Steven R. Cummings, MD Emeritus Professor of Medicine and Epidemiology, UCSF Director, SF Coordinating Center Mytrus, 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 Bladder From 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