Written by Benjamin Clark, Kiera Lander, and Toby Hobbs
August brought a busy month for health technology assessment (HTA), with developments spanning the EU, UK, and Ireland; including a new tool to help health technology developers navigate Joint Clinical Assessment (JCA) eligibility, and further information from NICE on the new EQ-5D-5L data set. Here’s our summary of the key updates, and their potential implications for evidence generation and submission strategies.
EU HTA: Making JCA scoping a little easier
One of the key EU HTA developments last month was the launch of a new tool, the JCA eligibility checker. The tool has been designed to take some of the guesswork out of JCA eligibility, enabling health technology developers to quickly assess whether a product is likely to fall within the scope of a JCA.
The EU survey-based tool asks a series of questions about the product and provides an early indication of whether it is likely to be in scope. While it does not replace formal confirmation from the HTA Secretariat, it offers a useful first step for health technology developers before submitting the more detailed Eligibility and Letter of Intent information. An early indication of JCA scope could also enable evidence teams to start planning sooner and identify potential requirements at an earlier stage.
NICE: Looking beyond clinical efficacy
NICE made three notable recommendations last month, with a common theme running through the decisions: the value of a treatment can extend well beyond its direct clinical effect.
NICE recommended once weekly insulin efsitora alfa (Onswik®) for people with type 2 diabetes, which is anticipated to reduce the number of annual injections from 365 to 52, while providing comparable glycaemic control to daily basal insulin. NICE recognised the benefits to patients and carers, with fewer injections causing less disruption to everyday life, improving treatment adherence, and requiring fewer visits from carers or healthcare workers.
NICE also recommended finerenone (Kerendia®) for chronic heart failure and acalabrutinib (Calquence®) with venetoclax (Venclyxto®) with or without obinutuzumab (Gazyvaro®) for untreated chronic lymphocytic leukaemia.
These decisions reinforce the importance of looking beyond traditional clinical endpoints when developing a submission strategy. Quality of life, adherence, treatment burden and healthcare resource use can all contribute to the overall value of a technology and should be considered from the outset.
NICE Listens: Opening the conversation on AI
AI was also on NICE’s agenda last month. Through its NICE Listens programme, NICE is seeking public views on the use of AI in health and care, with a particular focus on trust, transparency, fairness and accountability. At the same time, NICE is developing methods for evaluating both AI-enabled technologies and AI-generated evidence.
Leveraging AI to support with evidence generation for NICE reimbursement is fast-evolving and should be closely monitored by submitting companies. Technical performance will remain important, but questions around governance, bias, transparency and explainability are likely to play an increasingly important role in how AI-generated evidence is viewed by HTA agencies. Public engagement is expected to explore how people want AI to be used in health and social care, what role it should play in NICE’s work, and the principles NICE should follow when evaluating AI technologies.
NICE: Interim implementation of the new EQ-5D-5L value set
NICE has published an Interim methods statement setting out how the new EQ-5D-5L value set will be implemented in technology appraisals. The statement provides clarity on how the updated value set will be incorporated into economic evaluations while NICE moves towards full implementation.
For companies developing or updating NICE models, the transition to the new value set is an important methodological consideration. Submitting companies will want to understand which value set applies to their appraisal and whether the change could affect utility estimates, quality-adjusted life years (QALYs), and the overall cost-effectiveness results.
SMC: A clean sweep in August
The Scottish Medicines Consortium (SMC) had a particularly positive month, accepting all seven medicines it reviewed in August.
The decisions covered a broad range of indications, including generalised myasthenia gravis, allergic rhinitis, advanced neuroendocrine tumours, advanced endometrial cancer, giant cell arteritis, advanced Parkinson’s disease, and hyperkalaemia.
The seven accepted medicines were zilucoplan (Zilbrysq®), 12 SQ-HDM (Acarizax®), cabozantinib (Cabozantinib Ipsen®), dostarlimab (Jemperli®) with chemotherapy, upadacitinib (Rinvoq®), levodopa/carbidopa/entacapone (Lecigon®), and sodium zirconium cyclosilicate (Lokelma®).
The acceptance of seven medicines for routine use by NHS Scotland is notable, particularly given the breadth of indications covered, and provides an encouraging snapshot of the current Scottish HTA landscape.
NCPE: Two assessments, two different outcomes
In Ireland, the National Centre for Pharmacoeconomics (NCPE) completed two assessments in August, with both decisions highlighting the importance of the economic case.
Acalabrutinib (Calquence®) plus bendamustine and rituximab for previously untreated mantle cell lymphoma in transplant-ineligible adults was not recommended for reimbursement unless its cost-effectiveness improves relative to existing treatments.
The NCPE also recommended that Linzagolix Choline (Yselty®) be considered for reimbursement if cost-effectiveness can be improved relative to existing treatments in moderate-to-severe symptoms associated with uterine fibroids.
The final reimbursement decision for both treatments now sits with Ireland’s Health Service Executive (HSE). The two assessments are a reminder that strong clinical evidence alone does not guarantee a positive HTA outcome. For Irish submissions, a robust and well-supported economic case remains critical, particularly where there is uncertainty around comparative benefit or cost-effectiveness.
Looking ahead
Overall, the developments seen in August reinforce the importance of early strategy planning and a holistic approach to value demonstration. As HTA requirements continue to evolve, Companies that proactively adapt their evidence generation strategies and look beyond clinical outcomes alone will be best positioned for positive outcomes
If you would like to learn more about HTA submissions (including systematic reviews, health economic modelling, and medical writing), please contact us at Source Health Economics, a HEOR consultancy specialising in evidence generation, health economics, and communication.
