Pilot Studies for the Active Comparator Osteoporosis Large Simple Trial (ATLAST)

说明(申请人提供)：尽管用于预防和治疗骨质疏松症的已获批准疗法的数量不断增加，但人们对现有的和较新的抗骨质疏松症药物的相对骨折风险降低和长期安全性知之甚少。大型简单临床试验(LST)，也称为实用型试验，包括随机化，但使用最小的纳入/排除标准，允许对传统随机对照试验中经常被排除在外的“真实世界”患者群体进行研究。积极的骨质疏松症大型简单试验(ATLAST)将是对接受FDA批准的抗骨质疏松症治疗的患者进行的LST试验，该治疗将研究不同社区人群的比较有效性和安全性结果。作为一项研究设计效率，ATLAST将包括通过与Medicare医疗索赔和D部分处方药数据相联系对患者结果的纵向监测。为了促进未来ATLAST研究的成功实施，我们建议进行一项试点和可行性测试，重点是开发和完善基础设施和患者登记流程。我们建议开发和测试与Mytrus合作开发的基于网络的技术用于繁忙的社区医生办公室的可行性。这笔试点赠款的具体目标(SA)是：SA 1)开发完成ATLAST所需的新材料和修改现有材料，包括：a)定制和试点测试互动登记、知情同意和在安全的基于网络的数据收集平台上运行的随机化软件，b)获得与联邦医疗保险数据链接所需的同意和受保护的健康信息，以及c)开发以患者为基础的骨质疏松症结果后续调查；SA 2)完成为ATLAST研究开发的流程和方法的小型临床试验(即试点测试)，合作伙伴包括六个地理位置不同的社区一般做法，以测试36名患者对知情同意程序、随机化、后续调查和创建Medicare Finder档案的接受性和可行性。从ATLAST试点研究中吸取的经验教训将促进ATLAST的全面推广，这将在基于社区的LST和骨质疏松症比较有效性研究方面取得重大进展。

DESCRIPTION (provided by applicant): Despite continued increases in the number of approved therapeutics for prevention and treatment of osteoporosis, very little is known about the comparative fracture risk reduction and long-term safety of well-established and newer anti-osteoporosis drugs. Large simple clinical trials (LSTs), also known as pragmatic trials, include randomization but use minimal inclusion/exclusion criteria, allowing for study of 'real world' patient populations who are often excluded in traditional randomized controlled trials. The Active Comparator Osteoporosis Large Simple Trial (ATLAST) will be an LST of patients randomized to FDA approved anti-osteoporosis therapies that will study comparative effectiveness and safety outcomes in diverse community-based populations. As a study design efficiency, ATLAST will include the longitudinal surveillance of patient outcomes through linkage with Medicare medical claims and Part D prescription drug data. To facilitate the successful implementation of a future ATLAST study, we propose a pilot and feasibility testing that focuses on developing and refining the infrastructure and patient enrollment process. We propose to develop and test feasibility of using web- based technology developed in collaboration with Mytrus to be used in busy community physician offices. The specific aims (SAs) of this pilot grant are: SA 1) To develop new and to modify existing materials necessary for the completion of ATLAST including: a) customizing and pilot testing of interactive enrollment, informed consent, and randomization software running on a secure web-based data collection platform, b) obtaining consent and protected health information needed for linkage to Medicare data, and c) developing a patient-based osteoporosis outcomes follow-up survey; SA 2) To complete a mini-clinical trial (i.e. pilot test) of the processes and methods developed for the ATLAST study in partnerships with a sample of six geographically diverse community general practices to test the acceptance and feasibility of informed consent processes, randomization, follow-up surveys, and creation of Medicare finder files in 36 patients. Lessons learned from the ATLAST pilot study will facilitate the full- scale rollout of ATLAST that will provide major advances in community-based LSTs and osteoporosis comparative effectiveness research.