February 2014

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FDA and IOM Examine Risk-Benefit

On February 12, the U.S. Food and Drug Administration (FDA)held a workshop in partnership with the Institute of Medicine on characterizing and communicating uncertainty in the assessment of risk and benefits of pharmaceutical products. As part the fifth reauthorization of the Prescription Drug User Fee Act, FDA has worked to develop a structured approach to the assessment of benefits and risks in drug regulatory decision-making to provide more clarity and transparency in this aspect of the drug review process. FDA envisions that this framework will serve as a template for USE IN product reviews, as well as a vehicle for explaining the basis for FDA’s regulatory decisions. This workshop began addressing opportunities to advance the development of more systematic and structured approaches to characterizing and communicating risks and benefits across the lifecycle of a drug. During the workshop FDA officials presented on on FDA’s work to develop their new risk/benefit framework and highlighted case studies where FDA was faced with uncertainty about the risk and benefits of treatments prior to a drug’s approval and how they approached this uncertainty.

In November of 2012 ACT-AD submitted comments to the FDA urging them to continue making Alzheimer’s disease a priority with respect to their activities to develop an enhanced risk/benefit framework. These comments were reinforced in May 2013 when ACT-AD weighed in on FDA’s draft risk/benefit plan. In these comments ACT-AD requested additional details on how early in the drug development process patient views will be included in conversations between the FDA’s product review divisions and new drug sponsors, the mechanisms FDA plans to use to garner patient and caregiver input, and the amount of information the Agency intends to solicit from patients regarding their attitudes on the risks and benefits of new drugs.

Recordings from the FDA workshop can be viewed here.

ACT-AD’s Comments on Risk-Benefit can be viewed at http://act-ad.org/activities/.

Accelerating Medicines Partnerships Launches to Speed Drug Development

On February 4, the National Institutes of Health (NIH), 10 biopharmaceutical companies, and several nonprofit organizations announced a new partnership to more quickly identify and validate biomarkers for the development of diagnostics and therapies. The Accelerating Medicines Partnership (AMP) begins as a pilot program and $230 million over five years will be invested into promising biological targets for new drug therapies for Alzheimer’s disease, type 2 diabetes, rheumatoid arthritis and lupus. For Alzheimer’s disease specifically, a number of research initiatives have already been identified.

The AMP’s innovative approach relies on collaboration and openness. The 10 participating companies will join with NIH scientists and nonprofit partners in sharing costs, expertise, and resources in regular discussions and meetings to determine which of their previously proprietary findings are most likely to lead to effective treatments, and make them public. Once public, all researchers are free to utilize the findings through normal competitive development pathways. If the pilot is successful, the hope is that it will be used to advance effective treatments for other conditions.

For more information on the partnership and its launch, click here.

Academy of Radiology Research Holds Conference on Brain Imaging Advances

On February 3, the Academy of Radiology Research hosted a one-day symposium at the National Institutes of Health where imaging researchers, neuroscientists, patient advocates and NIH program staff explored the potential imaging connections between three pressing national research topics: autism, Alzheimer’s disease and traumatic brain injury. This conference was an interdisciplinary forum where partners could discuss ways to leverage current imaging research investments in these three areas.
Carolyn Meltzer, MD, of Emory University, chaired a session on Alzheimer’s disease. During this session, attendees heard three presentations on the importance of imaging in unlocking the mysteries of this disease. The first presentation was from Satoshi Minoshima, MD, PhD, of the University of Washington, Seattle. Dr. Minoshima spoke about molecular imaging’s role in understanding the pathophysiology of Alzheimer’s disease and in treatment development. Jeffrey Patrella, MD, of Duke University Medical Center, addressed functional MRI’s role in Alzheimer’s disease research and the need to study connections between Alzheimer’s neuropathology and network abnormalities in certain cognitive deficits. Cynthia Bens from the Alliance for Aging Research represented the ACT-AD Coalition at this symposium and presented on work the patient advocacy community has been engaged in to focus attention of regulators and payors on the importance of imaging to Alzheimer’s disease drug development .

For the symposium program, click here.

For Ms. Bens’ slides, click here.

Beginning with the End in Mind – Study Endpoints: Targeting Patient-Centered Outcomes

Study endpoint measures in clinical trials determine what conclusions can be made about treatment benefit. Measure selection varies depending upon the phase of clinical development and the specific objectives a trial is expected to achieve. On March 18-20, the Drug Industry Association will host a conference titled, “Beginning with the End in Mind-Study Endpoints: Targeting Patient-Centered Outcomes” that addresses the need to balance measurement objectives with various drug development stakeholder interests. The program is co-chaired and organized by representatives of FDA’s Study, Endpoints and Labeling Division at the Center for Drug Evaluation and Research. The meeting will explore tradeoffs and various stakeholder perspectives for developing a study endpoint measurement strategy.

ACT-AD has been given the opportunity to provide a patient advocacy perspective on the “context of use” for drug development tools, and respond on behalf of the Alzheimer’s advocacy community about the development and application of these tools in the context of clinical trials. Cynthia Bens from the Alliance for Aging Research will represent the ACT-AD Coalition at this conference.

For more information on the conference, click here.