1. Innovative Post-Market Research Methodologies to Access Patients
and Data Without Traditional Investigator Sites
Peri and post-market research is used to ensure that the mandate of patient centered outcomes is
addressed (for both payers and regulators), that prescribers can more readily distinguish between
therapeutic choices, and that payers understand the full impact of a drug when assessing their return on
investment. All of this information is used by sponsor organizations to understand the breadth and depth
of their product’s profile, and to properly position it in the marketplace.
The term ‘low-intervention’ has recently emerged from the EMA in its Regulation (EU) No. 536/2014,
published in May 2014. This term describes reseach in which the drug/therapy is used in accordance
with the approved product label, but does require some minimal diagnostic or monitoring procedures that
pose only minimal risk to patients. Examples of these minimal risk tests would include things like an eye
exam, a blood draw, a urine sample, a pulmonary function test, etc. In these study designs, very large
numbers of patients are often needed (into the thousands), and they can be followed either longitudinally
over the course of the disease, or for several years. In this circumstance, it would be overwhelming
expensive to recruit the very large numbers of investigator sites needed, and to pay those sites to follow
patients over multiple years.
Decreased Involvement from Traditional Investigator Sites
In the environment of low-intervention studies, I have seen and recommended various innovative
approaches to procure data from enormous numbers of patients. An example of this type of innovation is
to use various means to identify study patients without a traditional investigator. Options that have
been/are being implemented are:
• Utilize a large capacity clinical research unit (eg. a phase I unit) with capabilities for multiple forms
of clinical assessments, and arrange for large groups/cohorts of patients to have their minimal
diagnostic/monitoring assessments done there
• Involve pharmacists as the investigator where the research question does not involve a direct
clinical assessment
• Avoid the use of investigator sites all together by sending clinicians to patients’ homes or to a
central location such as a community center, rather than having patients come to a clinic.
In the 3rd option, traveling clinicians (i.e. nurses) can visit patients at a location convenient to the patient,
and perform straight forward clinical assessments and/or collect basic samples. Examples of what can be
done at patients’ homes include measures of height, weight, pulmonary function testing, as well as the
collection of blood and urine. This methodology is very conducive to patient retention in long-term
studies. It may of course also be viable and preferable to involve various combinations of the options
listed above depending on the study population and reseach question.
There are various logistic considerations that need to be considered in order for these methodologies to
be effective. These logistic considerations include the standardization of the equipment used to collect
the data, training of the staff who interact with the patients, clear protocol stipulations of how to handle
unsolicited clinical information including safety data, as well as integration of ‘mobile’ data into any other
data being collected in the study. In all cases, the methodologies employed must also align with the
standard of care in the particular region, cultural sensitivities, as well as be minimally intrusive to both
patients and clinicians alike.
On-Line ‘Virtual’ Data Collection of Patient Reported Outcomes
David Selkirk 30-Apr-2015 Page 1 of 2

2. Innovative Post-Market Research Methodologies to Access Patients
and Data Without Traditional Investigator Sites
Other types of post-market research collects Patient Reported Outcomes (PRO) as the key set of data,
rather than any type of clinical or laboratory assessments. In these investigations, it can be possible to
interact with patients in a completely virtual way. Study patients can be consented over the phone and/or
the internet, and patients subsequently complete questionnaires on-line at pre-determined timepoints. In
this setting, it is important to have both pro-active reminders (text, e-mail, phone), as well as reactive
mechanisms (same modalities) to prompt patients who are tardy in completing required data. The design
of the instrument is also very important as some regulators (specifically the FDA) consider a PRO
questionnaire completed on a desk-top computer to be a different modality than completing the same
questionnaire via a smart phone or tablet.
In all of these circumstances, it is important to have a clear plan to identify study patients. Social media
can be a powerful tool to access potential study patients in chronic conditions, as the world’s population is
now irrevocably linked to this medium for accessing information and sharing experiences with other
patients.
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