EMA celebrates 20 years, looks to complex future

LONDON – "The best way to celebrate is to start preparing the European Medicines Agency [EMA] for the future."

This was the call to arms from Guido Rasi, principal adviser in charge of strategy at the EMA, opening the agency's 20th anniversary celebration conference here on Wednesday.

Three words – complexity, opportunity, patients – sum up the key issues the EMA needs to address, Rasi said.

The future of developing medicines will be more complex, with more sophisticated molecules, cell-based therapies, delivery technologies, novel diagnostics and new methods for identifying patients.

At the same time disease classification is becoming ever more complex too.

As opportunities that the EMA needs to prepare for, Rasi cited personalized medicines and the potential to incorporate technologies such as big data and internet connected sensors into drug development strategies.

On patients, Rasi said one of the biggest achievements of the EMA is what it has done to bring patients into the regulatory process, to generate data and contribute to risk assessments.

The thread around these three is regulatory science, which the EMA and other agencies must advance and promote in the future.

"This brings everything together and teaches us how to evolve," Rasi said.

This message – that new regulatory science is needed to take medicines oversight forward in the future – was reiterated by other speakers including Margaret Hamburg, FDA commissioner, delivering a congratulatory speech, Elias Zerhouni, president of global R&D at Sanofi SA, addressing the conference on behalf of the industry, and Mark Walport the UK government's chief science adviser, speaking from the perspective of public health.

Hamburg pointed to dramatic shifts in terms of regulation that are arising as a result of the globalization of the pharmaceutical industry. This calls for stronger partnerships among regulators to share data, create standards and extract maximum value from strained regulatory budgets.

"We have to work collaboratively to make sure the best possible science underpins every facet of our work," Hamburg said.

The knowledge and tools needed to assess products and for the development of new products are extremely important, but at present there is a gap – their importance is under-appreciated and under-resourced. "It is time to come together to advance the field of regulatory science," said Hamburg.

So many of the questions the FDA and the EMA face are the same – the two agencies should cooperate, for example in pediatric drugs and rare diseases, to avoid duplication.

"When there are limited populations, you need global harmonization of development efforts," Hamburg said.

Zerhouni endorsed Hamburg's view, saying "There is no question that there is a need to work on regulatory science."

One example is in identifying biomarkers to be shared across the industry in a new "preclinical commons." This will require regulators to develop a consistent approach to assessment and validation.

"Biomarkers are only as good as the understanding of the fundamental underlying biology," Zerhouni said.

Regulatory agencies should use research to develop new ways of regulating, said Zerhouni. "Be a creative engine. If you don't keep pace, you will slow things down. If regulatory science lags behind it will definitely be a negative."

Zerhouni said that when he was head of the U.S. NIH, overseeing a $30 billion per annum research budget, he was shocked at how little investment there was in regulatory science. "There is a huge gap: you become a regulator without doing research in regulation," he said.

Walport reinforced the need for regulatory science and said he wanted its scope to be wider than nuts and bolts tools for regulatory reviews or pharmacovigilance. The fact is that patients are impatient, and for regulators to do their jobs properly, they need to understand the values of patients.

"Regulatory science needs to be about social science as much as biological science," Walport said.

Hamburg is about to bow out after six years at the head of the FDA. She said it is gratifying to see how the FDA/EMA relationship has evolved in that time. "Together we have made valuable progress at a critical point in time."

Hamburg added that her decision to step down was not an easy one – she remains committed to the cause and there is more to be done. However, "I leave behind a stronger and more engaged FDA," Hamburg concluded.

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