Patien Patient-managed managed registries registries How How can - PowerPoint PPT Presentation

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 Duchenne Parent Project Foundation 29 Classified as public by the European Medicines Agency

Why patient-managed registries are needed? Classified as public by the European Medicines Agency

Clinical data Patient reported data Hospitals Classified as public by the European Medicines Agency

Patient Orgs Classified as public by the European Medicines Agency

Technology barrier Low-hanging fruit effect Classified as public by the European Medicines Agency

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

Optimal (re)use of data for drug development and care Collection of data relevant to patients (PROMs) Position of the Placebo data should be made available DMD Patient Patients deciding about the use of their own community Data Patients are willing and interested to share data Clinical trial data should be returned to participants Classified as public by the European Medicines Agency

D U C H E N N E F A I R D A T A DECLARATION 1 . n P a t i e t d e r i v e d o r p r o v i d e d d a t a a r e n o t o wn e d b y t h o se wh o c o l l e c t t h e m, a n d t h e i r r e u se sh o u l d b e p r i m a r i l y c o n t r o l l e d b y t h e d o n o r s s o f 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 , c o m p a n i e s a n d h e a l t h p r o f e ssi o n a l s a r e c u st o d i a n s. 2 . T o e n a a b l e t h u e o p t i m l r e u se o f d a t a , t h e d a t a n e e d s t o b e F i n d a b l e , Ac c e ssi b l e , I n t e r o p 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 o f e ssi o n a l s, p a t n i e t s a n d i n p a r t i c l a r a l s o b y ma c h i n e s. 3 . a T h e o p t i m l r e u se o f d a t a sh o u l d b e su p p o r t e d a t a l l l e v e l s, b y p r o f e ssi o n a l s a n d c u st o d i a n s ( a l l o wf e d e r a t e d l e a r n i n g o n t h e d a t a u p o n r e q u e st , g i v e t h e d a t a t o t h e d o n o r i n F AI R f o r m a t wh e n a sk e d ) , c a r e p r o f e ssi o n 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 o r m a t wh e r e v e r p o ssi b l e ) , a s n a l y t i c e n v i r o n m e n t s ( a d a p t t o F AI R d a t a ) a n d r e g u l a t o r s ( d e m a n d F AI R d a t a t h r o u g h o u t a n d a o p t i m l l y u se t h e m i n t h e r e g u l a t o r y p r o c e ss) . T h e r e i s a n e e d t o e d u c a t e a l l st a k e h o l d e r s a b o 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 o r t a n c e ( ‘ F AI R Awa r e ’ ) 4 . O a p t i m l c a r e sh o u l d b e t a k e n t o r e st r i c t t h e n e e d t o r e v e a l t h e a c t u a l i d e y n t i t o f 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 o p r o t e c t p r i v a c y wi t h a l l p o ssi b l e m e a n s, n b u t we r e a l i z e p r i v a c y i s su b o r d i n a t e i n m a n y c a se s t o f a st - t r a c k i n g o f b e t t e r so l u t i o s f o r t h e d i se a se s we su f f e r f r o m. 5. T h e r e f o r e , t h e r i g h t t o a l l o w i i c a n d e n t i f t i o o f 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 o u l d a l so b e p l a c e d i n t h e h a n d s o f t h a t i n d i v i d u a l o r a c h o se 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 o o l s sh o u l d b e d e v e l o p e d t o e a n a b l e o p t i m l c o - i n n v e st i g a t i o b y r e se a r c h e r s, m e d i c a l a n d h e s a l t h c a r e p r o f e ssi o n a l s, c h a r i t i e , c o m p a n i e s, p a t n i e t s a n d m a c h i n e s t o f o r m a ' so c i a l h e a l t h i 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 o n s a n d c a r e . 7 . R e g u l a t o r s sh a o u l d o p t i m l l y e n a b l e f a st - t r a c k i n g o f k e y i n n t e r v e n t i o s a n d i n v o l v e c i t i z e n a n n d m a c l h i n e p a r t i c p at i o i n t h a t p r o c e ss t o t h e l a r g e st p o ssi b l e e x t e n t . 8 . T h e f i e d s h o u l d a c t i v e l y d i sc o u r a g e p u b l i sh i n g o f h e a l t h - r e l a t e d i n f o r m n a t i o 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 o u r n a l s. T h e se a r e v e r y d i u lt f f i c t o a c c e ss a n d u n d e r st a n d b y b o t h i n f o r m e d l a y p e o 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 o r ma n t i o s h o 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 o 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 o u l d h a v e g o o d d a t a st e wa r d sh i p f o l l o 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 a d i n t h e i r g r a n t c o n d n s . i t i o 10. T h e r o l e o f h e a l t h i n su r a n c u e i n st i t t i o n s/ c o m p a n i e s sh o u l d a l so b e ma d e c l e a r : t h e y sh o 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 o 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 i a b e n t l i f e fo r t h e m t o ' t h e i r ' c l i e n t s, wi l l n o t b e a b u se d , su c h a s f o 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 o n g e n e t p i c re d i sp o n si t i o . 11. He a l t h i n su r a n c u e i n st i t t i o n s/ c o m p a n i e s a n d g o v e r n me n t s sh o u l d j o i n f o r c e s a n d su p p o r t ( a l so f i n n c i a l l y ) t h e d e v e l o p m e n t o f t r u st e d e n v i r o 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 o r t h e b e t t e r m e n t o 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 o f c o st s t o k e e p a o p t i m l h e a l t h ca r e a f f o r d a b l e f o r a l l . Classified as public by the European Medicines Agency

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) Patient Data 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. Classified as public by the European Medicines Agency

Gives patients the power and control over the use of their own data. Gives patients the option to ‘get their data together’. See it as ‘storing in a locker’ Data platform 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 Classified as public by the European Medicines Agency