For drug companies developing new treatments for Duchenne muscular dystrophy, cTAP is an unparalleled resource for making clinical trials more effective and accelerating clinical progress. cTAP’s nimbleness in identifying and acting on priorities with near-term value for drug development ranks high in member-satisfaction.
We have assembled the largest database of de-identified longitudinal clinical data in the Duchenne community, currently more than 12,000 patient-visits representing real world evidence and natural history for almost 2000 patients with from 33 clinical centers in Europe and the U.S., as well as >3000 patient visits from almost 600 patients enrolled in clinical trials.
Functional outcomes include longitudinal measures of ambulation (6MWD, NSAA, TFTs), upper limb (PUL), respiration (FVC, % predicted FVC, MIP, MEP), cardiac (echo, LVEF, RVEF, left ventricle fractional shortening), and bone health (DEXA scans for bone density, body mass, % body fat, lean body mass).
The records for more than 90 percent of patients include detailed glucocorticoid dose and regimen history, and verified dystrophin genotype. Patient reported outcomes are also available for approx. 20% of patients. Typical duration of follow-up is 2-3 years; more than 400 boys have ≥3 years of follow-up; >100 boys have ≥5 years of follow-up.
cTAP combines the best of a consortium model with the best of a research collaboration model. We bring the benefits of cost-sharing, as seen in a consortium; the weight of a collective to have the most impact with regulators; the shared learning achieved by harnessing the expertise of members and shared recognition for all members for helping to bring drugs to patients sooner.
Moreover, to provide the flexibility that ensures any individual sponsor can realize the specific goals it seeks, the cTAP platform is modular: sponsors pay only for the areas they wish to fund.
Scientists, researchers and clinical registry investigators can all benefit greatly by participating in the cTAP collaboration. cTAP offers a neutral, third-party resource that makes it easy to collaborate with multiple pharmaceutical companies in a simultaneous yet fair process.
cTAP offers academic leaders and clinical networks the opportunity to access to advanced data science expertise. Researchers may even deploy cTAP’s collaborative analytic platform on their own curated data outside of cTAP. These capabilities simplify data pooling for academic collaborators, accelerated development, faster insights, and a lower barrier to sharing and replication of results.
Yet the cTAP platform and collaboration is set up to protect patient data and investigator control. De-identified patient data is shared only in a highly secure environment. Researchers retain full control of data use and seniority of authorship. cTAP can also provide support to collaborators to pay for data mining and processing, activities that are not always adequately supported by academic grants.
The payoffs of participation with cTAP are profound: researchers author more papers, promote their groups’ capabilities, access metrics demonstrating the value of curated data to funding agencies, and speed the development of new treatments for patients.
cTAP puts smart analytics and existing patient data to work to help guide faster, smarter clinical trials — harnessing the collective expertise and resources needed to accelerate the development of effective drugs that patients need.
Core to how cTAP achieves this is the power of collaboration: Everyone benefits from better understanding how to design trials that get to the right answer as efficiently as possible. Everyone can play a part in this “pre-competitive” effort to help patients access important therapies as quickly as possible — including the patient community.
cTAP’s ultimate goal is to serve the Duchenne patient community by making clinical trials more efficient and effective. Patients and patient organizations can lend a hand and help cTAP achieve that goal by staying informed on cTAP’s progress noted on the website and being ready to act when opportunities for patient community support arise. Continue to check the cTAP website for updates and information such as press releases and links to new scientific publications.
Some organizations also support cTAP activities financially, or participate by contributing their staff’s expertise to cTAP operations. If you would like to support cTAP directly, or think your programmatic staff should be involved, contact us for a conversation.