CDRC Annual Meeting Resources

Day 1 Panel Discussions: Session 1: Rare Diseases
Moderators: Matt Might (UAB) and Shira Strongin (FDA)
Panelists: Andy Crouse (UAB), Ethan Perlstein (Perlara), Eva Morava-Kozicz (Mayo), Lisa Schill (RASopathies Network), Omid Karkouti (Rarebase), Clare Thibodeaux (Cures Within Reach), Sandra Sermone (ADNP Kids Research Foundation)

Session 2: Rare Oncology
Moderators: Leslie Doros (FDA) and Sonia Singh (FDA)
Panelists: Bill Tap (MSKCC), Denise Reinke (U Michigan), Kris Ann Schultz (Children’s MN), Pan Pantziarka (Anticancer Fund), Breelyn Wilky (U Colorado)

Day 2 Panel Discussions: Session 3: Special Populations
Moderators: Matt Laughon (UNC) and Kate Borkowski (FDA)
Panelists: Anup Challa (Vanderbilt), Rachel Greenberg (Duke), Mili Duggal (FDA), Matt Robinson (Hopkins), Khyzer Aziz (Hopkins), Perdita Taylor-Zapata (NICHD), Kanwaljit Singh (INC), Prabha Viswanathan (FDA), Genny Taylor (UNC), David Kimberlin (UAB), Jason Lang (Duke), Barbara Goodman (Cures Within Reach)

Session 4: Regulatory
Moderators: David Simon (Harvard) and Marco Schito (C-Path)
Panelists: Jonathan Darrow (Harvard), John Liddicoat (Cambridge), Sundeep Agrawal (FDA), Daniel O’Connor (MHRA), Lydie Meheus (Anticancer Fund), Heather Stone (FDA), Perdita Taylor-Zapata (NICHD), Nitin Bagul (TGA), César Hernandez Garcia (AEMPS Spain), Momir Radulović (Slovenian Medicines Agency), Agnes Klein (Health Canada)

Day 3 Panel Discussions: Session 5: Electronic Health Records

Panel 1 Moderators: Smith Heavner (C-Path) and Aysun Tekin (Mayo Clinic)
Panelists: Rahul Kashyap (VIRUS Registry), Matt Robinson (JHU), Paul Nagy (JHU), Matt Roe (Verana Health), Laura Merson (Oxford)

Panel 2 Moderator: Laura Merson (Oxford) and Smith Heavner (C-Path)
Panelists: Will Stevens (RECOVERY Trial, University of Oxford), Ann-Marie Mallon (NHS Digital), Kalynn Kennon (IDDO), Miguel Pedrera Jimenez (Hospital 12 de Octubre, Madrid, Spain)

CDRC 2021 Annual Meeting: The Collaboratory

Session 1 

1. Shira Strongin’s Story
2. Congenital disorders of glycosylation
3. Individualized drug repurposing for the long tail of genetic diseases
4. Repurposing Ketamine from Club to Clinic
5. Repurposing- Case Study

Session 2

1. The Use of Repurposed Drugs in Sarcoma
2. Rare Oncology Session Sarcoma Drug Repurposing
3. Engaging Sarcoma Patient Advocates
4. Propanolol in Angiosarcoma- the story so far
5. From Bedside to Registry… and Back Again

Session 3

1. Ranking Diseases in Pregnancy for Drug Repurposing
2. Neonatal Global Rank Score Development
3. Congenital CMV Treatment Options
4. The Pediatric Trials Networks experience
5. Need to Develop Survey Tools
6. Repurposing of montelukast for infants with bronchopulmonary dysplasia

Session 4

1. Introduction & Work Group Goals
2. Role of non-profit organizations in non-commercial repurposing
3. Oncology Center of Excellence Project Renewal
4. Regulatory Views- ILAP Initiative
5. Closing the gap in generic drug repurposing
6. Policy Issues in Drug Repurposing

Session 5

1. Use of SDTM and ADaM with electronic healthcare data
2. The ISARIC COVID-19 Data Platform
3. Mapping EHR data to inform clinical characterization
4. SCCM Discovery VIRUS COVID-19 Registry
5. Virus Registry with OHDSI: Automating ETL from EMRs

C-Path’s Cure Drug Repurposing Collaboratory will host a three-day annual meeting in a virtual format, November 16-18, 2021.

Together, with key stakeholder groups including clinicians, researchers, foundations, nonprofits, patient advocates and regulators, CDRC will host a public webinar this November to discuss how the Collaboratory can capture real-world data that generate hypothesis regarding treatment efficacy using repurposed drugs to address diseases of high unmet clinical need. The meeting will include key opinion leaders from numerous disease communities as well as experts on automated EHR data extraction and ways in which these resources can be leveraged to develop partnerships to inform large adaptive platform trials.

What to expect:
Day One will be hosted by the Rare Diseases and Rare Oncology Coordinating Committees.

Topics will include:

• Repurposing case study
• AI tool predicted ketamine may help ADNP-driven autism
• The use of repurposed drugs in rare sarcoma subtypes and current examples
• Patient perspective on drug repurposing for sarcoma
• Registries for rare tumors and case report form

Day Two will be hosted by the Regulatory, Legislative and Policy working group and the Special Populations Coordinating Committee. Topics will include:

• Regulatory challenges in drug repurposing
• U.S. FDA Project Renewal to update drug labels
• MHRA ILAR Initiative to support alternative regulatory pathways
• Congenital CMV treatment options and existing guidelines
• Generating RWD to inform clinical trials
• Survey tools

Day Three will include sessions dedicated to electronic health record data extraction for COVID-19. Topics will include:

• Developing registries to leverage RWD from EHRs
• Data harmonization and common data models
• International collaboration and data sharing
• Automating extraction of EHR data

Agenda 

Speaker Bios