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#PatientsEU2020
MarisolMontolio,PhDresearch@duchenne-spain.orgScientificDirectoratDuchenneParentProjectSpainAdjunctProfessoratUniversityofBarcelona
#PatientsEU2020
Curator Spanish Duchenne, Becker and Carriers Registry
Adjunct Professor at University of Barcelona
Patient Advocacy BoardMember of the muscle Diseases Specialist GroupResearch Board
Scientific Director & Director of the technological department of Duchenne Parent Project Spain
Solve-RD
Scientific Advisor (merosin deficit children association)
Management Committee (COST Action [CA17103] )
External Scientific Board
#PatientsEU2020
RAREDISEASE
AFFECTS 350MILLIONPEOPLEINTHEWORLD
80%OFRAREDISEASESAREGENETIC
ONLY5%OFRAREDISEASESHAVEANAPPROVEDTREATMENT
8% OFTHEGLOBALPOPULATIONAFFECTEDBYARARE
DISEASE
OVER 7,000 RAREDISEASESINDENTIFIED
ARAREDISEASE,INEUROPE,ISADISEASETHATAFFECTSLESSTHAN1
INEVERY2,000CITIZENS
#PatientsEU2020
What is Duchenne Parent Project Spain?
#PatientsEU2020
CEO
DUCHENNEPARENTPROJECTSPAINDEPARTMENTS
FUNDRAISINGCOMMUNICATIONRESEARCH PSYCHO-
SOCIALTECHNOLOGICALINNOVATION
LEGALADVICE
PARENTSBOARD
TEAM
#PatientsEU2020
#PatientsEU2020
#PatientsEU2020
TOKNOWWHATISTHEPROBLEM
#PatientsEU2020
We have examined how collaboration with the pharmaceutical and
technological industry, universities and hospitals can produce profits. We
have seen how a focused and methodological approach with each of
them can generate knowledge and projects much faster than expected.
#PatientsEU2020
How the patient’s organisation can promote and accelerate the
development of a drug?
THEMODEL
#PatientsEU2020
EDUCATIONALPROGRAMS
PSYCHO-SOCIALPROGRAMS
TECHNOLOGYPROGRAMS
RESEARCHPROGRAMS
WITHAGLOBALVISIONOFTHEDISEASEWEWILLBEABLETOREACHOURGOAL
WECANNOTSEPARATETHEPROCESSWENEEDTOMAKEAHOMOGENEOUSSETOFALLTHESTEPS
#PatientsEU2020
DISCOVERY/PRECLINICAL STUDIES CLINICAL TRIALS MARKETING AUTHORISATION
RESEARCHPROJECTS
RESEARCHTOOLS
RESEARCHNETWORKING
RESEARCHEDUCATION
CLINICALTRIALS
SUPPORT
PATIENTSREGISTRY
EMACONTRIBUTION
#PatientsEU2020
RESEAR
CH-Strategy PATIENTS
REGISTRY
IMPROVE MUSCLE STATUS PREVENTION INFLAMMATION, FIBROSIS
DIAGNOSIS
RESEARCH TOOLS
ATTACK THE CAUSE
Support 5 Research Projects going on
Biobank (by collaborations), Animals Models (Mouse & Zebrafish), Cells lines, Screening Platforms and Patients Registry
SARA or Services Attached to the Registry of Associated Patients
CLINICAL TRIALSMARKETING AUTHORISA
TION
QuestionariesQuiz
6MWT+Natural History
DISCOVERY/PRECLINICAL STUDIES
Support 1 Clinical TrialSARA in 5 Clinicals Trials
QuestionariesQuiz
QuestionariesQuiz
QuestionariesQuiz
2 Research and 1 Technological Project going on
10 Research Projects going on
Patients Registry
Patients Registry5 Research Projects going on
#PatientsEU2020
ExtramuralResearch(2014-2020)
SelectedProjectsthroughcompetitivecallsforgrants
IntramuralResearch(2015-2020)
ProjectscreatedbyDPPE(2017-2019)
PatientsRegistry(2015)
§ 14 projects selected since 2014 for a total amount of ~ 1,5 million euro
§ 12 laboratories supported through DPPE grants§ Every 2 years: Call for traslational research projects in
the DMD/BMD field§ Review process by an independent scientific
committee (they can not access this funding)
§ 7 projects for a total amount of ~ 500,000€§ 3 laboratories currently supported through intramural
research§ DPPE: PI is the part- owner development§ Review process non competitive fund allocation
SARA(ServicesAttachedtotheRegistryofAssociatedPatients)tosupportClinicalTrials(2016)Projects
SelectedScientificCommitteeEvaluation
ApplicationSubmission
à à
ProjectsSelected
ScientificEvaluation
ApplicationSubmission
à à
RenewalProposals
àProjectSelected
ScientificEvaluation
Project
à à
LaboratoriesSelection (academical,industry..)
à
Clinical TrialSupport:Save drugs from ValleyofDeath(2016)
#PatientsEU2020
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#PatientsEU2020
RESEAR
CH-Registry
3ClinicalTrials
November2015
14ClinicalTrials
7Hospitals
January2020
hospital
hospital
hospital
hospital
pharma
pharma
pharmapharma
DMD SPAIN SITUATION
#PatientsEU2020
INDUSTRYHOSPITALSRESEARCHERS• Pharmaceuticals• TechnologyIndustry• DATAissharedwith
Treat-NMDglobal
• DATA is shared withTreat-NMD globalDuchenne Registry
• Collaborations withdifferent Universities
• Contactwithalltheneuromuscular referencehospitalsinSpain&clinicsteam• Weprovidespecificsupport(logistic…)
RESEAR
CH-Registry
THE DATA MAKES A DIFFERENCE:WORKING TOGETHER
SARAPROGRAM
#PatientsEU2020
What is SARA? SARA or Services Attached to the Registry of Associated Patients provides a central resource that offers advice and guidance to Patients, Industry and Spanish Hospitals on how to participate in a clinical trial in DMD or DMB.
SARA is associated with the Duchenne and Becker Spain Patient Registry and is related to all the key parts: Hospitals, Patients and Industry, facilitating communications among them to accelerate the clinical trial process.
Before the Duchenne and Becker Spain Patient Registry was launched, few clinical trials could be conducted in Spain for DMD or BMD. This meant that very few patients could participate in trials every year.
We work with the staff of all the Hospitals interested, coordinating the inclusion of patients from the Duchenne and Becker Spain Patient Registry, allowing the inclusion of any patient, regardless of the place where he lives in Spain. From the Duchenne and Becker Spain Patient Registry we want to give the greatest possible support to all families through SARA.
SARA includes parallel care activities to the clinical trial directed to children and their family members, to minimize the emotional and psychological effects of participation in the clinical trial.
"SARA guarantees that all patients with DMD or BMD in Spain, regardless of their age or city of residence, have access to clinical trials"
Duchenne Parent Project España CIF G25647587 [email protected] www.duchenne-spain.org
SARA Services Attached to the Registry of Associated Patients
DUCHENNE PARENT PROJECT ESPAÑA
Patients Registry DMD and BMD in Spain
With more than 250 patients registered in the last 3 years, it is included in Treat-NMD and the European Medicines Agency.
Services Attached to the Registry of Patients
This Service facilitates communications among all the stakeholders of clinical trials to accelerate the process.
What is SARA? SARA or Services Attached to the Registry of Associated Patients provides a central resource that offers advice and guidance to Patients, Industry and Spanish Hospitals on how to participate in a clinical trial in DMD or DMB.
SARA is associated with the Duchenne and Becker Spain Patient Registry and is related to all the key parts: Hospitals, Patients and Industry, facilitating communications among them to accelerate the clinical trial process.
Before the Duchenne and Becker Spain Patient Registry was launched, few clinical trials could be conducted in Spain for DMD or BMD. This meant that very few patients could participate in trials every year.
We work with the staff of all the Hospitals interested, coordinating the inclusion of patients from the Duchenne and Becker Spain Patient Registry, allowing the inclusion of any patient, regardless of the place where he lives in Spain. From the Duchenne and Becker Spain Patient Registry we want to give the greatest possible support to all families through SARA.
SARA includes parallel care activities to the clinical trial directed to children and their family members, to minimize the emotional and psychological effects of participation in the clinical trial.
"SARA guarantees that all patients with DMD or BMD in Spain, regardless of their age or city of residence, have access to clinical trials"
Duchenne Parent Project España CIF G25647587 [email protected] www.duchenne-spain.org
SARA Services Attached to the Registry of Associated Patients
DUCHENNE PARENT PROJECT ESPAÑA
Patients Registry DMD and BMD in Spain
With more than 250 patients registered in the last 3 years, it is included in Treat-NMD and the European Medicines Agency.
Services Attached to the Registry of Patients
This Service facilitates communications among all the stakeholders of clinical trials to accelerate the process.
RESEAR
CH-Registry SARA(ServicesAttachedtotheRegistryofAssociated
Patients)tosupportClinicalTrials
2year7 Hospitals
4Pharmaceuticals1 Foundation5 ClinicalTrials
THE DATA MAKES A DIFFERENCE:WORKING TOGETHER
#PatientsEU2020
OBJECTIVEUse networking with the
aim of establishing communication and
collaboration between the scientific community that
is investigating DMD/BMD, thus
facilitating research tools for a rapid advance in
these rare diseases.
4WORKSHOPS(4Years)
9Hospitals9Universities
4Industry(nopharma)1PatientOrganization
WORKSHOPS: Transfer of Scientific Knowledge in DMD
RESEAR
CH-Networking
#PatientsEU2020
OBJECTIVE
Use networking to unifyprotocols among all thehospitals to prevent thedeterioration of lungfunction in DMD
INTERNATIONAL MEETING: TRANSFER OF KNOWLEDGE ON
RESPIRATORY MANAGEMENT IN DMD
6Hospitals4Universities
1Pharmaceutical1Patient
Organization5Countries
WORKSHOPS: International meeting: transfer ofknowledge on respiratory management in DMD
RESEAR
CH-Networking
#PatientsEU2020
RedCOST:Delivery ofAntisenseRNATherapeutics
OBJECTIVECOST:ActionCA17103
Use networking and capacitybuilding in the field of nucleic acidtherapy delivery to allow RNA-targeting nucleic acid drugs to reachtheir full potential and become amainstream therapeutic option 25European countries
Canada,ChinaandUS75researchers53%female and29%ECIs3ONGsCompanies
Management Committee (COST Action [CA17103] )
RESEAR
CH-Networking
#PatientsEU2020
WEBINNARSERIES(2017-)16Countries74%Patients/Families15%Researchers12%ClinicsSocial,Research,Psycho…
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NEWSLETTER(2016-)
15Countries>800Users
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CH-Edu
catio
n
#PatientsEU2020
INTERNATIONALCONGRESSINTERNATIONALCONFERENCE
11Countries74%Patients/Families8%Pharmaceuticals8%Clinics6%Academic Researchers4%Students
EDUCATIONALPATIENTS/CLINICSPROGRAMS
FAQs (Frequenly AskedQuestions)Genetic CounsellingMasterclass
RAREDISEASESSUMMERSCHOOL
1University5Industry10Hospitals15Research Centers
PHARMACEUTICALSADVOCACY
2016- 2018IndividuallyAdvocacy2018- Part ofPatientCommunity Advisory Boards(CABs)
RESEAR
CH-Edu
catio
n
#PatientsEU2020
1. Patient EngagementNewsletter,WebSite,InternationalConference,Educational Programs,…
2.Patient Involvement:ResearchWorkshops,Intramural Projects,…
3.Patient Participation:SARAProject,Registry…
CHECKLIST
#PatientsEU2020
Haveastrategytobefeasibleandprofitable
Fillthegapinscience/business
Createownprojects
Searchforamonetaryreturnoftheprojectscreated
Thedatamakesadifference.Worktogether!
Ifyoudonothaveenoughmoney,youmusthaveenoughcontacts!
#PatientsEU2020
@MarisolMontolio@DPPSpain
@DPPSpain
DPPE
