Paris, 7 July 2020

The pharmaceutical strategy for Europe, to be published by the European Commission (EC) before the end of the year, should centre on transparency and robust evidence, support public health priorities and ensure evidence-based independent policy elaboration, assessments and surveillance of medicines and full access to clinical data.

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Prescrire’s response to the pharmaceutical strategy roadmap

The up-coming pharmaceutical strategy for Europe (the “Strategy”) should be based on overarching public health and general interest goals as well as on a shared commitment to transparency. Prescrire supports the overall goal of ensuring Europe’s supply of safe and affordable medicines (cf. below) as long as they have been proven to be effective.

1. Center Europe's pharmaceutical strategy around transparency and robust evidence

a) Strengthen the regulatory system for marketing authorisations by basing it on robust clinical trials and data

Marketing authorisations should rely on reliable, robust evidence. Prescrire calls on the EU to strengthen the regulatory standards and rules on drug evaluation. Solid evidence, based on comparative randomised clinical trials designed to meet health needs with meaningful endpoints should be required for marketing authorisations. Only such trials are fit for purpose and would avoid the release of drugs with unknown added therapeutic value, which may prove to be more harmful than existing products.

The Covid-19 pandemic illustrated the need for the conduct of large, well-designed, randomised clinical trials, which are the only way to provide robust data for drug evaluation, decision-making and clinical practice. The timely availability of genuine comparative trials data against standard treatment at the time of drug approval would represent an undeniable support towards an access to safe, effective and affordable medicines. As stated in the roadmap (page 1), to ensure sustainable health systems, new therapies need to be clinically better than existing alternatives as well as cost effective.

Public disclosure of clinical data and information, including clinical trial results and clinical study reports, is literally of vital interest for patients, healthcare professionals, researchers, health authorities, HTA bodies and independent drug bulletins. Prescrire calls for a swift implementation of the clinical trials Regulation.

b) Ensure that accelerated authorisations procedures are exceptions, and that they do not become the rule

Continuous trends of flexible and accelerated authorisations increase the uncertainty regarding the clinical value and safety of the drugs authorised that way.

Accelerated authorisations lead to a shift from pre-marketing approval evidence-collection to post-approval assessment. However, years of experience have shown that manufacturers fail to honour post-marketing commitments. Failure to respect post-marketing commitments should not be tolerated any longer: it should lead to a withdrawal of marketing authorisation.

Great hope is placed on the use and utility of “real life data”, “big data” and artificial intelligence to accelerated approvals yielding to faster access for patients. We call on for caution and urge European and national regulators not to weaken marketing authorization requirements by shifting the provision of evidence to real world data (after marketing authorization). As experienced during the Covid-10 pandemic (retraction of study in The Lancet) it is of outmost importance to consider very carefully the source and quality of big data, and to ensure access to the basic data and to the process used to analyze them.

Once marketing authorization has been granted, years can go by before studies of sufficient methodological quality are obtained and that even once a drug is proven to have severe or even fatal effects, it often takes months, if not years, to withdraw its marketing authorization.

Accelerated marketing authorisations should only be used as an exception, when genuine unmet medical needs are at stake, so as to prevent unnecessary exposure to avoidable harm.

2. Support public health and needs-driven research priorities

Unfortunately, the current EU framework of incentives and rewards that supports R&D, in particular for orphan drugs and paediatric medicines has either been abused or did not lead to new or better medicines. This legislation needs to be amended to put an end to the abuse.

Market failures (e.g. the lack of new antimicrobials, neglected diseases) show that the current pharmaceutical business model does not provide solutions for all public health needs. Policy makers should take this into account and explore other business models and solutions to support R&D projects related to public health needs, such as: collaboration with European academia centres, independent clinical research and the not-for-profit sector (e.g. DNDi).

In collaboration with the Member states, the Strategy should initiate a process to set up a list of priority medicines to be developed. European funds should be limited to the development of medicines for truly unmet public health needs. Granting of public funding should be attached with conditionalities: guarantee of affordability and availability, R&D costs transparency as well as price setting transparency. The European Commission should include such conditions in all forms of public funding and/or public-private partnership in pharmaceutical research. 

The Covid-19 pandemic pointed out the issues faced when trying to set up large European multi-site clinical trials. The Strategy should consider steps to be taken to facilitate the collaboration of national research institutions and to ease the conduct of large multi-national randomised clinical trials.

3. Manage and improve safety, including safety for medicines already on the market

Prescrire calls on the Commission to include the safety of medicines in the Strategy, by including the following:

• better prevention of errors linked to the use of existing drugs, notably through better packaging and product information (e.g. adapted packaging for specific patient categories like elderly people or children; safe dosing devices,…);
• better prevention of the adverse effects of existing drugs through more effective and transparent pharmacovigilance; and 
• improved labelling and patient information leaflets (e.g. providing more useful information for clinical practice including comparative information with other treatment options; clearly stating what is known, unknown and what is still under investigation, as well as report on the evidence and robustness of the data underlying the authorisation;…).

4. Focus on the availability and affordability of medicines

a) Ensure a sustainable access to affordable medicines through fair pricing

Prescrire calls on the Commission to include the following points in its Strategy, in order to improve the affordability of medicines:

• reconsidering IP rules (including SPC) and their impact on prices and competition and encourage the use of TRIPS flexibilities;
• strengthen the regulatory system for marketing authorisations by requiring the conduct of comparative randomised clinical trials (accelerated marketing authorisations should be strictly reserved for exceptional situations);
• requiring total transparency on the prices paid and discounts granted; transparency on cost of R&D;
• European cooperation on HTA should be supported. HTA plays an important role in the decision-making process on pricing and reimbursement of health technologies. HTA should be based on a robust methodology, rely on high standards and be carried out in an independent and transparent manner;
• reducing waste by increasing the use of generic drugs and biosimilars and encourage rational use of medicines.

b) Prevent medicine shortages

Medicine shortages are not a new problem. Among the different actions already under discussion, including the need for diversification of supply chains, the Commission should recall and clarify the legal obligations of marketing authorisation holders in respect to a timely delivery of critical medicines orders (Directive 2001/83/EC, article 81). If needed these rules should be reinforced. In case of non-respect of the obligations, appropriate sanctions should be applied.

5. Ensure independence of the European Medicines Agency and the EU's decision-making process

The announced revision of the European Medicines Agency (“EMA”) fee system is an excellent opportunity to put in place public funding for the EMA. The current funding system based on industry fees undermines the independence of the agency. To guarantee EMA's independence, and prevent sustainability issues due to fewer applications and subsequent fluctuations in fee revenues, any direct financial relationship between the Agency and the pharmaceutical industry should be avoided. This could be achieved by channelling industry fees to the European Commission, and by restructuring EMA's budget so that fees would make up but a small proportion of its overall budget.

The EU and the Member states should guarantee adequate public funding for the core missions of the EMA to ensure that the business interests of pharmaceutical companies do not override public health interests. Situations exposing the EMA and European Commission staff and experts to the risk of institutional capture and lobbying pressures that could compromise their impartiality and objectivity should be avoided.

Prescrire maintains that transparency, evidence based and independent policy elaboration, assessments and surveillance of medicines free from vested interests and full access to clinical data are a priority for the benefit of patients, healthcare professionals and the public in general. As such, they should be reflected in Europe's up-coming pharmaceutical strategy.

©Prescrire July 2020

> Download Prescrire's response (pdf, 151 Ko)

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