Euro Roundup: Commission publishes HTA guidance on joint clinical assessments, reporting rules

The European Commission has published two guidance documents to support implementation of the Health Technology Assessment Regulation (HTAR). The texts cover outcomes for joint clinical assessment (JCA) and reporting requirements.

When reviewing technologies under HTAR, the Member State Coordination Group on Health Technology Assessment identifies relevant parameters for the assessment scope. The scope should reflect member states’ needs in terms of parameters and information, data, analysis and other evidence to be submitted by the health technology developer (HTD).

The JCA guidance is intended to help member states define relevant outcomes during the scoping process. The document describes different types of outcomes, such as digital and patient-reported, before discussing points to note in the scoping process and requirements for reporting JCAs.

“Several outcomes are considered adequate in clinical studies and in HTA methodology to measure the clinical benefit to the patient,” the coordination group said. “Some outcomes may be fully acceptable as support for the risk/benefit ratio assessment of a certain therapy but are less suitable for the needs of JCA. This may be the case for surrogate outcomes.”

The guidance also covers another part of the JCA process that relates to outcomes. Later in the process, member states draw conclusions about the clinical value a technology adds within their national borders. Member states must give due consideration to JCA reports but need to reach their own conclusions on the clinical relevance or interpretation of the estimate of relative effectiveness and safety.

While the document is intended to help member states navigate two steps of the JCA process, the text also has implications for HTDs that are trying to bring products to market in the EU.

“The analysis and reporting recommendations for assessors are made with the implicit assumption that appropriate analyses and information is provided by the HTD. As such, this guidance also has practical implications for the submission dossier and assessment report which should be taken into account in the preparation of these documents and associated guidance,” the coordination group said.

The second document provides guidance on the reporting requirements for multiplicity issues and subgroup, sensitivity and post hoc analyses in JCAs. As the guidance explains, member states can assess evidence from individual studies within the framework of the original studies' statistical analysis plan, run a systematic review of a statistical summary of one or several studies or combine the approaches.

“These approaches impact the way in which different [member states] consider specific methodological issues such as multiplicity and subgroup, sensitivity and post hoc analyses. It is not the intent of this guidance to endorse a particular approach but to enable [member states] to draw their own conclusions at the national level,” the coordination group said.

The coordination group released the documents alongside advice on the scientific specifications of medicinal products subject to JCAs. The document is intended to clarify the scientific specifications of “new active substance” and “therapeutic indication of treatment of cancer” to determine if a medicinal product shall be subject to JCA between 12 January 2025 and 13 January 2030.

Press Release

EMA updates transparency rules, removing mechanism that delayed clinical data publication

The European Medicines Agency (EMA) has eliminated a deferral mechanism that allowed sponsors to delay publication of certain data and documents for up to seven years after a clinical trial’s completion.

Previously, sponsors could use the deferral mechanism to protect commercially confidential information. EMA said the new rules “strike a balance between transparency of information and protection of commercially confidential information” and benefit sponsors because they bring process simplifications. Following the changes, around 4,000 studies with issued decisions are publicly accessible.

“The removal of this mechanism will give stakeholders, including patients and healthcare professionals, quicker access to clinical trial information while commercially confidential information continues to be protected through redaction of documents,” EMA said in a summary of its management board meeting.

EMA has made the studies available through a new version of its Clinical Trials Information System (CTIS). The agency expects to add around 500 newly authorized trials to the portal each month. Some of the trials will be ongoing studies transitioned to CTIS from the Clinical Trials Directive.

Press Release

EMA creates templates to help drugmakers draft shortage prevention and mitigation plans

EMA has created templates for shortage prevention and mitigation plans. The agency is encouraging marketing authorization holders to draft plans to reduce the likelihood of shortages and limit the impact of any supply disruptions that do happen.

Shortage prevention plans are mandatory for medicines deemed critical in a health crisis and are part of the upcoming changes to the pharmaceutical legislation. Good practices on the prevention of shortages recommend having a plan in place. The template lists the information companies should include to simplify the creation of plans.

“Their ultimate objective is to reduce the likelihood of shortages by analyzing the risks in the supply chain and defining measures to be taken to mitigate the impact of the shortage by identifying particular existing vulnerabilities in the supply chain and addressing these risks. The degree of effort, formalization and documentation should be proportionate to the identified level of risk for each medicine,” EMA said.

The good shortage practices also recommend the creation of a shortage mitigation plan. In mitigation plans, companies formally identify signals and risks for the continued availability of their products and implement a procedure for mitigating the threats. EMA is advising companies to formally evaluate the effectiveness of mitigation plans and controls.

EMA Templates

SCHEER updates guidelines on phthalates in medical devices after public consultation

The Scientific Committee on Health, Environmental and Emerging Risks (SCHEER) has updated guidelines on assessing the benefits and risks of phthalates in certain medical devices.

SCHEER published its initial guidance on the topic in 2019 in response to evidence that phthalates can be carcinogenic, mutagenic, toxic to reproduction or have endocrine-disrupting properties. The updated document incorporates the experience of implementing the guidelines and insights from studies published in the past five years, plus feedback to a public consultation that SCHEER held earlier this year.

The updated document includes a section on the sources of evidence and SCHEER’s weight of evidence approach. SCHEER has also updated its framework for the evaluation of alternatives in the non-phthalate scenario, replacing the term “potential relevant” candidates with “most relevant” candidates. SCHEER explained the change in a statement.

“While a number of alternatives might be available, the focus should be limited to the likely most relevant alternatives based on a preliminary evaluation of the suitability of the available alternatives. This is to avoid unnecessary extensive evaluation of many alternatives,” SCHEER said.

Press Release, Updated Guidelines

Other News:

EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) has found patients may develop secondary malignancies after receiving CAR-T cell therapies. PRAC is recommending life-long monitoring of people who receive the cell therapies. The committee has also started a review of the risk of sudden drops in white blood cells in people taking the painkiller metamizole. Press Release

The Medical Device Coordination Group (MDCG) has updated its guidance on the designation, re-assessment and notification of conformity assessment bodies and notified bodies. The changes cover the conduct of joint assessments relating to extending the scope of designations. MDCG Guidance

Euro Roundup: Commission publishes HTA guidance on joint clinical assessments, reporting rules

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