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U.S. Department of Health and Human Services

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Critical Path To Personalized Medicine

<< Return to FY 2007 Budget Summary




Why is this initiative necessary?

Despite many serious unmet needs for new medical treatments and significant investments in basic biomedical research, a new wave of medical products capable of saving and extending lives is not reaching patients (or the FDA). The pace of new discoveries moving from the laboratory to clinical settings is not accelerating. FDA has acrucial and unique role to play in changing this situation.

Under the FDA Modernization Act of 1997 (P.L. 105-115), Congress expanded the FDA mission to include the promotion of public health,not only through the timely and efficient review of clinical research, but also though collaborations with partners in government, academia, and industry to further medical product development.Since then,FDA has made significant progress in accelerating the timeliness of premarket product review. However, the number of applications for new innovative products reaching the FDA has not accelerated.

The productivity of the medical product industry — the sector responsible for turning new scientific discoveries into treatments — is low and not improving. Despite advances in discovery, a drug starting human trials in 2000 was no more likely to reach the market than one entering trials in 1985 (roughly an 8 percent chance). Moreover, for pharmaceuticals, the product failure rate in phase 3 trials has increased to nearly 50 percent.

To correct this deficiency, there is an urgent need to modernize the medical product development process - the Critical Path - to make product development more predictable and less costly. Without a parallel focus and investment in the science of product development, we will not reap a full return on our investment in biomedical discovery - in the form of new products for patients.

FDA's Critical Path initiative will stimulate industry-wide efforts to identify the essential biomarkers and improved clinical trial designs that will accelerate product development. The initiative will also generate the information that the health care community needs to identify patients likely to benefit from a treatment and patients more likely to respond adversely to a product. Without clinically proven biomarkers and innovative trial designs, we cannot modernize medical product development and realize the potential of personalized medicine.

FDA is the only entity that sees the entire spectrum of scientific hurdles to effective project development, industry-wide. Thus, we are best positioned to identify projects and advance collaborations needed to overcome product development bottlenecks and support personalized medicine.


Critical Path to Personalized Medicine Funding History

  FY 2005
FY 2006*
FY 2007
$   $0 +$5,940,000
FTE   - 6

* $743,000 in earmarked, external funding provided in FY 2006


How does this initiative support Executive Branch public health priorities?

Accelerating the movement of discoveries into products will have benefits across a wide spectrum of health care products. For example, the Critical Path investment will lead to more rapid development of products that prevent or treat pandemic influenza and products that treat illnesses caused by bioterror agents, two Administration priorities. New trial designs and clear guidelines for using efficacy and safety biomarkers in product development will increase the pace and reduce the cost of developing safe and effective medical products.

In addition, this initiative advances medical research by:

  • Enabling the development of key tools, such as clinically proven biomarkers necessary to make medications safer and more effective because they are chosen based on the patient's personal characteristics.
  • Supporting FDA and industry's ability to translate research results more quickly into benefits for patients.
  • Accomplishing these goals through interdisciplinary and interagency collaboration.


What are the risks of not proceeding with the initiative?

The consequences of not receiving funds for this initiative include:

  • Continued stagnation in the success rate for new products and the rate that we translate new scientific knowledge into health benefits.
  • An inability to achieve the benefits of molecular medicine and further delay in the personalize, predictive and preemptive health care outcomes it could bring.
  • Continued focus on "blockbusters" to pay for high failure rate and inadequate attention to products aimed at many serious and fatal diseases.


What activities will these funds support?

FDA will use these funds to:

  • Expand, stimulate and manage scientific partnerships and targeted research that will modernize medical product development. The goal is to stimulate a new generation of scientific tools to: 1) enable product sponsors to predict and evaluate the safety and effectiveness of candidate products, 2) make product development less risky, and 3) enable individualization of therapy to improve effectiveness and avoid side effects
  • Translate the science developed by this initiative into FDA guidance to manufacturers that clarify the regulatory path for bringing products to market.


What results will FDA achieve?

The projects supported by these funds will produce an array of outputs, ranging from new biomarkers to technical standards; from the creation of publicly available databases and computer modeling tools, to new FDA guidances to assist industry with drug development. Examples include:

  • Medical Imaging Initiative — In FY 2007 FDA will support clinical trials to validate the use of positron emission tomography (PET) imaging as a surrogate endpoint for developing new cancer drugs. This activity will be a collaboration with NCI, CMS and other partners. We will also expand collaboration with NCI on qualifying cancer imaging for drug development to include additional imaging technologies. FY 2007 deliverables also include publishing technical standards (e.g., instrument calibration and settings) necessary to reliably use these technologies in drug development. This is a collaborative venture with professional groups and other government agencies. The goal is to publish additional standards for additional technologies over several years. These tools will enable drug developers to improve the efficacy of cancer drugs.
  • Improving Cardiac Drug Eluting Stent Design and Use — FY 2007 deliverables include developing preliminary components of a simulation model of drug eluting stent behavior in adults and children. A stent is a small, flexible, spring-like device used to support artery walls. Drug eluting stents (also known as drug coated stents or medicated stents), are coated with a drug that interferes with arterial re-blocking. Also in FY 2007, we will work to develop open source imaging software to assess drug-eluting stent performance and begin to develop guidance for industry on using the simulation model to predict device performance. The purpose of this project is to improve product performance and safety by predicting and avoiding product failures.
  • ECG Warehouse: Improving Drug Safety — FY 2007 deliverables include completing analytic tools to permit searches of electrocardiogram (ECG) data in new drug submissions for patterns associated with unsafe drugs. Also in FY 2007, we will establish partnerships with academia and industry for additional data analyses. The purpose of this initiative is to improve the ability to identify cardiac safety concerns before a drug is approved for marketing. This activity will also support our ability to detect post market safety signals.
  • Other Outputs — FY 2007 deliverables include publishing concept papers and draft guidances for industry on the framework for qualifying new safety and efficacy biomarkers for drug development, such as genomic and proteomic assays.


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