Working together to get medicines to market

The task of bringing new medicines to market represents a transatlantic regulatory challenge. According to global healthcare giant Roche, the solution may lie partly in reducing the discrepancies between EU and US approval pathways.

WORKING TOGETHER TO

GET MEDICINES TO MARKET

Getting new medicines to patients as quickly as possible is a priority for Roche and the industry. It is therefore a very positive development that new regulatory pathways have been put in place in both the US and the EU that aim to address this need. However, according to Sabine Atzor, head of EU Regulatory Policies in Roche Pharma Development, “Data show that in recent years it seems to have taken significantly less time to get marketing approval for a ‘new medical entity’ in the US. For European patients who urgently need new therapies, for instance to treat cancer, this relative delay in access may have a significant health outcome impact.”

Why the transatlantic discrepancy?

Three factors are likely to be the main reasons behind this. The first is that the US and EU regulatory authorities have different processes in place to address unmet medical needs in serious or life-threatening diseases such as cancer. The US has established a number of pathways – Fast track, Breakthrough Therapy Designation, Accelerated Approval and Priority Review – that ultimately allow for a more rapid review of a drug. In the EU there is Conditional Marketing Authorisation, Authorisation under Exceptional Circumstances, and Accelerated Review.

While these pathways have similarities in many respects, there are still fundamental differences between them leading to different timelines and outcomes in practice. As a result, streamlined cooperation between the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) – which would permit a more aligned approach to new drug approvals between countries – remains a challenge due to a lack of synchronisation. An alignment of procedures would be an ideal situation, and one that Roche would readily support, but this would certainly require a major political effort to make a reality.

Secondly, there are key differences in how decisions are made locally in terms of drug reimbursement, which ultimately determines how and when patients can access them. These decisions remain fragmented in the EU due to responsibilities at national level.

Finally, it appears there may be differences between the EU and the US regulatory authorities on how they apply the precautionary principle in regulatory decision-making. According to Sabine Atzor, “To be able to achieve the ultimate goal of maximum health benefit in this context, excessive risk aversion should be avoided. This requires an ongoing exchange on regulatory, scientific and ethical perspectives across regions to enhance mutual learning – an exchange into which Pharma companies are well placed to add value.”

How can these differences be addressed?

In March 2014, the EMA announced that it was going to pilot a new approach to bringing drugs to market called ‘adaptive licensing’. In essence, this will allow new drugs to be approved quickly based on initial evidence of benefit in a specific (most likely small, well defined) patient population, and then allow multiple additional approvals in other indications to be assessed as additional clinical data become available.

This process is unique as it is intended to bring all relevant stakeholders, including health technology assessment bodies (e.g. NICE in the UK), together at an early stage of the development of a product under ‘safe harbour’ conditions and thus allow early scientific exchange and cross-fertilisation of perspectives. Few candidates have in the meantime been selected for this pilot.

The pilot should go hand in hand with an analysis of key elements of the current regulatory system in Europe such as recently kicked off by the independent Escher platform. “Nearly 50 years after the adoption of the first pharmaceutical legislation in the EU, this initiative certainly provides for an excellent opportunity to ‘rethink’ the implementation of existing regulatory provisions, identify any gaps which need to be addressed, and reflect on the best future solutions,” says Sabine.

How far do the FDA and the EMA work together today?

Strong cooperation between the FDA and EMA is actually not a new topic. What was originally intended as a mutual recognition of inspection reports across the Atlantic in 1999 resulted in stronger cooperation between the two agencies in an exchange of Good Manufacturing Practice (GMP) inspection practices. This will hopefully now be underpinned with a stronger commitment on both sides under the EU–US Transatlantic Trade and Investment Partnership (EU-US TTIP), leading to an even more efficient and risk-based use of resources both by regulators and industry.

Exchanges between the FDA and EMA involving scientific committees have become the norm behind the scenes – particularly during the assessment of a medicinal product, encompassing exchange of assessment reports and review documents. In addition, the two agencies have developed common procedures for applications for orphan drug designation. They also share information on pharmacovigilance, scientific advice and biomarkers. All of these certainly deserve recognition.

Taking in a broader perspective on these topics, European citizens have elected a new European Parliament and a new European Commission has been appointed. Expectations remain high that these institutions will continue to drive the ‘Innovation Union’ agenda. In the area of pharmaceuticals, this will hopefully further enhance bilateral cooperation between the regions. n

This article was published on www.roche. com, where more information on these and other issues can be found.