The emerging role of wearable devices for real-world data collection: Engagement or Activation? October 18, 2016 Thom Schoenwaelder Vice President, PAREXEL Access, PAREXEL International

Today’s exploration Agenda How will RWD (real world data) be affected by the growth of wearables, biosensors, smart devices and apps? How will the use of RWD change utilization of Real World Evidence for access evaluation, and clinical development? Can access to this data change the use of information to engaging stakeholders and activate them to take action at the sub- population and broader level? Is this new trove of information able to change the threshold for evidence for access decisions and will it impact the definition of value moving forward?  

Definitions for today’s discussion For the sake of this discussion, Real World Data (RWD) will be defined as: Any patient generated data that is used for decision-making, collected outside of a randomized clinical trial Real World Evidence (RWE) : Relevant facts or data that has been analyzed using clinical, scientific and/or commercial rigor used in support of a conclusion or decision

What do we mean by “Wearables”? Wearables are devices worn, applied, or ingested that are designed to measure or capture health-related data in digital format without intervention by the subject and their associated apps. Examples include: Ingestible Sensors Smart Contact Lenses Biometric Watches Body-Worn Sensors Mobile Analytics Tools Wi-Fi Skin Sensors Info Source and Image Inspiration: Google; Samsung, Polar, MC10, Proteus Digital Health, CBS News, John Rogers

As wearables evolve generationally, more sophisticated data will become available With each successive generation of wearable technology the data collected is also becoming more clinically relevant, including: ECG Heart Rate Blood Flow Temperature CO2 and O2 levels Simulated Blood Pressure Real-time ECG Activity Tracker Wi-Fi & Bluetooth (It also tells the time)

Approximately between 4. 4 and 7 Approximately between 4.4 and 7.5% of all mobile apps are health related Source: Statista.com, used with permission

The use of health apps has more than doubled in the last two years

Reach of Mobile Apps Mobile Health Apps have a 35% Market reach in the Mobile app market The estimated number of mobile devices in the U.S. is approximately 4.43 billion in 2015 according to Statista Based on current estimated numbers this represents over 1.5 billion devices in the U.S. alone with some ability to collect health or fitness information, making the data collection potential enormous Source: Statista.com, used with permission

Changing the view of real world data

The Promise… Vast amounts of objective patient generated data creating innumerable benefits and deep insights.… The hope is: No patient interference in data collection Data collection up to 24/7/365 Broad-based, persistent longitudinal data Population based metrics resulting in actionable insights Identification of clinical endpoints based on data that could result in better outcomes Discovery of digital “flags” to uncover areas in need of further study To utilizing the data to prove product differentiation in a meaningful way Based on current state, there is a realization that the promise in unable to live up to the practice

In practice (for now)… All data are inherently flawed!... The reality is: Policies will need to be created to deal with this influx of data and the myriad issues it creates. HIPAA: Digital health companies are not bound by HIPAA, since the patient data is not connected to a protected “covered entity”, therefore digital health companies may use the data as they see fit No data collection guidelines or standards; data transparency Meager to non-existent quality control Data integrity No unified, integrated data aggregation or validation Little to no interoperability of devices or data sets real world data can serve to complement clinical trials, but will never replace them

Providers Patients Real World Data Policymakers Manufacturers Payers Data stakeholders Policymakers Manufacturers Payers Real World Data Patients Providers

All stakeholders may benefit from access to Real world data Payer: Provides evidence that a product will perform as expected in their populations; Enable clearer differentiation of products within a specific class; can provide like-to-like comparative evaluations of products to make coverage and reimbursement decisions Providers: Can gain a more well informed view of a product’s efficacy in a broader patient population or sub-population and can see results in patients that may have been otherwise excluded from a RCT Manufacturers: Will have the ability monitor the performance and safety of their products that could lead to patent extensions or new indications Policymakers: Will be able to combine RWD with other clinical and economic data to make prudent healthcare spending decisions Patients: Receive access to more effective treatments, more confidence that their treatment will perform as expected, with better outcomes

The issues are significant, however not insurmountable As the ubiquity and access to these increasingly relevant data becomes more widespread, the issues will have to be addressed by regulatory bodies in order for promise to meet practice: Questions around the adequacy of HIPAA are being discussed in light of the increasing volume of unregulated personal health data collected by digital health companies Deciding which data mining tools can be validated and used being used to uncover clinically rigorous and valuable insights Creation of accepted standards being developed around data transparency and integrity Establishment of regulatory guidelines allowing wearable RWD submission to be evaluated alongside other clinical data

A Global perspective on Real World Data

Regulatory View of Data evaluation (U.S.) COST-EFFECTIVENESS ANALYSIS REAL WORLD EVIDENCE OBSERVATIONAL STUDIES RANDOMIZED CLINICAL TRIALS

Health Technology Assessment View of data evaluation (ex-U.S.) REAL WORLD EVIDENCE OBSERVATIONAL STUDIES COST-EFFECTIVENESS ANALYSIS RANDOMIZED CLINICAL TRIALS

In conclusion

Real world data has significant potential, however there is more work to be done “We hope that there is patient- generated evidence that can be journal worthy and even find its way into regulatory submissions for new products“… ...“If we can get that trifecta of patient-generated data, outcomes data and medical records all in the mix, we'll have a really amazing real-world evidence base to use for all kinds of decisions.” Jeremy Gilbert, Vice President, PatientsLikeMe Source: ABBVIE Newsroom, Real-World Data Rounds Out Value Picture of Drugs; Jun 08, 2016

Questions?