By Kathy A. Grako, Ph.D., PMP, CCRP, Senior Clinical Strategy Scientist
In February the United States government announced a unique triad brought together to “accelerate the development of life saving drugs and to help identify new treatments and cures for diseases” under the Accelerated Medicine Partnership (AMP). FDA and NIH have teamed up with 10 industry heavy weights (AbbVie, Biogen Idec, Bristol-Myers Squibb, GlaxoSmithKline, Johnson & Johnson, Lilly, Merck, Pfizer, Sanofi, and Takeda) and 8 non-profits (Alzheimer’s Association, American Diabetes Association, Lupus Foundation of America, Foundation for the NIH, Geoffrey Beene Foundation, PhRMA, Rheumatology Research Foundation, and USAgainstAlzheimer’s) to identify biological processes and molecules related to Alzheimer’s, Type 2 Diabetes, and two autoimmune disorders: rheumatoid arthritis and lupus. Everyone wants to find the perfect biomarker for their indication; however, the sponsors I’ve spoken with do not want to take the time nor bear the cost alone to find that biomarker. Clinical trials are expensive enough without adding on detection and validation of a biomarker. The FDA has made improving the clinical trial process a priority for both sponsors and most importantly for the subjects enrolled in the clinical studies. Over a 3‑5 year period $230 million in possible grant funding will be managed by the Foundation for the NIH for pilot projects in the 3 chosen disease indications. Most importantly, all of the AMP data and analyses will be publicly accessible.
According to the NIH website (www.NIH.gov/AMP) AMP members will develop partnerships and will share costs, expertise, and resources to identify and validate new biological targets for diagnostics and drugs. Research highlights for each of the pilot diseases include:
(Total project funds $129.5 million: NIH = $67.6 million and Industry = $61.9 million)
- Identify an expanded set of biomarkers to predict clinical outcomes and incorporate them into 4 major NIH-funded clinical trials.
- Conduct large-scale, system biology analysis of human Alzheimer’s patient brain tissue samples to validate biological targets with key roles in disease progression, and increase understanding of molecular networks involved and identify new potential therapeutic targets.
Type 2 Diabetes
(Total project funds $58.4 million: NIH = $30.4 million and Industry = $28 million)
- Build a knowledge portal of DNA sequence, functional genomic and epigenomic information, and clinical data from studies on type 2 diabetes including heart and kidney complications. The portal will include existing and new data from 100,000-150,000 individuals and provide an opportunity to identify promising new therapeutic targets for diabetes.
- Focus on DNA regions thought critical for development or progression of the disease and search for natural variations for possible use in predicting drug development successes in targeted populations
Rheumatoid Arthritis and Lupus
(Total project funds $41.6 million: NIH = $20.9 million and Industry = $20.7 million)
- Collect and analyze tissue and blood samples from people with rheumatoid arthritis and lupus to focus on biological changes at the single cell level, to allow comparisons across diseases, and determine insights into key aspects of the disease process.
- Identify differences between rheumatoid arthritis patients who respond to current therapies and those who do not, and find a better systems-level understanding of disease mechanisms in rheumatoid arthritis and lupus.
The outcome for this unique open collaboration to identify and validate clinically relevant biomarkers are planned to be (1) an increased efficiency leading to shorter development time and improved rates of success leading to lower costs; (2) improving the process by identifying and understanding targets and biomarkers resulting in improved clinical trials; and (3) an increased number of more targeted therapies with improved efficacy because of a better understanding of disease biological pathways which in turn will decrease the number of failed Phase 2 and Phase 3 clinical trials and ultimately a more rational drug design with better targeted therapies.
If all goes as projected, the AMP program will be of great benefit and translate across multiple indications. We should all watch their progress and particularly the amount and quality of the publicly available data and the impact it should have on both research and development and clinical trial conduct over the upcoming years.
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