Feedback on the EFPSI workshop on regulatory statistics

Hi Alexander,

If I were able to attend the workshop, I would be most interested in the topic of the Role of Statisticians.  We can be consultants, contributors, collaborators and in some instances leaders, depending on where statisticians are positioned in their companies.  We can develop, test and/or apply new methods, depending on our training.  Because we carry the stigma of being support staff for clinical trials, a leadership role might require a new frame of mind.   Will you telling the audience what role statisticians most often play?

I liked this line from a recent post, “The understanding of data – its strengths and limitations –is a core competency of statisticians. Thus, they need to play a key role in helping others to understand and interpret the efficacy and safety data correctly.”

This competency sounds a lot like a data scientist, who seem to market themselves better than statisticians.  They remind us that they are in short supply, have a unique blend of cutting edge technical skills, can predict the future and use terms that corporate execs don’t understands such as support vector machines.

Perhaps Benefit-Risk involves more stakeholders than Data Science, making it more difficult to lead.  Perhaps by providing more clarity & transparency to decision-making, Benefit-Riskiticians are less mysterious than Data Scientists. Whatever the case, we can learn something from data scientists.  Will you be telling the audience how statisticians can better promote their abilities and have an equally sexy career as data scientists?

Kind regards,

Mike Colopy, PhD


What would you like to know about BR as a beginner?

I’m pretty excited as I was asked to present about benefit-risk at the first EFPSI workshop on regulatory statistics. The organizers have built a very good agenda with lots of good content and presenters.

I’ll talk after David Wright from the MHRA, who will give a regulatory perspective on the evolution of BR and the regulatory decision process. Georg Quartey (Roche) will speak after me about a variety of case studies. Thus, I’m in the middle. We agreed, that each presenter will have 20 minutes to speak and that the session will end with a 30 minutes podium discussion.

The goal of my presentation is to provide the perspective off an industry statistician on benefit-risk. I’m thinking especially about those statisticians, that rarely touched this topic in the past. My draft title is “What’s happening in benefit-risk and what is our role as a statistician?”

FDA on further Benefit-Risk framework – an outlook

PDUFA VI Draft Goals Letter just posted online

The Draft Goals Letter was just posted online here. There are lots of interesting things in this. Here is a quote from the Regulatory Decision Tools Subgroup that is especially interesting for Benefit-Risk:

“FDA discussed its proposal to explore the appropriateness of incorporating additional decision-analytic approaches that could inform FDA’s regulatory decision-making through the B-R Framework. As part of this proposal, the agency proposed convening at least one workshop to review potential approaches, such as the use of trial endpoints that capture ranked benefit and harm outcomes at the patient-level, and their applicability within FDA’s drug regulatory context. Industry indicated that the value in this proposed area of exploration would need to be weighed against other biostatistics-related proposals under discussion.”

FDA publishes draft guidance on BR assessment including patient perspective

The FDA published a draft guidance on “Factors to Consider Regarding Benefit- 11 Risk in Medical Device Product Availability, Compliance, and Enforcement Decisions”. Notably it contains a section called “How FDA Considers Benefit-Risk in Patient Focused Medical Device Product Availability, Compliance, and Enforcement Decisions”. It also discusses patient preferences on the benefit, patient tolerances of risk, patient impact, and patient preferences for availability.

ISPOR publishes two reports on the use of MCDA

The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) has published two reports on the use of multiple criteria decision analyses (MCDA). ISPOR uses MCDA as a collective heading for a set of techniques, that use structured, explicit approaches to decisions involving multiple criteria which aim it is to improve the quality of decision making. These reports result from a task force being established in 2014. The goal of this task force was to provide a foundational report on the topic and focus also on initial recommendations on how best to use MCDA methods to support health care decision making.