Saturday, August 23, 2014

Crystal Balls, Ebola, and Pharmaceutical Development in 2020

It is always safe to make predictions about what the future will be like in ten years, because if it turns out that you were wrong, the odds are no one will remember. If it turns out that you were right, you can seize the opportunity to remind everyone of your remarkable prescience. With that as prologue, I will start by revisiting a prediction I made sixteen years ago about a date still six years from now. In April of 1998, at a conference entitled NEXTMED:  The Future of Medicine, I made a prediction for the year 2020 (20/20 vision was a popular theme in the futurist business back then). Actually I made several predictions, but I have carefully selected the one which has the best chance of proving accurate. In my talk I included the Ebola virus as an example of the progress I foresaw in our ability to respond to future threats:

Immunology at the Rainbow’s End: A Push-Button Vaccine Machine

It is a few years off, but obviously the science of predicting protein structure from a gene sequence is moving rapidly; and, well within the time frame spanned by this talk, it will be a reality. At that point, the window will slam shut on the possibility of our being overrun by a third-world virus, another HIV or, worse, a more widespread and contagious Ebola. Within days of the first cases being picked up, a blood sample of a victim would be sufficient to do a full genomic analysis of the pathogen, the pathogen’s proteins would be fully analyzed both for their function and their antigenicity, the most antigenic regions would then be synthesized with an appropriate adjuvant, and a very effective vaccine would be coming off the production line a week or two later.

Thursday, August 14, 2014

Drug Safety and FDA Approval Times: It Is MUCH More Complicated Than the HEALTH AFFAIRS Study or the FORBES Response

In my blog post of March 26, 2014, I commented on a New England Journal of Medicine article authored by Darrow, Avorn, and Kesselheim that focused on the serious safety issues arising from the FDA’s accelerated drug approval programs for “breakthrough” drugs.  It is clear that the expedited approval of drugs based on surrogate endpoints can result in marketing approval for drugs with questionable risk/benefit ratios.  However, in the past week a different controversy has arisen over the relationship between drug safety and FDA approval times, sparked by an article in Health Affairs by Cassie Frank and others entitled Era Of Faster FDA Drug Approval Has Also Seen Increased Black-Box Warnings And Market Withdrawals.  The Frank article prompted a response from John R. Graham in Forbes with a title that evidences his disagreement with Frank’s group: Faster FDA Approvals Have Not Caused More Drug Safety Problems.  So who is right?  Actually, my answer is “Neither article sheds much light on the FDA role in drug safety.” It is complicated, like so many problems in pharmaceutical policy.

Monday, August 11, 2014

Ebola Biologic Stirs Bioethics Discussion

I was quoted (and my expertise inaccurately described) in the San Diego Union-Tribune’s article about the ethical issues raised by the experimental ZMapp biologic for the treatment of persons infected by the Ebola virus.  I am not a specialist in bioethics, which is the description provided for me in the article, and only claim to know something about the ethical issues that are raised in drug development.  The otherwise reasonably well-written story is here. There has been a fair amount written about the ethical issues in this situation, where there are very limited amounts of a drug that has only animal data supporting its safety and efficacy.  The New York Times article by Andrew Pollock is here. Arthur Caplan, who has moved to NYU since his infamous involvement in the tragic Jesse Gelsinger gene therapy death at the University of Pennsylvania, is quoted at the very end of Pollock’s article.  Caplan expresses concern about the appropriate allocation of resources to therapy research versus public health in the expanding Ebola epidemic.  He may be correct that expenditures for drug research and development will do little for the current outbreak, but that is largely irrelevant.  I doubt that the development and scale-up of Mapp Biopharmaceutical’s biologic is diverting significant resources from the public health measures that Caplan favors.

Thursday, August 7, 2014

Norway Leads the Way in Biosimilars: The NOR-SWITCH Study!

On July 24, 2014, Novartis announced that the FDA had accepted for filing the first application seeking marketing approval in the U.S. for a biosimilar version of filgrastim (Neupogen).  In my May 19, 2014, post “A Few Thoughts About Biosimilars” I discussed the problem of driving down the price of these somewhat cheaper, but still very expensive, drugs.  Biosimilars have been available in Europe for some time but none have been approved yet in the U.S.  In this post I will discuss a different but related problem in biosimilars development, which is built into the Biologics Price Competition and Innovation Act (BPCIA).