Real world evidence and the 21st Century Cures Act

20160731-img_2972-3Late last year, US Congress passed the 21st Century Cures Act,* a bipartisan bill that would increase funding for medical research, reevaluate the current mental health systems in the US and enhance the regulatory requirements for pharmaceuticals and medical devices. I am compelled to write a piece on a very interesting amendment to the law: “The Utilization of Real World Evidence” (Sec 3022). I am also going to attempt to describe the “so what?” of this important amendment in terms of the impact and opportunity for post-doctorate students in academia.

You may ask what Real World Evidence (RWE) is… According to the amendment, RWE is defined as “data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than randomized clinical trials…   The sources of real world evidence, including ongoing safety surveillance, observational studies, registries, claims, and patient-centered outcomes research activities.”

Let’s dissect this into laymen’s terms. Traditionally, pharmaceutical products have been approved by the Federal Food and Drug Administration (FDA) solely on data generated from controlled clinical trials where study subjects (patients) are monitored closely by clinicians. Additionally, patients are refrained from taking any other drugs or sometimes even diets and activities are being closely monitored and controlled by the study protocol. In some ways, this is the best methodology to test the “chemical” efficacy and safety of the drug but it lacks the true effectiveness and long-term safety of said drugs.

For example, Joe Smith has diabetes and enrolled in a clinical trial in Philadelphia, PA for 3 months. During the trial, his diabetic condition improved. The drug was ultimately approved. Joe would get this new diabetic drug at the pharmacy in New York City. A few months later, his diabetic condition actually worsened. What happened?

Here was the problem… Joe was a security guard in New York City mostly taking night shifts 5 days out of the week. During the day, he would sleep until 1PM. He would then have a large meal at a local diner. Time for exercising was not possible as he also had a second job at a local retail store. He would eat dinner late just before he started his night shift as a security guard. As you can see, the clinical trial that got the drug approved did not represent the “real world” situation for Joe.

By utilizing non-traditional data and analytics, the industry can finally solve this problem. Fundamentally, over the last 5 years or so, it has been a period of data liberation in the industry. Next time you go to the doctor’s office, does your doctor have an iPad to enter your symptoms and drugs that you are taking? When you call your insurance company, do they have all the details about your health history? When you go to Walgreens / CVS, are you surprised that they can see what other drugs you are taking and which doctors you are associated with? Also think about you day-to-day activities and ask yourself these questions… Do you have your smart phone with you and it is tracking your geolocation? Do you wear a Fitbit/Garmin to track your steps? Do you use Google and that it is collecting data on what you search for? Do you use app on your phone and that it is collecting data on how you play your games and therefore your cognitive level? All of these generate “real world data”! Many companies will clean the data to rid of confidential information and then provide them for public use.

Sounds fascinating? Indeed, it is! In fact, this is a nascent space in the industry that we are all still trying to develop best practices. I would imagine that the callout of RWE in the 21st Century Cures Act will transpire new opportunities for the industry and for people who may be interested to become a trailblazer in this space. Critics of the 21st Century Cures Act argue that the utilization of RWE could give pharmaceutical and medical device industries too much leverage to obtain product approval without highest level of evidence. However, my view is that traditional clinical trials MUST still be conducted to test the efficacy and safety of products. The utilization of RWE can only enhance our understanding of the product in a heterogenic world which translates to better long-term benefits for the patients.

So what? How does this affect post-doctorate candidates in academia?

On the surface, it may seem Real World Evidence is too far outside what post-docs do every day. However, a career-minded post-doc should take notes that this is the future of research and healthcare. As I mentioned above, RWE is a nascent space. It will disrupt the industry. Disruption also means opportunities. Many jobs will be created over the next many years in RWE, from clinical trials research and operations, to wearables-related opportunities, to jobs related to advanced data/analytics.   Also, I believe that biopharma industry will begin to increase their investments and to partner with academia for many new studies in RWE. In fact, just a quick search of academic RWE partnership with biopharma on the internet returns a large number of hits. Post-docs need to step out of their comfort zone to learn more about RWE, seek opportunities which may have been there all along (i.e., existing partnership between their institutions and biopharma), or leverage their analytical skills into big data. Objective for the post-doc is to be that group of trailblazers to set the gold-standard of RWE.

As I close out this blog post, I look at my Garmin watch and realize that I really need to do my steps and that I need to have a better sleep tonight before my doctor calls on me next month when I do my annual physicals.

*The 21st Century Cures Act was passed in the Senate on 12/7/16 with  a vote of 94 to 5 in favor. This vote followed the a 392 to 26 House vote the previous week. The bill was signed into law on 12/13/16 by President Obama.


Keith Ho, Associate Director Worldwide Medical, Biogen. Keith Ho is currently a member of the real world data strategy team at Biogen. By leveraging both traditional and non-traditional data and analytics methodologies, Keith and his team provide insights from the real world use of medicine and therefore delivering more patient-centric value to the products in the market. Keith has also held roles in R&D, Marketing and Corporate Strategy at various large pharmaceutical companies such as Merck and Bristol-Myers Squibb.

Note, all views expressed are the personal opinions of the author and don’t represent an official stance from AFS.

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