Irfan Khan

As a clinical researcher, cardiologist, and technologist, I want to commend the groundbreaking work of Dr. Martin’s MyStudies team, the FDA Catalyst team for providing the necessary data framework, and Health and Human Services (HHS) for the support of the project as covered in David Martin’s article “MyStudies Platform Brings Patient Experience to Drug Development.” Above all, the FDA’s Patient EngageÂ­ment Advisory Council-comprised of actual patients-deserves specific credit for bringing the patient voice into the design thinking for technologies in clinical research. This important and empowering project is very much a sign of the frame shift that is now upon us guiding how patients evaluate and engage clinical trials.

	In this project, we can also take encouragement from the clear validation by FDA of mobile input as a reasonable and appropriate media for collecÂ­tion of patient feedback in clinical trials-an apÂ­proach that maximizes collection opportunity and minimizes patient burden. Surely this is the path forward to making the collection ePRO and eCOA as streamlined as possible-as noted by recent advances from Medidata and others.

	And in reviewing the MyStudies capabilities and functionality, additional opportunities to fully capture patient voice present themselves. As Craig Lipset, until recently Pfizer’s head of clinical inÂ­novation, has observed, “There are two flavors to patient experience data-the first flavor includes FDA initiatives like MyStudies as well as patient-focused drug development (PFDD) that focus on the experience of patients with a specific medical condition or the patient experience while using a medication. These are natural and modern extenÂ­sions of patient-reported outcomes (PROs).”

	Lipset goes on to draw a distinction between this data set and the experience data of participaÂ­tion in clinical research itself. “Concurrently, reÂ­search sponsors are concerned with the subjecÂ­tive experience of patients within clinical research studies. This flavor is an extension of sponsor initiatives themed around patient centricity and patient engagement.”

	Today, this second patient experience data set-which would simply ask, “What is it like to participate in a clinical trial? Would you recomÂ­mend it to a friend or family?”-is the last unÂ­tapped data space that could radically transform clinical trial development and execution.

	And unlike our current propensity to collect and then sequester data from patients, this second patient experience data set would do its greatest good for the greatest number of people if it was patient-facing and transparent from inception.

	What would be some components of such an approach to capturing this deeper, more generÂ­alizable patient experience? As noted, it would start by being patient-facing and delivering a more user intent-oriented search experience than our current best options, like the compreÂ­hensive but often overwhelming clinicaltrials.gov. In other words, it would answer the questions that patients already ask as consumers, “what should I do?” and “what do people like me think about this option?”

	While we get this information shopping for shoes, cars, and even doctors, there is no equivalent for evaluating clinical research as a care option. The patient-as-consumer alÂ­ready has a framework in mind for online supÂ­port-from Amazon to Healthgrades-and that is peer-to-peer education and support in the form of ratings and reviews. The transformaÂ­tion we all hope for in patient perceptions of clinical research, ranging from concerns of plaÂ­cebo, guinea pigs, and Tuskegee, is not going to happen as a result of greater data downloads or white papers. It will only happen when the approximately five million Americans who’ve already participated in clinical trials can directly share their experiences with the next five milÂ­lion people considering participating-in a way that is easily digested and resonates.

	As we celebrate the work of the MyStudies team and address the patient experience transÂ­formation upon us, it is exciting to realize we’re at the beginning of the next great opportunity to enhance how we bring new medicines to market.

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The transformation in patient perceptions will only happen when the five million Americans who’ve already participated in clinical trials can directly share their experiences with the next five million people considering participating.

Patient Experience Comes to Drug Development

	As a clinical researcher, cardiologist, and technologist, I want to commend the groundbreaking work of Dr. Martin’s MyStudies team, the FDA Catalyst team for providing the necessary data framework, and Health and Human Services for the support of the project as covered in David Martin’s article 

“MyStudies Platform Brings Patient Experience to Drug Development.”

 Above all, the FDA’s Patient Engagement Advisory Council-comprised of actual patients-deserves specific credit for bringing the patient voice into the design thinking for technologies in clinical research. This important and empowering project is very much a sign of the frame shift that is now upon us guiding how patients evaluate and engage clinical trials.

	In this project we can also take encouragement from the clear validation by the FDA of mobile input as a reasonable and appropriate media for collection of patient feedback in clinical trials-an approach that maximizes collection opportunity and minimizes patient burden. Surely this is the path forward to making the collection ePRO and eCOA as streamlined as possible-as noted by recent advances from Medidata and others.

	And in reviewing the MyStudies capabilities and functionality, additional opportunities to fully capture patient voice present themselves. As Craig Lipset, until recently Pfizer’s Head of Clinical Innovation, has observed, “there are two flavors to patient experience data–the first flavor includes FDA initiatives like MyStudies as well as Patient-Focused Drug Development (PFDD) that focus on the experience of patients with a specific medical condition or the patient experience while using a medication. These are natural and modern extensions of Patient-Reported Outcomes (PROs).” 

	Lipset goes on to draw a distinction between this dataset and the experience data of participation in clinical research itself. “Concurrently, research sponsors are concerned with the subjective experience of patients within clinical research studies.  This flavor is an extension of sponsor initiatives themed around patient-centricity and patient-engagement.”

	Today, this second patient experience data set, which would simply ask – “Wwhat is it like to participate in a clinical trial? Would you recommend it to a friend or family?” – is the last untapped dataspace that could radically transform clinical trial development and execution.

	And unlike our current propensity to collect and then sequester data from patients – this second patient experience dataset would do its greatest good for greatest number of people if it was patient facing and transparent from inception. 

	What would be some components of such an approach to capturing this deeper, more generalizable patient experience? As noted, it would start by being patient-facing and deliver a more user intent-oriented search experience than our current best options, like the comprehensive but often overwhelming, clinicaltrials.gov. In other words, it would answer the questions that patients already ask as consumers – “what should I do?” and “what do people like me think about this option?” 

	While we get this information shopping for shoes, cars, and even doctors-there is no equivalent for evaluating clinical research as a care option. The patient-as-consumer already has a framework in mind for online support–from Amazon to Healthgrades–and that is peer-to-peer education and support in the form of ratings and reviews. The transformation we all hope for in patient perceptions of clinical research, ranging from concerns of placebo, guinea pigs, and Tuskegee, is not going to happen as a result of greater data downloads or white papers.  It will only happen when the approximately five million Americans who’ve already participated in clinical trials can directly share their experiences with the next five million participants people considering participating - in a way that is easily digested and resonates.

	As we celebrate the work of the MyStudies team and address the Patient Experience transformation already upon us, it is exciting to realize we’re at the very beginning of the next great opportunity to enhance how we bring new medicines to market.

Irfan Khan, writes that though we celebrate MyStudies' teams work, the FDA’s Patient Engagement Advisory Council deserves specific credit for bringing the patient voice into the design thinking for technologies in clinical research. 