more HTA bodies: the Institut national d’excellence en santé et en services sociaux (INESSS) in Quebec and Pharmac in New Zealand. [11]

The collaboration has prioritised work in 3 areas for 2023-2024: a) work sharing; partners will explore the feasibility of work sharing to support HTA decision making and improve their agency’s capacity, b) horizon scanning; to identify and prepare for the opportunities and challenges of the future and c) science and methods development. [11]

[1] https://www.nice.org.uk/news/article/nice-launches-first-modular-update-to-its-methods-and-processes

[2] https://www.scottishmedicines.org.uk/about-us/latest-update/speeding-up-access-to-new-medicines-in-scotland/

[3] https://www.ema.europa.eu/en/news/towards-permanent-collaboration-framework-ema-and-health-technology-assessment-bodies

[4] https://www.ema.europa.eu/en/news/darwin-eur-has-completed-its-first-studies-and-calling-new-data-partners

[5] https://www.ema.europa.eu/en/news/darwin-eur-continues-expanding-its-capacity-deliver-real-world-data-studies

[6] https://icer.org/news-insights/press-releases/icer-launches-a-patient-council-to-amplify-the-patient-voice-in-value-assessment/

[7] https://icer.org/assessment/icer-phti-assessment-framework-fordigital-health-technologies/

[8] https://phti.com/announcement/peterson-health-technology-institute-releases-new-digital-health-assessment-framework/

[9] https://www.cadth.ca/news/cadths-time-limited-recommendation-category-aims-support-earlier-access-promising-drugs

[10] CADTH, ICER, NICE. 2023. Position statement: Confidentiality of clinical evidence informing health technology assessment decision making, https://icer.org/wp-content/uploads/2023/04/Position-Statement_confidential-data_FINAL.pdf

[11] https://www.nice.org.uk/news/article/international-health-technology-assessment-collaboration-expands