TREAT-NMD and post-marketing surveillance for NMD drugs
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1 | TREAT-NMD and post-marketing surveillance for NMD drugs
Transcript of TREAT-NMD and post-marketing surveillance for NMD drugs
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TREAT-NMD and post-marketing surveillance for NMD drugs
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What is post-marketing surveillance?
Post marketing surveillance (PMS) is the collection of safety and efficacy data on a drug that has received (conditional) marketing approval for treating patients.
PMS data has to be collected on every patient receiving the drug, or an agreed number of these patients, and collection of data can last for up to 10-15 years.
Why does post-marketing surveillance data need to be collected?
Post-marketing surveillance data shows how a drug performs once it moves into the ‘normal clinical setting’. This data includes measures of safety (side effects), as well as efficacy (improvements in patient quality of life)
What is the role of EMA or FDA?
New therapies are only available after pharmaceutical companies have received marketing authorization (or conditional marketing authorization) from regulatory authorities, such as the European Medicines Agency (EMA) and/or the Food and Drug Administration (FDA) in the US. Other countries have similar regulatory agencies.
These regulatory authorities mandate post-marketing surveillance of authorized drugs.
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How post-marketing surveillance data is
collected and requirements
• Data is normally collected by clinicians, and in some cases, the patients
themselves.
• Any clinical centre that sees a patient receiving a new therapy will normally enter into a contract to agree to the reporting of data for PMS purposes.
• Generally, clinicians are compensated for their time to enter data into a database.
• Regulatory compliant data capture, storage and processing in a certified database system
• Accurate and reliable measures of patient health and well being (Quality controlled)
• Technical solution needs to be accessible, sustainable, and resource-sparing to patients and families
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The context for TREAT-NMD and CINRG
involvement in developing a platform
Pharmaceutical regulators, patient organizations, and clinical trial networks would like to see disease-specific post-marketing registries rather than product/drug-specific registries.
A disease-specific platform would reduce fragmentation of data collection, allow comparisons across different therapeutic interventions, and reduce the burden on patients and their clinicians.
This approach has been highly encouraged in a number of reports, including:
• EUCERD Recommendations on RD Patient Registration and Data Collection (2013)
• EUCERD/EMA meeting report on public-private partnerships for registries in rare diseases (2011)
Post-marketing surveillance of drugs for neuromuscular diseases
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How does this fit with TREAT-NMD affiliated
registries? • Several years of discussion about position of TREAT-NMD
affiliated registries have already taken place (facilitated by Filippo Buccella)
• Inquiries lead to financial return to the registries and this can be further expanded in the PMS platform
• Moving from the current TREAT-NMD affiliated registry system to a PMS compliant system has great opportunities for the registries Currently the TREAT-NMD registries have been set up to
primarily support clinical trial readiness and few provide longitudinal data
• But presents significant challenges Due to the strict regulatory environment in which PMS takes
place
CINRG is a clinical trial network that has that expertise
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What would be the advantages and opportunities of
having a disease-specific (shared) PMS platform?
• Greater transparency, faster reporting, reduced fragmentation of
data and greater efficiency for the pharmaceutical industry.
• This model would also reduce the burden on patients and clinicians, harmonising the whole data collection process.
• A disease specific platform could also include additional information from already existing patient registries.
• Involvement in a PMS platform would give visibility, show innovation and provide the potential for investment
• For some registries, little would need to change, others would need to link with clinical data collection/ natural history data collection in a way that has not happened to date
• Affiliation with a regulatory compliant single database system is necessary for the PMS data to be collected and stored in a compliant manner
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What we need to consider to be suitable for
PMS
• TREAT-NMD registries have much of the infrastructure and networks needed to enrol patients into a PMS study (as they do for clinical trials)
• However, the TREAT-NMD registries do not have the regulatory aspects currently in place (compliance monitoring, training/reliability, data acquisition and data management structures) that PMS requires
• DMD registries are organised on national level: a PMS registry is likely to require international data co-ordination
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How to develop a system to deliver on
PMS? • Regulatory compliance
• Clinician entry of data
• Contracting issues
• Partnership with clinical trial
network: CINRG that has been
running clinical trials and natural
history studies in a compliant
system
• Partnership with or development
of links with clinician entered
data
• Establishment of company to
deal with contracting and liability
issues
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PMS and Aggregation of NH data Supported
by Parent Foundation (FED) • PMS platform business model and regulatory seminars
• Consensus meeting between CINRG, TREAT-NMD and PTC on design of a PMS platform (March 2015)
First concept for STRIDE-NMD discussed
• PTC contracted with CINRG and TREAT-NMD to begin delivery of the STRIDE platform
STRIDE-Translarna™
• NH Data Convergence meeting at WMS (Oct 2015)
Compare different data elements collected by NH studies
How might they correspond and be merged?
• TREAT-NMD, CINRG and UPPMD met in Newcastle to discuss possible platform solutions for PMS (Nov 2015)
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Legacy / ongoing natural history studies
Other
NH
data
Add. NH
data
CINRG
DNHS Regulatory Compliant
Disease Specific Postmarketing
Protocols/Registries
Current situation: TREAT-NMD global registries, TREAT-NMD care and trial site registry, legacy and ongoing natural history studies (i.e. CINRG) are data silos with some common elements and components that can be linked for new efforts in post-marketing regulatory complaint registries. Proposal: STRIDE-NMD (Strategic Targeting of Registries and International Datasets of Excellence in Neuromuscular Disorders)
TREAT-NMD Global
Registries
TREAT-NMD Care and Trial Site Registry (CTSR)
Can currently deliver : site identification and training, patient identification, legacy NH data and regulatory compliant data capture Aim to deliver: linked resources for easier data collection, data access and linkage across registry types
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Post-marketing surveillance of drugs for neuromuscular diseases
Phase 1: Connecting national registries and natural history
studies (phase 0 - mapping study – In Progress)
Phase 2: Connecting additional resources and clinical user
interface
Phase 3: Expansion of model to additional drugs and
diseases
The context for TREAT-NMD and CINRG
involvement in developing a platform
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Legacy and ongoing natural history studies
Other
NH
data
CINRG
DNHS
Add. NH
data
TREAT-NMD Global Registries
Regulatory Compliant Disease Specific Postmarketing
Protocols/Registries
TREAT-NMD Care and Trial Site Registry
(CTSR)
Clinical user interface
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EMA Collaborative Framework for Patient Registries
Cross-Committee Task Force on Patient Registries Cross-Committee Task Force established • Develop strategy paper • Proposal for Pilot phase to test alternative regulatory
approaches
Objectives of Strategy on Patient Registries Primary objective: Develop and test EU collaborative framework for patient registries that would facilitate collection and analysis of high quality data to inform regulatory decisions; Long term objective: Test the feasibility of integrating registries in the adaptive licensing pilot, the one-stop shop strategy and the joint discussions between regulators and HTA bodies/payers.
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Aim of pilot phase
To test different methodological elements of the strategy on registries that may facilitate use of existing registries (or development of a new registry) to help MAAs/MAHs meet regulators’ needs for additional data in the post-marketing phase. Pilot phase to start end of 2015 after selecting products
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PTC and Translarna™ – First Opportunity!
• CINRG and Quintiles are providing PTC with the regulatory compliant database for Translarna™
• TREAT-NMD has negotiated a contract with PTC to provide additional support to PTC and the database Identifying clinical sites in countries where Translarna™ is
available
Providing training to sites
Developing a communications plan for PTC to inform sites and patients
Deliver the STRIDE platform!
• This is a great opportunity for us to build a regulatory compliant PMS platform for ALL NMDs
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The regulatory compliant platform leveraging
the disease and network expertise from
CINRG – clinical trial network
• Academic based clinical trial network founded in 1999
at Children’s National in Washington DC, governed
through by-laws and an elected Executive Committee
• Key accomplishments related to post-marketing:
15 years of sustainability as a NMD clinical trial network
12 completed and 7 active NMD research studies
Clinical study protocol templates, manuals, and common case
report form sets used across studies
Expert database development and management
Global utilization of data entry platform
Regulatory compliant tracking for personnel and data
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CINRG Data Management and
Operations Systems
• OpenClinica – Electronic data capture (EDC) for clinical data
21 CFR Part 11 Compliant
• REDCap – EDC for patient outcomes
21 CFR Part 11 Ready
• Evolve – Clinical Trial Management System
Goal: Develop open access tools that benefit the international
neuromuscular research community
