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U.S. Department of Health and Human Services

Science & Research

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I. Accelerating the Delivery of New Medical Treatments to Patients

Table of Contents: Advancing Regulatory Science for Public Health

Recent U.S. investments in biomedical research have dramatically expanded our understanding of biology and disease. Still, the development of new therapies is in decline, and the cost of bringing them to market has soared. In short, we must take advantage of every opportunity to improve the effectiveness and outcomes of healthcare and address growing threats to the strength and innovation of the U.S. biotechnology industry in order to ensure that the best medical treatments are made available to patients in a timely fashion.

What has FDA done?

As a result of the successes of the Critical Path Initiative and other projects, FDA scientists have been instrumental in a number of regulatory science achievements aimed at increasing the practical value of basic discoveries.  This is done to ensure that patients have access to the most cutting-edge medical treatment possible.  By leveraging existing resources and harnessing the vast knowledge of scientists in its Centers, FDA has established itself as an integral part of the healthcare paradigm.  Some of the aforementioned achievements are highlighted below:

• Methods for stem cell characterization.
In collaboration with other scientists, FDA scientists developed a method and identified gene biomarkers for “stemness”— that is, for the extent of differentiation in several lines of stem cells. The scientists developed tests and standards to establish the presence of different types of stem cells. These methods can now be used to ensure that undifferentiated stem cells, which may increase the risk of stem cell treatments, do not contaminate final, more differentiated stem cell products that are administered to patients. (Bhattacharya, et al, Blood 2004. 103:2956-2964.)

• Personalized treatment for cancer
The "I-SPY 2 TRIAL," launched in March 2010, represents a groundbreaking new clinical trial model that will help scientists quickly and efficiently test the most promising drugs in development for women with higher risk, rapidly growing breast cancers.   During the trial, drugs in development are individually targeted to the biology of each woman’s tumor using specific genetic or biological markers, known as “biomarkers.”  By applying an innovative trial design, researchers will use data from one set of patients’ treatments to treat other patients – more quickly eliminating ineffective treatments and drugs.  The I-SPY 2 trial was developed under the Biomarkers Consortium, a unique public-private partnership that includes the FDA, the National Institutes of Health, and major pharmaceutical companies, led by the Foundation for the National Institutes of Health.

What can FDA do with increased investment in regulatory science?

Although developments in science and technology hold great potential, the ways in which new therapies are developed and tested remain underdeveloped and underappreciated. As FDA’s independent Science Board found: “American lives are at risk” because

While the world of drug discovery and development has undergone revolutionary change — shifting from cellular to molecular and gene-based approaches — FDA’s evaluation methods have remained largely unchanged over the last half-century. 1  

Without advances in regulatory science, promising medical therapies may be discarded during the developmental process simply because we lack the tools to recognize their potential, or outdated evaluation methods may unnecessarily delay their approval. Conversely, countless dollars and years may be wasted assessing a novel therapy that is later shown to be unsafe or ineffective.

With creative advances in regulatory science, we can change the landscape entirely. We can modernize product development and develop new tools, standards, assays, disease models and science-based pathways to improve the speed, efficiency, predictability, capacity and quality of the entire process, from development to evaluation to manufacturing. Here are some areas in which FDA can make a lasting impact:

• Safer pain medications 
We are facing a global epidemic of prescription pain medicine abuse and misuse. At the same time, patients in agonizing pain are often left undertreated. New pain pathways have been discovered and new medicines are being developed that can help. But to accelerate the delivery of new treatments to patients, we need to find better pain models, measurement tools (including patient-reported assessments) and clinical trial designs to enable development of effective medications with less potential for abuse.

• Vaccines, drugs and diagnostics for tuberculosis 
FDA is supporting exciting research into treatments to prevent, diagnose and treat drug resistant tuberculosis (TB) and to develop TB vaccines, needed to protect millions of people everywhere, including U.S. citizens, travelers and troops. We must develop approaches that can more quickly evaluate TB treatments and vaccines, including identifying markers that can predict cure and protection. We must also develop tests that can more rapidly and accurately diagnose TB, including those that determine when TB is resistant to certain treatments.

• Greater availability of generic drugs
Generic drugs make up more than 70 percent of the prescriptions filled in this country and, for many, are the only solution to affordable treatment. Still, many products do not have generic alternatives even though patents for the reference products have expired. More generic products could be made available if the difficulty in determining bioequivalence for some products could be overcome, but metered-dose inhalers, dry-powder inhalers, certain topical products and products that are not systemically absorbed present challenges in determining bioequivalence. We must enhance our research into developing validated methods for determining bioequivalence for these products so that quality, lower-cost generic products can become more widely available.

• Modernized manufacturing and product quality
FDA is leading efforts on “Quality by Design (QbD),” which applies regulatory science to modernize the understanding and control of medical product manufacturing processes. Advances in regulatory science will not only ensure better quality, but could also lower development and manufacturing costs. Areas of investigation supported by FDA include (1) continuous processing, in which materials constantly flow in and out of the equipment and reduce overall manufacturing time and cost; (2) the use of process analytical technology (PAT) to monitor and control manufacturing processes as opposed to just testing products; and (3) new statistical approaches to detect changes in process or product quality. Applying these approaches will help control complex manufacturing processes, enhance their efficiency and provide more reliable products to patients. In addition, new technologies such as flexible manufacturing facilities and the use of modular and disposable equipment can speed production of products in routine and emergency situations.

There is much at stake for public health.  FDA will continue to strengthen mission-critical science within the agency while exploring new and exciting partnerships with other government entities, industry, and academia with the intent of transforming lives and safeguarding the public’s health.


1  FDA Science Board, FDA Science and Mission at Risk, Report of the Subcommittee on Science and Technology, November 2007.

Next Section: Improving Pediatric and Child Health