Web-based Assessment of Patient Reported Outcomes (PRO) Data in Cancer Patients: Potential Impact on Drug Approvals and Health Care Reimbursements

Scott K. Dessain, M.D., Ph.D.

At the EXL Pharma 5th Annual Digital Pharma East conference in Philadelphia on October 19, 2011, I was impressed by the approaches being taken to authentically engage the pharmaceutical customer. Something was missing, though, from many of these efforts: learning from the patients.

There is an important synergy between the need to communicate meaningfully with patients and the need to understand what they can tell us about their health, illness and treatments.  This information is called patient reported outcomes (PRO) data, and it is changing every aspect of the health care field.

I’m not the type of person who would generally be contributing to this blog. Most of the contributors are thought leaders in social media, marketing, and communications, whereas I’m an academic oncologist and medical researcher at Lankenau Hospital in the suburbs of Philadelphia, Pennsylvania.

I’ve come to start a discussion and gain your insights on a web-based format for PRO data collection and how such a website can incorporate key teaching, compliance, and relationship-building components to strengthen the provider/patient relationship and help support the reasonable use of health care dollars.

What is PRO data and why does it matter?

PRO data refers to information elicited from the patient that reflects the patient’s personal experience of their medical condition. It includes things only a patient can interpret and report, e.g., fatigue, pain, cognitive and social functioning, physical functioning, emotional state, appetite, and sleep quality.

Why do we spend so much money on healthcare in the United States without getting measurably better outcomes than are obtained in more frugal countries? How can we be sure that the money we are spending is worthwhile?

As we try to improve the cost-effectiveness of our healthcare system, it will become increasingly important to know if what we are doing makes sense. This isn’t just because we want to make sure we’re doing what’s best for patients. It’s also because those paying the bills will want us to justify the costs.

This is why PRO data matters. Basically, healthcare payers won’t pay for medical treatments that do not improve quality of life for patients, but we simply do not have enough of this kind of data.

One of my physician colleagues said recently at lunch, “I saw 7 patients in clinic this morning and didn’t make a single evidence-based decision.”  It is not that he is not a medical scholar. The fact is that much of what we do in medicine is not validated by clinical data. This is especially the case for clinical PRO data.

How I got into this

My immunology research laboratory works on therapeutics for biodefense, infectious disease, and cancer. This means we try to understand how the human immune system uses antibodies to fight disease and use that understanding to make drugs for people.

I also founded a biotech company, Immunome Inc. (www.immunomeinc.com), which provides human monoclonal antibodies for others that are looking to diagnose or treat human diseases.  When I started Immunome, I never guessed that I would need to stick my head out of my science cocoon.

But something has been nagging at me as my company strives to create $1B antibody therapeutics. What happens if we are successful? What if the thousands of other biotech companies in the United States also create $1B drugs?

Who is going to pay for all these drugs, in a society strained by an overwhelmed budget and debt? How are we going to know that our therapies are worth the money, or even how much to pay for them?

A lot will depend on our ability to demonstrate to payers that we are improving the quality of life (QOL) for our patients. We are already seeing how QOL data can have important financial implications for the biotechnology and pharmaceutical industry.

PRO data and the FDA

Genentech recently lost the FDA indication for giving bevacizumab, Avastin, to patients with metastatic breast cancer. This is a decision that will cost the company over $1B annually, and it means that we may have difficulty obtaining insurance company reimbursement for patients who need the drug.  (I honestly don’t know if that was the right decision, but it seems clear that some women do very well with it).

Data in multiple studies showed that patients who received Avastin had a longer period of survival without progression of their cancer. However, the overall survival (the average time that the patients lived after starting treatment) for these patients was not improved. In their presentation to the FDA Oncology Drugs Advisory Committee in July, 2010, (http://www.fda.gov/AdvisoryCommittees/Calendar/ucm213724.htm) Genentech argued that the quality of life for patients who have a longer progression free survival is better. This may make intuitive sense.  It means that women who receive the drug would have a longer time without facing the inevitable progression of their fatal disease.

But Dr. Wyndham Wilson of the NCI asked,  “So, are there data either from the study are from past studies in metastatic breast cancer where progression free survival translates to an improvement in patient reported outcomes?”  Dr. Sandra Horning of Genentech noted that they had evidence from one study, AVADO, which demonstrated that quality of life in the Avastin group was not worse.  But she further commented on the challenges in obtaining quality of life (QOL) data, due to need to measure different symptoms and because it is “collected at intervals that may or may not capture quality-of-life changes.”

Everyone learned a valuable lesson from this. If we want our drugs to be paid for in the future, we will need to demonstrate that they make a difference in a way that is important to the patient.

The medical research community has been alert to this problem for some time. I searched the National Institutes of Health clinical trials database (clinicaltrials.gov) website for “quality of life” and found over 11,000 trials that are measuring some aspect of “quality of life”. Of these, cancer trials numbered 3396.

The FDA in 2009 provided guidance on this issue:

“Findings measured by a well-defined and reliable PRO instrument in appropriately designed investigations can be used to support a claim in medical product labeling”


So, PRO data is going to be helpful and/or essential to the approval of drugs in the future.

A clinical trial for PRO data collection on the web

At Digital Pharma East conference I presented a talk entitled “Examining A Clinical Trial that Uses Web-Based Video To Capture Quality Of Life Data In Cancer Patients”. The potential for using web communication in medicine is untapped and vast. Many people, especially patients, are in contact with the web through some device. A web-based application crosses socioeconomic boundaries because it can be accessed via mobile and other web-enabled devices.

Can we create a web-based collection method for PRO data that is timely, accessible, comfortable, and effective? What would be the best format for this?

Survey questions asked by a person in a video format would be an improvement over a paper survey. The web-based survey could be answered on any platform, it could be taken at a time convenient for the patient, and the data would be securely and seamlessly entered into the clinical trial database.

My group at Lankenau has written a study of a web-based survey.  It has been recorded in a video format by ehealthcareTV, who will be hosting the website and collecting the data. We will be testing the prototype study in the clinic soon. Watch this space for further updates as we develop the survey method.

Why is this trial important?

Even as the health care financing is undergoing tremendous change, our patients are changing, as well.  They are becoming increasingly comfortable with and reliant on the web for communication and medical information. They are becoming more involved in the medical decision process. Lastly, they are struggling along with health care providers to ensure that what we do, and what gets reimbursed, makes sense for them and for society.

We need to find the best ways to use media and the web to build trust and learn what our patients can teach us.  The web is one of the best ways to communicate with our patients. We need to learn how to use the web to better listen to them.

How you can help

We’ve started with quality of life assessments in cancer patients.  We’re interested in learning what other types of data would particularly benefit from a web-based survey method. Also, could a web-based survey be used to simultaneously convey positive messages (lifestyle, encouragement). These could help improve patient awareness and health, as well as building trust between caregivers and patients.  Do you have any ideas or suggestions? For further information, contact me or check out the website: www.eHealthcareTV.com.

Thank you to my friend Christine Cavalier, M.Ed., an expert in Social Media Education and Psychology (www.purplecar.net), for her contribution to the study design and to this article.

Scott K. Dessain, M.D., Ph.D. is a co-founder of eHealthcareTV and an Associate Professor at the Lankenau Institute for Medical Research.

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