Changing the Context of European Pharma

It seems only yesterday that the future for European pharma was personalized  medicine. The European Union first gave the term official status in a formal paper in 2008, entitled a Renewed Vision for the Pharmaceutical Sector, in which the Commission included a section on ‘Towards more personalized  medicines’. A high-profile conference in Brussels in 2011 constituted a groundbreaking attempt at capturing the concept for the EU. Medicine would never be the same again. No more blockbusters for huge populations; instead, stratification of patients would transform care. And around the same time, a plethora of new groups began to appear, all claiming to hold the key to developing this emerging discipline.

The EU, which likes to see itself as a leader in such things, had already promised –in its 2008 communication - to bring some further definition to the concept, with an official paper on the subject. But that took much longer than expected –until 2013 - to struggle into existence, and its birth provided something of a disappointment. In form it had been downgraded merely to a working document for European Commission staff, in designation it had shifted to acquire the bizarre title of "the 'omics paper", and in content it amounted to little more than a statement of the dilemmas linked to the subject, and at best a shopping list of what might be done – without much in the way of plans for action.

Meanwhile, other discussions were getting underway under a different heading – alternative pathways. And over the last couple of years it is this approach (often under its fuller title of Medicine adaptive pathways to patients – MAPPs) that has become the term on everyone's lips in Europe. Driven largely by the European Medicines Agency's work in bringing together companies, regulators and payers in a pilot project with half-a-dozen real cases, MAPPs is being hailed as the new dawn of pharmaceutical development, the new holy grail of healthcare.

Or at least it was. Then came President Obama's state of the union address in January, with his announcement of an initiative in precision medicine. That set the cat among the European pigeons, mainly because it looked like (and was even expressed like) a direct threat to every other drug-producing region in the world, and partly because it gave a new jolt to the terminology. Stratified was out, a long time ago, it seemed. Personalized  had been and almost gone. But even MAPPs was pushed aside by Obama's seizing of the high ground. Now Europe is looking around uncomfortably wondering if it is backing the wrong horse in the innovation sweepstakes.

How much of this debate is just, as Hamlet might say, "Words, words, words"?

Who could offer a better answer than an official who has for years been in charge of the personalized  medicines unit in the European Commission and is now in charge of an organisation with a big stake in MAPPs? Step forward Irene Norstedt, who is now, since the end of 2014, the acting executive director of the Innovative Medicines Initiative – the EU's multi-billion public-private partnership in drug development.

For Norstedt, MAPPs is "just one component of personalized  medicine". She sees personalized  medicine as much broader, covering everything from basic research to patient access, developing clinical trials methodology for stratification of patients, investigating what sort of biomarkers are able to identify susceptible patients… In her view, "personalized  medicine is a medical approach that starts with the patients, and science drives the strategy". Norstedt sees the battle to find - and validate - new biomarkers as the bottleneck: it is the biomarkers that crucially allow better stratification of patients. "When you have those you can choose your patients, and the risks are reduced for going further down the MAPPs route".

But she admits that the differences in terminology don't help. Obama's decision to use the term "precision medicine" has merit, Norstedt recognises. "'Personalized  medicine' can give the misleading impression that it's medicine personalized  for individuals", she says.

"What's in a name?", as another Shakespearean hero mused. Quite a lot, in drug development…