One way to improve clinical trial reporting: a Yelp-style rating system

This piece originally appeared in the Timmerman Report.

STAT recently published an in-depth report about the many research centers that don’t bother to publicly disclose the results of their clinical trials, even though they are required to do so. This follows on a New England Journal of Medicine article back in March that had a similar analysis of the lack of reporting and publication of clinical trial data to clinicaltrials.gov.

Most observers of biomedical research would agree that getting clinical trial data out about what happened in a trial is pretty important, whether the trial succeeded or failed. After all, biomedical translational research is most meaningful when done on human subjects and negative information can be quite informative and useful. Animal models are nice, but translation of results from animals to humans is a spotty proposition at best. We need to know what’s working, and what’s not, to know how to best allocate our research resources and how to treat patients.

The lack of reporting is an embarrassment for research. It’s also understandable, because so far the FDA hasn’t used its authority to punish anyone for delayed reporting. Nobody appears to have lost any research funding because they failed to post trial results in a timely manner. Universities told STAT their researchers were “too busy,” given other constraints on their time, to report their results. So what really seems to be going on is that reporting is prioritized below most other activities in clinical research.

It was interesting and eye-opening that industry fared better than academia in both the STAT story and the NEJM article with respect to how many studies have been reported. Having seen the industry process first-hand, I’d speculate that (at least for positive trials) there’s a much stronger incentive to get data out in public. Successful trial results can create buzz among clinicians and patients, revving up trial enrollment which can then help get a new drug on the market faster, and convince people to use it when it’s available. It may be that in academia the effort of getting trial results in the required format for clinicaltrials.gov is perceived as too much work, relative to the rewards. Academics are naturally going to spend more energy on directly rewarded activities like writing grant proposals and writing peer-reviewed scientific publications that help them win even more grants, promotions, and other accolades. Well okay. If this is the case, then figuring out new incentives may be key.

So what would work? Anyone who participates in a clinical trial is providing time, may be subject to risks and often is asked to provide samples that are biobanked to support future exploratory and translational research. It’s like when people donate to food banks. I’m pretty sure they mean that food to be eaten and not to sit on a shelf. These participants in clinical trials deserve to have their volunteerism rewarded.

This got me thinking about how to empower patients to get more of what they want. Patient-centered research is a buzzword these days, and for good reason. Patients have at times been an afterthought in the biomedical research enterprise. I thought of services like Yelp and Uber and Angie’s List and other peer-to-peer systems that allow users to get information, provide feedback and give ratings to specific providers. And I wondered: could this be a way to apply pressure to clinical trial researchers to improve their reporting? Continue reading

The power law relationship in drug development

All opinions are my own and do not necessarily reflect those of Novo Nordisk.

A few weeks ago a friend and I had the great opportunity to go see Nate Silver speak at the University of Washington. He’s a funny, engaging speaker, and for someone like me who makes his living generating and analyzing data, Silver’s work in sports, politics and other fields has been inspirational.  Much of his talk covered elements of his book, The Signal and the Noise, which I read over a year ago. It was good to get a refresher. One of the elements that particularly struck me this time around, to the point that I took a picture of his slide, was the concept of the power law and its empirical relationship to so many of the phenomena we deal with in life.

Nate Silver graph small

Figure 1: Slide from Nate Silver’s talk demonstrating the power law relationship in business–how often the last 20% of accuracy (or quality or sales or…) comes from the last 80% of effort.

Because I spend way too much time thinking about the business of drug development, I started thinking of how this concept applies to our industry and specifically the problem the industry is facing with creating innovative medicines.

Continue reading

The fall and rise of the LEGO Kingdom: A review of “Brick by Brick”

All opinions are my own and do not necessarily reflect those of Novo Nordisk.

When people ask me what I did growing up, they expect me to say “surf.” I know this because when I tell them what I did for fun their next question is always, “What, you didn’t surf?” I didn’t. Still haven’t learned. Instead I did a lot of the things boys all over the US did. I watched TV. I hung out at the mall and at fast food restaurants. And I played with LEGO.

The brick fundamentally hasn’t changed since I was a kid. My son has a bunch and the basic essence is still snapping things together with that satisfying “click,” and the gradual accretion of form and function from individual, generic elements. Kind of like how life evolves, you know? And yet at the same time LEGO has undergone great changes in packaging, themes, toy categories, and target audiences. Today it’s one of the most respected and recognized toy brands in the world. But something I hadn’t realized until reading “Brick by Brick” by David Robertson and Bill Breen is how close LEGO actually came to crashing and burning in the 90s and early aughts, before recovering to once again become a commercial powerhouse.

Continue reading

The Aussie pipeline to the slopes of British Columbia

All opinions are my own and do not necessarily reflect those of Novo Nordisk

So this past Christmas, I decided to go downhill skiing.  I’ve gotten away from the sport for a few years, and felt it was time for a reintroduction. The slopes are full of older skiers, so I know skiing is something I should be able to keep doing for a good while longer, as long as I don’t get too rusty. Also as long as my knees hold out. And to get back into it I figured a trip to Silver Star in British Columbia would be ideal. I last visited this ski area over ten years ago, but remembered being very impressed by the slopes, the snow, the people and the facilities.

I booked a trip and that was my first hint of an Aussie connection.  Everyone I spoke to on the phone had that distinctive twang that’s mangled in so many Outback Steakhouse commercials.  When I arrived on the 21st of December, almost every Silver Star employee I met had come from Down Under. Continue reading

Genetic counseling at Illumina

All opinions of the interviewer are my own and do not necessarily reflect those of Novo Nordisk.

Illumina is the industry leader in high-throughput sequencing platforms and over the past decade has employed a fascinating mix of innovation, creativity in approach, community engagement and aggressive exploration into different business areas. I recently had the opportunity to interview Erica Ramos, who works as a clinical genomics specialist and certified genetic counselor in the professional services group within Illumina’s CLIA lab, about Illumina and genetic counseling.

Kyle Serikawa:  Can you describe what Illumina is doing in the field of genetic counseling? That is, are you creating a genetic counseling service, or advocating an increase in training of genetic counselors, or creating materials to facilitate counseling?

Erica Ramos: So Illumina has four full time genetic counselors as part of their services group. We don’t provide direct services to patients; Illumina’s model is to provide support to the providers, the physicians. We support what’s being done in the genetics core at Illumina. As for training, we offer opportunities for that. Every year we welcome a second year student in genetic counseling for a 10 week, part time rotation. We’ve done about 5 of those so far. It’s an opportunity for those students to see how genetic counseling skills can be applied to a non-clinical setting. We see the internships as a way to engage these people who will go on to become genetic counselors. Illumina is also a very active in the genetics community, including membership in the American College of Medical Genetics and other organizations.

KS: Given the current landscape of, for example, exome and whole-genome sequencing, it seems like genetic literacy will become an increasingly important skill—both for understanding how genetic variants can be interpreted and also how genetic information will be communicated. How is Illumina thinking about educational needs in genetics?

ER: The genetics community as a whole is concerned about the need for wider understanding of genetics to help inform medical practice. From Illumina’s standpoint, one of the things we can do is to support the internships I’ve described as a way to provide exposure to non-clinical roles for genetic counselors, which broadens the potential market. Also, we’re providing a training option that maybe not all academic programs can support. At the same time, the universities themselves can see the developing need, and through supply and demand we hope to see an increase in the number of genetic counselors being trained.

There is also need for the education and updating of other professions. Physicians, nurse practitioners and others. Illumina has put on the “Understand Your Genome” symposia to work with providers who don’t currently have as deep an understanding as they would like.

KS: How do you see genetic counseling as synergizing with Illumina’s business interests? Continue reading