#hcsm – TrialSphere

Hi Everybody!

Recently, I was fortunate enough to be included in the 2016 NYU Healthcare Makerthon – an initiative between the NYU Entrepreneurial Institute & the NYU Langone School of Medicine. The goal of the Makerthon is to solve an important, health-related challenge by completing some step-work:

Identify the challenge,
recruit a quality team and then,
together, develop a solution into a viable venture.

My challenge is to “Re-imagine the medical research process to give participants a better experience.”

Does the number “$2.8 billion” sound familiar? If it does, you might work in clinical research. If it doesn’t, that number (and 10-15 years) is what it takes to get a therapy FDA-approved and to the public. PhRMA companies spent almost $60 billion on R&D in 2015. The NIH kicked-in an additional $31 billion.

Anyone who has ever looked at the numbers or attended a study Investigator’s Meeting knows, poor study enrollment and retention account for a huge portion of those costs.

Clinical trials account for nearly 40% of the US pharma research budget and total around $7B per year. The estimated cost of patient recruitment is 40% of the total budget, or $1.89B.

If a potential therapy is able to pass a recruitment feasibility assessment, the costs of enrollment often leads to study discontinuations and therapies that “die on the vine”. Additionally, sponsors may be forced to loosen study eligibility criteria to improve enrollment, only to hand the FDA under-powered data that is too weak to properly evaluate.

Before anyone clicks the back button because they (understandably) have a tough time feeling empathetic over Big Pharma’s recruitment woes, let me assure you I’m not trying to inspire empathy. There is more to the story.

Most of my research background has been limited to clinical trials with pharmacological interventions. But, more recently, I’ve been involved with other types of medical research at NYUMC’s Dept. of Radiology and Center for Biomedical Imaging. We have an amazing team of brilliant scientists, physicists, doctors & engineers (think Matt Damon in Good Will Hunting) – all working to improve patient outcomes through innovative imaging methods. The way I like to explain it to people who don’t regularly write mathematical formulas on window panes, is to try to remember what a television’s picture looked like in the 80’s and compare it with what you see today when you walk into Best Buy. There’s HD, 4K, curved screens, amazing sound, 3-D and VR. The quality of TV sets and peripherals has improved dramatically since Johnny Carson was on weeknights at 11:30 ET.

That’s essentially what our researchers are doing with medical imaging technology like MRI, PET, post-processing software, sequence development & 3-D printing. We move forward by recruiting study participants who graciously volunteer for imaging scan(s).

Some participants have a diagnosis such as Alzheimer’s, cancer, osteoarthritis or Parkinson’s. Others are healthy volunteers whose images are used for comparisons. From what i’ve observed, the participant’s experience is usually good enough for them to refer their friends and family to us.

I use imaging research as an example, hoping to soothe any lingering beliefs that medical research is confined to corporate interests. I admit, it’s easy to become jaded with all the negative press surrounding the pharmaceutical industry. But, to their credit, Hep C (associated with 19,659 deaths in 2014) is now curable in > 90% of patients (within < 12 weeks of treatment).

New pharmaceutical therapies have also contributed to a huge reduction in the amount of cancer, autoimmune and heart disease-related deaths over the last decade. But meanwhile, all over the world, researchers are developing technologies, medicines, prosthetics, implants, procedures, apps, software & naturalistic therapies (just to name a few) aimed at lengthening and improving the quality of human lives. Sure, it’s not all altruistic, but if people are earning a healthy living by developing these therapies, IMHO, it’s as it should be.

It seems to me that finding a solution to research recruitment/engagement challenge could cut R&D costs, resulting in lower healthcare costs and faster delivery of therapies to the patients who need them. But I digress.

After brainstorming potential solutions and getting some ideas on paper, I discovered the WordPress forums and managed to put some of my ideas into a website. I’m not a web guy and while building it, I wasn’t sure what may take shape. But I really enjoyed it as a creative outlet and it was therapeutic for me. TrialSphere also gave me an opportunity to shed some light on an idea that I had found tough to articulate.

Joining NYU Langone School of Medicine has exposed me to more opportunities than I can keep tabs on, including this Makerthon challenge. On 10/26/16, Makerthon participants gave brief overviews of their challenges to an audience of mostly NYU & NYU Langone Medical Center community members. After we said our piece, two Apple, Inc. engineers presented the capabilities of iOS, ResearchKit, CareKit and HealthKit. These open-source development platforms are powerful and the timing is perfect to build them into the research recruitment/engagement space. With the right team, we could leverage these platforms to build a simple, intuitive, powerful and scalable solution for this challenge.

My next step in the Makerthon project is recruiting a team of 3-5 people to join me. Ideally, I need a developer, designer and someone with general business skills. None of these skills are part of my background, but I can sure help navigating the research environment.

The Makerthon’s Phase II takes place November 18-20 (RSVP here.). This phase involves working with top healthcare investors, entrepreneurs and startup experts to help develop the idea into a viable venture. The top 4 teams selected by a panel of healthcare startup experts will receive cash prizes totaling $10,000 and advance to Phase III – “Venture Support” (Jan. 19-Feb. 19).

So, here is my call to YOU for action:

If you’re a developer, designer, business person, or,
if you have a desire to crack this challenge and believe you have something to contribute … please don’t hesitate to contact me directly or leave a comment below.

Enjoy the Cubs winning the World Series and Happy Halloween Everyone!

Many Thanks,

Steve Stork

A new challenge has been presented to researchers conducting placebo controlled studies: The potential of social media unblinding a clinical research study.

But, what is unblinding?

Unblinding is when either a volunteer or a clinician become aware of which treatment the volunteer is receiving.

Being “blinded” to whether someone is receiving active study drug or a placebo (i.e. “sugar pill”) is necessary to determine if a treatment is effective. In order for FDA to approve a new medication, they need evidence that it can treat disease symptoms more effectively than a dummy pill. Interestingly, a dummy pill is often quite effective at treating a number of medical conditions, though the reason for this is still unclear.

If someone believes they are receiving treatment that will make them feel better, often times they do feel better. So much so that the disease may slow down or even go away completely. If a researcher knows that a participant is receiving active study drug, they may be inclined to interact with them in such a way as to amplify the effect of the drug (i.e. “I definitely think you’re getting better.” or “This drug is promising. I expect you to improve soon”. This bias can skew the study data, rendering it worthless. This is why the “blind” must be maintained if we are to understand the drug’s value.

Research Volunteers are now able to connect and communicate with each other using faster technologies which speak to broader audiences. The risk of research volunteers sharing sensitive study information has always existed, whether volunteers are talking among themselves in a waiting room, attending support groups or, more recently, engaging in conversations on Facebook, Twitter, or health-oriented online communities. The exchanges are typically not moderated and many tech savvy Research Volunteers are understandably eager to share their experiences with others.

Scenario
A research volunteer participating in an online forum may share that they received an injection at their randomization visit in which they experienced no side effects. Meanwhile, another forum participant may share that the injection they received gave them a burning sensation when administered. The two can engage in conversation about their experience while trying to determine who is receiving study drug and who is receiving placebo. This scenario could jeopardize the blind and once this information hits the internet, there is usually no way for anyone to come along and remove their comments or posts.

This is a real concern and it’s happening now. See The Wall Street Journal July 2014 article – Researchers Fret as Social Media Lift Veil on Drug Trials.

The Center for Information and Study on Clinical Research Participation (CISCRP) has also produced some short, volunteer-facing videos which attempt to educate Research Volunteers on this problem. Also, some Informed Consent Forms now contain language warning volunteers against sharing study-related information over the internet.

Even with these measures in place, the exchange of study related information may be difficult to stop. While education may be helpful, there is no guarantee that people will comply or fully understand the issue of sharing sensitive study information. If a health-related website took initiative and attempted to moderate comments and posts from their users, those users can always create their own community with no moderation or rules.

“The FDA is going to have to figure out how to accommodate social media.”

– Richard Garr – President/CEO Neuralstem Inc.

While TrialSphere seeks to bridge the communication gap between Research Professionals & Research Volunteers, we understand that the information shared through forums, messages and comments could be sensitive.

Comments and posts are moderated by TrialSphere staff members who have years of experience working in clinical research. We have also added the ability for all Research Professional members to moderate the material posted by any Research Volunteer members. If a Research Professional comes across a comment or post by a Research Volunteer that looks like it may contain sensitive study information, that Research Professional may flag it for deletion. Research Volunteer members are made aware of the possibility that their posts and/or comments may be deleted by Research Professional members, in the interest of preserving the integrity of the study data. Research Volunteers seem to be understanding of this.

We know that this issue is an ongoing discussion and we welcome your questions and comments below.

If you would like to learn more about Social Media, Clinical Research & Unblinding, check out:

Society For Participatory Medicine – Website

Nature Medicine – Engage with research participants about social media – Article by Craig H. Lipset, MPH – Head of Clinical Innovation Within Worldwide Research & Development at Pfizer

Tag: #hcsm

Design, Development, Business & Creative Help Wanted: NYU Healthcare Makerthon