Experiences Good Practices and Lessons Learned Lana Skirboll VP Science Policy Sanofi October 28 2016 National Health Council 1 Some of the real and potential uses of PFDD 2 Questions being raised about PFDD in regulatory decisionmaking ID: 731819
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Patient Engagement in Drug Development:Experiences, Good Practices and Lessons Learned
Lana SkirbollVP Science PolicySanofiOctober 28, 2016, National Health Council
1Slide2
Some of the (real and potential) uses of PFDD
2Slide3
Questions being raised about PFDD in regulatory decision-makingFDA use of PFDD in regulatory decision making is still evolving.It premature to talk about ‘good practices’ when ‘practice’ itself is still emerging.The many sources of PFDD innovation3
GOAL:
An agreed upon framework for:
the collection of valid and reproducible patient voice data and
how it will be used in the review and approval of drugs and devices.Slide4
The Perpetual Conundrum: Validated PV Data
The
collection of valid and reproducible patient voice data
using tools and methods that are
both valid and reproducible
Is it a reasonable goal that the right tools/methodologies could reach all (or most) the patients, especially in this time of social media and internet connectivity?
How do we do this without further slowing the process?
How to get there?
We need behavioral scientists who know how to develop valid tools and methodologies that are fit for purpose and that represent human opinion and the components of human decision making.
We need to test tools them, refine them and validate them for use by ALL.
FDA needs a validation process so we do not have the reinvent the wheel.Slide5
5
Burden
of disease
Burden of
treatment
Tolerance for risk
Expectation of benefit
A validated survey tool that reaches 1000’s of patients!!!
Querying patients one at a time
VS
One Example: A Validated Survey Tool?Slide6
PFDD -- Why bother? (Motherhood and Apple Pie!)Patients benefit from being heardIndustry can develop better products
FDA hears from the all the stakeholdersPayers ensure they are paying for what matters
Physicians deliver the best treatment option
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7Back-up slidesSlide8
External stimuli – Cures and Innovation Bills21st Century Cures – would:define patient experience datarequire FDA to establish process to guide entity wishing to develop patient experience dataallow FDA to request patient experience datarequire FDA to publish draft guidance on the use of patient experience data, registries, patient beliefs on B-R
require FDA to hold regular workshops to gain input on methodologies and collection of patient experience data. Senate Innovation Bill – would:define patient data (as well as patient experience data)require FDA to make public a statement on patient experience data and related information submitted as part of a drug/biologic application including whether it was reviewed as part of the
application
Require FDA to publish guidance as above
PDUFA VI
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External stimuli – PDUFA VIPDUFA VIProvides resources to increase FDA staff capacity/capabilities within review divisions Requires FDA to develop a series of guidance documents: Comprehensive and representative patient/caregiver data collection and taxonomy
Methods to identify what impacts are most important to patients Out
of list of impacts, what to measure in a clinical
trial
Methods
to incorporate/develop COAs into endpoints robust enough for regulatory
decision-making
Requires FDA to
maintain
a repository of tools for stakeholders
revise
relevant MAPPs and SOPPs
h
old
a public meeting to gather input on enhancing patient engagement in clinical trials
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