The European Medicines Agency (EMA) will this week publish the much-awaited results of a pilot project on the early marketing authorisation of drugs, EURACTIV has learnt.
In March 2014, EMA launched the “adaptive pathways” pilot project to explore ways to speed up patients’ access to new drugs by gathering evidence through real-life use, after they have been placed on the market, to supplement data from clinical trials.
“We are planning to publish the report on the adaptive pathways pilot next week,” Rebecca Harding from EMA’s Media and Public Relations Service told euractiv.com on Friday (29 July).
Previously, the European Consumers’ Organisation (BEUC) blamed EMA for a lack of transparency, as there has been no public debate to date. “The EMA still has not published its report about how the project has been going so far, which was due in early 2016,” BEUC said in a policy paper.
A growing number of patients face diseases that can be treated with medicines that are not yet available on the pharmaceutical market. The purpose of this project is to speed up the procedures of approving new drugs for this kind of patients under the existing regulatory framework for medicines.
According to EMA, the concept applies primarily to treatments in areas of high medical need, “where it is difficult to collect data via traditional routes and where large clinical trials would unnecessarily expose patients who are unlikely to benefit from the medicine”.
The exception, not the rule
BEUC is concerned that this new fast-track procedure could expose patients to unnecessary health risks, as these medicines would be put on the market before the available data on their safety is complete.
European consumers are also wondering what the added value of such a project would be. EU pharmaceutical legislation already includes several provisions to foster early access to new medicines.
According to BEUC, fast track procedures should be “the exception, not the rule”. The organisation said that the scope of the procedure, as well as the criteria for selecting the products to which it will apply, remain unclear.
“If this fast-track approval procedure is meant to serve only the drugs that are urgently needed, the EMA should clarify this scope. Otherwise drug makers could start requesting hasty approvals for other drugs and expose consumers to unnecessary risks,” BEUC said. It added that fast track approvals should only be used for a very limited range of medicines and only when there is no other available alternative.
On a government level, EU health ministers recently (17 June) called for a more cautious approach on all early access schemes, thought they did not specifically mention the ‘adaptive pathways’ programme.
“The exact conditions for the inclusion of innovative and specialised medicinal products in the existing schemes of early marketing authorisation could be further clarified in order to improve transparency, to ensure a continuous positive benefit risk balance of medicinal products put on the market under special conditions and to focus on medicinal products of major therapeutic interest for public health or to meet unmet medical needs of patients,” EU health ministers said in their conclusions.
BEUC also raised concerns about the issue of post-marketing control of such medicines, as part of the data gathered during clinical trials will be replaced with the collection of “real world evidence data”. Practically, this means that additional data will need to be provided by pharmaceutical companies.
Referring to several studies, the EU consumers’ organisation said that the current system monitoring the drugs that have already entered the market was remarkably weak.
“Some studies indicate that the current system does not promptly react to ineffective medicines, not even when the use of a drug is associated with increased mortality […]. Withdrawal of products suspected of adverse reactions (ADR) has not improved over the last 60 years,” BEUC said.
According to the European Commission, approximately 5% of all hospital admissions are caused by ADR and about 5% of hospitalised patients suffer from ADR.
“In the EU, the average time to conduct post-marketing studies attached to conditional marketing authorisation is four years. There are delays or discrepancies in the fulfillment of these obligations in more than one third of the authorisation procedures,” BEUC added.
EU health ministers also raised the issue of post-marketing control.
“The post-market compliance with certain obligations for marketing authorisation holders is not always optimal. This may result in independent research data and information from patient registries not being structurally generated, collected and made available for research and proof of effectiveness and safety,” health ministers noted.
The European Parliament is also working on an own initiative report on access to medicines. A vote in the ENVI committee is expected on 7-8 November.
- 7-8 November: European Parliament's ENVI committee expected to vote on access to medicines report.
- European Commission: Regulation on pharmacovigilance of medicinal products for human use
- European Council: Council conclusions on strengthening the balance in the pharmaceutical systems in the EU and its Member States
- European Parliament: EU options for improving access to medicines
- European Medicines Agency: Support for early access
- European Medicines Agency: Adaptive pathways
European Consumers' Organisation