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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 Many Sources of Subjects • Web: search engines, Craig’s list, health sites, online communities • Medical groups, HMOs, practice networks • Pharmacy databases, e.g. Medco • Recruitment companies Study Drug Is Shipped to Homes • Overnight courier • Signature required • Participant enters study drug ID to confirm receipt Points and Issues What Can be Tested? • • • • 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 D2P Trials in Medical Practice • 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 What Can’t be a D2P Study? • 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 D2P Studies and Elderly, Low SES, Some Ethnic Groups, • 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 Summary • The D2P method may make large CER studies more affordable and ‘real world’