Euro Roundup: EFPIA slams pharma legislation, offers laundry list of changes

The European Federation of Pharmaceutical Industries and Associations (EFPIA) has published its official response to the European Commission’s draft pharmaceutical legislation. The response brings together all of the trade group’s criticisms and features a long list of recommendations for how to revise the legislation.

Pharma companies and trade groups began attacking the legislation before it was officially published in April 2023 and kept up their criticism once the draft was available to the public. Now, EFPIA has collated all of the concerns into a 62-page response with numerous recommendations, including the need for clarification of expedited regulatory pathways, a stronger and more predictable incentive system and a new definition of “unmet medical need.”

In a shorter document published alongside the full response, EFPIA makes the case that changing regulatory data protection and orphan market exclusivity will accelerate the decline in the European Union’s (EU) share of the global research and development investment. EFPIA also cautioned against the Bolar genetic drug exemption expansion.

“The Bolar exemption expansion beyond marketing approval poses a threat to the enforcement of fully valid [intellectual property] rights, further jeopardizing EU competitiveness. Timely generic competition already exists. This expansion undermines the integrity of the IP system innovators rely on to continue their substantial investment in researching and developing novel therapies in Europe for European patients,” EFPIA wrote.

Other points made in the short document cover pediatric medicines and the environment. On pediatric medicines, EFPIA warned that “the absence of a clear obligations framework in their development will hinder rather than support new developments,” while on the environment it said “the possibility to refuse or restrict marketing authorization solely on environmental grounds poses a significant threat to the established authorization system and could delay or prevent patient access to life-saving medicines.”

On the issue of drug shortages, EFPIA argues that solutions “should be proportionate to the risk, improve cooperation, and leverage digital technologies,” adding that creating “prevention plans for all medicines without a risk-based approach would strain resources for both industry and regulators.”

EFPIA report, Short response

ABPI warns UK drug pricing changes could deter investment in drug development

A report commissioned by the Association of the British Pharmaceutical Industry (ABPI) has raised “serious concerns” about plans to revise a drug pricing and access scheme, questioning the assumptions that underpin the proposals and warning that the changes could limit patients’ access to treatment and clinical trials.

ABPI asked NERA Economic Consulting to examine the economic reasoning and analysis behind plans put forward by the UK Department of Health and Social Care in July 2023. The resulting report identified five ways that the proposals and associated impact assessment are “flawed.” ABPI is pushing for the issues raised in the report to be “fully addressed before any final decisions are taken.”

The five concerns identified in the report are:

the annual growth cap mechanism creates uncertainty for investors
the Life Cycle Adjustment imposes higher rebates on allegedly less competitive drug segments at a later stage of maturity and will likely fail to improve investment conditions
a failure to consider alternative policy options
a lack of transparency in the impact assessment
a lack of independent scrutiny of the impact on business

NERA outlined how the annual growth cap creates uncertainty. “Two per cent is too low to allow for upward pressures on total sales that are driven by factors outside the control of the pharmaceutical industry, such as general inflation and a growing volume of demand for branded medicines from the NHS. This has led to volatile and rising payment percentages in recent years. Volatile and rising payment percentages create uncertainty,” NERA wrote.

In NERA’s view, the uncertainty is “likely to lead companies to launch fewer medicines in the UK market,” an outcome that is inconsistent with the goal of ensuring medicines are available on reasonable terms. “It may also reduce investment in the UK life sciences sector, for example due to fewer clinical trials or if negative perceptions of the UK regulatory environment become the deciding factor in a close decision between two investment locations,” the report states.

Overall, the proposals focus on one of the objectives of the scheme -- limiting growth in the cost of branded medicines -- at the expense of ensuring reasonable terms that account for the cost of research and development, and supporting the life sciences sector, according to the NERA report.

The UK is accepting feedback on the proposals until 10 October 2023.

ABPI press release, NERA report

Q&A update clarifies how Clinical Trials Regulation applies to radiopharmaceuticals

The European Commission has updated questions and answers on the Clinical Trials Regulation (CTR). Officials have added a new query that addresses the impact of the regulation on radiopharmaceuticals.

According to the question, CTR “mentions only some aspects specific to radiopharmaceuticals, it does not specify any documentation regarding exposure to ionizing radiation in clinical trials.” That lack of detail raised the question of whether “sponsors are no longer expected to present such information in the protocol and/or application.”

The Commission responded that the “sponsor is expected to include information on exposure to ionizing radiation in the protocol,” in line with a CTR annex. The specific information that sponsors should include depends on the “situation of exposure.”

In the updated advice document, the Commission divides exposure to ionizing radiation into two main situations -- radiodiagnostic procedures and radiotherapeutic procedures -- and outlines the information that sponsors should provide about each type of application. For example, sponsors of radiotherapeutic trials should describe dosimetric procedures and dose limits to risk organs.

The Commission also updated its answer to a question about the need to obtain informed consent when a minor participating in a clinical trial “reaches the age of legal competence.” Since the age is set by national law and varies from 12 years to 18 years, the Commission has clarified that the information sheet and consent form should be appropriate for the age of the participant.

Q&A document

EMA urges authorization holders to prioritize nitrosamine notifications

The European Medicines Agency (EMA) has told marketing authorization holders that have yet to notify national authorities about nitrosamine impurities to “do so as a matter of priority.”

The EU deadlines for evaluating the risk of impurities, completing confirmatory testing and updating marketing authorizations have now passed. With the final deadline for chemically synthesized medicines passing on 1 October 2023, EMA has updated its nitrosamine impurity page to explain the need for companies that missed the targets to act now. EMA also outlined the need to take precautionary measures to mitigate the risk of nitrosamine impurities.

Officials published the update alongside a revised version of a question and answer document about nitrosamine impurities. The updated document features a revised response to a question about how and when companies should report risk evaluations and confirmatory testing to national authorities.

EMA notice, Q&A document

Euro Roundup: EFPIA slams pharma legislation, offers laundry list of changes

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