You would think the sheer number of diseases and medical should yield plenty of opportunities to come up with new treatments. Yet there is a worrying lack of new billion-dollar "blockbuster" medicines in the pharma sector.
The recent tragic outbreak of Ebola in West Africa, which showed that there were only a few experimental treatments available for a condition first diagnosed decades before, has shone a light on some of the reasons the pace of innovation in the industry may be slowing.
"Historically, diseases of the poor have not been as well researched as diseases of the rich," Dr Ben Goldacre, author of Bad Pharma and leading pharma activist, told CNBC.
The rushing forward of untested medical treatments during the Ebola outbreak was the most interesting aspect of the epidemic, particularly if it leads to similar accelerations of the clinical trial process elsewhere, according to Goldacre.
While the number of new treatments hasn't really dropped off, but they are being developed for smaller numbers of people, and it is still difficult and expensive to get them to market.
Regulation is more onerous than ever, and the typical final-stage submission of a medicine to the U.S. Food and Drug Administration runs to more than a million pages.
Molecular level
The landscape of medicine is changing, with a move away from tradition small molecule drugs (that's pills to you and me) to biological drugs: antibodies or human proteins. These have the advantage of being easier to prove that they might work in early stage trials – but at the moment there are certain conditions, such as Alzheimer's, which they won't be able to treat because they don't act directly on cells.
There are also issues around the lack of available subjects to test early-stage drugs for diseases which don't already have effective treatments. There is also a general movement away from animal testing on ethical grounds – plus many of these conditions are difficult to model in animals.
Beyond the threat of epidemics such as Ebola, the growth of drug-resistant infections and the lack of new antibiotics coming to market is one of the most worrying side-effects of the apparent lack of innovation. Antibiotics don't just aid common, relatively mild conditions like skin conditions or minor chest infections, they are also needed for more serious conditions like tuberculosis and to help minimize the risk of infection during many important surgeries. Antiobiotic resistant bacteria already cause 2 million illnesses and 23,000 deaths in the U.S. annually, according to the Centers for Disease Control and Prevention.
It is likely to be decades before the necessary changes to the industry model are made, according to Richard Seabrook, head of business development at the Wellcome Trust, the U.K. non-profit research organisation.
"What we need is better understanding of the disease process," he told CNBC.
"Pharmaceutical companies need to be better-informed about what molecules are likely to work."
Sharing and caring
One way the industry is trying to tackle the problem is by sharing more information about their clinical trials and early-stage research – a tricky business in an industry whose lifeblood is intellectual property, and where companies fiercely guard early-stage research.
An increasingly vocal section of the industry is arguing for greater co-operation and sharing of data.
Dalvir Gill, chief executive of TransCelerate, an industry joint venture which encourages co-operation over things like the designing of clinical trial processes or the computer system into which clinical trial data is entered.
"It's no longer a sustainable system whereby the amount of money spent is in sums that we can hardly imagine, just to get one drug to the point where it can go to market," Gill told CNBC.
"We have shown that we can collaborate, if we get the right forum."
Goldacre, a noted industry critic who leads the All Trials movement which campaigns for all clinical trial results to be disclosed, said: "We need more and more companies to take this more seriously."
It is the lack of affordable innovation rather than innovation per se which is the problem, according to Julian Remnant, partner and life Sciences R&D lead at Deloitte.
"It's an industry without a competitor or peer," Remnant pointed out.
Roche's ongoing bitter battle with the U.K.'s drug pricing body NICE, over whether the U.K.'s health service will pay for its £90,000 per patient breast cancer drug Kadcyla, may help establish the potential rewards of future innovation, Goldacre said.
"Roche is negotiating a new norm for what you can charge, where the cost of the drug isn't the manufacturing, it's the cost of developing it," he said.
- By CNBC's Catherine Boyle
