Monday, April 18, 2016

Rethinking Clinical Trials

Here is the link to my article that appeared today in El Mundo's (translation  below) supplement on innovation and technology (Innovadores). El Mundo is one of the most prestigious newspapers in Spain. 

Much has been said about the major discoveries that will change our lives. These range from treatments for cancer to those that change the course of the increasing number of rare and new diseases. Developing cures or treatments aligns with the business goal of developing a product or intervention that will improve lives and generate revenue for the innovator company. 

Given all the products that are in the discovery pipeline it is clear that companies want to develop new products that improve or enhance lives. And of course the product must be priced so that it is affordable; without sales there are no revenues. Unfortunately, too often in the discussion of the need to develop new treatments the arguments about price are at best myopic. One way to manage prices is to decrease the cost of drug development that according to industry estimates is about $1.2 to $1.3 billion. Advances in different fields like engineering, information technology, and telecommunications need to be applied to drug development.

Other opportunities lie in building new methodologies for clinical trials so that we know more precisely the characteristics of people who will benefit from the treatments that are in development. What we know is that the bulk of trials are based on models of drug and treatment development that ignore that health is very individualized.

There is much upbeat talk about the promise of precision medicine or personalized medicine but it will remain just talk as long as the clinical trials that underlie the development process continue to be structured in a way that is neither precise nor personalized. The innovator working with regulatory agencies has to rethink these critical aspects of development.

The use of animal studies has long been an essential part of basic research. Yet even in these often seminal efforts there have been huge omissions that effect everything from validity to reliability. Reardon [1]pointed out that, “In 2014, the NIH [United States National Institutes of Health] began requiring researchers to include female animals in studies, and giving out supplementary grants to those who complained about the cost." The omission of female animals was not good science.

Likewise factors that also effect outcomes in animal studies are what the animal has been fed and its living conditions.  Research indicates that variations in these conditions change the health and longevity of the animals, which impacts on the outcomes and the ability to replicate the research. Although leading experts indicate that animal studies are becoming less relevant we still often extrapolate from animals to humans.

When it comes to studies of people our failures to develop targeted treatments and interventions are compromised even further. To develop precision or personalized medicine clinical trials must at a minimum include and analyze information using race, ethnicity, and gender. These are characteristics that are essential to understanding the success or failure of treatments. The process will be enhanced as information about the individual’s genetic and microbiome become more available.

The solution is not to spend more money on larger clinical trials but to use the advances in all of the sciences and design trials that are better defined, that are inclusive, and carefully monitored. This will make it possible to develop medicines and treatments that meet the health needs of the individual, the revenue goals of the innovators, and most importantly, continue to support future discoveries to benefit us all.

[1] Reardon, S. "A mouse's house may ruin studies— Environmental factors lie behind many irreproducible rodent experiments." Nature. February 18, 2016 vol. 530 Pg. 254

Thursday, January 7, 2016

Sugar and the 2015-2020 Dietary Guidelines

We are all too familiar with sugar as a driver for diabetes and excess weight. Yang et al[1]  piled on even more data to raise our concerns about sugar when their research found that people who had more sugar in their diet were more likely to die sooner from cardiovascular disease.

So today when I listened to the Dietary Guidelines Stakeholders Briefing convened by USDA and DHHS I was very interested in the recommendations on sugar. I knew that in March 2015 the World Health Organization released a new guideline that recommended adults and children reduce their daily intake of free sugars to less than 10% of their total energy intake and that a further reduction to below 5% or roughly 25 grams (6 teaspoons) per day would provide additional health benefits.  I wanted to understand the rationale on deciding on the U.S. guideline of 10% instead of 5%. 

I was glad to be part of this briefing and liked the transparency of of being encouraged to type in our questions. As I typed in my question I could see the questions that others had asked and was looking forward to the answers about soy, caffeine, etc. When the moment for Q & A’s came up I was shocked that none of the questions that were posted were asked. I typed my question in again just in case someone had missed it…and still no response. I sent an email to and am still waiting for a response. A simple answer would be informative.  But I guess it is all related to why the 2015-2020 Dietary Guidelines were not released until 2016.

[1] Yang, Q., Zhang, Z., Gregg, E.W., Flanders, W., Merritt, R., Hu, F.B. “Added Sugar Intake and Cardiovascular Diseases Mortality Among US Adults,” JAMA Internal Medicine. 2014;174(4) Pgs. 516-524. doi:10.1001/jamainternmed.2013.13563.

Tuesday, December 29, 2015

Myths We Love

Megan Scudellari’s, “Myths that will not die,” (Nature, Dec. 17, 2015 Vol. 528 pages 322-325) focused on five myths: (1) screening saves lives for all types of cancer; (2) antioxidants are good and free radicals are bad; (3) humans have exceptionally large brains; (4) individuals learn best when taught in their preferred learning style; and, (5) the human population is growing exponentially.

Each statement had some initial evidence to support it but over time more research challenged the initial outcomes. What was compelling as I read the article was realizing that there were factors that seem to perpetuate if not strengthen a myth. These factors include a vocal community of supporters; the development of products, industries, and research lines to address or remedy the situation; and, attempts to discredit new research. As a result, myths like lies that are repeated, become accepted as truth. In the end the damage is done to all of us because rather than moving forward with science we are in a chokehold because of what others want us to believe.

As I look forward to 2016 and beyond, my hope is that as our knowledge grows we will use information wisely and change what we know and espouse. "Trust, but verify," should apply to much of what we do in health so that we can achieve the healthier and longer lives we all want.

Wednesday, August 26, 2015

Me and Burgers

I love hamburgers but lately I only eat them at home. I do not want to seem like a wuss but it seemed to me that when I ate burgers outside my home my microbiome let me know that they were displeased with my selection. Some of the people I know thought that I was just being picky when I said that I preferred meat that did not have added antibiotics or hormones.  Today's Washington Post made clear that my microbiome led me to a healthier choice.

Sunday, August 9, 2015

Coca-Cola Funds Global Energy Balance Network

WHO is recommending limits in sugar intake because of the worldwide increase in people who have excess weight. Drinking one typical can of sugar sweetened beverage exceeds the limit. Nevertheless, it seems that Coca-Cola is interested in research that documents that excess weight is not due to bad diets but to not enough exercise They just funded the Global Energy Balance Network to lead this work. 

Thursday, May 7, 2015

How to Reach the Hispanic Community...

Here is the link to my article that appeared on May 6, 2015 in El Mundo, Section on Business and Innovation, Madrid, Spain. .  The translation is below. 

Reaching Everybody

I was having a conversation with the CEO of a major company about how to reach 57 million peple in the United States. When I mentioned that I was talking about reaching the Hispanic consumer there was a pause and a palpable change in the conversation. The enthusiastic CEO went into automatic mode accentuated by polite comments that indicated interest and understanding. Then the entire project was handed off to the person responsible for Diversity — a death knell for meaningful work.

The Chief Diversity Officer is a C suite position without budget responsibilities that lacks the clout that is necessary to execute. These positions are meant to deflect challenges to the status quo rather than define the possibilities and the opportunities in a growing market. With neither a place in the chain of command nor the portfolio that could make the necessary investments it is a place where ideas go to die. The Diversity person is there to make the company appear like they are doing something to serve their diverse consumer base.

Just like EEO Offices of decades past or the more recent renaming of similar offices as Minority, Disparities, Equity, or some other politically palatable name of the moment these offices do little more than develop plans and do training. Most companies take a 20th century approach and establish an employee group that is made up of the targeted community to advise them; hire outside expertise on the targeted group; create an advisory group of stakeholders; create a diversity, health equity, multicultural, or alliance development office; translate existing documents, webpages, etc. to the target language; add pictures of the target group; add food selections at corporate cafeterias that cover a variety of countries; and, sponsor “Month of…” events.

While these may be well-intentioned the totality of activities make it obvious that what has been accomplished is mostly veneer. Even worse they can be seen as a defensive response devoid of the tools to create the type of meaningful inclusion that produces financial results. This is bad for business and for our economy.

Reaching Hispanics or any target group means reaching individuals with an image, work, and product that resonates with who they are and what they want. It takes more than a diversity office to do that…it takes leadership and the commitment by every person in a company.  That leadership is hard to come by when Hispanics are one in six persons in the U.S. but at Fortune 500 companies are less than 2% of the CEOs and less than 4% of the Boards of Directors.

Trust and brand loyalty go hand-on-hand and today each individual wants to know that a company knows who they are and what they want. Too often I hear that Hispanics are too diverse and because of that it is hard/incorrect/disrespectful to get a single message that resonates with everyone. That type of strategy reveals a lack of understanding of the American marketplace.

With the rare exception of being in a building that is on fire and yelling ”Fire,” it is unlikely that a single word can reach everyone. The era of the single message went the way of having only a handful of major networks. Each person wants and expects to be reached with a message that is tailored to them; that is why Google has been so successful. Google knows each of their users and craft messages for them.

So when I am asked how do you reach Hispanics? It’s the same strategy that you use for everybody—use language and images that are meaningful to the individual, make clear that you understand what they want and desire, and as a result earn trust and respect. You do different things at different times. Reaching everybody is no longer a one shot deal.

Thursday, March 5, 2015

Big Data and You

El Mundo, one of the most prestigious newspapers in Spain, created a daily supplement on innovation and entrepreneurship (title Innovadores) and as an advisor to Bankinter's Future Forum I wrote this article on "Big Data and You."
Here is the translation:

Big Data and You

We all accept that there is an abundance of data that are used to define everything about us. The data that are collected are then harvested by all types of entities and put together in ways that communicate more to others than we may want to share. But with the promise that the data cannot be traced back to the individual we release much of what is private about our lives. Nowhere is this more evident than in the health care setting where data are being merged from different sources to produce predictive models for deciding upon the type of treatment an individual should receive.

The use of big health data has been heralded as a giant leap forward in that information from different people can be used to give a fuller picture of what are the risk factors that we need to pay attention to. With these aggregated data and the risk profiles they provide health care providers at every level are equipped with an array of protocols about how to treat a patient. These data can also provide new and compelling alternatives to what would be considered the usual standard of care.

But we must proceed with great caution.  Recent research[1] funded by the National Heart Blood Lung Institute in the U.S. documented that commonly used risk assessments of atherosclerotic cardiovascular disease (ASCVD) overestimated the risk by 37 to 154% in men and from 8 to 67% in women. That translates into a huge amount of people getting treatment they did not need.

Big data are the results of combining lots of smaller bits of information. How big data are gathered and how they are merged can become a problem. This is made worse if at the point of care risk scores replace talking to the patient or the ever elusive listening to what the patient says. The promise of health care providers to “Do no harm” can be compromised by the overreliance on tools that are meant to add to the clinical conversation and not dominate them.

Jane L Delgado, PhD, MS,
President and CEO
National Alliance for Hispanic Health
Washington, DC

[1] Andrew P. DeFilippis, MD, MSc*; Rebekah Young, PhD*; Christopher J. Carrubba, MD; John W. McEvoy, MB, BCh, BAO; Matthew J. Budoff, MD; Roger S. Blumenthal, MD; Richard A. Kronmal, PhD; Robyn L. McClelland, PhD; Khurram Nasir, MD, MPH; and Michael J. Blaha, MD, MPH. An Analysis of Calibration and Discrimination Among Multiple Cardiovascular Risk Scores in a Modern Multiethnic Cohort. Ann Intern Med. 2015;162(4):266-275. doi:10.7326/M14-1281