Overview
To increase the awareness of Therapeutic Area specific knowledge relevant for Statistical Programmers, Statisticians and Data Managers.
Objectives
To prepare a comprehensive indication specific information related to:-
- Description of Disease
- Agency guidelines applicable for the corresponding Disease or Indication
- Clinical Trial Endpoints
- Clinical Trial Designs
- Data Collection
- Data Challenges
- SDTM Data
- ADaM Data
- Statistical Analysis
To review and maintain information on indication specifics
Alzheimer's Disease
Urgency/Background
There is unprecedented urgency and unmet medical need in Alzheimer's disease (AD). AD is the sixth-leading cause of death in the United States and is the only leading cause of death without any means to effectively prevent, cure or even slow progression. More than 5 million Americans are living with Alzheimer's disease. Without the development of important medical breakthroughs, by 2050, the number of people with Alzheimer's disease could reach 13.8 million. The human suffering of both patients and caregivers, and the economic burden on the nations' health care system and government programs is enormous. Wit the baby boomers reaching the age of elevated risk, we are running out of time. This is a crisis. We need to take action and do what we can to advance AD research... to discover the effective treatments that we want and need. Statistical urgency?
Proposal
The proposal is to form a Scientific Working Group (SWG) for the advancement of AD treatments led by statisticians from industry, academic and government sectors. The focus of this SWG will be research collaboration to develop analytical approaches in relevant areas of AD, with the primary goals of advancing the understanding of the disease and enabling the next generation of breakthrough treatments. The working group will initially focus on specific topics that are currently of great importance and interest to the AD research and medical product development communities. For example, as the AD pharmaceutical development field is moving away from symptomatic treatments, and focusing more on disease modification, how can we do a better job of demonstrating these disease modifying effects? Currently, there is limited research and publication on how to define and demonstrate AD disease modification. Concerted effort on this topic will potentially provide guidelines for future clinical trial design and regulatory evidence for decisions on new disease modifying drugs. This will likely have wide-reaching impacts beyond Alzheimer’s and will be potentially applicable to a number of other neurodegenerative diseases. Other examples of areas within AD that may have the potential to influence and improve AD research are listed below under “Topics.” As AD is an evolving field of research and development, there will be many opportunities for statisticians to take leadership roles in developing novel trial designs and statistical methodologies to help with the search for effective treatments.
Topics
Primary analysis/missing data/estimands in ADGoals
The AD Scientific Working Group has the following goals:
- Assume a statistical leadership role in advancing the AD field in the areas of research and drug development
- Form a broad-based AD Stats Community that will stay current, connected and collaborative