The Impact of Patient Public Involvement on Clinical Research

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1 The Impact of Patient Public Involvement on Clinical Research Dr Sue Pavitt Reader in Applied Health Sciences Leeds Institute of Health Sciences University of Leeds

2 Overview Aim To understand how Patient Public Involvement (PPI ) can impact of clinical research Objectives Introduction to Patient Public Involvement (PPI) PPI impact on clinical trials - case studies Study Design Study literature & informed consent Study logistics Recruitment & Retention

3 Who are the PP in PPI? By Public and Patient we mean: Patients / potential patients People who use health and social services Carers (unpaid) Family members Disabled persons Members of the public who have been targeted by a specific research grant programme Organisations that represent patients and public s views Individuals/groups that are affected by health or clinical issues

4 What is the I in PPI? An active partnership between the public and researchers in the research process Research to, about or for the public Research with or by the public RAISES PATIENT PUBLIC PERCEPTION OF CLINICAL TRIALS

5 Benefits of PPI User involvement in clinical research is valuable and ensures: vdifferent perspectives heard vresearch priorities identified by medics are also important and relevant to users voutcomes important to users are measured vimproved research design vimproved trial logistics vaccess to patients - via peer networks vaccess hard-to-reach patient groups veffective dissemination Improved research that addresses: patient needs, achieves recruitment & retention and delivers to target

6 PPI in Designing a Trial Questions asked in Designing a Trial 4 th Question what do patients think? qare we asking the right question to improve the health and quality of life for patients?

7 Case Study 1: Getting the right research question(s) q Example from Oral Cancer Oncologist focus 5-year survival at any cost Patient focus quality of life issues function & disfigurement v Trial designed to address future patients needs - providing complete impact of treatment on survival and quality of life v Improved Recruitment & Retention - patient sees trial as important

8 Case Study 2- SWITCH PPI Impact on Trial Design: Ethics, safety and cost implications SWITCH RCT N=870 Patients with Rheumatoid Arthritis who have failed a TNF antagonist Biologic Therapy N=290 Alternative Mechanism TNF N=290 Abatacept Schedule 14 Tx; IV 90 mins N=290 Rituximab-Comparator Schedule 2 Tx;IV 405 mins Infliximab Schedule 8 Tx IV 225 mins Adalimumab Schedule 26 Tx SC 30 mins v Funders suggested the SWITCH trial Tx should be blinded. PPI representatives argued: v Unethical & safety concerns - drug treatment schedule different for Tx arms v 71 treatments/patient (average 4.5 fold higher than non-blinded) v 78.5 hours of treatment/patient (average 4.3 fold higher than non-blinded) v Trial costs would be significantly higher v Saved 2.5m in Tx delivery costs alone Etanercept Schedule 52 Tx SC 30 mins Certolizumab Schedule 26 Tx SC 30 mins v Lose pragmatic design - results may not be transferable to everyday practice

9 SWITCH PPI impact on trial design Patient friendly design & value for money PPI improved trial design and retained a more ethical, safer and pragmatic drug trial ØEquivalence design provides more drug options to patients ØParticipant friendly trial keeps refusals to a minimum ØMinimises Tx appointments from weekly to actual recommended Tx schedule for each drug Ø Improved fundng success reduced budget by several million ØImproved patient safety fewer intravenous infusions

10 Case Study 3: Lessons learnt from the ProtecT Trial: impact on trial literature ProtecT Prostate testing for cancer treatment v Recruiters found it difficult to explain the uncertainty about treatment and did not present options equally v Non-treatment arm described as watchful waiting v Patients interpreted as if clinicians would watch while I die v PPI changed descriptor to active monitoring v Recruitment rates increased from 40% to 70%

11 Case Study 4: PPI Impact on Trial Literature IMPROVDENT An RCT to improve the fit of dentures by testing two dental impression materials Trial Literature Reviewed Recommendations to make the Patient Information Leaflets more understandable Improved Trial Operations Trial is explained better results in improved uptake Timely ethics approval Early attainment of recruitment to schedule

12 Case Study 4: PPI Impact on Trial operations / logistics Trial Operational input Appointments available largely between 10am - 3pm Accommodates travel to appointments on Senior Citizen Bus Pass Improved Trial Operations Trial is user friendly Participants less inconvenienced Few cancelled appointments Recruitment to schedule

13 Better designed trials improve Recruitment & Retention RECRUITMENT 2/3rds of trials fail to reach target and require expensive extensions Expensive extensions or Underpowered trials RETENTION 1/3 rd enrolees drop out AWARENESS ADDITIONAL SUPPORT

14 The Future - PPI Integral to Clinical Trial Design & Recruitment Strategy q Trial designed to take account of patients needs q Trial operations / logistics made patient friendly q Trial literature simplified Ensuring informed consent Improved Recruitment & Retention Patient Benefit Improved Health Improved Delivery of Trials & Evidencebased Medicine

15 Any Questions? Dr Sue Pavitt Leeds Institute of Health Sciences +44 (0)