European Regulatory Roundup: Report Calls to Minimize Amount of Data Generated by Sponsors (28 November 2016)

Regulatory NewsRegulatory News | 28 November 2016 |  By 

Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news.

EU Report Calls on Regulators, HTAs to Minimize Sponsor Data-Generation Requirements

The European Parliament’s policy department has published a report recommending regulators and health technology assessment (HTA) bodies take steps to minimize the amount of data demanded of drug developers. Members of the Directorate for Economic and Scientific Policies made the proposal in a report on the links between pharmaceutical R&D models and access to affordable medicines.

Most of the more than 130-page report describes the current drug R&D and market access situation, plus the initiatives already in place to try to optimize the process. This information will support discussions at the Committee on Environment, Public Health and Food Safety (ENVI), the European Parliament group for which the report was written.

In the final few pages, the authors put forward some policy proposals for ENVI members and their peers. The suggestions include ideas that could affect regulators in Europe.

“Attention must be paid to commercially unattractive medicines with a valuable unmet medical need. However, regulators should avoid making any additional requirements for data collection for new medicines as this could result in a decline in productivity of new medicines,” the authors wrote. “This means that when [randomized controlled trials] aiming for more subgroups to demonstrate patient benefit are combined with increasing data requirements to demonstrate safety and efficacy, pharmaceutical companies have to collect more data on more patients.”

The authors are concerned this could both increase the cost of developing a drug and reduce the likelihood of it succeeding. These forces could make drug developers wary of advancing certain drugs, resulting in longer waits for effective treatments for some unmet medical needs.

Such concerns are underpinned by a belief that clinical data requirements are already weighing on sponsors. This belief stems from an analysis of the reimbursement landscape in Europe. The report’s authors are the latest observers to suggest HTAs and drug developers alike would benefit from the standardization of the clinical data requirements for reimbursement. Done correctly, the authors think standardization could save governments and drugmakers money while also shortening the path to market for new products. 

The suggestions feed into a debate about how best to ensure patients have access to new medicines at a time when national healthcare budgets are struggling to keep up with the rising cost of looking after aging populations. A separate report published by ENVI last month called for harmonization of pricing and reimbursement criteria. 

ENVI Report

CHMP Starts Consultation on Depression Drug Clinical Trial Guidelines

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has begun the process of revising the guideline covering clinical trials of depression drugs. CHMP thinks the text needs revisions to the definition and identification of partial responders.

Changes to sections about partial and nonresponders with major depressive disorder (MDD) are at the top of the short list of planned revisions. CHMP framed the changes as a way to ensure the guideline supports development of drugs for the 50% of MDD patients who experience incomplete responses or resistance to treatment. Doctors are looking to combination treatments to help these patients, and CHMP wants trial designs to reflect this clinical practice trend. Changes to the diagnostic criteria for subpopulations of MDD patients are also planned.

CHMP wants to use the revision process as an opportunity to reflect the interest in treating symptoms associated with MDD. Specifically, CHMP thinks new guidance is needed on how to design trials to support claims for efficacy in MDD patients in terms of cognitive function. This will entail defining cognitive disturbances in depression and detailing appropriate trial design strategies.

The proposed changes come relatively shortly after CHMP last updated the guideline. CHMP adopted the most recent version of the guideline in 2013. The need to revise the text three years later reflects the ongoing difficulties in treating depression, fast-changing clinical practices and the move toward treatment of specific symptoms. That shift toward symptom-specific treatments was accelerated by the fifth edition of the Diagnostic and Statistical Manual of Mental Disorders, which was published two weeks before CHMP adopted the current guideline.

CHMP has also published a draft concept paper on the need to revise the guideline on clinical trials of migraine treatments. The current guideline, which came into force in 2007, lacks details of how drug developers can generate data to support a claim of treatment of chronic migraine. Botox is approved in the United States and Europe as a treatment for chronic migraine, but there is hope for the development of new drugs. The condition was added to the International Classification of Headache Disorders in 2013

The comment periods for the depression and migraine drafts close, respectively, on 28 February and 31 January.

Concept Paper, More

EMA Details New Opportunities to Reduce Animal Testing

The EMA has published a draft reflection paper detailing opportunities to replace, reduce and refine animal testing. The document lists the main animal tests requested by regulators today alongside current and future opportunities to adopt alternatives.

A handful of CHMP working parties and committees have put together details of tests in their fields to build on the 2014 draft guideline on regulatory acceptance of testing approaches to advance 3R: replacement, reduction and refinement.

For example, the CHMP Safety Working Party has outlined its take on 3R opportunities and repeated dose toxicity tests. Today, drug developers can perform the test on one species of animal if they can clearly justify the decision to the regulator. When CHMP adopted guidance on the test in 2010, it said two species, including one non-rodent, must be used. The new draft paper suggests exposure-based setting of the maximum tolerated dose could provide a further 3R opportunity.

Similar current and future opportunities are listed for an array of regulatory tests, although the scope to adopt 3R practices is greater in some areas than others. As it stands, there are no 3R opportunities for carcinogenicity and reproductive tests. Regulators are working to create opportunities, though. CHMP highlights work to define conditions under which two-year rat studies can be eliminated as a future opportunity in carcinogenicity testing.   

The regulator is accepting feedback on the draft until the end of May.

Reflection Paper

EMA Updates Post-Authorization Procedural Advice About Orphan Drugs

EMA has updated its post-authorization procedural advice for users of the centralized procedure to clarify its stance on matters relating to orphan drug designations. The new sections cover the maintenance of orphan designations when filing for type II variations and extension applications.

Whether a company is applying for a type II variation or extension application, the regulatory process and factors to consider are the same.

If the application includes a new orphan therapeutic indication, the sponsor should give the Committee for Orphan Medicinal Products (COMP) a maintenance report. COMP wants to confirm the maintenance of the orphan designation before it rules on a new indication. 

The process is slightly different for new indications that fall within an already authorized orphan status. In such situations, COMP will assess whether the variation raises “justified and serious” doubts regarding the meeting of orphan designation criteria. Sponsors should include justification to show no such doubts exist when submitting their variations.

EMA Advice


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