EuropaBio cautiously optimistic on clinical trials and personalised medicine for 2013


1/31/13 EuropaBio cautiously optimistic on clinical trials and personalised medicine for 2013 - APM Health Europe

Thursday, 24 January 2013 07:00 GMT
EuropaBio cautiously optimistic on clinical trials and personalised medicine for 2013
by Peter O'Donnell

BRUSSELS, Jan 24 (APM) - Europe's leading biotechnology industry association is hopeful of improvements in the EU regulatory system for clinical trials and personalised medicine, its director general, Nathalie Moll, has said.

Setting out EuropaBio's "top priorities" for 2013, Moll told APM the proposed update of the rules on clinical trials, which is now going through the EU's legislative machinery, offers "an improved, simplified and more efficient regulatory framework."

She said it has the potential to "achieve more harmonisation, transparency and consistency in the approval and conduct of clinical trials across the EU, while maintaining high standards of patient safety and robustness and reliability of clinical data".

This could "go a long way towards strengthening Europe's position as a leader in the research and development of innovative, new medicines".

But she warned: "Greater care should be taken in legislative drafting to ensure that the obligations and requirements are sufficiently precise, clear and unconditional."

Confidentiality was one of the areas she outlined as needing further attention. "Assurance should be provided that personal data, intellectual property, regulatory and commercially confidential information and know-how of applicants contained in the EU database will be protected".

She also underlined the need for "a faster process to extend a clinical trial to additional member states" once a clinical trial authorisation has been granted.

Provisions for regular review - and updating, where needed - of the new rules could also prove inadequate, she said. It is important to ensure that the new regulation "always facilitates the development of innovative medicines and treatments for patients".

In addition, there is no scientific justification for the longer assessment timelines proposed for advanced therapy medicinal products, according to EuropaBio.


EuropaBio is a strong supporter of efforts now being made in the EU "to push the development of personalised medicine up the policy agenda and to engage broadly with stakeholders", said Moll.

It could provide "more value for money due to improved drug effectiveness by ensuring that patients are treated with the medication that their bodies are best able to react to", she says.

Moll sees one of the main hurdles as the difficulties in costing diagnostics: "Implementing personalised medicine in healthcare is potentially a costly investment; it requires testing a whole patient population to identify groups of responding patients or to screen out patients likely to suffer adverse reactions or who need different dosing."

Evaluation of cost-effectiveness of particular molecular diagnostic approaches is also problematic: "Diagnostics are normally supported by analytical performance data and rarely by clinical or outcome data, but in order to perform a health technology assessment you need clinical or outcome data too."

"There needs to be better coordination and a more standardised approach to assessment", to create a more integrated approach to health technology assessment (HTA) and subsequently, market access and reimbursement, she says. "For personalised medicine to be successful and available to the patients most in need, opportunities must be found within the current HTA system."

In particular, she argues, "current models of HTA need to be modified so as not to focus only on short-term solutions, and instead embrace personalised medicine through the concept of overall economic value, instead of lowest price".

The fragmented nature of EU regulation - which currently separates approval of drugs and companion diagnostics - is another challenge that she identifies. "It is very costly to produce better evidence on the clinical utility of genomic tests for cancer prior to obtaining reimbursement", she says, holding out some hope that the current proposal for a regulation on in vitro diagnostics could "pave the way for a more structured and clear regulatory pathway".

EuropaBio is a member of the recently-formed European Alliance for Personalised Medicine (EAPM), and says it will be "contributing actively" to an EAPM conference on 'Innovation & Patient Access to Personalised Medicine' in Dublin in March.


The EU could create new opportunities for advanced industries such as biopharmaceuticals if it manages to agree on adequate funding for its new research programme, Horizon 2020, according to EuropaBio.

But "an effective research and innovation funding policy such as Horizon 2020 must be matched by a coherent and workable regulatory framework that ensures that the ideas and products invented in Europe are also put on the market in Europe to the benefit of our citizens and of our economy", argues Moll.

EuropaBio wants a new public private partnership (PPP) to be created under Horizon 2020 to build on the work of the Innovative Medicines Initiative (IMI), which it sees as having been a "clear driver of innovation within the healthcare sector". A new PPP for innovative healthcare research, said Moll, should be "well-funded, with clear goals and strategies formulated following a sincere reflection on the successes and difficulties confronted by the IMI".


[32825] 24/01/2013 07:00 GMT
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