From The Editor | May 13, 2016

Clinical News Roundup: Industry Seeks FDA Guidance On mHealth Technologies

Ed Miseta

By Ed Miseta, Chief Editor, Clinical Leader
Follow Me On Twitter @EdClinical


Industry Seeks FDA Guidance On mHealth

Wearable and mobile devices are a part of the clinical trials environment and will play an even bigger role in the future. Fitbits, mobile apps, smartphones, and tablets are all finding a home in patient’s lives, and, consequently, clinical trials. While many industry professionals believe FDA has been clear in its guidance on incorporating these devices into trials, others believe more needs to be done before they will be comfortable moving forward. 

In October 2015, the FDA put out a request for more information on the technologies and innovations being used. FDA is most interested in the challenges companies face when collecting data from trial participants’ own devices. According to a report on, over 40 comments were posted. Representatives from sponsor companies (Pfizer and GSK) and industry groups such as ACRO (Association of Clinical Research Organizations) and AdvaMed (Advanced Medical Technology Association) contributed their thoughts tot he FDA. 

AdvaMed recommends FDA monitor new technology use and allow the situation “to develop on a case-by-case basis” to promote the use of the technologies. However, the group also believes guidance will eventually be needed for sponsors, investigators, and institutional review boards (IRBs) to ensure patient privacy and cybersecurity. ACRO believes FDA needs to provide clear guidance in terms of security and monitoring requirements, noting the agency “needs to be willing to share the progress being made in the industry, and provide feedback on the use of new technologies and inspection results.”

Medidata, a provider of clinical research technology solutions, called on FDA to address industry apprehension over whether regulatory agencies will consider data gathered via non-traditional tools or if existing regulations will prohibit or discourage using mHealth tools. The company notes some sponsors are waiting for others to proceed, while others want to pursue mHealth but are concerned because they don’t feel they have enough information to give FDA in order to have an informed discussion. Medidata recommends FDA evaluate how to open up communications with sponsors to seek formal feedback before conducting an mHealth-enabled clinical trial.

 Will Bitcoin Improve Transparency In Clinical Trial Research? is reporting that two clinicians have devised a system which prevents clinical trial documents from being altered to make new medicines appear to be more effective than they actually are. The result of the trial, which was on cardiovascular diabetes and ethanol, has passed peer review on open science publishing platform F1000Research

Undeclared protocol changes continue to be an issue in clinical research. If a medicine is found to be ineffective, researchers can pursue new health outcomes until a positive result is found. They could then report only the positive findings. The problem persists despite mandates requiring that all trials be registered prior to the start of experiments.

Using blockchain, a decentralized database of bitcoin transactions, every transaction is publically recorded, timestamped, and stored across a large, international network of computers. This makes it impossible for researchers to tamper with the records. The approach by Greg Irving of the University of Cambridge and John Holden, a General Practitioner, involves converting a clinical trial document into a bitcoin to take advantage of its blockchain infrastructure.

Under the system, the original clinical protocol is given a unique digital signature using an online tool called the SHA256 Calculator. This signature is converted into a public bitcoin key using Strongcoin, another online tool. This transaction is timestamped and recorded as a blockchain, and multiple copies are stored in a computer network. By generating a new bitcoin key using the text of the document, anyone can confirm if alterations to the original text have been made.

New Software Can Change Future Of Drug Discovery

Producing a new drug that can be tested on humans can take decades and cost billions. Sometimes, finding patients to take the drug can take just as long. But IEEE SPECTRUM is reporting tech startup twoXAR, founded by two computer scientists, may have found a way to make that process more efficient.

twoXAR is developing an algorithm to search through potential drug candidate data. Andrew M. Radin, cofounder and chief business officer for twoXAR, notes computers usually augment what researchers are already doing. He notes drug discovery researchers build molecular models and then decide which ones to test, instead of letting computers crunch the data and make the decision.

When CEO Andrew A. Radin (same name, no relation) attended Stanford graduate school, an assignment asked him to come up with an idea for a medical problem that could be solved by computer science. His idea involved using an algorithm to merge and mine a number of disparate biomedical databases to make predictions about what drugs were most likely to be successful in testing as particular disease therapies. The databases used included, but weren’t limited to, clinical record searches, molecular similarity models, and gene expression information. He later teamed with the younger Andrew Radin to start twoXAR.

The technology can combine any number of public and proprietary data sets to rank the probability of a particular drug candidate being effective in treating a particular disease.

Wearables And Social Media Lead Clinical Trial Trends

A new survey from the Association of Clinical Research Organizations (ACRO) finds wearables and social media to be the leading trends in clinical trials. The trends are driven by a rapid increase in the use of wearable technology and an upsurge in social media for patient recruitment.

"ACRO members are at the forefront of developing and implementing new clinical trial technologies to enhance efficiency and improve the patient experience," says John Lewis, senior VP, policy & public affairs at ACRO. "But the biopharmaceutical industry is heavily regulated so there are several areas where we are requesting that the FDA consult with stakeholders and provide additional guidance to encourage the adoption of these exciting technologies."

Other areas where ACRO members identified substantial benefit/increase in adoption were: risk-based monitoring; electronic informed consent (eConsent); and the use of near real-time trial data analytics. Lewis noted that the FDA has already provided guidance on risk-based monitoring and eConsent and the industry has been actively implementing these tools and technologies. ACRO's members have invested in developing a variety of real-time clinical trial data analytics platforms which are supporting risk-based monitoring, adaptive trial designs and other innovations.

Staffing And Budget Constraints Increase Use Of RBM

Cutting Edge Information reports 80 percent of large- and mid-sized companies are using risk-based monitoring (RBM) as an alternative to in-person monitoring in clinical trials. The survey also found RBM being used by only 38 percent of small companies.

In many cases, constrained staffing and budgetary resources have persuaded companies to consider RBM. Surveyed teams at Top 10 and Top 50 organizations rated RBM less challenging than did similar teams at small organizations and biotech firms. According to interviewed executives, many companies are open to RBM but may be uncertain how to best utilize it. Additionally, cutting back on existing, in-person monitoring practices can be difficult.

"Even companies that don't formally conduct RBM activities benefit from some of its practices," said Sarah Ray, senior research analyst at Cutting Edge Information. "For example, teams are more likely to frequent sites that generate higher numbers of queries. Companies often review data and adjust planned monthly site visits based on what that data reveals."

Seven Technology Trends That Will Revolutionize ePRO Assessments

The eClinical solutions market is currently valued at $3 billion per year, and is expected to grow to almost $6 billion per year in the next four years. According to MedCity News, the fastest growing segment of that market is electronic patient reported outcomes (ePRO), which currently takes in over $500 million per year. The FDA, payers, and the changing technology landscape, particularly the use of handhelds and wearables, are driving the trend.

Zikria Syed, technologist and life sciences entrepreneur, believes electronic clinical outcomes assessment (eCOA), also known as ePRO, is ripe for a technology upgrade. He identifies seven technology trends that will revolutionize these solutions, and clinical trials, in coming years.

  1. Consumerization of the user experience: users bring their own computing devices and apps to the trial. A large ePRO trial can involve tens of thousands of users and go on for years. Reduced training and on-boarding costs and improved engagement can have a significant positive impact on the trial.
  2. Pharma’s move to the cloud is underway, with companies like Veeva providing a multi-tenant cloud solution. ePRO solutions with a large patient user base are suited for a move to the cloud.
  3. Real-time insights and feedback improves usability and becomes more important when engaging a large base of users. Consumer health apps are providing continuous feedback to users and increasing patient engagement.
  4. The use of mobile and tablets in trials is just underway, but will continue to increase. A recent survey of 1,500 clinical trial site contacts by Worldwide Clinical Trials found investigators still prefer paper over electronic entry. As tablets and mobile devices replace the clipboard, solution providers will need to continue to improve the usability of these devices.
  5. Bring your own device (BYOD) is also a growing trend and can reduce the cost of eCOA solutions, where the supplying of devices accounts for about a third of the cost.
  6. Device integration will be a growing area of importance, as the wearables market grew to 78.1 million units in 2015. Smartphone users are also using those devices to track physical activity. Technology in healthcare will continue to be a frontier for invention, which will require sponsors to carefully evaluate new devices and appropriately incorporate them into trial designs.
  7. Big data is also having an impact on the industry. In eCOA solutions, wearables will create large sets of new data about patient health, mobility, activity, and the safety and efficacy of treatments. Big data can also be used for study design and patient/investigator selection to find the best match for trials.