A New Innova+ve Approach to Integrate Pa+ent Preferences into the Sta+s+cal Design for Clinical Trials: A Collabora+ve Case Study

A New Innova+ve Approach to Integrate Pa+ent Preferences into the Sta+s+cal Design for Clinical Trials: A Collabora+ve Case Study A Collabora)on of the Medical Device Innova)on Consor)um, FDA, MIT, RTI Health Solu)ons and the Michael J. Fox Founda)on for Parkinson s Research #Patients2018

Our Speakers Heather Benz, FDA Center for Devices and Radiological Health Margaret Sheehan, The Michael J. Fox Foundation for Parkinson s Research Lauren McLaughlin, The Michael J. Fox Foundation for Parkinson s Research Stephanie Christopher, Medical Device Innovation Consortium Annie Saha, FDA Center for Devices and Radiological Health #Patients2018

Heather Benz, FDA Center for Devices and Radiological Health #Patients2018

Pa)ents are at the Heart of What We Do CDRH Vision: Patients in the U.S. have access to high-quality, safe, and effective medical devices of public health importance first in the world 4

What if pa)ents urgency for new therapeu+c op+ons and tolerance of uncertainty were taken into account when designing and sizing clinical trials? 5

Specific Aims www.fda.gov 6

Iden+fying Outcomes 1 Ac+vity Par+cipants www.fda.gov Iden)fy and develop preliminary list of poten)al amributes through literature review Iden)fy Refine Priori)ze Define Sponsors Pa)ents Physicians Sponsors Pa)ents Physicians Pa)ents Physicians Sponsors 7

Iden+fying Outcomes Refine and clarify amributes through 2 discussion and focus groups Ac+vity Par+cipants www.fda.gov Iden)fy Refine Sponsors Pa)ents Physicians Sponsors 8

Iden+fying Outcomes Priori)ze amributes by using ranking surveys 3 groups and discussion Ac+vity Par+cipants www.fda.gov Iden)fy Refine Priori)ze Sponsors Pa)ents Physicians Sponsors Pa)ents Physicians 9

Iden+fying Outcomes Develop unbiased, pa)ent-centered descrip)ons in discussion groups 4 Ac+vity Par+cipants www.fda.gov Iden)fy Refine Priori)ze Define Sponsors Pa)ents Physicians Sponsors Pa)ents Physicians Pa)ents Physicians Sponsors 10

Margaret Sheehan, The Michael J. Fox Foundation for Parkinson s Research #Patients2018

Lauren McLaughlin, The Michael J. Fox Foundation for Parkinson s Research #Patients2018

THE MICHAEL J. FOX FOUNDATION FOR PARKINSON S RESEARCH

MJFF IS THE WORLD S LARGEST NONPROFIT FUNDER OF PD RESEARCH Our Mission The Michael J. Fox Foundation is dedicated to finding a cure for Parkinson s disease through an aggressively funded research agenda and to ensuring the development of improved therapies for those living with Parkinson s today. Vital Stats» Founded in 2000 by actor Michael J. Fox» Public charity» More than 85,000 donors in 2016 (individuals, corporations, nonprofits)» No chapters: team of 125 based in NYC» 4,200 grassroots fundraisers reaching 175,000 supporters worldwide in 2016» Over 30,000 emails were sent to members of Congress on critical Parkinson s issues in 2016» Nearly $750 million in research programs funded to date» $90 million in research programs funded in 2016» More than 2,600 projects funded to date» More than 600 active grants in current portfolio» 34% of funded projects are led by researchers outside the United States» Fund academics, biotechs and pharma 14

PATIENTS NEEDS DRIVE OUR EFFORTS MJFF was founded by a person with Parkinson s disease. Assessing all potential projects through a patientfocused lens, everything we do is driven by the many unmet medical needs of Parkinson s patients today. 15

WE TAKE A HOLISTIC APPROACH TO OUR WORK We drive promising therapeutic targets while also investing to make PD drug development faster, cheaper, and more attractive. Our work is not done. Therefore, we seek collaboration to accelerate ongoing activities and launch initiatives in parallel. 16

Stephanie Christopher, Medical Device Innovation Consortium #Patients2018

A novel method (and a novel team) for incorporation of patient preferences in clinical trial design

Why MDIC? MDIC is a 501(c)(3) non-profit organization and is the first-ever public-private partnership created with the sole objective of advancing regulatory science of medical devices for patient benefit

What is Regulatory Science? FDA Strategic Plan, August 2011 Advancing Regulatory Science at FDA The science of developing new tools, standards, and approaches to assess the safety, efficacy, quality, and performance of FDA-regulated products Benefits patients by speeding the rate of important technologies reaching market Reduces time and resources needed for device development, assessment, and review. For example: Ø Can lead to quicker, more efficient device approvals Ø Can decrease the size and duration of premarket clinical trials Faster, Safer, More Cost-effective www.mdic.org

Why this matters to MDIC members Meeting patient needs CDRH commitment to advancing use of patient preference Potential for smaller clinical trials http://www.milkenreview.org/articles/p-values-vs-patient-values

Annie Saha, FDA Center for Devices and Radiological Health #Patients2018

Looking Forward CDRH is commimed to integra)ng the pa)ent voice into our regulatory decision-making CDRH encourages pa)ent groups, industry, and others to interact with us early to discuss how pa)ent input can be used for regulatory purposes Partnerships are key to advancing the science of pa)ent input Other ways to incorporate pa)ent input Improve communica)on of benefit-risk informa)on to pa)ents Incorporate pa)ent input to inform clinical trial design Encourage PRO development and use 23

Pa)ents are at the Heart of What We Do CDRH Vision: Patients in the U.S. have access to high-quality, safe, and effective medical devices of public health importance first in the world 24

Tool Box Notes For our toolbox contribu)on, we would like to share a handout we created on the first steps for sponsors to consider when thinking about launching a pa)ent preference study. You can download the tool at: hmp://mdic.org/wp-content/uploads/2016/04/ Pa)ent-Preference-Study-Design-20171102.pdf #Patients2018

Our Speakers Heather Benz, FDA Center for Devices and Radiological Health Margaret Sheehan, The Michael J. Fox Foundation for Parkinson s Research Lauren McLaughlin, The Michael J. Fox Foundation for Parkinson s Research Stephanie Christopher, Medical Device Innovation Consortium Annie Saha, FDA Center for Devices and Radiological Health #Patients2018

Join us May 18 to hear the final results of the project hmp://mdic.org/spi/pcor/workshop #Patients2018