The most valuable lessons I learned were in the audience of policy meetings at the Brookings Insititution, National Press Club, and the White House. Discussions (often heated) about health economics and outcomes research led by the formal medical director of CMS at an advisory council meeting were eye opening. Once you learn--you can't go back. I can't pretend that diabetes is just diabetes and a pill or injection is the answer.
I recently approached the mic at a session sponsored by the Johns Hopkins Institute for Basic Biomedical Sciences. Weighty Matters: Recent Advances in Metabolism, Obesity and Diabetes Research was targeted to journalists. Except there was limited or no data. No skill session on how to evaluate clinical data. I asked about a bariatric procedure and the risk of malabsorption of nutrients if indeed the digestive tract was abbreviated. You could hear crickets.
My goal is to unpack the science. If we understand the evidence--both what we know and don't know--better decisions will be made. Better articles, education, and recommendations for patients at the point of care.
Recommending gastric bypass as a national solution for our diabetes epidemic is bad medicine and bad economics.-- Mark Hyman, MD
I was in...
I did not attend. I will be staying up-to-date and writing about the sessions but not with my peers in a fully-staffed press room. Sometimes the personal over-rides the professional and you have to face the reality of the situation. A few client projects escalated requiring close oversight. I have been at conferences trying to juggle phone conversations, go-to-meetings, and deadlines and it isn't pretty.
I made a tough call. Tough because I thrive for the live connection. Access is my secret sauce and I use it liberally.
Lucky for me, I have been thinking about writing more specifically about diabetes. What a perfect time to slow down and create a narrative. But first, we need context. A level set of sorts to set the stage for how we think about clinical evidence. We also need to consider how we utilize surrogate measures to determine clinical efficacy and safety in clinical trials and what that means for the real world population (outside of a controlled clinical trial).
Comparison of international agencies: concerns with surrogate outcomes. Y axis: Drug submission. X axis: Agency. *No: no (e2) = implicit no “evidence 2”; no (ref) = implicit no “reference”; no (e) = explicit no “evidence 1”; no (e1 + e2) = explicit no “evidence 1” and implicit no “evidence 2”; Yes: yes (e1) = implicit yes “evidence 1”; yes (e2) = implicit yes “evidence 2”; yes (used) = implicit yes “used before”; yes (ref) = implicit yes “reference”; yes (e) = explicit yes; Not identified: N/S = no statement; N/A = not applicable; Red shades = negative statements of surrogate acceptability; Green shade = positive statement of surrogate acceptability; HbA1c = hemoglobin A1c; 6MWD = 6 minute walk distance; composite = histology, virology, serology; SVR = sustained virological response; CDR = Common Drug Review; HC = Health Canada; FDA = Food and Drug Administration; EMA = European Medicines Agency; NICE = National Institute for Health and Clinical Excellence; PBS = Pharmaceutical Benefit Scheme; SMC = Scottish Medicines Consortium.v
Surrogate outcomes are defined as a laboratory measurement or a physical sign used as a substitute for a clinically meaningful endpoint that measures directly how a patient feels, functions or survives, and that is expected to predict the effect of the therapy.
Surrogates are often biomarkers such as hemoglobin A1C [HbA1c], blood pressure, lipid levels, etc. Surrogate outcomes are used in clinical trials for reasons of efficiency and practicality; they can be measured with fewer patients, less invasiveness and a shorter observation period.
Where surrogate outcomes have validated links with final endpoints, their use can greatly facilitate clinical research. However, in the absence of validated links, there can be uncertainty about patient benefit; and even where the epidemiologic basis is sound, long-term safety and other unanticipated issues may predominate.
For example, while blood pressure is conclusively linked to cardiovascular morbidity and mortality, antihypertensive drugs do not necessarily reduce morbidity or mortality as expected.-- Surrogate outcomes: experiences at the Common Drug Review
Looking at a therapeutic area pipe-line and deciding what the gaps should be is like locking the barn door after the cows have wandered off. You need to look at the data--be open to the bounty of freely available datasets. Be open to making a difference. Let's figure it out together... moo.
2016 Banting Medal for Scientific Achievement
The statistics (at least to me) point to need for population health interventions and prevention strategies. Health costs are unsustainable and the diversion of funds looking for cures downstream of deleterious social correlates leaves me gobsmacked.
Thoughtful discussions about content development and outcomes analytics that apply the principles and frameworks of health policy and economics to persistent and perplexing health and health care problems...