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In a year in which several Big Pharma players felt pain as the Vioxx imbroglio settled out – take August's $253 million settlement against Merck, for example – news in funding, policy, and strategy, from government agencies to biotechs, suggests that the industry is still as robust as ever. Here's our unofficial Top 10 list of developments, in no particular order.
1. THE RISE OF FOUNDATIONS
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Keep it under your hat, but this guy Bill Gates may be onto something. In 2005, the Bill and Melinda Gates Foundation made announcements about two immense groups of international grants for health-related research and treatment: $258.3 million for malaria, and $436 million for challenges in global health. That's just a fraction of the $5.5 billion in such grants the foundation has made since its inception.
The foundation isn't the only one increasing its funding of research in the United States; research support from private foundations, voluntary health organizations, and institutes increased 36% from $1.8 billion in 1994 to $2.5 billion in 2003, the latest figures available, when adjusted for inflation, according to a
2. EU REGULATES REGENERATIVE MEDICINE
One of the bigger milestones for the European biotech industry in 2005 came toward the end of the year when the European Commission published long-awaited draft regulation governing what they call "advanced therapies" – including gene therapy, cell therapy and tissue engineering. The proposal, published in mid-November, aims to guarantee health protection for patients treated with these therapies, plus making it easier for companies to get products to market by establishing an EU-wide regulatory framework for their authorization, supervision and post-authorization vigilance.
"It's very important," says Aurelie Vandeputte, Manager of the Healthcare Council at EuropaBio, the EU biotech industry group. "It will help ensure patient safety because you have regulation at the EU level." It will also help even out a rugged playing-field for companies who currently have to contend with vastly differing regulations in different EU member states, she says.
3. 454: BUILT FOR SEQUENCING SPEED
© 2005 Nature Publishing Group
When 454 Life Sciences announced in May that it had inked a $62 million, 5-year deal with Roche for the promotion of its fast genome sequencing system, it was one of several high points in a good year for the Branford, Conn.-based subsidiary of CuraGen.
The 454 sequencing system uses picoliter-scale technology, light emitting sequencing chemistries and top-notch informatics to allow fast, whole-genome sequencing. The company sold its first system in March, to the Broad Institute of MIT and Harvard.
In September, the company gave the genomics world a black and white illustration of the technology's use. In a paper published in
4. BIOSIMILARS ARE DIFFERENT
With billions of dollars of biopharmaceuticals due to come off patent in the next few years, "biosimilars" – follow-on versions of original biologicals – are set to be increasingly big news. In 2005, the European Medicines Agency (EMEA) set the pace in developing a clear regulatory path for this type of drug, the biopharmaceutical version of generics.
"Biosimilars are in essence a new, third class of medicinal products," says Thomas Bols, director of government affairs for Europe at the US biotech firm Amgen. "We have now innovative, generic and biosimilar products. For biosimilars to get approved, a new legislative and regulatory pathway had to be created as both the approval schemes for innovative and generic medicines are not appropriate."
In this, the EU is taking a lead. "The EU is the first region in the world – before the US in any case – which has now adopted a legislative and regulatory pathway for the approval of biosimilar medicines," Bols points out.
5. CONFLICTED INTERESTS
After nearly two years of investigations, congressional hearings, and internal turmoil, the National Institutes of Health in August issued final conflict of interest regulations for its employees, easing restrictions on ownership of stock in drug and biotech companies and allowing most staff scientists to interact with colleagues in nonprofit and scientific organizations. The rules relaxed more stringent regulations announced in February that would have limited stock held by all NIH employees and totally ban all outside consulting, even with nonprofit organizations. Those rules were imposed after media and congressional reports revealed that some NIH scientists had accepted lucrative consulting contracts with outside companies. But the rules were relaxed after scientists reported widespread dissatisfaction and a few senior officials said they would quit. The new rules limit stock ownership for only 200 senior NIH scientists and employees but ban any consulting with pharmaceutical and biotech companies. Senior employees have until February 2006 to divest of their biotech and drug company holdings in excess of $15,000.
6. TYSABRI SUSPENDED
When Cambridge, Mass.-based firm Biogen-Idec voluntarily pulled its monoclonal antibody Tysabri (natalizumab) from the US market in late February, it was clearly very bad news. The decision to withdraw the multiple sclerosis drug from market was made after one fatal case and one additional case of progressive multifocal leukoencephalopathy (PML) were confirmed in patients receiving the drug. But as well as being a downer for the company and its marketing partner, Ireland's Elan Pharmaceuticals, the setback for Tysabri may have been something of a wakeup call for the biotech industry: Biologics can, despite their specificity, cause adverse events.
After reaching that nadir in February, however, there are signs the Tysabri story may have a happier ending. Safety testing this year has revealed just one other case of PML. J.P. Morgan Securities upgraded shares of the company to "overweight" from "neutral," and in October, Forbes suggested the drug may be back on the market before the end of 2006.
7. TERROR TACTICS EVOLVE
When the New York Stock Exchange postponed a planned listing of Huntingdon Life Sciences in September, the biotech industry saw it as a worrying sign that biomedical research had taken a backseat to the tactics of animal extremists. The decision had been preceded by a violent and threatening campaign against the company by the Animal Liberation Front, and prompted Biotechnology Industry Organization (BIO) chief executive James Greenwood to write to NYSE officials warning that "allowing terrorists to hobble the marketplace is equivalent to surrendering democracy."
The government is concerned as well: In May, John Lewis, US Federal Bureau of Investigations deputy assistant director for counter-terrorism, told the Senate Energy and Natural Resources Committee that the FBI is investigating more than 150 cases associated with animal rights and eco-terrorism in the United States.
Meanwhile, companies are fighting back, making more use of laws passed to protect them from terrorists like Stop Huntingdon Animal Cruelty-USA, which has been the subject of injunctions.
8. BIOTECH IN AFRICA
On the opening day of an agricultural science and technology meeting in Burkina Faso, on June 21, Pamela Bridgewater – US deputy assistant secretary of state for African affairs – announced that the presidents of four African countries had voiced their support for agricultural biotech. The heads of state of Mali, Niger, Ghana, and Burkina Faso all stressed to US officials how important they felt it was to focus on the uses of biotechnology to improve health and well-being of their populations, according to a report posted on several US Embassy Web sites.
The report makes no mention of any concrete actions being planned by those governments, but their statement comes at a time when recognition of the value of science is gaining political support in the continent of Africa. In September, the governments of more than 40 African countries pledged their support for a $160 million plan to boost science and technology across the continent. In terms of spending, that plan puts the "safe development and application of biotechnology" at the top of the pile, on an equal footing with securing and sustaining water.
9. MONEY FLOWS FROM OFF SHORE
In June this year, newspapers in Britain and beyond reported that a private equity group based in Bermuda – Celtic Pharma – was going to buy the British biotech company Xenova for up to £26.1 million. David Oxlade, chief executive of Xenova, told
Oxlade went on to suggest that the deal might set a precedent in the biotech sector. Certainly Celtic, founded in December 2004, has its sights set on rapid expansion – it has raised some $300 million so far, and has its sights set on $1 billion by next year.
10. FDA APPROVES DRUG FOR BLACK PATIENTS
Nitromed, an emerging firm based in Lexington, Mass., made history in July this year when the Food and Drug Administration approved its BiDil drug for the treatment of heart failure in self-identified black patients. The first racially targeted drug, BiDil was said by the FDA to represent a step toward the holy grail of personalized medicine.
Robert Temple, FDA associate director of medical policy, said "the information presented to the FDA clearly showed that blacks suffering from heart failure will now have an additional safe and effective option for treating their condition. In the future, we hope to discover characteristics that identify people of any race who might be helped by Bidil."
But for John Hodgson of UK life sciences consultancy Critical I, the approval with a racial indication was "odd." "What the analysis didn't separate out was the very likely fact that the African-American group, matched age for age, were sicker in the first place," he says. In his view, the data suggest that the effect of the drug could be specific to the sicker population rather than a specific race group. "It's a very bad example, I think, of pharmacogenetics being used, and a very diverse group being lumped together in an inappropriate way."