These are my picks for the emerging trends and issues in orphan drug research and biotechnology in the U.S. in 2005. New drugs, biotech start-ups and mergers, FDA and pharmaceutical reform--take a look at what will be important this year.
1. Amazing new orphan drugs coming to market
Two orphan drugs in development in 2004, Yondelis and Aplidin, were derived from marine biology, of all things. Other completely-new (novel) drugs are in the development pipeline. In 2004 the FDA granted orphan designation to nearly 150 drugs and treatments, allowing them to proceed with clinical trials. No doubt 2005 will witness more major breakthroughs in treatment of orphan diseases.2. Biotechnology start-ups rising
Venture capitalists are once again becoming strong players in biotechnology research and development. VC firms invested a record $5 billion in the biotechnology sector in 2004, and the trend is expected to continue in 2005. This is encouraging news, since a number of biotechnology firms in the past were founded solely for orphan drug development. Let's hope we see more of the same.
3. Mergers and acquisitions continue
In 2004, biotechnology and biopharmaceutical firms continued to grow and develop, and significant mergers and acquisitions occurred. Look for more of these in 2005, as companies add research and development strength. Overall, biotechnology industry revenues are projected to rise almost 20 percent this year.
4. Congress considers FDA and pharmaceutical reform
Recent testimony by an FDA scientist brought the FDA drug approval process to the attention of Congress, which is now looking at reform for the agency, especially in light of safety problems with drugs already on the market such as Vioxx. Efforts to reform the FDA could conceivably spread to the orphan drug approval process. Also, regulatory reforms that affect pharmaceutical companies could have a great impact on biotechnology firms, since the two often work together in orphan drug development.5. State funding for biotechnology
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$3 billion measure was approved in California in 2004 to fund stem cell research. New Jersey, Connecticut, Wisconsin, and Maryland are considering similar proposals. Technically, the California measure allows funding of other forms of biotechnology research as well. This provision is important because stem cell research is only a small part of biotechnology. The Biotechnology Industry Organization says it will encourage states to consider not limiting funding strictly to stem cell research.
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