steven r. cummings, md emeritus professor of medicine and epidemiology, ucsf director, sf...
Post on 19-Dec-2015
217 views
TRANSCRIPT
![Page 1: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/1.jpg)
Steven R. Cummings, MDEmeritus Professor of Medicine and Epidemiology, UCSF
Director, SF Coordinating CenterMytrus, Founder & CEO
![Page 2: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/2.jpg)
Purpose and Outline• Describe a method that may conduct trials
more efficiently• Demonstrate the method developed by a
start-up, Mytrus• Discuss its limitations
![Page 3: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/3.jpg)
Financial Interest
Founder and stock holder in Mytrus
![Page 4: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/4.jpg)
Comparative Effectiveness Trials• Generally large… and expensive• Ideally ‘real world’
![Page 5: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/5.jpg)
Bricks-and-Mortar Multicenter Trials
• Bricks-and-mortar clinical sites – including academic sites – are expensive
• Sites account for ~75% of trials costs
![Page 6: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/6.jpg)
Bricks-and-Mortar Multicenter Trials
• Recruitment is limited to subjects who live near the sites
• Research sites typically enroll select patients; not “real world”
![Page 7: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/7.jpg)
Direct-to-Participant (D2P) Trials• One clinical & coordinating site• Connects to participants at home by web,
phone, and wireless technologies
![Page 8: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/8.jpg)
Direct-to-Participant (D2P) Trials• All parts of trials can be done from home:
eligibility, consent, labs, drug delivery, efficacy and safety endpoints
![Page 9: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/9.jpg)
Potential value• 20-60% less expensive especially for large
studies• May recruit more rapidly• More ‘real world’
![Page 10: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/10.jpg)
First D2P Trial: KALM Trial (2000)• Nutraceuticals for insomnia or anxiety• All on-line: eligibility, consent, drug delivery,
endpoints, safety management• 391 recruited from 45 states in 8 weeks
1747
![Page 11: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/11.jpg)
Key Support for the D2P Method• FDA supports the method; approved a trial• Trials have been approved by national (WIRB)
and local (UCSF, UC Davis…) IRBs
![Page 12: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/12.jpg)
How it is done
Trial of Detrol for Overactive BladderFrom the Subject’s Point of View
![Page 13: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/13.jpg)
• Web: search engines, Craig’s list, health sites, online communities
• Medical groups, HMOs, practice networks• Pharmacy databases, e.g. Medco• Recruitment companies
Many Sources of Subjects
![Page 14: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/14.jpg)
Study Drug Is Shipped to Homes• Overnight courier• Signature required• Participant enters study drug ID to confirm
receipt
![Page 15: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/15.jpg)
Points and Issues
![Page 16: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/16.jpg)
• Labs and many in home measurements• Behavioral interventions• Injectable drugs given by home nurses• Clinical endpoints – Self report confirmed by medical records– Medical databases, eg Medicare, EMRs
What Can be Tested?
![Page 17: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/17.jpg)
• Medical practices can participate without a research infrastructure
• Identify patients who may qualify• Give patients an iPad with the system in the
office; continue follow-up on line at home• The patient is managed by the central site• eg, a UAB trial will enroll 8,000 from primary
care offices to compare 2 osteoporosis drugs
D2P Trials in Medical Practice
![Page 18: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/18.jpg)
• Not for trials that require specialized examinations at baseline and all follow-up visits
• Less efficient for complex trials– If there are many procedures and hurdles, most
subjects need personal help
What Can’t be a D2P Study?
![Page 19: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/19.jpg)
• No data yet; web use is growing• Most parts can be done from home by phone;
using cell-phones may broaden participation• With a large denominator, trials might
selectively over-enroll some groups
D2P Studies and Elderly, Low SES, Some Ethnic Groups,
![Page 20: Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, Founder & CEO](https://reader036.vdocument.in/reader036/viewer/2022062516/56649d355503460f94a0d509/html5/thumbnails/20.jpg)
• The D2P method may make large CER studies more affordable and ‘real world’
Summary