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Post Marketing Translarna Registry
Proprietary and Confidential1
9th TGDOC Curator MeetingLeuven, Belgium
Lauren Morgenroth, MS, CGCCOO TRiNDS
Tuesday September 20th, 2016
Disclosures• Employee of TRiNDS, LLC (new specialized neuromuscular contract
research organization (CRO)) which serves the academic clinical trial network, Cooperative International Neuromuscular Research Group (CINRG), as its Coordinating Center
• The CINRG network and its clinical sites are the recipient of grants from NIH, DOD, PPMD, FED, MDA, FSH Society, and FSHD Global
• CINRG and TRiNDS have business relationships with several pharmaceutical companies for data summaries, study design, study planning, study operations, data management, and statistical analysis
• 2015 Funding from FED for post marketing research and training• Consulting agreement with Marathon
Key Points and Conclusions• De-risk infrastructure with pragmatic partnership with academic
researchers and patient registries• Conservation of patient, family, and physician resources by
leverage of existing tools and networks: TREAT-NMD, CINRG/TRiNDS, Regulatory Expert CRO (Quintiles, etc.)
• Build platform that can reinvigorate research and share information with the public and community at large
• Maintain successful post marketing study and allow expansion to additional studies
Proprietary and Confidential3
Traditional Challenges: Orphan Drug Development
• Academic medicine has domain expertise in rare disease• Private industry develops drugs for rare disease:
• Focus on ‘value’ of drug development program• Typically little rare disease domain expertise
• Pragmatic partnership of academic medicine and industry seems to make sense however there are some barriers:
• Need of industry to control aspects of drug development• Need of academic researchers to be better versed in regulatory
requirements• Industry default to use contract research organizations (CROs) that have
expertise in drug development, but not in neuromuscular disease
Proprietary and Confidential4
Review of Post Marketing Efforts
Proprietary and Confidential5
• Meeting of KOLs• Existing
resources
Information Gathering
• Consulting firm assessment
• Meetings with key Pharma
Market Research • Webinars
• Presentations at curator meeting
Education
• Joining of resources: TREAT-NMD and CINRG
Platform Development • PTC: PASS
• Continued education and updates
Maintenance
2014 2015 2016
Post Marketing Resources • TREAT-NMD: http://www.treat-nmd.eu/resources/post-marketing-
surveillance/introduction/• EMA:
http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/q_and_a/q_and_a_detail_000037.jsp
• FDA: http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Post-marketingPhaseIVCommitments/
• Pharma: http://www.pfizer.com/research/pmc/what_pmc, Shire, Amgen, Janssen, Novartis, etc.
Proprietary and Confidential6
EMA Guidance – Updated in 2016
Proprietary and Confidential7
FDA Guidance
Proprietary and Confidential8
FDA Post Marketing Database
Proprietary and Confidential9
http://www.accessdata.fda.gov/scripts/cder/pmc/
Example from FDA Database • NDA Approval Date: 01/31/2012• Postmarketing requirement: Conduct an open-label, single-arm trial to
evaluate the long-term safety of Kalydeco…the trial will enroll pediatric patients < 18 years of age and who are registered in the US Cystic Fibrosis Foundation (CFF) Patient Registry. Patients will be followed for at least 3 years and long-term efficacy will also be assessed. The trial assessments will comprise the data collected as part of the CFF registry…results of this trial will be presented in the context of observational data on the same outcomes for all patients in the CFF registry…who are treated with Kalydeco (ivacaftor).
• Projected Completion Date: 12/31/2020
Proprietary and Confidential10
TRiNDS – Official Coordinating Center of CINRG
• Cooperative International Neuromuscular Research Group (CINRG)
• Academic based clinical trial network launched in 2000• Multi-disciplinary and cross-institutional network of clinicians and
scientists• Study the cause(s), pathogenesis and clinical outcomes of
neuromuscular disorders through controlled clinical studies
• Access to over 25 specialized neuromuscular clinical sites around the world
Proprietary and Confidential11
TRiNDS – CINRG Organization
Proprietary and Confidential12
Patient Support Organizations
CINRG Clinical Study Sites
CINRG Executive Committee (EC)
CINRG Publication Subcommittee
(CPS)
CINRG Outcomes Subcommittee
(COS)
CINRG Data and Safety Monitoring
Board (DSMB)
TRiNDS – New Contract Research Organization (CRO)
• TRiNDS: Therapeutic Research in Neuromuscular Disorders Solutions
• Specialized neuromuscular CRO providing four main solutions to pharmaceutical and biotechnology industries as well as academic investigators and clinical centers for their clinical study needs:1. Clinical Operations 2. Data Management 3. Biostatistics4. Study Measurements
Proprietary and Confidential13
Neuromuscular Domain Expertise
• TRiNDS team completed 12 neuromuscular clinical studies• Diseases: Duchenne, Becker, limb girdle, and facioscapulohumeral• Type: Observational, pilot, phase II, phase III, and post marketing
• Network of certified physical therapists • Clinical study protocol templates, manuals, and common case
report forms• Domain specific site monitoring, centralized data processing and
collection of site documents across over 25 clinical centers
Proprietary and Confidential14 14
Data Management • Specialized neuromuscular and rare disease expert data
managers that provide a full range of data services• TRiNDS uses OpenClinica for electronic data capture (EDC)
• 21 CFR Part 11 and GCP Compliant• Online data entry• Fully integrated system including a randomization and online participant
survey modules• Network of trained study team members for data entry and queries
• CINRG Quantitative Measurement System (CQMS) connectivity
Proprietary and Confidential16
Data Management
Study Measurements• Specialized neuromuscular expertise to select the most
appropriate study measurements across the entire disease spectrum: Biomarkers Strength and function Pulmonary Cardiac Patient reported outcomes Quality of life
Proprietary and Confidential18
Study Measurements
Natural History: Foundation and Validation
Proprietary and Confidential19
EndpointDevelopment
StudyDesign Clinical Trials Post
Marketing
Natural History Pathophysiology Effects of intervention
PTC: Translarna™ Post Marketing Commitments
• Purpose: Post-approval safety study (PASS) per pharmacovigilance risk assessment committee (PRAC) of EMA to gather data on Translarna (ataluren) safety, effectiveness, and prescription patterns in routine clinical practice
• Type: Observational [Patient Registry]• Design: Prospective• Duration: 5 years• Estimated Enrollment: 200 • Estimated Study Start – End: April 2015 – April 2022• Information from: https://clinicaltrials.gov/ct2/show/NCT02369731
Proprietary and Confidential20
Key Outcome Measures• Primary: Incidence of adverse events • Secondary:
• Change in laboratory parameters • Change in blood pressure • Prescriber and patient compliance per approved labelling
• Other: Patient health management measures
• Important note: No protocol-mandated procedures, it is expected that physicians and other caregivers follow published treatment guidelines and standards of care
• Information from: https://clinicaltrials.gov/ct2/show/NCT02369731
Proprietary and Confidential21
OpenClinica Database
Proprietary and Confidential22
OpenClinica Database (2)
Proprietary and Confidential23
Integrated Monitoring to Database
Proprietary and Confidential24
Registry of Translarna™ PASS Status• Key partnerships: PTC, TREAT-NMD, CINRG/TRiNDS, and
Quintiles• Status as of September 2016
• 10 active sites across 3 different countries• France: 4 sites (1 patient)• Germany: 5 sites (17 patients)• Austria: 1 site (3 patients)
• 21 enrolled participants • Other countries under activation, in total 60 sites will participate
across 16 different countries in Europe and outside Europe• Questions: Abdallah Delage [email protected]
Proprietary and Confidential25