Euro Roundup: EFPIA warns HTA changes could hamper drug access

EFPIA has warned that an upcoming approach to health technology assessments (HTAs) “would hamper access to some of the most cutting-edge products in the research pipeline” if applied next year without tweaks.

The trade group issued the warning in response to the findings of a report that it commissioned. For the report, Evidera applied the EUnetHTA 21 proposed methods guidelines to three approved cancer drugs to simulate a joint clinical assessment (JCA) report. The new HTA regulation, which will take effect in early 2025, makes a JCA of the relative effectiveness of the product a key pillar of the process. Oncology drugs and advanced therapy medicinal products are set to be the first treatments to undergo JCAs.

Evidera’s simulations highlighted the potential for the PICO framework to create complexity. Using the framework, research questions are defined in terms of the populations, the intervention, comparators and outcomes (PICO). Evidera found 57 potential combinations in one of the simulated assessments.

There were fewer PICOs for the other drugs, 16 and 22, but Evidera found “all three technologies would require the use of indirect treatment comparisons or network meta-analyses” to address questions about the combinations. Evidence from a randomized trial was available for the drug with 57 PICOs but “the direct evidence only addressed one of the many potential comparators in an evolving treatment environment,” Evidera said.

Evidera attributed the 57 PICOs to “the availability of multiple alternate treatment options, including targeted therapies and other chemotherapy regimens.” EFPIA said large numbers of PICOs are “expected to happen frequently when utilizing the additive proposed scoping approach in the highly dynamic oncology landscape.”

The report features proposals for how to improve the process. One idea is to have health technology developers (HTDs) propose an evidence-based base-case PICO for the JCA based on objective, verifiable data on which patient is most likely to receive the new technology.

“This can be used to propose relevant comparators that reflect the main standards of care used across the EU,” Evidera said. “The evidence-based HTD base-case PICO, and contextual background, can be used by JCA assessors as a starting point to enable more transparent and efficient scoping.”

Evidera is also proposing to involve the HTD, clinical experts and patients in scoping meetings in the belief it can ensure “the most representative European treatment practices and relevant patient outcomes are reflected in the final scope.” HTDs should be included because they can share information “on the clinical data in context of the disease, clinical practice evolution through the development program, available evidence within and outside of trials, and regulatory strategy and timelines.”

EFPIA now wants to work with the Coordination Group, the European Commission and the HTA Stakeholder Network “to help define optimal implementing arrangements and guidelines.”

Press Release, EFPIA Report

EDQM shares advice on how to avoid Certification of Suitability rejections

The European Directorate for the Quality of Medicines & HealthCare (EDQM) has published advice on how to avoid rejection when applying for the revision or renewal of Certificates of Suitability (CEPs).

EDQM used the notice to remind CEP holders of a guideline that lists different notifications and their associated conditions. By default, CEP holders should classify any change that is neither a notification nor a major change as a minor change. The exception is “editorial changes for which specific guidance is given in the policy document,” EDQM said.

“Any submission of a notification which includes changes not classifiable as a notification will be rejected and the changes will then need to be resubmitted using the correct classification (with associated documentation and fee),” EDQM said.

The directorate highlighted the need to submit minor revisions for revised discussions on impurities and for the submission of nitrosamine risk assessments. EDQM also reminded CEP holders about the need to classify any changes to the synthesis or control strategy that they make to cut or eliminate the risk of nitrosamine impurities or other mutagenic impurities.

EDQM Notice

EMA seeks applicants for third phase of high-risk device pilot, adds HTA element

The European Medicines Agency (EMA) has invited EU-based manufacturers and their authorized representatives to apply for the third phase of the high-risk medical device pilot by 30 June.

EMA is running the pilot to enable expert panels to give scientific advice to manufacturers of high-risk medical devices. The agency asked developers of class III devices and class IIb active devices intended to administer and/or remove medicinal products to apply to take part in the pilot in February 2023. EMA planned to provide free advice on clinical development to 10 companies.

For the third phase of the pilot, EMA is asking applicants to say if they would be willing to have an HTA body observe their project. The agency will tell applicants interested in being observed by a HTA if it will be possible to involve a body.

The next batch of companies will follow the six applications that the expert panels picked in the first round. Those applications focused on fields such as the circulatory system, orthopedics, neurology and dentistry. EMA plans to share information on the progress made in the second round of the pilot “in due course.”

EMA Notice

Ireland’s HPRA moving to slot allocation for certain new product authorisations

Ireland’s Health Products Regulatory Authority (HPRA) is planning to allocate expected submission dates to companies applying for national product authorization and through the decentralized procedure.

HPRA sees the transition towards a system of allocating authorization slots in an agreed-upon month to “facilitate capacity planning for efficient assessment of these applications.” The agency said the new system will “also help maintain access for products on both the Irish and European markets.”

In a new guide to national authorizations, HPRA asked applicants to submit requests “as early as possible and no later than two months prior to the preferred submission date of the application.” The agency has provided a common request form for applicants who need to justify the product's relevance to the Irish market.

HPRA will give successful applicants the next available slot in a six-month time window and ask them to pay a non-refundable booking fee of €1,000 ($1,084) to secure the slot. Once the agency receives the submission, it will offset the booking fee against the full application fee. Slots can only be used for the specific active substances and dosage forms named in the initial application.

HPRA Notice, Guide

EDQM publishes procedures for nitrosamine drug substance-related impurities

EDQM has published analytical procedures for the determination of nitrosamine drug substance-related impurities (NDSRIs) and intermediate-related contaminants.

The official medicines control laboratories (OMCLs) of the General European OMCL Network began developing procedures for the determination of N-nitrosamine contaminants in 2018. Recent reports of NDSRI contamination in medicinal products led the OMCLs to expand their work to cover the creation of in-house procedures for the detection of those impurities.

EDQM has published six procedures stemming from the OMCLs’ work on the impurities. One of the contaminants, NTTP in tablets containing sitagliptin, derives from an active pharmaceutical ingredient intermediate and is detected using a different technique than the other impurities. The other procedures cover products that contain ramipril, fluoxetine, sertraline, duloxetine and hydrochlorothiazide.

EDQM Notice

Euro Roundup: EFPIA warns HTA changes could hamper drug access

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