Dhavalkumar Patel, of the Novartis Institutes for Biomedical Research, said the future of innovation in healthcare lies in finding new medicines that treat several rare illnesses, but this takes longer than simply developing a single drug for a major disease.
Patel told a healthcare conference in Brussels yesterday (3 December), hosted by the Friends of Europe think-tank, that US regulators grant five-year extensions to patent holders, allowing pharma companies to search for new applications for their products. In the EU, the maximum extension is just one year, he said.
"We want to move away from the blockbuster era where one medicine treats one condition and makes a billion dollars. In future, medicines need to be more personalised and should be tailored to reflect age, gender and ethnicity," he said.
He cited the example of a new treatment for Muckle-Wells syndrome, a condition which affects just a few hundred people. This can be treated by targeting a key component of the body's immune system known as the IL-1 pathway.
"We know Mucle-Wells is IL-1 dependent. But this pathway may also be important in several other rare illnesses like juvenile arthritis and some forms of gout and diabetes," said Patel.
Intensive research in this area could deliver the potential for treating tens of millions of patients, making it attractive to investors.
"It's just too expensive to develop a drug for a condition that affects 1,000 people worldwide – that wouldn't be sustainable. But many conditions are influenced by the same biological pathway so if one molecule could treat several rare conditions, it becomes sustainable," he said.
Short patent extensions a 'roadblock' to research
According to Patel, one "roadblock" to innovation of this kind is the length of patent protection provided in Europe. "We'd like additional years to develop other uses," he said.
Many patients are still not treated with the drugs available today, Patel noted, and there are populations that the industry has neglected. "We need to change that," he said.
"We are trying to develop medicines for smaller indications where there is an unmet need. To make this worthwhile, companies are focusing on the underlying mechanisms of diseases in the hope that they can uncover a drug which could treat multiple illnesses," Patel told the conference.
Patel was merely voicing a change of attitude found across the pharmaceutical sector, where companies like Pfizer have been snapping up smaller biotech firms focused on niche areas.
The shift in approach comes at a time when blockbuster drugs for cholesterol, stroke and heartburn continue to rake in billions of euros in profits, but there are few new blockbusters in the research pipeline.
Nonetheless, this trend could be good news for patients with rare diseases, which have often been neglected as research funds were poured into the search for curing illnesses affecting millions of people.



