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Jeana H FrostCommunicatie, Amsterdam Center for Social MediaVU University Amsterdam
Participatory medicine and using Patient Reported Outcomes to inform Off-Label Prescribing
Previous work: Interested in patient-led inquiry into health (graduate school, PLM, VU)
Psychology, learning sciences, communications
Talk:
1. Understudied indications: Off-label prescribing
2. The opportunities available to collect data from patients to inform medical practice
3. Example: Evaluation of one data set of off-label use
Off-label prescribing
• Before a drug is brought to market in the U.S., Federal Drug Administration (FDA) legislates 3 phases of testing including efficacy testing for a single purpose
• Once a drug is brought to market, physicians are free to prescribe drugs for both the tested purpose and for “off-label” purposes/indications although pharmaceutical companies cannot promote off-label uses
Current situation
• Clinicians are innovators testing novel uses of existing drugs within their practice
(Demonaco, Ali, & von Hippel, 2006)
• Patients increasingly are forcing and even steering this innovation
Current situation• 21% of treatment instances are off-label
• 73% based on little to no scientific evidence(Radley, Finkelstein, & Safford, 2006)
Why?
The cost of gathering evidence
• $15,700 per patient phase 1 trial
• $26,000 phase 3 (Goldfarb, 2006)
• $100 – 800 million to bring a drug to market (Fee, 2007)
• Drugs are commonly off-patent
• Possible disincentive, could remove an existing product line
The cost of not doing so…
Patients at risk of unnecessary and even harmful treatments
One example: In one year (2002), 63% of 392,000 terbutaline
prescriptions were for pregnant women (Young, Adams, 2003)
No demonstrated benefits and instances of real harm (Haas, 2007; Nanda et al, 2002; CTAF, 2002)
Evidence is lacking but individual experiences are not.Opportunity to collect patient outcomes
Convergence of factors
1. Patient Reported Outcomes (PROs)
2. Patients going online
3. People adding personal data to larger databases online
1. Patient Reported Outcomes (PROs)• Have strong prognostic value (Rothman,
2007)
• Less prone to memory bias (Hufford, 2002)
• Provide a sense of anonymity to research subjects and are less vulnerable to misrepresentation of sensitive topics (Turner, 1998)
1. Patient Reported Outcomes (PROs)• Food and Drug Administration (FDA) and
the European Medicines Agency (EMEA) have released guidelines on how to standardize PROs for use within research and drug labeling.
• Usage of PROs common in domains ranging from oncology (Basch, 2009) to allergy treatment (Baiardini, 2010)
2. The Rise of ePatients
• 74% of American adults go online
• 61% of adults look online for health information, termed "e-patients”
(Fox, 2009)
Similar access in the Netherlands
2. The Rise of ePatientsHealth consumers are interested in patient
generated information, of e-patients…• 41% have read someone else's commentary or
experience about health or medical issues online• 24% have consulted rankings or reviews online
of doctors or other providers. • 24% have consulted rankings or reviews online
of hospitals or other medical facilities.
(Fox, 2009 - Pew Internet and Life)
2. The Rise of ePatients
But few are actively writing or creating new health content: of e-patients …
• 6% have tagged or categorized health or medical content• 6% have posted in an online discussion, listserv, or other online group
forum. • 5% have posted comments about health on a blog. • 5% have posted a review online of a doctor. • 4% have posted a review online of a hospital. • 4% have shared photos, videos or audio files online about health or
medical issues.
In sum, 37% of adults, or 60% of e-patients, have done at least one of the above activities.
(Fox, 2009 - Pew Internet and Life)
An aside: Benefits to patients of participating in online discussions
• Patients learn from one another not only “medical” information but also how to live with a condition
(Hoch)
• Participation heightens levels of knowledge, well-being, and empowerment
(Barak et al, 2008)
• Concerns about negative effects of participation have not been proven e.g. communities self-correct
(Esquivel et. al. 2006)
New sources of authority in healthcarePatients themselves as experts“Americans' pursuit of health takes place within a
widening network of both online and offline sources. Whereas someone may have in the past called a health professional, their Mom, or a good friend, they now are also reading blogs, listening to podcasts, updating their social network profile, and posting comments. And many people, once they find health information online, talk with someone about it offline.”
Susannah Fox, The Social Life of Health Information Pew Internet Report
The Result: People, globally and of all ages, trust providers and a “people like me” equally
2008 Edelman Trust Barometer
3. People also putting structured data online: tracking outcomes
3. Significance to patients: Participatory Research “As these [life raft] examples show, patient-driven research will become
more and more important, and will provide an example of the way things will go in the future. As other patient groups begin generating their own medical data, it will change the relationships between research professionals, clinicians, and patients quite dramatically.
Gilles Frydman, President of Association of Online Cancer Resources
PatientsLikeMe ALS Membersorganized to track outcomes on an experimental treatment
One company focuses on using these outcomes
PatientsLikeMe case study
•Started in 2006
•Now over 100,000 members
•Began by building communities for conditions, now building a broader platform
Opportunity
SHAREPatients sharing detailed health data is what makes our communities unique. This information is the basis of the PatientsLikeMe network and validates each individual.
LEARNThe information shared creates a new knowledge about the real-world treatments, symptoms, and reality of living with illness. Patients learn about their disease and themselves in context of the community.
FIND Patients find other patients like them. They discover what options are available for treatment and if their experience with their disease is normal. They can reach out to others like them for advice and insight.
Core Platform
How it works
Value of PatientsLikeMe:Some Results from a User Survey
1,323 Users completed the survey
(19% response rate)• 71% helpful in learning about the symptoms
they are experiencing• 67% they are getting better care by recording
symptoms over time• 69% agreed with “I feel more comfortable
asking for information on sensitive topics such as sex, sexuality, religion, or spirituality ” than online and offline support groups
Using the platform to better understand off-label prescriptions:Studied two drugs prescribed off-label1. Amitriptyline
2. Modafinil
The Amitriptyline Case
• The drug most commonly prescribed off-label (Radley, et. al. 2006)
• One FDA approved purpose (depression)
• 14 Amitriptyline clinical trials recruiting subjects (clinicaltrials.gov)
Method
Analyzed site data
• Treatment histories– Prevalence – Treatment purpose
• Drug evaluations – Efficacy– Side-effects
• At time of analysis 53,926 site members
Amitriptyline Dataset
• 1,394 treatment reports for amitriptyline
• 865 currently taking the drug
• 2% of members
• 1,197 evaluations of Amitriptyline
• 5 PatientsLikeMe communities MS, Parkinson’s, Mood Conditions, ALS, Fibromyalgia
At time of analysis there were 53,926 site members
Online version
Results: Purposes
Common purposes:1. Insomnia and other sleep problems (27%)
2. Pain (17%)
3. Depression (9%)
91% for off-label purposes
Of note:8% of ALS patients report taking the drug
40% of them to treat excess saliva
Results: side effects
Most commonly reported side effects:– Feeling sleepy, groggy, or drowsy (46%)– Dry mouth (29%) – Weight gain (22%)
Based on 270 side-effect reports
Results: Efficacy of Amitriptyline
Drug rated more effective for off-label compared to approved purpose:
Based on: 70 reports for purpose of depression
And, 520 reports for off-label purposes
Purpose Moderate or Major Effect
Off–label indications 52%
Depression 40 %
Study Conclusions
• Patient platforms offer a unique data source for understanding utilization and treatment experience across populations
• Can access more cases than traditional trials with a low barrier to participation
• This patient reported outcomes from these sites can provide evidence about: – Secondary uses of drugs (excess saliva) – Drugs that are off-patent and therefore unlikely to be
studied systematically
The Problem in 1870’s and Now?"Medical knowledge is at a low ebb among us," said Mr. Bulstrode,
…"I, for my part, hail the advent of Mr. Lydgate. I hope to find good reason for confiding the new hospital to his management."
"That is all very fine," replied Mr. Standish, who was not fond of Mr. Bulstrode; "if you like him to try experiments on your hospital patients, and kill a few people for charity I have no objection. But I am not going to hand money out of my purse to have experiments tried on me. I like treatment that has been tested a little."
"Well, you know, Standish, every dose you take is an experiment-an experiment, you know," said Mr. Brooke, nodding towards the lawyer.
George Eliot, Middlemarch 1876
Conclusions
Number of challenges for data collection•Rise of personalized medicine•Growing concern about the ethics of clinical trials
Need for novel solutions Patients are motivated to participate and have a unique perspectiveIncorporating the patient voice should be a part of those solutions
“To listen well is as powerful a means of communication and influence as to talk well.”
John Marshall
