Daunting but common challenges currently face many biotech, pharmaceutical, and device firms. These companies are encountering
a restless public, worried investors, and a skeptical, publicity-hungry Congress that are all concerned about product safety
and the reliability of regulators' scrutiny. Individual firms are arming themselves to fight their own particular safety issues,
financial challenges, or public relations blunders. R&D costs are up, yet biopharmaceutical productivity is down. Given this
landscape, industry observers are beginning to conclude that collaborating in key pre-competitive areas might actually benefit
the industry as a whole by creating a much stronger arena for future competition.
Ernst R. Berndt, Ph.D.
In mid-August, the MIT Center for Biomedical Innovation (CBI) hosted a day-long forum (full video at http://cbi.mit.edu/) that explored the relationship between innovation and safety, and the associated risks faced by patients, investors, payers,
regulators and industry executives. The newly-launched center exemplified the collaborative principles it professes by engaging
speakers from industry, government, and academia as it addressed the challenges of developing, producing, and distributing
safe medicines. A number of the panels explored specific collaborative mechanisms that, if properly constructed, will significantly
reduce the inherent risks in biomedical innovation, as well as enhance the overall productivity of the discovery, development,
and commercialization processes.
The MIT-CBI Forum focused on recent scientific, bioengineering, informatic, and process advances that will improve the benefit/risk
ratio of therapeutic innovation in the areas of:
- Biosafety testing, from pre-clinical analyses to product launch
- Manufacturing scale-up and product distribution systems
- Pharmaco-vigilance and post-launch market surveillance.
One of the themes that emerged from the panel discussions was the likelihood of significantly increased and changing roles
for information technology in the drug development and post-launch surveillance processes. While industry and patient advocacy
groups have long argued that FDA should bundle rapid approval of innovative medicines with more intensive and comprehensive
post-launch monitoring, the recent market withdrawals of Vioxx and Bextra for safety reasons has heightened the public's awareness
of the need for effective surveillance.
Patient registries can provide useful information, but as panelists noted, not only are they costly to develop, but frequently
only after they are functioning is it realized that several critically important questions have been inadvertently omitted.
An alternative possibility involves linking databases from the clinical development process to multi-million-member medical
claims databases, but this raises at least two additional concerns. First, to facilitate effective surveillance, the various
medical claims databases and patient registries must be able to communicate with each other, which requires collaboration
on standards not only among drug developers, but also with insurers/payers, else, as CBI Executive Director Dr. Frank Douglas
noted, "we will erect a 21st century Tower of Babel." Second, since data from such sources typically are observational rather
than experimentally randomized, criteria must be developed that spell out the evidentiary standards that would warrant conditions
under which there is sufficient evidence for FDA or the manufacturer to issue new warnings or even withdraw a medicine from
the market for safety reasons. Developing bio-informatic statistical tools and software that reliably distinguish random and
innocuous chance events from evidence suggestive of genuine medical adverse events requires collaboration not only among institutions
but also across disciplines. Further, as CMS Administrator Dr. Mark McClellan noted in the previous year's event at MIT, the
sharing of post-launch surveillance data offers opportunities for enhanced collaboration and contributions from ex-US healthcare
Dr. Douglas closed the forum by noting that "the approach to solutions is actually very simple and yet at the same time very
difficult. It has to do with sociology. Are we willing in a real sense to collaborate in order to compete? That is the paradigm
shift. CBI is in a strange way attempting to tackle that problem." The opportunity costs of not collaborating on pre-competitive
issues facing the industry and society are staggering. Could a "safe haven" be created where data could be generated, analyzed,
and shared and standards established to the benefit of all? "Our CBI Stakeholders from industry and government," Douglas continued,
"have said resoundingly that if CBI could find those areas where we can collaborate to compete, it could indeed make a tremendous
contribution to all working in the healthcare space."
Ernst R. Berndt, Ph.D., director and program leader, MIT Center for Biomedical Innovation and Professor of Applied Economics, MIT Sloan School
of Management; Cambridge, MA; 617.253.2665; fax 617.258.6855; email@example.com