Global (coordination) of MitoPatients Registry: a challenge

5th Italian MeetingMitochondrial Medicine

June 5th 2015 – Bologna

Piero Santantonio

Summary1. Mitocon is involved in Patients clinical Registry projects since 2011

(Italian Network)

2. In yearly meetings IMP, the International Federation of Patients Associations (until now 12 national patients associations from Europe, America, Australia and Asia) for the last 2 years patients registries issue was always present in agenda and the subject discussed

3. In many part of the world researchers and patients associations are working on different registries for two main purpose:• clinical registries• patients driven registries for «counting» mito-patients popolation

and gather informations about state of thier health and quality of their life,.

4. IMP decided in the last meeting in Tampere in june 2014 to start a project titled: Global (coordination of) patient registries

Use of Mito-Patients data

Lobby

Clinical trial

Pharma needs

Social needs

(assoc.)Fund

RaisingPolitical and

Public Health

ClinicalRegistries

Other National/

Global Rare disease

registries

PatientsRegistries

Mito-Patients data: a possible path

Universe of Mito-patients data

Anagr.

basic

specific

(family, country, social

context, ...)

Clinical

symptons

rough

detaile

d

diagnosys

clinical

rough

det.

biochemical

rough

det.

genetics

rough

det.

Bio bank

rough

det.

therapy

rough

det.

follow up

rough

det.

QoL

rough

det.

Social needs

rough

det.

Data needed in Clinical Registries

Universe of Mito-patients data

Anagr.

basic

specific

(family,

territ.

context, ...)

Clinical

symptons

rough

detaile

d

diagnosys

clinical

rough

det.

biochemical

rough

det.

genetics

rough

det.

Bio bank

rough

det.

therapy

rough

det.

follow up

rough

det.

QoL

rough

det.

Social needs

rough

det.

Data needed in Patients Registries

Universe of Mito-patients data

Anagr.

basic

specific

(family,

territ.

context, ...)

Clinical

symptons

rough

detaile

d

diagnosys

clinical

rough

det.

biochemical

rough

det.

genetics

rough

det.

Bio bank

rough

det.

therapy

rough

det.

follow up

rough

det.

QoL

rough

det.

Social needs

rough

det.

Nowadays situation

ClinicalRegistries

Other National/ Global Rare

disease registries

PatientsRegistries

QoLProj.

A possible approach to build a patients registry coherent with other registries

ClinicalReg. #1Data Set

ClinicalReg. #2

Data Set

Patient Reg. #1 Data Set

1. collect data set from active Registries (clinical, patients, «Global»,

2. Involve the main actors:• Patients Ass. because

of QoL and Social needs data

• Clinicians because of clinical data

3. find a possible recomanded data set for Mito Patients (RED) and Clinical Registries (YELLOW)

Recomandeddata set for

ClinicalRegistries

Recom. Data Set for

Patients Reg.

Where we areNation Resp. Data

Type driven by N° of patients

Biobank linked

Germany T. Klopstock Clinical/ Research German consortium 1.000

Yes(~ 90% pts )

UK R. Mc Farland

Clinical/ Research MRC/ Newcastle 1.100

Yes(~ 50%pts)

USA M. Hirano Clinical/ Research Columbia Univ. 500 Yes

USA P. Yeske Patients UMDF500~400

caregiver

~ 100 family

-

USA Amy Holbert Research Rare Diseases C.R.N.

NAMDC 1159 No

Italy M Mancuso Clinical/ Research MITOCON 1.300 Not directly

What we have planned to do• Compare database• Map the data correspondences• Give a sort of minimum standard of patients

registries database• Search for a system or a SW to merge/compare

data• Build a patients-driven Registry for patients

associations purpose, above all with extended QoL data

Where are we?1. review and compare clinical part of the data set

(almost concluded)2. define Patients data set (almost concluded,

Scientific comitee supervision requested)3. review already existing SW which can manage such

patients defined data (ongoing)4. define policy and rules of use of data and

involvement of national associations (to be done)5. define a budget (to be done)6. Plan the following steps (to be done in next IMP

meeting)

Further implementation forItalian Patients Registry1. Mitocon will support the «maintenance» of the

actual Italian Registry• fund for each new patients registration• fund for new Mito-Italian-Centre• fund for research projects (i.e. pediatrics data analysis)

2. Start with Patients Section and Patients inclusion in Patients Registry Project• New QoL section for patients• Call for patients collaboration for personal and QoL

data (NO CLINICAL)

Patients Section

THANK YOU