The days of the $1 billion blockbuster drug are over, according to a senior industry researcher. Big pharma is now investing in finding multiple uses for medicines developed to treat rare diseases, but is pushing the EU for regulatory changes to make research worthwhile.
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 longer “data exclusivity” periods than the EU, allowing pharma companies to search for new applications for their products. In the US, the period of data exclusivity is ten years; in the EU, the maximum period is just five years, 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.
Regulatory environment a ‘roadblock’ to research
According to Patel, one “roadblock” to innovation of this kind is the length of data exclusivity provided in Europe. During this period, competitors cannot refer to the safety, efficacy and quality of a new drug for the purpose of obtaining marketing approval for a rival product.
Currently, the European regulatory framework allows for only one year of additional exclusivity for any new therapeutic indication of a molecule that has already been authorised. In the US, an additional three years can be given, at the discretion of the regulator.
“We’d like additional years [in the EU] 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.
Christoph Thalheim, secretary-general of the European Multiple Sclerosis Platform (EMSP), said patient groups would welcome all kinds of research, whether it is directed towards common or rare diseases.
He said people with rare diseases have been frustrated that treatments for their illnesses have not always been seen as worth investing in. Discussing the prospect of finding new applications for existing medicines, he highlighted growing interest in a blood pressure treatment which could help MS patients.
Elizabeth Docteur, an independent health consultant formerly of the OECD, said innovation is critical in medicine but noted that the sector is markedly different to other industries.
"When it comes to computers, innovation has made them faster and cheaper. But in healthcare, medicines have become better but are more expensive, and they take up an increasing proportion of spending," she said.
Docteur said spending "smarter" will be crucial in order to get more in return for investment in medical innovation. She pointed to differences in drug costs across the EU, and said 80% of the price of medicines goes to the innovator in some countries, while in others, patent holders get just 50%. Where a large proportion of the price goes to wholesalers, retailers and the taxman, it provides less incentive for innovation.
According to Docteur, governments should do their own cost-benefit analysis when setting a price for new innovative medicines. National authorities tend to follow the price of a new product in countries where it is initially launched, but companies have been savvy enough to select countries where they will get a favourable price for the launch. She said the EU can do more to support research comparing the true added value of a new drug.
Bulgarian liberal MEP Antonyia Parvanova said much was being done to support innovation in the health sector, including the EU's Innovative Medicines Initiative and funding from the Framework Programme for Science and Research (FP7).
She said in Israel, 10% of the health budget is earmarked for research and European governments could take a leaf out of Israel's book on this score. Parvanova also questioned the rationale of the French government spending €1.5 billion on swine flu medication while devoting just €770 million to cancer research.
"When we talk about added value, we have to ask what will make a better contribution to public health so that we can strike the right balance," she said.
On the issue of extending patents, Parvanova said any more in this direction would likely meet with resistance from generic companies who rely on off-patent medicines to make money.
She was also critical of EU health ministers' failure to reach agreement on a proposal for cross-border healthcare at their meeting in Brussels on 1 December. "This was a very bad decision and sent the wrong message on the day the Lisbon Treaty came into force," she said.
Wim Oosterom of PricewaterhouseCoopers said the discussion on innovation in healthcare should be broadened to include organisational innovation. He said investment in monitoring technology and efforts to increase patient compliance with medicines would lead to better value.
Oosterom said treating and monitoring patients at home is cheaper for governments and preferred by patients, but hospitals have no incentive to switch from inpatient care to homecare.
"One of the main hurdles to innovation in this area is that we currently pay providers [hospitals] instead of paying per patient. Why should a hospital pay for homecare if it means less money for the hospital? Money should follow the patient regardless of where they are treated," he said.
A 'blockbuster' drug is usually defined as a single medicine that treats one disease and generates revenue of more than $1 billion per year (€664 million). Bestselling medicines like the cholesterol-busting drug Lipitor can clock up sales of almost €8 million a year, although it comes off patent in a couple of years' time.
Nevertheless, there has been much angst in the pharmaceutical sector in recent years as the pipeline of new groundbreaking medicines for common illnesses appears to be drying up. This comes at a time when advances in gene therapy and personalised medicine suggest the days of finding a mass-market drug to sell to large volumes of patients may be coming to an end.
Pharmaceutical firms have been criticised in the past for their quest to find the next blockbuster for chronic conditions affecting patients in the developed world. This, say critics, has come at the expense of people with rare diseases, because it has not always been viewed as economically worthwhile to research uncommon illnesses or those affecting poorer populations.
Business & Industry
- European Federation of Pharmaceutical Industires and Associations (EFPIA)
- European Generic Medicines Association (EGA)
- European Biopharmaceutical Enterprises (EBE)
- Novartis Pharmaceuticals
Think tanks & Academia
- National Public Radio:Pfizer Piles Into Biotech With Deal For Gaucher's Drug(1 Dec. 2009)
- Wall Street Journal:Lipitor & the era of mega-blockbusters(30 Nov. 2009)