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STREAM THREE: Jeff Keefer, Inclusion in Clinical Trials

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Canadian Organization for Rare Disorders
Canadian Organization for Rare Disorders

A Rare International Dialogue (Saturday May 11, 2019) Ethnic and Diversity Issues Inclusion in Clinical Trials - Jeff Keefer, IQVIA

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Copyright © 2019 IQVIA. All rights reserved. Jeffrey Keefer MD, PhD Pediatric and Rare Disease Center of Excellence May 2019 Driving Diversity By Increasing Access to Clinical Trials
2 Why Does Diversity In Trials Matter? 1. From the FDA: “It is important to test drugs and medical products in the people they are meant to help. FDA works to make sure that people of different ages, races, ethnic groups, and genders are included in clinical trials.” 2. People with rare disorders deserve access to clinical trials regardless of age, race, ethnic group, geography and gender -  The ability to participate in finding cures -  Potential to benefit from the drug being tested ›  Open label extension access -  Risk/harm
3 More than 4400 clinical trials have been started for rare diseases in the past 5 years and currently there are 2150 active clinical trials globally Rare Disease Trials by Country (2014-2018) Progress in National Diversity Confidential: For internal use onlySource: Analytical Operations analysis of MARS Dashboard
Situation Solution Result •  Traditional enrollment in sickle cell disease (SCD) trials in the US and EU for extremely slow •  Difficult Indication and complex protocol •  Why are we not performing the study in countries with highest disease burden? •  Build research infrastructure in Sub- Saharan Africa for SCD •  Engaged investigators •  Eager patients •  Solutions to patient logistics •  Distance from sites •  Lack of internet/phone Sickle Cell Disease in Pediatrics Providing clinical trial access and novel therapies to those who most need it Region Protocol Deviations/ Subject ePRO compliance (%) SAE’s/ subject Africa 0.91 99 0.36 US/Canada 2.91 92 0.75 EU 1.04 93 1.96 •  Rapid recruitment •  High data quality •  Now standard practice to include sub-Saharan Africa in SCD trials
5 Established regional presence providing full service solutions to deliver quality data and new treatments to the populations that need it most Sub-Saharan Africa Clinical Operations BENEFITS OF CONDUCTING CLINICAL TRIALS IN THE REGION q  Potential to provide treatment options to patients who can truly benefit q  Relationships with eager investigators, emerging site infrastructure q  Experience working across pharma, biotech and public/ private partnership structures q  Regional teams, supported by global infrastructure and SOPs IQVIA Clinical Office locations Home-based permanent CRA staff IQVIA clinical trial experience
6 Beyond country selection: Influencing diversity in clinical trials
7 What Influences Diversity In A Rare Disease Trial? Chance Based on Investigator Patient Pool Protocol Directed Targeted Advertising •  Advocacy groups •  Social media •  Standard advertising Geography Study Burden
8 Identifying People With Rare Diseases Using Data
9 Increasing Access to Research with Data and Novel Trial Models >Real world data supports patient referrals Research site in big city >Precision enrollment solution >Training research naïve sites >Virtual trial model >Home healthcare Care in patient home Provide care local to patient Open site closer to patient
10 Local Care Team Health Care Practitioner Home Health Nurse Phlebotomist Recruitment ExpertPatient Guide Virtual PI and Study Team Study Concierge Patient Mobile Health Devices Electrocardiography Blood Pressure Monitor Continuous Glucose Monitor Actigraphy Spirometry Primary Investigator Smartphone The Virtual Trial: Putting the Patient at the Center
11 Increasing Access to Research with Data and Novel Trial Models >Real world data supports patient referrals Research site in big city >Precision enrollment solution >Training research naïve sites >Virtual trial model >Home healthcare Care in patient home Provide care local to patient Open site closer to patient
12 Patient Identification and Referral Boost Patient Access to Trial Traditional approach New approach Access to Potential Patients PatientPotential 226% Increase >80 patient lives impacted with access to treatment 89% patients enrolled globally from referral sources 37% physician specialists referred a patient (US high-touch referral support) •  Global study of rare form of blindness •  Cross-border referrals coordinated with sponsor •  Multi-channel efforts leverage sponsor, advocacy, and investigator relationships •  Public physician registry data confirms engaged specialists, but lacks insight on patient activity •  Unmet need for treatment options generating high level of physician interest
13 •  Collaborated with travel concierge vendor ü Travel concierge and site portal to facilitate patient travel ü Prepaid debit card with notice for meals, etc. ü Manage patient stipends Reducing Patient Burden Increases Potential for Participation Challenge Solution Extremely limited trial population •  US-only study in ultra rare disease •  Critical to capitalize on all enrollment opportunities Complicated treatment Limited sites participating •  Highly-specialized experts referring to few specific centers able to provide treatment •  Patients requiring mix of travel support based upon geography 13
14 Joint aches Thick, coarse oil skin Sleep apnea Fatigue and weak Excess sweat Presents to Primary Physician with symptoms John Presents: Nov 2018 Referred to Cardiologist Referred to Dermatologist 1 Feb 2019 Referred to ENT specialist Mar 2020 Referred to Rheumatologist Jul 2022 Finds Dermatologist 2 John is Diagnosed: Sep 2023 Diagnosis of Acromegaly from Endocrinologist (ICD-10 code E22.0) Aug 2019 Apr 2021 Referred to Endocrinologist Jul 2023 Average 4.8 Years and 7.4 physicians to diagnosis John’s symptoms fluctuate, Disease progresses John may be given additional treatment or may be eligible for clinical research study Improved Access to Clinical Trials Will Drive Diversity

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STREAM THREE: Jeff Keefer, Inclusion in Clinical Trials

  • 1. Copyright © 2019 IQVIA. All rights reserved. Jeffrey Keefer MD, PhD Pediatric and Rare Disease Center of Excellence May 2019 Driving Diversity By Increasing Access to Clinical Trials
  • 2. 2 Why Does Diversity In Trials Matter? 1. From the FDA: “It is important to test drugs and medical products in the people they are meant to help. FDA works to make sure that people of different ages, races, ethnic groups, and genders are included in clinical trials.” 2. People with rare disorders deserve access to clinical trials regardless of age, race, ethnic group, geography and gender -  The ability to participate in finding cures -  Potential to benefit from the drug being tested ›  Open label extension access -  Risk/harm
  • 3. 3 More than 4400 clinical trials have been started for rare diseases in the past 5 years and currently there are 2150 active clinical trials globally Rare Disease Trials by Country (2014-2018) Progress in National Diversity Confidential: For internal use onlySource: Analytical Operations analysis of MARS Dashboard
  • 4. Situation Solution Result •  Traditional enrollment in sickle cell disease (SCD) trials in the US and EU for extremely slow •  Difficult Indication and complex protocol •  Why are we not performing the study in countries with highest disease burden? •  Build research infrastructure in Sub- Saharan Africa for SCD •  Engaged investigators •  Eager patients •  Solutions to patient logistics •  Distance from sites •  Lack of internet/phone Sickle Cell Disease in Pediatrics Providing clinical trial access and novel therapies to those who most need it Region Protocol Deviations/ Subject ePRO compliance (%) SAE’s/ subject Africa 0.91 99 0.36 US/Canada 2.91 92 0.75 EU 1.04 93 1.96 •  Rapid recruitment •  High data quality •  Now standard practice to include sub-Saharan Africa in SCD trials
  • 5. 5 Established regional presence providing full service solutions to deliver quality data and new treatments to the populations that need it most Sub-Saharan Africa Clinical Operations BENEFITS OF CONDUCTING CLINICAL TRIALS IN THE REGION q  Potential to provide treatment options to patients who can truly benefit q  Relationships with eager investigators, emerging site infrastructure q  Experience working across pharma, biotech and public/ private partnership structures q  Regional teams, supported by global infrastructure and SOPs IQVIA Clinical Office locations Home-based permanent CRA staff IQVIA clinical trial experience
  • 6. 6 Beyond country selection: Influencing diversity in clinical trials
  • 7. 7 What Influences Diversity In A Rare Disease Trial? Chance Based on Investigator Patient Pool Protocol Directed Targeted Advertising •  Advocacy groups •  Social media •  Standard advertising Geography Study Burden
  • 8. 8 Identifying People With Rare Diseases Using Data
  • 9. 9 Increasing Access to Research with Data and Novel Trial Models >Real world data supports patient referrals Research site in big city >Precision enrollment solution >Training research naïve sites >Virtual trial model >Home healthcare Care in patient home Provide care local to patient Open site closer to patient
  • 10. 10 Local Care Team Health Care Practitioner Home Health Nurse Phlebotomist Recruitment ExpertPatient Guide Virtual PI and Study Team Study Concierge Patient Mobile Health Devices Electrocardiography Blood Pressure Monitor Continuous Glucose Monitor Actigraphy Spirometry Primary Investigator Smartphone The Virtual Trial: Putting the Patient at the Center
  • 11. 11 Increasing Access to Research with Data and Novel Trial Models >Real world data supports patient referrals Research site in big city >Precision enrollment solution >Training research naïve sites >Virtual trial model >Home healthcare Care in patient home Provide care local to patient Open site closer to patient
  • 12. 12 Patient Identification and Referral Boost Patient Access to Trial Traditional approach New approach Access to Potential Patients PatientPotential 226% Increase >80 patient lives impacted with access to treatment 89% patients enrolled globally from referral sources 37% physician specialists referred a patient (US high-touch referral support) •  Global study of rare form of blindness •  Cross-border referrals coordinated with sponsor •  Multi-channel efforts leverage sponsor, advocacy, and investigator relationships •  Public physician registry data confirms engaged specialists, but lacks insight on patient activity •  Unmet need for treatment options generating high level of physician interest
  • 13. 13 •  Collaborated with travel concierge vendor ü Travel concierge and site portal to facilitate patient travel ü Prepaid debit card with notice for meals, etc. ü Manage patient stipends Reducing Patient Burden Increases Potential for Participation Challenge Solution Extremely limited trial population •  US-only study in ultra rare disease •  Critical to capitalize on all enrollment opportunities Complicated treatment Limited sites participating •  Highly-specialized experts referring to few specific centers able to provide treatment •  Patients requiring mix of travel support based upon geography 13
  • 14. 14 Joint aches Thick, coarse oil skin Sleep apnea Fatigue and weak Excess sweat Presents to Primary Physician with symptoms John Presents: Nov 2018 Referred to Cardiologist Referred to Dermatologist 1 Feb 2019 Referred to ENT specialist Mar 2020 Referred to Rheumatologist Jul 2022 Finds Dermatologist 2 John is Diagnosed: Sep 2023 Diagnosis of Acromegaly from Endocrinologist (ICD-10 code E22.0) Aug 2019 Apr 2021 Referred to Endocrinologist Jul 2023 Average 4.8 Years and 7.4 physicians to diagnosis John’s symptoms fluctuate, Disease progresses John may be given additional treatment or may be eligible for clinical research study Improved Access to Clinical Trials Will Drive Diversity