Patients As Data Source


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Patients As Data Source

  1. 1. Participatory medicine and usingPatient Reported Outcomes to informOff-Label PrescribingJeana H FrostCommunicatie, Amsterdam Center for Social MediaVU University Amsterdam
  2. 2. Previous work:Interested in patient-led inquiry into health(graduate school, PLM, VU)Psychology, learning sciences, communications
  3. 3. Talk:1. Understudied indications: Off-label prescribing2. The opportunities available to collect data from patients to inform medical practice3. Example: Evaluation of one data set of off-label use
  4. 4. 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
  5. 5. 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
  6. 6. Current situation• 21% of treatment instances are off-label• 73% based on little to no scientific evidence(Radley, Finkelstein, & Safford, 2006)Why?
  7. 7. 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
  8. 8. The cost of not doing so…Patients at risk of unnecessary and even harmful treatmentsOne 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)
  9. 9. Evidence is lacking but individualexperiences are not.Opportunity to collect patient outcomes
  10. 10. Convergence of factors1. Patient Reported Outcomes (PROs)2. Patients going online3. People adding personal data to larger databases online
  11. 11. 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)
  12. 12. 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)
  13. 13. 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
  14. 14. 2. The Rise of ePatientsHealth consumers are interested in patient generated information, of e-patients…• 41% have read someone elses 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)
  15. 15. 2. The Rise of ePatientsBut 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)
  16. 16. 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)
  17. 17. 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
  18. 18. The Result:People, globally and of all ages, trustproviders and a “people like me” equally 2008 Edelman Trust Barometer
  19. 19. 3. People also putting structured dataonline: tracking outcomes
  20. 20. 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 Members organized to track outcomes on an experimental treatment
  21. 21. One company focuses on using theseoutcomesPatientsLikeMe case study•Started in 2006•Now over 100,000 members•Began by building communities for conditions,now building a broader platform
  22. 22. Opportunity
  23. 23. Core PlatformSHARE FIND LEARNPatients sharing detailed Patients find other patients like The information shared createshealth data is what makes them. They discover what a new knowledge about theour communities unique. options are available for real-world treatments,This information is the basis treatment and if their experience symptoms, and reality of livingof the PatientsLikeMe with their disease is normal. They with illness. Patients learn aboutnetwork and validates each can reach out to others like them their disease and themselves inindividual. for advice and insight. context of the community.
  24. 24. How it works
  25. 25. Value of PatientsLikeMe:Some Results from a User Survey1,323 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
  26. 26. Using the platform to better understandoff-label prescriptions:Studied two drugs prescribed off-label• Amitriptyline• Modafinil
  27. 27. 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 (
  28. 28. MethodAnalyzed site data• Treatment histories – Prevalence – Treatment purpose• Drug evaluations – Efficacy – Side-effects• At time of analysis 53,926 site members
  29. 29. 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
  30. 30. Online version
  31. 31. Results: PurposesCommon purposes: – Insomnia and other sleep problems (27%) – Pain (17%) – Depression (9%)91% for off-label purposesOf note: 8% of ALS patients report taking the drug 40% of them to treat excess saliva
  32. 32. Results: side effectsMost commonly reported side effects: – Feeling sleepy, groggy, or drowsy (46%) – Dry mouth (29%) – Weight gain (22%)Based on 270 side-effect reports
  33. 33. Results: Efficacy of AmitriptylineDrug rated more effective for off-labelcompared to approved purpose: Purpose Moderate or Major Effect Off–label indications 52% Depression 40 %Based on: 70 reports for purpose of depressionAnd, 520 reports for off-label purposes
  34. 34. 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
  35. 35. 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
  36. 36. ConclusionsNumber 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 aunique perspectiveIncorporating the patient voice should be a partof those solutions
  37. 37. Thank Youj.h.frost@vu.nlVU Amsterdam KankerNL
  38. 38. “To listen well is as powerful ameans of communication andinfluence as to talk well.”John Marshall