Are you curious how to evaluate the significance of a new medicine or medical device?
GenAI notice - NO AI-powered tools were used to generated this article or its contents but some IAAE colleagues did kindly review it to make it better!
This article is a follow on to a previous article called Patient Stories and Stories of Patience - how Pharma 4.0 leaders share purpose with their teams and how you can too.
It is a common challenge, and increasingly important for all humans to believability-weight information, knowledge, and wisdom. Thanks to Ray Dalio for this useful concept. In some matters doing so is trivial and inconsequential, in others, significant and sometimes critical. Without going down a rabbit hole to explain epistemology (a branch of philosophy that examines the nature, origin, and limits of knowledge) let's rather take an example of an announcement about a recent medical innovation and follow several steps to evaluate the significance by contextualizing it using other sources of public information and knowledge.
What would serve as a good example to explore?
The Ceo of Sanofi, Paul Hudson, recently shared on LinkedIn in the post shown below that, together with Regeneron, Sanofi gained regulatory approval from the US FDA and the China NMPA, for the first-ever biologic which will treat certain adults with uncontrolled Chronic Obstructive Pulmonary Disease (COPD). This news, which was released at the end of September 2024, follows a similar approval in the European Union.
For those of us who aren't trained doctors, or know of anyone directly impacted by COPD, it is not intuitive to understand the significance of an announcement like this. But this doesn't mean we can't or shouldn't try to evaluate significance. Let's start by finding a definition by a credible authority such as the Mayo Clinic to gain an understanding of COPD:
“Chronic obstructive pulmonary disease (COPD) is an ongoing lung condition caused by damage to the lungs. The damage results in swelling and irritation, also called inflammation, inside the airways that limit airflow into and out of the lungs. This limited airflow is known as obstruction. Symptoms include trouble breathing, a daily cough that brings up mucus and a tight, whistling sound in the lungs called wheezing. COPD is most often caused by long-term exposure to irritating smoke, fumes, dust or chemicals. The most common cause is cigarette smoke.” Mayo Clinic, Overview of COPD
Now that we know a little more what COPD is, how can we next understand how serious COPD is as a leading cause of death?
The Lancet is a credible weekly peer-reviewed general medical journal which published the latest ‘Global Burden’ study of 2021. The chart below shows that COPD, a non-communicable disease, has a descreasing rate of death per 100,000 people over the last thirty years, and has been in the top five leading causes of death since 1990.
Now we can better understand the significance of this announcement, and acknowledge the value of future availability of new treatments in China, the EU, and the US is excellent news.
Futher research would need to be carried out on the estimated numbers of people currently impacted, access to this medicine, pricing, and other aspects such as demonstrated benefit once treated. Nonetheless this is significant to a very large number of people (when you consider a global population now over 8 billion human beings).
To gain an even better understanding of the significance, can we understand more about how serious COPD is as a cause of illness before death?
This is where the concepts of Global Burden of Disease and Disability Adjusted Life Years (DALYs) are very helpful. The definition from the Our World in Data website explains below:
“To assess the health of a population, it’s straightforward to focus on mortality, or concepts like life expectancy, which are based on mortality estimates. But this does not take into account the suffering that diseases cause the people who live with them. By looking at both mortality and morbidity (the prevalent diseases), we can have a more comprehensive understanding of health outcomes. The sum of mortality and morbidity is called the “burden of disease” by researchers, and can be measured by a metric called “Disability Adjusted Life Years” (DALYs). DALYs are standardized units to measure lost health. They help compare the burden of different diseases in different countries, populations, and times. Conceptually, one DALY represents one lost year of healthy life – it is the equivalent of losing one year in good health because of either premature death or disease or disability.” Source: Our World in Data - Burden of Disease
The image below produced by Public Health England is helpful to illustrate these concepts.
Recommended by LinkedIn
Additionally, how can we determine over time if the treatment of COPD is effective?
This is where the Global Burden of Disease comparison tool (developed by The Institute for Health Metrics and Evaluation is informative. The graphic below shows color shading by category of disease (blue represents non-communicable diseases) and the darker shading represents a positive % change between 1991 and 2021. COPD represents 2.77% of the total Disability Adjusted Life Years.
For a fascinating story about the origins of the concept of DALYs, consider reading or listening to Epic Measures: One Doctor, Seven Billion Patients. This account by Jeremy Smith covers Christopher Murray (the current IHME Director) and the work by him, Alan Lopez, and hundreds of other colleagues over several decades on Global Burden of Disease studies was funded by the Bill & Melinda Gates Foundation.
So is this particular annoucement significant?
Yes, yes it it. Although COPD has descreased as a cause of illness and death per 100,000 people since 1991 it is still in the top five leading causes of death and represents 2.77% of the total Disability Adjusted Life Years for all people on earth. Having access to new treatment such as this by Sanofi and Regeneron in the US, EU and China is a significant step of progress but more work remains to be done to expand access to other nations.
Disclaimer! This has been a short evaluation by someone who is neither a doctor nor epidemiologist of a recent announcement that is far from complete or rigorous. There are likely dozens of points that would benefit from further explanation or caveats. However, my purpose of sharing this article that illustrates steps to better understand context and begin to evaluate signficance. I invite you to share in the comments ways that could improve this approach (or better sources to reference) as well as any share links to other examples that you have come across that could help better understand the link between medical innovation and health impact.
This article was written by Malcolm Jeffers, Transformation Strategist and Head of Research with the International Academy of Automation Engineering. Although Sanofi is represented on the IAAE Life Sciences Advisory Board this example was chosen as a recent relevant example. For future articles I invite you to follow me here on LinkedIn and also consider subscribing to the IAAE email newsletter for articles by other IAAE thought leaders.
Sources
Paul Hudson, CEO Sanofi Approval announcement (LinkedIn, Accessed 27SEP24)
Overview of Chronic obstructive pulmonary disease (COPD) (Mayo Clinic, Accessed 27SEP24)
Epic Measures: One Doctor. Seven Billion Patients (Amazon Audible)