Embedding patient centricity Understanding the role of real-world evidence in global market access strategies. Copyright 2014 Quintiles

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1 Embedding patient centricity Understanding the role of real-world evidence in global market access strategies Copyright 2014 Quintiles

2 Agenda Introduction Real-world evidence: Increasing access to innovative treatments Louise Parmenter The evolution of the patient voice: View point of an ex-regulator Stella Blackburn Q&A These materials have been prepared solely for educational purposes to contribute to the understanding of patient centricity and real-world evidence. Quintiles makes no representations or warranties about the accuracy or suitability of any information in the materials; all such content is provided to participants on an as is basis. QUINTILES HEREBY DISCLAIMS ALL WARRANTIES REGARDING THE CONTENTS OF THESE MATERIALS, INCLUDING WITHOUT LIMITATION, ALL WARRANTIES OF TITLE, NON-INFRINGEMENT, MERCHANTABILITY, AND FITNESS FOR A PARTICULAR PURPOSE 2

3 Today s speakers Louise Parmenter, Global Head of Operations, Epidemiology and Outcomes Research, Real-World and Late Phase at Quintiles Specialist in real-world and late phase research with 22 years of related global operational and strategic experience. She is currently responsible for a team of epidemiologists and outcomes researchers primarily based in the United States with growing teams in Europe and Asia. The team s focus areas include safety, comparative effectiveness, design and implementation of Risk Evaluation and Mitigation Strategies (REMS), outcomes research and methods development. In this capacity, Dr. Parmenter is responsible for study design, scientific oversight and study publication. Stella Blackburn, Vice President, Global Head of Risk Management, Real-World and Late Phase Research at Quintiles Leads risk management efforts in North America, Europe and Asia-Pacific for the Quintiles real-world and late phase research group. In her role, Dr. Blackburn develops multidisciplinary benefit risk management services, reviews and assists customers with interpretation of pharmacovigilance legislation, and ensures compliance with regulations and best practices in the conduct of pharmacovigilance and risk management activities for the company s real-world and late phase research efforts. With more than two decades of experience in the pharmacovigilance and pharmacoepidemiology fields, Dr. Blackburn joined Quintiles from the European Medicines Agency (EMA) where she served for more than 16 years in various roles, most recently as the EMA Risk Management Development and Scientific Lead 3

4 Real-world evidence increasing access to innovative treatments 4

5 As a form of practice it seeks to focus medical attention on the individual patient s needs and concerns rather than the doctor s. Defining Patient-Centered Medicine Bard, Charles L, Defining Patient Centered Medicine : New England Journal of Medicine ;9 5

6 Benefits of patient-centered approaches to healthcare Improvement Physician satisfaction Patients emotional state Medication adherence Patient satisfaction and empowerment Malpractice complaints Consultation time Symptom severity Healthcare resource use Healthcare costs Reduction HUDON, C., FORTIN, M., HAGGERTY, J. L., LAMBERT, M. & POITRAS, M.-E Measuring Patients Perceptions of Patient-Centered Care: A Systematic Review of Tools for Family Medicine. Annals of Family Medicine, 9,

7 Examples of decision-makers promoting patient-centered approaches Patient-Centered Outcomes Research Institute (PCORI) European Medicines Agency 7

8 PCORI s research project agenda Research shall be designed, as appropriate, to take into account the potential for differences in the effectiveness of health care treatments, services, and items as used with various subpopulations, such as racial and ethnic minorities, women, age, and groups of individuals with different comorbidities, genetic and molecular sub-types, or quality of life preferences and include members of such subpopulations as subjects in the research as feasible and appropriate. US Patient Protection and Affordable Care Act US Social Security Administration Sec [42 U.S.C. 1320e] Accessed 19 April

9 European Medicines Agency adaptive pathways to patients (formally Adaptive Licensing ) March 2014: Adaptive Licensing December 2014: Adaptive Pathways Regulators, companies, Health Technology Assessment bodies (HTAs) and patient representatives Explore ways to optimise development pathways and potentially accelerate patients access to medicines. This faster access may be achieved by shorter time to approval and/or reimbursement decision for targeted groups of patients. Adaptive pathways to patients: report on the initial experience of the pilot project December EMA/758619/

10 So how does a patient centered approach impact the role of real-world evidence in global market access strategies? 10

11 A paradigm shift in thinking Market Access Patient- Centric Thinking Patient Access John Glasspool Sep 20, 2013 Pharmaceutical Executive. Market Access is Dead: Patient Access is the New Prescription for Healthcare Marketing 11

12 A Paradigm Shift in Thinking Market Access Patient- Centric Thinking Patient Access John Glasspool Sep 20, 2013 Pharmaceutical Executive. Market Access is Dead: Patient Access is the New Prescription for Healthcare Marketing 12

13 Drives a new patient-centered approach Research and Development License Market Access Commercial availability Commercial Ethical sales 13

14 Drives a new patient-centered approach Research and Development Patient Outcomes Market Access Patient Outcomes Commercial Patient Outcomes 14

15 Pfizer Exubera First U.S. inhaled insulin for diabetes FDA approval in January 2006 Anticipated blockbuster Withdrawn from the market in October 2007 Lack of consumer demand This amazing science, incredible engineering, and true breakthrough frankly did not resonate with patients and their lifestyle. We realized we hadn't asked all the right questions of the right patients at the right time, and yet we continued pushing forward. Pfizer s Chief Medical Officer Pfizer: Putting The Patient At The Center Of Its Drug Development Universe. Life Science Leader December 24,

16 Pfizer Xeljanz (tofacitinib) Indicated for rheumatoid arthritis FDA approval in November 2012 A year later, the FDA approved a supplemental new drug application (snda) including additional patientreported outcomes data. Eight domains of the 36 item Medical Outcomes Study Short- Form Health Survey (SF-36) now appear in the label. We worked to include things that are not usually part of a clinical trial. That's a soup-tonuts example of listening to patients about what's important to them, including it in clinical trials, collecting and analyzing the data, and then sharing it back with the patient community Pfizer s Chief Medical Officer Pfizer: Putting The Patient At The Center Of Its Drug Development Universe. Lifescience Leader December 24,

17 Lilly to use real-world data in diabetes collaboration Lilly is to use data collected in an everyday setting as part of a collaboration with the T1D Exchange to research new ways to improve care for people with type 1 diabetes. PM Live April

18 10 steps towards patient access Toward Patient-Centered Drug Development in Oncology. Ethan Basch New England Journal of Medicine August :5 18

19 Key steps towards patient access Step Appoint senior executives to lead patient engagement efforts and focus all teams on patient outcomes Use real-world studies to establish burden of disease, resource utilization, population, subpopulation, and individual patient characteristics, and treatment pathways Timing Throughout drug development Throughout drug development Discuss plans for measuring and analyzing patient-centered outcomes at structured meetings between drug-development team, regulatory agency and where possible, payers Throughout drug development Engage patients representative of the target population Generate evidence in patients representative of target population likely to use the product Throughout drug development Throughout drug development Adapted from Toward Patient-Centered Drug Development in Oncology. Ethan Basch New England Journal of Medicine August :5 19

20 Key steps towards patient access Step Identify patient-centered outcomes (symptom, functional, and other outcomes affected by a disease or product and important to patients) through direct patient feedback Develop or select measures to evaluate outcomes using established qualitative and quantitative methods Include PRO and other patient-centered measures in pivotal trials, with protocol-specified plans for statistical analysis as well as minimizing and handling missing data Include PROs in drug labels to help patients and providers with decision making Evaluate product use in the real-world: safety & effectiveness; compliance, adherence, persistence; treatment satisfaction using robust research methods Timing Before pivotal trials Before pivotal trials Pivotal trials Regulatory review Post-launch Adapted from Toward Patient-Centered Drug Development in Oncology. Ethan Basch New England Journal of Medicine August :5 20

21 The evolution of the patient voice a viewpoint from an ex-regulator 21

22 L état, c est moi 22

23 Key stakeholders in drug development Biopharma Doctors Regulators Patients Drug Development Payers 23

24 Patient needs Medicines to meet unmet medical needs Affordable medicines Medicines with predictable outcomes Medicines with fewer side effects 24

25 Patient needs Medicines to meet unmet medical needs Affordable medicines Medicines with predictable outcomes Medicines with fewer side effects But do we really know what patients want? 25

26 Key milestones of EMA interactions with patients and consumers Slide from EMA website accessed 20/04/

27 Current representation on PCWP Age Platform Europe Alzheimer Europe European AIDS treatment group European Cancer Patient Coalition European Consumers Organisation European Federation of Allergy and Airways Diseases Patients Associations European Federation of Neurological Associations European Heart Network European Institute of Women s Health European Multiple Scelerosis Platform European Organisation for Rare Diseases European Patients Forum European Prostate Cancer Coalition European Public Health Alliance Health Action International Europe International Alliance of Patients Organisations International Diabetes Federation European Region International Patient Organisation for Primary Immunodeficiencies Patients Network for Medical Research and Health 27

28 Involvement of patients at the EMA Part of the EMA Management Board (2 members) Inclusion in scientific committees as members COMP 3 members PDCO 3 members CHMP CAT 2 members PRAC 1 member 28

29 Involvement of patients at the EMA Contributions on disease and product-specific questions Scientific Advice Working Party (SAWP) Scientific Advisory Groups (SAG) Consultation on guidelines and policies Review of documents destined for the public Dissemination of information Participation in workshops, networks and research projects 29

30 The patients and representatives in the room, in their statements, defended the position that at that late stage of the disease even small effects providing longer independent use of arms and hands, or preserving the ability to feed and drink from a cup on their own, would represent a significant and important effect. Taken from EPAR for Translarna 30

31 Key contributions from patients What benefits of a medicine are important for patients? What level of risk is acceptable? What level of risk minimisation is acceptable? Which medicines/treatments should be funded? 31

32 Challenges Are patient representatives really representative? Who owns patient data? 32

33 WP4: The PROTECT Pregnancy Study New tools for data collection from consumers The PROTECT project has received support from the Innovative Medicines Initiative Joint Undertaking ( under Grant Agreement n , resources of which are composed of financial contribution from the European Union's Seventh Framework Programme (FP7/ ) and EFPIA companies in kind contribution.

34 New tools for data collection from consumers An exploratory study of self-reported medication use in pregnant women and pregnancy outcomes Objectives To assess the extent to which data collected directly from pregnant women via the Internet and IVRS provides information on medication use and other potential risk factors throughout pregnancy, and is suitable for research purposes 34

35 An exploratory study of self-reported medication use in pregnant women and pregnancy outcomes Questions: Can we get data earlier in pregnancy than traditional routes? How consistently and for how long will women provide the data requested? How representative are the women? How important are data not captured by EHR or pharmacy databases? Is the information of sufficient quality to be used for pharmacovigilance? 35

36 Data collection flow IVRS Baseline questionnaire 2 weekly Web Baseline questionnaire Follow up Follow up Follow up Follow up Pregnancy outcome Satisfaction Q Follow up Follow up 4 weekly Discontinuation Questionnaire 36

37 Data collected Baseline Demographics Lifestyle and risk factors Pregnancy-related information Medications, supplements, vaccinations, contraception, medical/cosmetic procedures, herbals, others Follow-up Change to expected date of delivery Changes to previous medications New problems and medications Vaccinations and others End of study Pregnancy outcomes: live birth, miscarriage/still birth, termination, ectopic pregnancy Birth defects Satisfaction questions 37

38 Work package 4 - Study population 4 countries: Denmark Poland The Netherlands United Kingdom 2065 women provided data Self identified as pregnant Recruited directly, without intervention of HCP 38

39 Data which may not be found elsewhere DK % (n) NL % (n) PL % (n) UK % (n) All % (n) Stopped medications when pregnant 15 (95) 11 (51) 11 (27) 18 (126) 14 (299) Stopped doctor s prescribed medications 5 (31) 4 (19) 5 (11) 6 (39) 5 (100) Used medications from other people 0.3 (2) 1 (3) 0.4 (1) 1 (5) 0.5 (11) Had vaccinations 14 (92) 4 (19) 3 (6) 49 (345) 22 (462) Stopped smoking 17 (106) 14 (68) 19 (45) 16 (114) 16 (333) Reported illicit drug use 1 (6) 0.4 (2) 0.4 (1) 1 (8) 1 (17) 39

40 Conclusion It is possible to recruit women early in pregnancy before they may have consulted HCPs Direct to consumer studies offer important benefits in collecting certain data not found in EHR 40

41 Conclusion Biopharma Regulators Doctors Patients Payers 41

42 Audience questions 42