Steven R. Cummings, MDEmeritus Professor of Medicine and Epidemiology, UCSF

Director, SF Coordinating CenterMytrus, Founder & CEO

Purpose and Outline• Describe a method that may conduct trials

more efficiently• Demonstrate the method developed by a

start-up, Mytrus• Discuss its limitations

Financial Interest

Founder and stock holder in Mytrus

Comparative Effectiveness Trials• Generally large… and expensive• Ideally ‘real world’

Bricks-and-Mortar Multicenter Trials

• Bricks-and-mortar clinical sites – including academic sites – are expensive

• Sites account for ~75% of trials costs

Bricks-and-Mortar Multicenter Trials

• Recruitment is limited to subjects who live near the sites

• Research sites typically enroll select patients; not “real world”

Direct-to-Participant (D2P) Trials• One clinical & coordinating site• Connects to participants at home by web,

phone, and wireless technologies

Direct-to-Participant (D2P) Trials• All parts of trials can be done from home:

eligibility, consent, labs, drug delivery, efficacy and safety endpoints

Potential value• 20-60% less expensive especially for large

studies• May recruit more rapidly• More ‘real world’

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

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

How it is done

Trial of Detrol for Overactive BladderFrom the Subject’s Point of View

• 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

Study Drug Is Shipped to Homes• Overnight courier• Signature required• Participant enters study drug ID to confirm

receipt

Points and Issues

• 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?

• 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

• 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?

• 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,

• The D2P method may make large CER studies more affordable and ‘real world’

Summary