Archive for the 'healthstream' Category

Patient-Reported Data: FDA Cares, So Should You

The FDA has long recognized patient-reported data in clinical trials.  Many widely used drugs have been approved primarily based on patient-reported improvements.  Migraine drugs are typically approved using patient diaries measuring levels of pain relief, phonophobia, photophobia and nausea; the primary endpoint of depression trials is the change from baseline in a standard depression rating scale; Pfizer’s Lyrica (pregabalin) showed efficacy in neuropathic pain based on a difference in average self-reported pain scores between patients treated with Lyrica and placebo; and the list goes on.  Whether FDA’s acceptance of these patient-reported endpoints is due to FDA’s belief in the value of patient-reported outcomes (PROs), or due to the lack of a ‘harder’ endpoint for these trials is debatable.  However, below is a selected list of drugs primarily approved based on PROs, and their peak WW sales.  One can see that relying on patient-reported clinical data has not hindered the success of these products.

FDA Guidance on Patient-Reported Outcome Data

In December 2009, FDA issued a guidance document instructing industry on how to use PROs in drug development and clinical trials.  Although FDA has historically accepted patient-reported outcomes for approval when a more objective endpoint is lacking, they have been very reluctant to include secondary PRO endpoints (such as improvements in quality of life) in approved product labeling.  Some in industry have viewed this new guidance as sign of a shift within FDA toward the importance of patient experience and quality of life measures.  If true, this could have a broad impact on the way drugs are designed, developed and marketed.

To date, PRO data has been underutilized in clinical practice.  However, if FDA begins to recognize PRO measures such as quality of life, pharma companies will begin to generate data on their drugs’ impact on these measures. Physicians will have additional data available when deciding which drug might be best for their patients and will begin to rely more heavily on PRO data.

Importance of PRO Measures

There are many reasons to value PRO measurements.  Some treatment effects are only known to the patient, and the patient provides a unique perspective on treatment effectiveness that goes beyond traditional clinical measurements.  European pricing authorities have long valued the impact of quality of life on drug evaluation, using the quality-adjusted life year (QALY) measurement of disease burden, which includes both the quality and the quantity of life lived.  But even for those who do not believe a focus on the patient is reason enough, there is evidence of the clinical importance of PRO measures.  A study of 293 heart failure patients showed that patient-reported functional status was a prognostic predictor of hospitalizations, quality of life and death; and a meta-analysis of oncology research from 1982-2008 showed a correlation between patient-reported quality of life and survival in cancer patients.

Opportunity for mHealth

All of this, of course, creates a tremendous opportunity for companies working to collect patient-reported data, and I have previously written about the value of longitudinal personal health data streams, or healthstreams, that include both physician/system-generated data (EMR), combined with more frequently added patient-generated data.  Traditionally, patient-reported data has been captured disproportionaley via written diaries.  However, since the most common measures for a PRO instrument are based on the Likert scale and Visual Analog Scale (VAS), it seems clear that any PRO data commonly captured via written patient diaries can be more easily and efficiently captured by electronic means, and work is being done to validate electronic capture methods.

In the future, we will see this type of PRO data regularly captured by patients via mobile devices and mobile apps, and this data will become a common tool for physicians in clinical practice.  Some companies, such as Ringful Health and ReliefInsite (recently acquired by PatientsLikeMe), are already moving in this direction with mobile versions of asthma diaries and pain journals.

Patient-reported outcomes data is valuable and gaining importance in the practice of medicine.  When you see your first pharmaceutical ad featuring claims of improvements to quality of life, you will know the era of patient-reported outcomes has arrived.


Completing the Picture: The Value of User-Generated Health Data Streams

The healthcare industry is undergoing a dramatic and necessary transformation. Empowered patients are taking action and participating in their own healthcare, marking a significant change from the long-standing paternalistic relationship between physician and patient.  Underlying this transformation is a new found access to health information via the internet and personal health data via consumer health devices and applications.

Much has been written about this long coming transformation, which has spawned the Health 2.0 movement, a successful conference series of the same name started by Mathew Holt and Indu Subaiya, the peer reviewed Journal of Participatory Medicine and a series of grassroots unconferences championed by Mark Scrimshire.

To date, many have focused on the challenge of digitizing the vast amounts of health data in the current health system and on the one-way transfer of existing data from the system to the patient via EMRs and PHRsGoogle and Microsoft have launched data warehouses for personal health information, and a few large institutions have embraced this one-way flow of information. The Mayo Clinic recently partnered with Microsoft HealthVault to provide patients access to their medical records, and Kaiser Permanente has been very successful with their “My Health Manager” offering.

Providing access to health information and health data is a logical first step.  The foundation of the modern health industry is health data.   Each time a prescription is dispensed (3.8 billion times in 2007), a medical claim is submitted, or a lab is ordered, a piece of personal health data is generated.  Evidence-based treatment guidelines often focus on specific health data values, such as BP, LDL or HbA1c, for treatment recommendations.  And a modern medical record is simply an aggregation of health data generated at the point of care, including patient reported symptoms, diagnoses, treatments, test results, physician commentary and observations.  However, medical records are punctuated data streams with large gaps in data from time periods in between physician visits.  It is the doctor’s difficult job to fill in these gaps with imperfect information recounted by the patient to complete the picture and treat the patient effectively.

Focusing only on the EMR, or the one-way transmission of personal health data from the system to the patient, misses half of the health data equation.  With increasing frequency, patients are beginning to generate and report their own personal health data.  Sites such as CureTogether and PatientsLikeMe are collecting patient reported data, analyzing the data and generating valuable services for their members.  And recently, we have seen a rapid rise in ‘health trackers’ to record health and wellness information (MedHelp, TheCarrot, DailyMile, hLog, Polka, Ringful).  Data is also being collected and uploaded in the home by personal health devices such as BP monitors with SD cards, WiFi weight scales, USB glucose meters and activity monitors, some now sold at Best Buy.  To date, these patient generated data streams have existed in silos, both disconnected from traditional data streams generated within the current health system and from data generated on other sites.

In the future, each individual’s traditional health data stream (EMR) and user-generated data streams will merge into one personal health data stream, or healthstream, and this healthstream will be owned solely by the patient.  Diagnoses, treatment decisions and lab results will be overlaid on top of more frequently added data from the patient including symptoms, adherence to medication, daily weight, diet, mood, activity levels, sleep patterns, etc.  This complete longitudinal data stream will be valuable to both the physician and patient and become a part of everyday medical practice.  Physicians will prescribe trackers, apps and home health devices, along with their standard Rx, and will receive a report at the patient’s next visit.  Rather than relying on recollections, physicians will have more complete pictures of their patients, and patients will begin to feel like partners in their own care.

This longitudinal healthstream will become a valuable asset for the patient, and will increase in value with each added data point.  Patients will have the right to grant permission to use all or part of their healthstream for medical care, for research and discovery, or even for profit.  I will explore the topics of permission and use of the healthstream in future posts.

For another view of the future of the healthstream, see Dr. Vijay Goel’s post “HealthStreaming: What data would you need in your stream to make your health decisions?”

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