“We as innovators have to make decisions and investments that are increasingly large in a world that is divergent in policies, and synchronisation,” Elias Zerhouni, President of Global R&D at pharma giant Sanofi told delegates at the EMA’s 20th Anniversary Conference in London last month.
“This leads investment boards to make decisions which I deplore. The trend today is to go towards more narrow, more orphan, more specialty drugs at the expense of primary care drugs. If you look at the WHO projections, my thesis is this is going towards the iceberg – we are going in the wrong direction.
"At the end of the day, the diseases where I see approvals coming through affect less than 20% of the population - 80% is not affected."
“I had trouble convincing my organisation to fund the dengue vaccine, one of the first decisions I made [after being appointed President, Global R&D in January 2011]. Why was that? Because you have a 45,000 patient trial which is a huge endeavour and at the same time a very high risk investment.”
In Europe, 327 applications for orphan drugs - those for diseases affecting no more than five in 10,000 - were made in 2014, up from 201 in 2013, and of the 82 drugs approved last year, 17 were for such diseases.
The focus on smaller patient populations is being driven by pharma looking to refill depleted pipelines, coupled with the high prices associated with such treatments, Alistair Kent, Director of the Genetic Alliance UK, told in-Pharmatechnologist.com earlier this year.
Co-captains on the Titanic
To help lower development costs and allow more high-risk investment, he pleaded for the audience to embrace “the total system, not just at one piece of the system,” and called on greater collaboration between both industry and regulators.
Earlier Guido Rasi, Principal Adviser in Charge of Strategy at the European Medicines Agency (EMA) asked Zerhouni: "If Sanofi is the Titanic, are we [regulators] passengers on the ship or are we the iceberg?"
Zerhouni modified the metaphor saying regulators were alongside the company, and the industry as a whole, at the wheel of the ship.
“Frankly if we don’t steer in the same direction we will hit the iceberg and the iceberg to me is the disease burden that is affecting the world – in particular rising burden of chronic diseases and their impact on healthcare systems.”
While he applauded advances by the EMA and US FDA, such as the ongoing shifs to regulatory science and adaptive pathway approaches, he called on his “co-captains” to collaborate further to allow pharma to concentrate investment in innovation.
There are more opportunities to exchange information and communicate than ever and this should lead to regulatory convergence, but unfortunately it is not, he said, and there is still an “enormous cost” imposed on the innovator.
“I spend 20% of our R&D budget trying to mix and match what I need to do between different systems. Pharma companies are global and are not going to develop drugs for one population as they can’t recover the innovation that way.”