[Editor's Note: We asked selected Xconomists a series of questions designed to zero in on the big issues of the year, including "What would you be willing to throw a punch over?"]

The FDA is the easy target for those of us who are in drug development. But what I am most mad about is the unwillingness of our industry to focus and invest in innovative, high-impact drugs instead of “me too” knockoffs. Thus, my “anger” is focused on institutions that myopically insist on investing in low-risk, copycat ideas that undermine the true strength of our industry over the long term.

Risk-taking and innovation in drug discovery are critical to our field. Aspiring merely to be fast followers instead of striving for innovation is putting our competitive advantage at risk. The entrepreneurial spirit has been key to America’s success in many industries, especially biotech, but there have been several recent events that are stifling innovation.

The downturn in the economy has obviously dampened the financing of new ideas. Changes to patent law arguably encourage long, drawn-out opposition hearings that could be detrimental to the smaller inventor. However, my key concern for our industry is the lack of new and innovative drugs, despite the stunning pace of scientific progress over the last several decades. Not enough companies are focused on innovation and not enough people in charge are taking the right level of risk to actually come up with a new idea that can have a substantial impact on human health. Innovation is the alpha and the omega of our industry and should always be cherished as a way to create valuable and meaningful high-impact drugs.

Comments (1) | Reprints | Share:          

After consulting with the Oracle at the Temple of Apollo at Delphi, here are a few of the trends I was told to watch for in 2011…

Two revolutionizing pharmaceutical frontiers are on the horizon—RNA therapeutics and stem cell therapies:

—RNA therapeutics will become a big reality in 2011. Mipomersen, a LDL lowering drug from Isis, completed four Phase III trials and is expected to be approved as the first major RNA therapeutic. There will be more than two dozen clinical trials currently involving RNA therapeutics, including siRNA drugs. In 2011, the microRNA therapeutic platform will mature further and will advance in the clinic.

—Stem cell biology is rapidly advancing toward human patient trials. For example, Geron has just initiated a Phase 1 clinical trial in spinal cord injury using the injection of functional replacement cells manufactured from human embryonic stem cells.

Also, more effective and efficient clinical trials will remain a high priority for the drug industry, and new predictive biomarkers will be key:

—Predictive and companion molecular biomarkers will continue to be extremely important for drug development and clinical trials. Drug companies now understand the model of “one drug fits all” is not effective or economical. The new model is a more personalized approach that tries to identify the right patient cohort for a specific drug. Molecular biomarkers are an approach to identify which patient will respond, or are responding, to a particular drug treatment regimen. There is a possibility that drug developers will not even consider initiating a clinical development program without a biomarker associated with it.

—Biomarkers predictive of disease state and outcomes will be highly sought after.

New genomic technologies will emerge from centralized core facilities and spread into everyday laboratories. For example:

—Deep genomic sequencing using a platform that sits on a lab bench will replace large capillary electrophoresis equipment and potentially even chip-based array technology.

And my final prediction for 2011:

—All biotech companies will be very, very successful :-)…if they make the right choices…and pay homage to the oracle.

[Editor's Note: This is part of a series of posts from Xconomists and other technology and life sciences leaders from around the U.S. who are weighing in with the top surprises they've seen in their respective fields in the past year, or the major things to watch for in 2011.]

Comments (2) | Reprints | Share:          

…is coming soon.

Comments | Reprints | Share: