#Patients2018

ANewInnova+veApproachtoIntegratePa+entPreferencesintotheSta+s+calDesignforClinicalTrials:ACollabora+ve

CaseStudy

ACollabora)onoftheMedicalDeviceInnova)onConsor)um,FDA,MIT,RTIHealthSolu)onsandtheMichael

J.FoxFounda)onforParkinson’sResearch

#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

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Pa)entsareattheHeartofWhatWeDo

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

5

Whatifpa)ents’urgencyfornewtherapeu+cop+onsandtoleranceofuncertaintyweretakenintoaccount

whendesigningandsizingclinicaltrials?

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SpecificAims

www.fda.gov

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Iden+fyingOutcomes

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8

Iden+fyingOutcomes

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Par+cipants

RefineandclarifyaMributesthroughdiscussionandfocusgroups2

9

Iden+fyingOutcomes

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Developunbiased,pa)ent-centereddescrip)onsindiscussiongroups4

#Patients2018

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

#Patients2018

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

THE MICHAEL J. FOX FOUNDATION FOR PARKINSON’S RESEARCH

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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

MJFF IS THE WORLD’S LARGEST NONPROFIT FUNDER OF PD RESEARCH

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MJFF was founded by a person with Parkinson’s disease. Assessing all potential projects through a patient-focused lens, everything we do is driven by the many unmet medical needs of Parkinson’s patients today.

PATIENTS’ NEEDS DRIVE OUR EFFORTS

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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.

WE TAKE A HOLISTIC APPROACH TO OUR WORK

#Patients2018

Stephanie Christopher, Medical Device Innovation Consortium

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? 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 pre-

market clinical trials

Faster, Safer, More Cost-effective FDA Strategic Plan, August 2011 Advancing Regulatory Science at FDA

www.MDIC.org

Why this matters to MDIC members

http://www.milkenreview.org/articles/p-values-vs-patient-values

• Meeting patient needs

• CDRH commitment to advancing use of patient preference

• Potential for smaller clinical trials

#Patients2018

Annie Saha, FDA Center for Devices and Radiological Health

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LookingForward

•  CDRHiscommiMedtointegra)ngthepa)entvoiceintoourregulatorydecision-making

•  CDRHencouragespa)entgroups,industry,andotherstointeractwithusearlytodiscusshowpa)entinputcanbeusedforregulatorypurposes

•  Partnershipsarekeytoadvancingthescienceofpa)entinput•  Otherwaystoincorporatepa)entinput

–  Improvecommunica)onofbenefit-riskinforma)ontopa)ents–  Incorporatepa)entinputtoinformclinicaltrialdesign–  EncouragePROdevelopmentanduse

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Pa)entsareattheHeartofWhatWeDo

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

#Patients2018

ToolBoxNotes

Forourtoolboxcontribu)on,wewouldliketoshareahandoutwecreatedonthefirststepsforsponsorstoconsiderwhenthinkingaboutlaunchingapa)entpreferencestudy.Youcandownloadthetoolat: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

JoinusMay18tohearthefinalresultsoftheproject

hMp://mdic.org/spi/pcor/workshop