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Patient Engagement: Co-designing outcomes that matter, why and how

Bray Patrick-Lake, MFS
Duke Clinical Research Institute

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Patient Engagement: Co-designing outcomes that matter, why and how

  1. 1. Patient Engagement: Co-designing outcomes that matter, why and how Bray Patrick-Lake, MFS Director of Stakeholder Engagement, Duke Clinical Research Institute
  2. 2. The current research and health care system is broken. Patients and research participants are over it. 2
  3. 3. What we dream of … 3 Precision health fueled by engaging, impactful research that is person-centered and efficient, which generates evidence- based prevention strategies, reliable and timely diagnosis, and effective treatment options that are responsive to individual needs, characteristics, and preferences. Yet … Precision health fueled by engaging, impactful research that is person-centered and efficient, which leads to evidence-based prevention strategies, reliable and timely diagnosis, and effective treatment options that are responsive to individual needs, characteristics, and preferences.
  4. 4. It’s hard to be healthy when the understanding of a person’s health is dangerously segmented and unidimensional. – Incomplete and disintegrated health records – Incomprehensibly short-sighted snapshots of health absent of the dynamic family and community frameworks that people are embedded in – Tunnel-vision practice of “our best guess” medicine divided by disease/body part • Misses important holistic views for prevention, diagnosis and treatment • Leaves people at risk of preventable adverse events • Fails to identify opportunities to participate in research • Failure to develop medical practices and products with greatest benefit to patients 4
  5. 5. IT TAKES 12 YEARS OR LONGER TO BRING A NEW TREATMENT TO PATIENTS The drug development pyramid 1 2 2-5 5-10 10-20 Substances 10,000-30,000 Substances Synthesis, Examination & Screening Preclinical Tests (Animal) Clinical Tests (Human) Product surveillance Phase IV Phase III Phase II Phase I Basic Research Development Introduction Registration PHARM R&D 0 2 3 4 6 9 8 11 12 5 7 10 1 Years Briggs. Effective Use of Health Technology Assessment to Maximize Market Access. http://www.iconplc.com/icon-files/insight-newsletter/June11/effective.html. Accessed 16 Nov 2012
  6. 6. Based on my personal characteristics, what can I expect my outcomes to be? WE DON’T KNOW.  Therapies reach market after study in cleanest population possible (often lacking critical diversity)  Research results often an average; some receive benefit, others harm  Subgroup analysis on single variables  Treatment timing, combinations and sequencing often not studied  Waste of finite resources treating patients who: • aren’t at risk; • won’t benefit; and whose • transition to disease could likely have been prevented. 6
  7. 7. Failure to Ascertain Interpatient Variability WHY? When we take a traditional research approach looking only at clinical and claims data two patients may look identical. Both live 10 years and have the same number of hospital admissions, but one has a miserable life and the other a high quality life.
  8. 8. 8 It’s time for more relevant and impactful research that uses an integrated approach to understanding health and disease. Unique Me In 3D diet behavior wearables environment exercise lifestyle work pets family Clinical Data social media appsclaims data genetics consumer data
  9. 9. You can’t truly understand a person’s health without taking into account the health of their community. 9
  10. 10. 10 When Your Zip Code Says You’re Going to Die Earlier Than Others Like You Why are some neighborhoods and people so much healthier than others?
  11. 11. 11 Mapping Life Expectancy  Virginia Commonwealth University, Center on Society and Health, Mapping Life Expectancy RWJF funded project Education and income • communities with weak tax bases cannot support high-quality schools and jobs are often scarce in neighborhoods with struggling economies. Unsafe or unhealthy housing • exposes residents to allergens and other hazards like overcrowding. Stores and restaurants selling unhealthy food may outnumber markets with fresh produce or restaurants with nutritious food. Opportunities for residents to exercise, walk, or cycle may be limited • some neighborhoods are unsafe for children to play outside Proximity to highways, factories, or other sources of toxic agents • exposes residents to pollutants Access to primary care doctors and good hospitals limited Unreliable or expensive public transit • isolates residents from good jobs, health and child care, and social services Residential segregation • features that isolate communities (e.g., highways) can limit social cohesion, stifle economic growth, and perpetuate cycles of poverty
  12. 12. To create outcomes that matter we have to understand people’s lives; and to do that we must engage with people and see the world through their eyes. 12
  13. 13. “If patient engagement were a drug, it would be the blockbuster drug of the century and malpractice not to use it.” Leonard Kish (2012)
  14. 14. There has been a significant shift in how research sponsors and investigators engage with patients, moving away from the traditional approach of keeping patients at arm’s length as passive subjects in clinical trials, toward one of recognizing them as active partners across the drug research and development continuum. As patient organizations, industry leaders and regulatory officials work to achieve patient centricity and develop mechanisms for ensuring the patient voice is incorporated into all aspects of therapy development, evidence of the positive impact of patient engagement is mounting. Background
  15. 15. What’s driving patient engagement?  PCORI and PCORnet/PCRF  Patient-focused Drug Development  NIH funding requirements  Precision Medicine Initiative Cohort Program (All of Us)  21st Century Cures Act  Clinical Trials Transformation Initiative  FasterCures  National Health Council
  16. 16. Background American Cancer Society (ACS) forms to raise disease awareness 1913 1946 ACS raises $1 million for research ACS lobbies for passage of National Cancer Act; NCI receives funding to expand 1971 1983 Orphan Drug Act passed Act Up attends AIDS Clinical Trials Group meeting 1989 1995 Genentech works with NBCC on expanded access for Herceptin Advancing Breakthrough Therapies for Patients; FDASIA Act 2012 2013 FDA Patient- Focused Drug Development; Benefit-Risk CFF sells drug royalties for $3B; PPMD submits FDA draft guidance on therapy development 2014 Many of today’s patient groups serve as active partners in the clinical trial enterprise and invest private funding in milestone driven research with focus on leveraging their assets to de-risk research and increase return on investment.
  17. 17. https://www.fda.gov/downloads/Drugs/NewsEvents/UCM603130.pdf
  18. 18. CTTI patient group engagement across the R&D continuum From BENCH to BEDSIDE and BACK Pre- Discovery Pre- Clinical Phase 1/2/3 FDA Review & Approval PAS/ Outcomes www.ctti-clinicaltrials.org/projects/patient-groups-clinical-trials • Fundraising and direct funding for research • Providing translational tools (assays, cell & animal models, bio-samples, biomarkers, etc.) • Helping define study’s eligibility criteria • Natural history database & patient registry support • Input on meaningful clinical endpoints • Assistance re informed consent form • Working with FDA re benefit-risk and draft guidance • Accompanying sponsor to Pre-IND FDA mtg to advocate for study • Input re interest of research question to patient community • Providing data on unmet need & therapeutic burden • Fundraising and direct funding for research to identify target molecules • Facilitating collaboration with NIH • Characterizing the disease & relevant mechanisms of action • Fundraising & direct funding for research, trial operations support • Assistance in selecting & recruiting optimum clinical sites • Clinical infrastructure support • Helping educate/motivate patient community & recruit for trials • Providing patient feedback on participant experience • Serving on Data & Safety Monitoring Board • Input for any trial adaptations or modifications • Accompanying sponsor to milestone meetings, e.g., after phase 2 &3 • Providing public testimony at the FDA Advisory Committee & other FDA hearings • Preparing submission for newborn screening when appropriate • Serving on post-market surveillance initiatives • Helping return study results to participants • Co-presenting results • Publications/communications re results • Feedback on how patient community views results • Natural history database & registry support • Working with payers re reimbursement
  19. 19. Patient Input in Device Total Product Life Cycle O’Callaghan 6 Oct 15 CDRH
  20. 20. Patient Partners Are Providing Unique Value  De-risking early-stage development with funding and public-private partnerships for basic, translational, and early clinical research  Reducing uncertainty in the regulatory process by working closely with the regulators throughout the entire R&D process  Helping to develop more effective, efficient trials with a greater chance of success through: – better questions and study design – efficient recruitment and improved retention – fewer protocol amendments – procedures that are better-suited to the patient – clinical endpoints that are well-grounded in the natural history of the disease – potential benefits that are most important to the patient
  21. 21. CTTI PGCT Economic Model Overview  Main drivers of drug valuation are cost, revenue, timing, risk and intangibles -- patient engagement impacts on them all  Expected net present value (ENPV) modeling can account for most drivers in a clear and well-accepted summary metric  For engagement activities resulting in avoiding an amendment and/or an improved patient trial experience, the benefits in cost and ENPV vastly outweigh the resources spent on engagement  ENPV modeling and similar approaches can support sponsor decisions to increase patient engagement throughout development www.ctti-clinicaltrials.org/projects/patient-groups-clinical-trials
  22. 22. What can happen when patients provide input on study design? Consider two major mechanisms that can impact on project value: 1. May avoid a protocol amendment – ~70% of phase 2 and 3 trials have at least one amendment* – ~22% of amendments are due to recruitment difficulty or feedback from sites or investigators* 2. Improving the patient experience – Making the informed consent easier to understand – Simplify the eligibility criteria – Making the trial less demanding for patients – Providing in-trial feedback to patients *Getz K, et al,. Measuring the incidence, causes, and repercussions of protocol amendments. Drug Inform J. 2011;45:265-275. Getz K, et. al. The impact of protocol amendments on clinical trial performance and cost. Ther Innov Regul Sci 2016; 50(4):436-441.
  23. 23. Why are we talking about it?  Amendments cost an average of $500,000  8 weeks to implement each amendment  Typical phase 3 study has 4 amendments  40% of amendments deemed “avoidable” Source: Ken Getz, Tufts CSDD, “The State of the Clinical Research Enterprise.” NINDS INSPIRE Workshop, June 2013. Many amendments occur before the first patient is enrolled and could have been prevented with better planning and engagement of the patient community.
  24. 24. Impact of avoiding an amendment Prob NPV ($MM) Succeed Fail Succeed Regulatory Approval Fail Succeed Phase 3 Fail Phase 2 6.5 years $121 MM Avoid a phase 2 amendment • Save $0.5MM • Avoid 3 month delay* Avoid a phase 3 amendment • Save $2.1MM • Avoid 3 month delay* * Getz K, et. al. The impact of protocol amendments on clinical trial performance and cost. Ther Innov Regul Sci 2016; 50(4):436-441. 493 -4 -44 -27 15% 67% 2% 16% 88% 12% 52% 48% 33% 67%
  25. 25. Impact of improving the patient experience  Examples: – Making the informed consent easier to understand – Making the trial less demanding for patients – Providing in-trial feedback to patients Succeed Fail Succeed Regulatory Approval Fail Succeed Phase 3 Fail Phase 2 NPV ($MM) 493 -4 -44 -27 15% 67% 2% 16% 88% 12% 52% 48% 33% 67% • Faster enrollment • Better compliance • Fewer dropouts • Faster enrollment • Better compliance • Fewer dropouts
  26. 26. Economic Model Results Millions of Dollars Phase II Phase III Phase II Phase III NPV Impact + $24.5 +$32.0 +$38.2 +$30.9 ENPV Impact +$3.8 +$15.0 +$30.1 +$57.0 Improve Patient ExperienceAvoid One Amendment Phase 2 Impact  10x benefit in cost  700x benefit in ENPV  1240x benefit in NPV Phase 3 Impact  42x benefit in cost  1500x benefit in ENPV  1300x benefit in NPV OVERALL
  27. 27. Where to start ???
  28. 28. Principles for Patient-centered measurement American Institutes for Research  Patient-centered  Driven by patients’ expressed needs—not assumptions about what matters to them  Makes a difference for and benefits patients  Focuses on structures, processes and outcomes that patients care about, not what the system says they should care about Measurement that driven by patients’ expressed preferences, needs, and values that informs progress toward better health, better care, and lower costs. https://aircpce.org/sites/default/files/PCM%20Principles_April182017_FINAL.pdf
  29. 29. The myths of PROs  MYTH: PROs are by definition patient-centered.  MYTH: If patients are reporting on an outcome, it matters to them.  MYTH: If a PROM is published, it is automatically validated for any use case. C. Grossman, FasterCures
  30. 30. Principles of patient-centered measurement Patients’ goals, preferences, and priorities drive what is measured and how performance is assessed. Patient- Centered Measurement Patient- Driven Holistic TransparentComprehensible & Timely Co- Created Patients are equal partners in measure development and have decision- making authority about how data is collected, reported, and used. Patients and other stakeholders get timely, easy-to-understand data to inform decision-making and quality improvement. Measurement recognizes that patients are whole people and considers their circumstances, life and health histories, and experiences within and outside of the health care system. Patients have access to the same data as other stakeholders and understand how data is used to inform decision- making around care practices and policies. https://aircpce.org/sites/default/files/PCM%20Principles_April182 017_FINAL.pdf
  31. 31. DCRI Guiding Principles for Engagement  People come first. Always.  We recognize that people are embedded in dynamic family and community frameworks that we honor and respect across the continuum of life care.  People are our partners in research; not our subjects. We believe in taking every opportunity to co-learn. We engage participants, families, and community members in our research design, conduct, oversight, and dissemination activities.  We are transparent and trustworthy. We communicate to research participants how valuable their contributions are to science and medicine. We take the time to thank research participants, update them on progress, and share our findings in language understandable to everyone.  We create value. We work to return results in a responsible and meaningful manner and maximize what can be learned by sharing data with other researchers. We give back.  We are not transactional in our approach. We encourage and incentivize collaborations with people and communities that look past the end of a project or last study visit. We create opportunities to continue co-learning and working in partnership with Participants, families, and community members to improve health outcomes.
  32. 32. Taking the first step Start small: Pick one project, area of care, group of patients, or type of measurement to focus on first. Ask, and listen: Reach out to patients and families to ask for their perspectives. Invite partnership in projects. Keep an open mind, and listen carefully to responses about what is important to them. Examine current practices: Who decides which measures to collect and report? Who has input on those decisions? Are patients and families represented? What are their unmet needs? https://aircpce.org/sites/default/files/PCM%20Principles_April182 017_FINAL.pdf
  33. 33. Quotes from Diabetes Patients  You could argue that people with decent A1C don't need to change what they're doing - but that doesn't take into account the incredible amount of work to get that "good" a1c. So, optimizing for reducing amount of work (actions throughout the day, reducing cognitive burden, emotions, and overall improving QOL) is something that many people would choose, and even sacrifice some small changes/gains in A1C for.  In addition to QOL issues, which are significant, there’s evidence that shows damage to vascular endothelial tissue and brain structure as a result of extreme highs and lows.  Time in range is probably the best example. HCPs care about A1C but often ignore TIR. TIR is the amount of time spent within goal limits, such as 80-180 mg/DL. People can have a great A1C, but be constantly running high or low, or both.
  34. 34. Quotes from Diabetes Patients  Disease Burden - Few, if any, researchers look at the impact of multiple drug/device therapies on a sense of disease burden. Over time, many of us require medications to treat complications and/or other conditions. Few people look at the impact of the complexity of treatment on the ability to adhere to regimens and/or function in daily life. There are a variety of things that would be interesting to study here that nobody is focused on doing.
  35. 35. The first call from dad, Lisa Berdan Essays From the Front Lines of Health Care doi:10.1016/j.ahj.2011.10.006
  36. 36. Use caution when developing the field  I have diabetes-related kidney disease, which can cause anemia. I take an erythropoietin stimulating agent (ESA) and iron infusions, which help tremendously … problem is, it’s difficult to get insurance to cover the meds if your hemoglobin is in a normal range. […] Doctors latch on to specific measures, which tend to get “calcified.” Insurance companies then have research that backs up their need to restrict access to expensive drugs.
  37. 37. Aligning on unmet medical need • Treat or prevent a condition with no current therapy • Unsatisfactorily effective therapies • Unsatisfactory effect on serious outcomes • Serious or unacceptable side effects, risks or harms (short or long-term) • Burdensome therapies • Inaccessible therapies (site of delivery, shortages, cost) Business case for tightly aligned views of patient need: • (Potential) access to expedited regulatory pathways • Market differentiation • Stronger value proposition with payers • Better adherence • Better health outcomes fastercures.org/programs/patients-count
  38. 38. Frank Rockhold, PhD | GlaxoSmithKline Patient benefit-risk trade-offs fastercures.org/programs/patients-count
  39. 39. The Voice of the Patient using stated- preference methods http://www.parentprojectmd.org/site/PageServer?pagename=nws_index
  40. 40. There are examples of using PRO symptoms and physical function in FDA labelling  Emicizumab- (Hemlibra®)  Hemophilia A  Primary Endpoint: Annualized Bleeding Rate  PRO supported the primary endpoint * NOTE: Labeling focused on symptoms and the physical function component of the instrument
  41. 41. Raising the Bar on Research Partnerships with Patients & Community Unique living laboratory in which to execute a robust, multi-faceted engagement program.  Help sponsors and investigators co-design, pilot and scale successful engagement models, methods, and technologies across the R&D continuum in partnership with people  Shift practice to solving for the problem (not the proposal) and engaging from bench to bedside and back – always.  Further the science of participant engagement by evaluating partnerships with participant and community stakeholders and creating measures for engagement success RESEARCH TOGETHER strives to create a gold standard of partnership with patients and community members in research design, conduct, oversight and dissemination to improve participant experience and the impact of research.
  42. 42.  Passionate (or frustrated) patients in clinic  Guidestar.org or charitynavigator.org  Scientific meetings or disease-based advocacy events  Partnering for Cures, DIA, AdvaMed  Health Research Alliance, FasterCures TRAIN group, Genetic Alliance, NORD  Online patient communities, Facebook, Twitter  Google patient bloggers  FDA, PCORI, NIH panels and meetings Connecting with Patients and Advocacy Groups Bray Patrick-Lake, Patients as P-values Partners
  43. 43. PATIENT ENGAGEMENT RESEARCH DESIGN Access breadth and depth of thought leadership who uniquely understand the science of participant engagement from research development through implementation and evaluation. “Tiger Team” of participant engagement & communications experts • Engagement and communications strategy, support, and evaluation • Digital engagement and community building • User-centered design • Frictionless research participation • Empiric development of consent process and forms • Patient preferences • Behavioral economics • Return of value • Dissemination of research results https://dcri.org/insights/stakeholder-engagement Reach out at Duke for assistance
  44. 44. In Sum • It is time to be bold about doing things differently to do justice to people as unique, multidimensional individuals embedded in dynamic family and community frameworks. • While new methods are being developed and evaluated, patient engagement is here to stay. It’s time to work hand-in-hand with patients as a norm. • Together, we must generate novel ideas and keep listening, studying, innovating, and adapting research to benefit patients and ensure we are meeting community and scientific needs.
  45. 45. Thank You! bray.patrick-lake@duke.edu DCRI-ResearchTogether@duke.edu