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Sep 09, 2022 179 likes •328 views

Patien Patient-managed managed registries registries How How can we can we get high qualit get high quality y data from pat data from patients ients for precis for precision ion medicine? medicine? Elizabeth Vroom Julian Isla

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Patien Patient-managed managed registries registries

How How can we can we get high qualit get high quality y data from pat data from patients ients for precis for precision ion medicine? medicine?

Julian Isla Foundation 29 Elizabeth Vroom Duchenne Parent Project

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Why patient-managed registries are needed?

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Hospitals Clinical data Patient reported data

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Patient Orgs

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Technology barrier Low-hanging fruit effect

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Duchenne Data

Genetic defect Duchenne Muscular Dystrophy (DMD) discovered in 1986 Natural History studies started as early as 1969 Patients see approx 10 different health care professionals per year Data collected by HCP, patient registries and research databases. Clinicaltrials.gov shows 271 clinical trials for DMD data kept by companies

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Position of the DMD Patient community

Optimal (re)use of data for drug development and care Collection of data relevant to patients (PROMs) Placebo data should be made available Patients deciding about the use of their own Data Patients are willing and interested to share data Clinical trial data should be returned to participants

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D U C H E N N E F A I R D A T A DECLARATION

1 . P a t i e n t d e r i v e d

  • r

p r

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i d e d d a t a a r e n

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t r

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l e d b y t h e d

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t h e se d a t a . R e se a r c h e r s, c h a r i t i e s , c

  • m

p a n i e s a n d h e a l t h p r

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e ssi

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a l s a r e c u st

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i a n s. 2 . T

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n a b l e t h e

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t i m a l r e u se

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d a t a , t h e d a t a n e e d s t

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e F i n d a b l e , Ac c e ssi b l e , I n t e r

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e r a b l e a n d R e u sa b l e ( i . e . F AI R ) b y m e d i c a l p r

  • f

e ssi

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a l s, p a t i e n t s a n d i n p a r t i c u l a r a l s

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y ma c h i n e s. 3 . T h e

  • p

t i m a l r e u se

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d a t a sh

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l d b e su p p

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t e d a t a l l l e v e l s, b y p r

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e ssi

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a l s a n d c u st

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i a n s ( a l l

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e d e r a t e d l e a r n i n g

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t h e d a t a u p

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r e q u e st , g i v e t h e d a t a t

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h e d

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i n F AI R f

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m a t wh e n a sk e d ) , c a r e p r

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e ssi

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a l s ( c a p t u r e d a t a a t t h e so u r c e i n F AI R f

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m a t wh e r e v e r p

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b l e ) , a n a l y t i c s e n v i r

  • n

m e n t s ( a d a p t t

  • F

AI R d a t a ) a n d r e g u l a t

  • r

s ( d e m a n d F AI R d a t a t h r

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g h

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t a n d

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t i m a l l y u se t h e m i n t h e r e g u l a t

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y p r

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e ss) . T h e r e i s a n e e d t

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d u c a t e a l l st a k e h

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d e r s a b

  • u

t t h e F AI R p r i n c i p l e s a n d t h e i r i m p

  • r

t a n c e ( ‘ F AI R Awa r e ’ ) 4 . O p t i m a l c a r e sh

  • u

l d b e t a k e n t

  • r

e st r i c t t h e n e e d t

  • r

e v e a l t h e a c t u a l i d e n t i t y

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i n d i v i d u a l s a sso c i a t e d wi t h c e r t a i n d a t a , a n d t

  • p

r

  • t

e c t p r i v a c y wi t h a l l p

  • ssi

b l e m e a n s, b u t we r e a l i z e p r i v a c y i s su b

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d i n a t e i n m a n y c a se s t

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a st

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r a c k i n g

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b e t t e r so l u t i

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s f

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t h e d i se a se s we su f f e r f r

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5. T h e r e f

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e , t h e r i g h t t

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l l

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i d e n t i f i c a t i

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t h e i n d i v i d u a l a sso c i a t e d wi t h c e r t a i n d a t a sh

  • u

l d a l so b e p l a c e d i n t h e h a n d s

  • f

t h a t i n d i v i d u a l

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a c h

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n t r u st e d p a r t y . 6. T e c h n i q u e s a n d t

  • l

s sh

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l d b e d e v e l

  • p

e d t

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n a b l e

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t i m a l c

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n v e st i g a t i

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b y r e se a r c h e r s, m e d i c a l a n d h e a l t h c a r e p r

  • f

e ssi

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a l s, c h a r i t i e s , c

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p a n i e s, p a t i e n t s a n d m a c h i n e s t

  • f
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m a ' so c i a l h e a l t h m a c h i n e ' a i m e d a t b e t t e r so l u t i

  • n

s a n d c a r e . 7 . R e g u l a t

  • r

s sh

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l d

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t i m a l l y e n a b l e f a st

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r a c k i n g

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k e y i n t e r v e n t i

  • n

s a n d i n v

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v e c i t i z e n a n d m a c h i n e p a r t i c i p at i

  • n

i n t h a t p r

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e ss t

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h e l a r g e st p

  • ssi

b l e e x t e n t . 8 . T h e f i e l d s h

  • u

l d a c t i v e l y d i sc

  • u

r a g e p u b l i sh i n g

  • f

h e a l t h

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e l a t e d i n f

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m a t i

  • n

e x c l u si v e l y i n c l a ssi c a l n a r r a t i v e j

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r n a l s. T h e se a r e v e r y d i f f i c u lt t

  • a

c c e ss a n d u n d e r st a n d b y b

  • t

h i n f

  • r

m e d l a y p e

  • p

l e a n d b y m a c h i n e s. I n st e a d , d a t a a n d i n f

  • r

ma t i

  • n

s h

  • u

l d b e p u b l i sh e d i n a wa y t h a t m a k e s i t mo r e r e a d i l y r e u sa b l e b y

  • t

h e r s t h a n a sm a l l i n n e r c i r c l e . 9 . F u n d i n g a g e n c i e s sh

  • u

l d h a v e g

  • d

d a t a st e wa r d sh i p f

  • l

l

  • wi

n g t h e F AI R p r i n c i p l e s i n c l u d e d i n t h e i r g r a n t c

  • n

d i t i

  • n s .

10. T h e r

  • l

e

  • f

h e a l t h i n su r a n c e i n st i t u t i

  • n

s/ c

  • m

p a n i e s sh

  • u

l d a l so b e ma d e c l e a r : t h e y sh

  • u

l d p u b l i c l y st a t e t h a t h a v i n g f u l l a c c e ss t

  • r

e a l wo r l d d a t a , e v e n wh e n t h e se a r e r e

  • i

d e n t i f i a b l e fo r t h e m t

  • '

t h e i r ' c l i e n t s, wi l l n

  • t

b e a b u se d , su c h a s f

  • r

e x a m p l e i n c r e a se p r e m i u ms b a se d

  • n

g e n e t i c p re d i sp

  • si

t i

  • n

. 11. He a l t h i n su r a n c e i n st i t u t i

  • n s/

c

  • m

p a n i e s a n d g

  • v

e r n me n t s sh

  • u

l d j

  • i

n f

  • r

c e s a n d su p p

  • r

t ( a l so f i n a n c i a l l y ) t h e d e v e l

  • p

m e n t

  • f

t r u st e d e n v i r

  • n

me n t s wh e r e r e a l wo r l d c i t i z e n d a t a c a n b e m a x i m a l l y r e u se d f

  • r

t h e b e t t e r m e n t

  • f

h e a l t h c a r e a n d t h e m a ssi v e sa v i n g

  • f

c

  • st

s t

  • k

e e p

  • p

t i m a l h e a l t h ca r e a f f

  • r

d a b l e f

  • r

a l l .

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Patient Data

Patient derived or provided data are not owned by those who collected them, and their reuse should be primarily controlled by the donors of these data. Researchers and Health professionals are custodians. (GDPR) To enable the optimal reuse of real world data, the data needs to be Findable, Accessible, Interoperable and reusable by Medical professionals, Patients and in particular also by machines.

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Data platform

Gives patients the power and control over the use of their

  • wn data.

Gives patients the option to ‘get their data together’. See it as ‘storing in a locker’ Have all their data with them at all times (emergencies) Patient data can be used for questions relevant to the patient community whether it is development of new drugs, new technologies or about daily life. Will facilitate the adoption of emerging technologies regarding data collection and enable their optimal application in health research, care and drug development

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Health Data Locker

  • Patients need a safe place to store their information.
  • A health data locker provides:
  • Interoperability
  • Ownership
  • Portability
  • FAIR principles implementation:
  • Findable, Accessible, Interoperable, Reusable
  • Patients don’t give data; they allow requestors to visit it
  • A Blockchain registry can help with this feature
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Privacy and security by design

Privacy and security are critical features for DDP. A security breach could put the project at risk. Privacy and security design is being done from the beginning:

  • GDPR

assessment using a PIA (Privacy Impact Assessment).

  • Security certification. An external provider with expertise

in security runs technical security tests.

  • A DPO is responsible for data protection
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Focusing on conversations

We don’t want to expose Duchenne patients to a form with hundred of data items We are exploring conversational interfaces where the form is replaced by a chat bot The chat bot can ask questions to the patient and

  • ther interfaces like Alexa from Amazon, Siri or

Google Assistant can be used.

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Thank you

elizabeth.vroom@worldduchenne.org julian.isla@foundation29.org