LONDON – The EMA is ready and prepared to go ahead with its policy of releasing clinical study dossiers in full once a product receives marketing approval and will publish the first report in the second half of 2016.

This "pioneering approach" to clinical trials data transparency will be "of benefit to all," said Guido Rasi, EMA executive director. Academics will have access to data for research and companies will save time in development by having information about previous trials and what did and did not work.

Rasi told BioWorld Today he believes there is support from the majority of industry for the EMA's approach. While there are technical issues to sort out in terms of ensuring patient privacy, the approach for handling commercially confidential data is now settled.

"I can't be 100 percent sure companies will be 100 percent happy. But I'm quite sure that after a while everyone will be sure this is the way to go," Rasi said.

Rasi made these comments as he set out his vision for the next five years at the head of the EMA. On Nov. 16 he began a new term in office after a one-year suspension from the post over a row about the objectivity of his initial recruitment in 2011. (See BioWorld Today, Oct. 2, 2015.)

The suspension had been "a year of transition" and "time to reflect," said Rasi, noting, "there are more changes happening in medicines development today than in my 35 years in public health, either as a doctor, a researcher or a regulator." The EMA has to be prepared to handle these changes, of which Rasi specified three in particular.

First, understanding of the human body and the underlying science has "vastly evolved" and regulators need new approaches to handle this and to steer R&D toward unmet medical needs.

Second, the EMA needs to respond to the globalization of drug development and manufacture. Eighty five percent of medicines on the market in Europe have at least one step in their production process outside Europe and as a result, "supply chains are complex and extremely vulnerable to disruption," Rasi said.

The third key shift is the mounting threat to the sustainability of Europe's publicly funded health care systems. In addition to an aging population, the increasing incidence of chronic disease and higher public expectations, the rising cost of medicines is a factor here.

"The pricing of medicines is clearly outside our remit, but I feel we must contribute to ensuring sustainability," said Rasi. A more efficient regulatory system might be one route to bringing down the cost of development and ensuring medicines are affordable.

The EMA will frame its response to the explosion of biology, to globalization and to the fragility of health care systems, around five policy building blocks, of which clinical trials data transparency is one.

Another building block will involve putting the focus on drugs with the potential to make real improvements in patients' lives, and without compromising safety, to speed up the regulatory process and access.

This will involve mechanisms such as adaptive pathways, through which the EMA aims to grant early approval in a subgroup of patients and then extend the label as real world evidence of how a product performs when it is on the market accumulates.

Another scheme, Prime (Priority Medicines), to be launched next year, will provide additional support in the form of early dialogue with EMA experts and chaperoning through the regulatory process for drugs with the potential to address unmet medical needs. (See BioWorld Today, Nov. 11, 2015.)

The EMA also intends to spur research in areas of unmet need, with Rasi citing antibiotics as one area of focus.

In addition, EMA will seek to speed up access by intensifying its relationship with national health technology (HTA) assessment bodies, so that trials can be designed not only to deliver clinical proof of efficacy, but to answer HTA questions about incremental benefits and cost effectiveness.

A third building block will be increasing patient involvement, "in all aspects of our work" and in "ensuring their views and needs are taken into account," said Rasi. Patients should have support to contribute in this way. "It's a true problem. We need [patients] to have time to become a little bit expert and to go beyond the emotion of being a patient," Rasi said.

It will be important to ensure patients are independent of external influences, and given this Rasi suggested that there should be public funding to provide the support patients need to get involved in the regulatory process.

Fourth, EMA will look beyond the data amassed in formal clinical trials and deploy all the information available to study the impact a drug has in real life. The agency will use its power to mandate the collection of in-market data and develop improved methods for assessing risks and benefits across a drug's life span.

Finally, EMA will make greater efforts to collaborate with its international counterparts and with national regulatory bodies in Europe, to foster the best use of resources and offer expertise to countries with less-developed regulatory systems. Rasi said the EMA is in the process of finalizing a joint strategy with Europe's national regulators on pipeline monitoring and horizon scanning to help with resource allocation.