Now that I’ve had the chance to settle in to the blogosphere, I’d like to tell you a little more about what I hope to accomplish with “Trials Without Tribulations.” It is obvious to me (and others in the industry, see here, here, and here) that biopharma faces a number of sizable challenges. Tribulations, if you will. I hope TWT can shine a spotlight on these challenges and stimulate discussion about developing solutions. Here’s a rundown of some major obstacles:
- The traditional discrete-phase development model. Routine insistence on pauses between phases assumes that merely waiting will enable greater understanding of data from the previous trial. With essentially unlimited computing power to run sophisticated analytics that are standing by for database lock, why can’t we understand everything there is to learn from trial data rapidly enough to proceed without a discernible pause between phases?
- A lack of global harmonization in development standards. Officially, we have globally harmonized standards. In practice, we have big differences, even among the major regulatory bodies that speak of harmonizing standards. An obvious example is device regulation in the US vs. Europe. Do historical and cultural context make these differences inevitable, or can standards become more uniform and global approvals become the norm?
- Lock-in development arrangements between big pharma companies and CROs. Lock-ins may provide volume discounts, but they also chain pharma to big CROs that have profited hugely from the development approach that has reduced productivity in the last 25 years. Is pharma looking for greater efficiency in precisely the wrong place? Would there be greater savings and better results from staying flexible and selecting the optimal partner for each program?
- Failing to identify the optimal dose before phase III trials. To avoid the cost of additional dosing arms, companies have likely been testing too few dosing options. This is an area where adaptive methods can help. The proposed HIV vaccine trial that starts with multiple vaccines and prunes candidate vaccines may be a sign of things to come. Is there a way to test more candidates and doses without a linear increase in the number of trials and trial costs?
- Wasted time and cost of excessive SDV and on-site monitoring. Right now, most pharma companies and CROs are failing to take full advantage of technology that optimizes monitoring—a basic but costly activity. When will EMRs universally have the ability to grant monitors selective access to patient records, enabling remote SDV of far more data? When will we become good enough at remote monitoring to make site visits unnecessary? What changes in technology and processes are necessary to reach that goal?
- Efficiency challenges in creating companion diagnostics for new targeted therapeutics. Companion diagnostics obviously improve targeting, but developing both the diagnostic and the drug increases costs and complexity. How can we handle such parallel development projects more efficiently?
- Allowing our development processes to block the transition to personalized medicine. Our industry relies on methods that will never be efficient enough for profitable development of personalized medicines. We’re leaving blockbusters behind but not even getting close to a new era in which companies thrive by producing greater numbers of smaller-market drugs. How can the industry (and regulators) move beyond development methods that only a blockbuster can pay for?
I have already started to address some of these issues, such as the impact technology and information flow can have on monitoring, enrollment and more. But I plan to scrutinize each of these topics even more comprehensively in the coming months and hope that in doing so, TWT will engender productive dialogue and eventually, solutions to the daunting hurdles on the track.