Regenerative medicine researchers are making enormous strides to discover more about diseases and disease treatments. From heart regeneration to cancer immunotherapies to gene therapies, they are doing some of the most exciting research today, and some of them are even succeeding… Or are they?
I do not want to keep you in suspense: They are succeeding, but they are not done yet.
I imagine many bench scientists would define success as translation to the clinic. Many clinical scientists and physicians would see positive Phase 3 clinical trial data as success. When I first started as a regulator, I remember thinking a regulatory market authorization was success. As I moved along in my career, I thought a recommendation from the Canadian Agency for Drugs and Technologies in Health (CADTH) Common Drug Review was success. It seems we all think we see the light at the end of the tunnel, but our perspectives are limited.
Scientific breakthroughs are not the end of the tunnel, nor are regulatory market authorizations, and reimbursement decisions are not the end of the tunnel either… neither is provincial formulary listing. Implementation is the end of the tunnel AND the entire tunnel is a toll route that requires valuable information as currency.
If we all step back to the broadest perspective, we would recognize that ultimate success is only realized with the appropriate use of the health products we are developing. This would mean that the science is good, the benefit-risk profile is positive, that the price makes sense to all involved, and that the users/recipients are able to put this all together to successfully treat the patient. Anything less is a failure of some degree, although not in the normative sense. What is more, the failures along the way represent the opportunity cost of developing and using another product or procedure that could have been a success for them or someone else.
A potential solution rests in this idea of “precision medicine.” Although this term is often misused and flawed because it does not recognize that “precision medicine” is something physicians always strive for, it does convey a fundamental concept. It suggests that we need a focus on getting the right drug to the right person under the right circumstances. To this end, precision medicine can involve companion diagnostics that identify genomic information correlated to drug safety or effectiveness. This proposition is not simple and is not cheap.
It may be possible to get genetic information for $129, but it costs hundreds more than that to turn raw data into meaningful information, and it costs millions more to develop this technology in the first place. It involves collecting, documenting and applying good biological samples and data. Enter new Canadian funding initiatives, and groups like Genome Canada. They recognize that the potential for precision medicine requires significant investment, and together with Precision Medicine Policy Network, Institute of Health Economics, Genome Alberta, Genome Quebec, Alberta Innovates, Paceomics, CIHR, and the University of Alberta, they hosted a forward-thinking workshop to consider how this investment can be better informed.
Truly enlightened investment in technology will consider the concepts of value of information (VOI), and the value of implementation (VOImp). At its simplest, this means that people investing in technology adoption (including drug formulary managers) should understand the potential benefits of spending more money to get additional information to reduce uncertainty or improve appropriate uptake/use. Understanding these terms can help grant applicants address emerging funding agency requirements, support efficient translational pathways to clinical use, support reimbursement negotiations, and inform implementation plans.
I am fortunate to have attended this precision medicine workshop on VOI and VOImp, and I look forward to figuring out how to apply key concepts earlier in product development for more efficient commercialization and translation.
Latest posts by Patrick Bedford (see all)
- Successful translation? Consider the value of implementation - February 26, 2018