EMA Goes Off-Script

The august institutions of the European Union are profoundly attached to orthodoxy, and deeply resistant to stepping out of line, putting their heads up above the parapet, or indulging in speculation. So it was a bit of a surprise to see the European Medicines Agency depart from the tradition of tame complacency in its annual report published in mid-May, and throw caution to the winds with some distinctly non-official reflections from some distinctly non-official sources.  

Peter O'Donnell

"What are the most significant developments in public health and how is the regulatory world changing in Europe and beyond?” it muses, way beyond its core brief of the scientific evaluation of medicines. And it goes on to offer what it describes as some "engaging thoughts" on alignment of data for health technology assessment, what big data might or might not bring, and the potential of immunotherapies.   The "engaging thoughts" are delivered – and this is even more heretical – by outsiders. Niklas Hedberg, the chief pharmacist of the Swedish Dental and Pharmaceuticals Benefits Agency; Professor Jan Schellens, Head of Clinical Pharmacology at the Netherlands Cancer Institute, and – heresy on heresy – a non-European, Frank Pasquale, Professor of Law at the University of Maryland. Hedberg strays straight into a minefield when he says that regulators and HTA agencies share "a common goal to enable safe, efficient and affordable medicines with added value for the patient and the healthcare system, to reach patients in the EU in an equal and timely manner." It may seem anodyne as a statement, but at a time when everyone is watching for signs of a shift by EMA into economic considerations, to see a reference in the EMA annual report to "affordable medicines" is bound to set a few alarm bells ringing. Spiros Vamvakas, head of EMA’s Scientific Advice Office, joins the conversation – and treads also into hallowed territory – with his own insistence on the need to "reduce delays between a medicine’s marketing authorization and decisions on reimbursement." The issue is not far either from the thinking of Schellens in his observations on immunotherapies. "The identification of patients who will show a good response to a treatment is one of the key elements taken into account by healthcare systems in the context of reimbursement decisions."   Pasquale too transgresses, breaching the decorum that restricts most formal EU documents to dull conformity. In his reflections, he freestyles on the risks of contamination or subversion of science from abuses of new technologies, and comes close to shooting the fox of big data. He is, he said, concerned over recent cases of "patients connecting through social networks and deciding to improvise a clinical study where they would take a lowered dose of their treatment to see if it is just as effective as a higher dose. There is also an issue around the quality of the data collected through big-data applications deployed at the population level. We cannot permit an ‘n = all’ mentality that would dispense with established statistical methods," he insists.   Surprising and refreshing candor. But then surprise and candor have taken hold of EMA. Kent Woods, the retiring chairman of the agency's management board, opens his foreword to the annual report with the surprisingly candid remark that  "one unprecedented event overshadowed the agency’s work in 2015" – the removal and eventual reinstatement of Guido Rasi as executive director. He also casually lets the cat out of the bag about how the EMA circumvented the administrative decision to remove Rasi: "During 'this unsettled period,' leadership passed to Rasi's deputy, Andreas Pott, "supported by Guido Rasi as Principal Adviser in charge of Strategy." And Rasi has been reappointed with a mandate that runs to 2020. Look out for more surprises and more candor.   Peter O'Donnell is a freelance journalist who specializes in European health affairs and is based in Brussels, Belgium.