Overview

Launched in 2014, the Accelerating Medicines Partnership® (AMP®) program is a public-private partnership between the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), multiple biopharmaceutical and life science companies, non-profit and other organizations to transform the current model for developing new diagnostics and treatments. Current AMP projects include:

• Alzheimer’s disease (AD 1.0 Biomarkers in Clinical Trials and AD 2.0)
• Autoimmune and Immune-Mediated Diseases
• Autoimmune disorders of rheumatoid arthritis and systemic lupus erythematosus (lupus) (RA/Lupus)
• Bespoke Gene Therapy Consortium (BGTC)
• Common Metabolic Diseases (CMD)
• Heart Failure
• Parkinson’s disease (PD)
• Schizophrenia (SCZ)

AMP partners share a common goal of increasing the number of new diagnostics and therapies for patients and reduce the time and cost of developing them. The AMP program aims to improve understanding of therapeutically relevant biological pathways and validate information that could be relevant for the development of multiple therapeutics.

Through this cross-sector partnership, managed through the Foundation for the NIH (FNIH), NIH and AMP partners are sharing expertise and resources — over $830 million to date, which includes in kind contributions — in an integrated governance structure that enables the best-informed contributions to science from all participants.

Current AMP Partners

Timeline

The Challenge

As a result of technological revolutions in genomics, proteomics, imaging, and more, researchers have been able to identify changes in genes, proteins, and other molecules that cause disease and influence disease progression. Clinicians use this information to determine the presence of disease through biomarkers with the use of diagnostic tests. Biopharmaceutical companies use it to develop therapies that target these specific genes and molecules to interfere with their processes and modify the course of disease.

While technological advances have produced a wealth of data on the biological cause of disease, moving these discoveries into treatments has been far more difficult. For some diseases, the challenge is identifying which biological insights will lead to effective drug targets, when choosing the wrong target can result in failures late in the drug development process, costing time, money, and ultimately, lives. For other diseases — such as very rare diseases — designing a treatment like a gene therapy that is capable of modifying the course of a disease is straightforward, but moving that potential treatment from laboratory testing to human trials is prohibitively difficult. Developing a new drug — from early discovery through Food and Drug Administration (FDA) approval — takes well over a decade and has a failure rate of more than 95 percent. Consequently, each success costs more than $1 billion. The most expensive failures happen in late phase clinical trials, with a lack of drug efficacy currently estimated as responsible for 59 percent of Phase II failures and 52 percent of Phase III failures.¹ Therefore, it’s essential to do a better job of pinpointing the right biological targets early in the process.

The entire biomedical research community and the public have a shared interest in compressing the timelines, reducing the costs, and increasing the success rates of new targeted therapies. Given the amount and complexity of the data, this goal will require a systematic approach in which government, academia, industry, and patient groups work collaboratively.

¹J Arrowsmith, P Miller. Trial Watch: Phase II and Phase II attrition rates 2011-2012. Nat. Rev. Drug Discov, 12, 569, (2013).

The Impact

By optimizing the process for identifying and validating clinically relevant disease targets for drug design, AMP aims to:

Increase efficiency:

• Shorter development time: accelerating the hard work of sifting through a large number of candidates to identify the best biological targets for drug development could shave months or even years off of the early stages of discovery.
• Improved prospects for success: with disease targets and biomarkers that have been validated rigorously with human data, higher confidence about efficacy should be achieved, allowing researchers to move the most promising compounds quickly into the pipeline with the expectation of fewer failures in late stage clinical trials.
• Lower costs: shorter development timeframes and fewer late-stage drug failures should reduce the cost of delivering new and effective medicines to patients.

Improve the process:

• Better understanding of biological targets and identification of valid biomarkers will enable more robust clinical trials — in part by testing therapies on patients most likely to respond to them based on the molecular profiles of their disease.

Increase the number and effectiveness of new targeted therapies:

• Understanding the biological pathways underlying disease and the specific biological targets that can alter disease will lead to more rational drug design and better tailored therapies.
• Reducing the number of failures in Phase II and Phase III clinical trials will increase the number of new drugs developed.
• The increase in expected returns will likely make drug development a more attractive investment.

Governance

Steering committees for each of the projects, with representation from the partners participating in those projects, meet regularly to review ongoing progress and milestones. The steering committees are managed by FNIH under the direction of an AMP Executive Committee comprised of representatives from NIH, participating industry leaders, FDA, European Medicines Agency, and non-profit and other organizations.

ACCELERATING MEDICINES PARTNERSHIP and AMP are registered service marks of the U.S. Department of Health and Human Services.