​​Building Blocks to Further Precision Medicine

Cancer Progress Report 2015: Contents

In this section you will learn:

  • Increasing federal support for biomedical research is crucial to advancing precision medicine and making continued progress against cancer.
  • Regulatory science and policy play a key role in advancing precision medicine and making continued progress against cancer.
  • Federal support is needed to continue to develop and train the biomedical research workforce of tomorrow.
  • Patient and caregiver perspectives need to be considered as an integral part of advancing precision medicine.
  • Precision medicine can play an important role in the prediction and prevention of disease.

Thanks to the efforts of countless researchers across the entire biomedical research continuum, we have made great progress in our understanding of the molecular and genetic mechanisms underlying the collection of diseases that we call cancer, which in turn has made possible the development of new methods for preventing, detecting, diagnosing, and treating cancer.

In fact, in the 12 months between Aug. 1, 2014, and July 31, 2015, the FDA approved nine new anticancer therapeutics, one new cancer prevention vaccine, and one new cancer screening test (see Table 1​). During this period, the FDA also approved new uses for six previously approved anticancer therapeutics and one imaging agent.

This progress would not have been possible without federal support for the NIH, NCI, and FDA.

Nowhere is this progress more apparent than in the emerging field of precision medicine. At its very essence, precision medicine is treating patients based on the characteristics that distinguish that individual from other patients with the same disease, and the field of oncology has been leading the way in the development of precision treatments (see Treating Cancer More Precisely).

On Jan. 30, 2015, President Obama announced plans for a new Precision Medicine Initiative that would capitalize on the existing foundation of precision oncology, with the goal of extending precision medicine treatments to all forms of cancer and many other diseases. To make this a reality will require robust, sustained, and predictable funding increases for the NIH and NCI, who are leading this effort. Additionally, it is essential to develop mechanisms to involve patients more directly in the development of new treatments (see sidebar on Building Blocks to Further Precision Medicine​​).

Robust, Sustained, and Predictable Funding Increases for Biomedical Research

Federal grants from the NIH and NCI represent the lifeblood of biomedical research and form the foundation upon which the majority of scientific and medical discoveries are made. Bipartisan support for the NIH and NCI since President Nixon signed the National Cancer Act into law 44 years ago has resulted in extraordinary progress against cancer, as detailed in this report. In addition to saving lives, the federal investments in cancer research have also spurred our economy by creating jobs and establishing entirely new industries, such as the biotechnology industry.

Although prior federal investments in the NIH and NCI have led to significant progress, the purchasing power of the NIH has decreased by 25 percent since 2004 (see Figure 18​​). To regain momentum and accelerate the pace of progress, we must provide the NIH and NCI with the resources it needs to continue to fund lifesaving research.

Enhancing Support for Regulatory Science and Policy Activities at the FDA

The FDA represents an integral part of the biomedical research community (see sidebar on The Biomedical Research Community), and the support of this critical agency through government funding is crucial in continuing our progress against cancer and the delivery of safe, effective, and precise medicines to patients residing in the United States.

To keep pace with the rapid progress we are seeing in biomedical research, particularly in the area of precision medicine, the FDA is increasingly focused on advancing regulatory science, which is the study of developing new tools, standards, and approaches to assess the safety, efficacy, quality, validity, and performance of medical products. The regulatory science initiatives of the FDA are aimed at promoting and developing evidence-based regulatory policies that balance innovation and the expedited approval of medical products with patient safety (166).

It is imperative that the FDA is supported in its efforts to keep abreast of the latest scientific and technological progress through discourse, cooperation, and collaboration with academia, industry, patient advocacy groups, and the government.

Therefore, to achieve the aforementioned goals, Congress and the administration must equip the FDA with the resources the agency needs to support the regulatory processes and professional development of staff.

IncreasingPatient Participation in Precision Medicine Initiatives

The significant progress made in our understanding of the biology of cancer has led to the advent of precision medicine (see Figure 5​; Transforming Lives Through Precision Medicine). To fully realize the promise of precision medicine, patients should be equal partners in this initiativ​e. Patients are already involved in various aspects of biomedical research through their participation in activities including clinical trials and grant review, and as members of Institutional Review Boards (IRBs) and advisory committees. To make the development of new precision medicine treatments more patient-centric, we need to better understand how patients are engaged in their own treatment decisions.

Through its Patient-focused Drug Development initiative (169), the FDA has formally started gathering patient and caregiver perspectives related to their disease burden, impact on quality of life, and unmet needs. Efforts such as these, which better integrate the patient perspective into research, clinical trials, and the review and approval processes, should be encouraged so that patients can become equal partners in our joint quest for better treatments.

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Leadership in the Local and Global Economies

The impact of federal support for the NIH, NCI, and FDA reaches well beyond the research laboratory and the clinic. The medical research enterprise supports hundreds of thousands of jobs in communities across the United States, and for every dollar invested in the NIH, an estimated $2.21 is returned in the form of local economic growth (170). The discoveries that are fueled by these investments have led to the remarkable progress that we have seen in areas such as precision medicine. For example, the sequencing of the human genome has been hailed by many as the landmark scientific achievement of the 20th century. These types of breakthroughs are what has positioned the United States as the world leader in biomedical research. 

This leadership position, however, is in jeopardy as federal investments in biomedical research have been flat since 2004, while other countries are investing significantly in science, technology, and biomedical research (171). Thus, to maintain the pace of scientific progress for patient benefit, we must follow suit. Such a renewed investment in biomedical research would strengthen the position of the United States as the global leader in science and technology and would ensure U.S. economic leadership in the 21st century.

Developing and Training the Workforce of Tomorrow

Performing world-class research requires recruiting, training, and retaining the best minds across multiple disciplines, and this means providing adequate support in the form of training and research grants that will support individual researchers at all stages of their careers. Since 2003, stagnant funding for the NIH and NCI has resulted in a steady decline in the ability of these institutions to award research grants. In fact, in 2014, a grant application to the NCI had a one-in-seven chance of being funded, one of the lowest “success rates” on record for the NCI (172).

For researchers who are just beginning their careers, securing a federally funded grant directly is essential for their ability to continue a career in research. For more established researchers, failure to renew their grant funding can leave them unable to support new graduate students, postdoctoral scientists, and expert laboratory staff. This entire situation underscores the importance of providing robust, sustained, and predictable increases to the NIH and NCI.

Precision Prevention

It has been estimated that more than 50 percent of all U.S. cancer cases could be prevented through a combination of lifestyle modification and regular screening (173). Therefore, the best way to reduce death and disability due to these particular cancers is to take some of the necessary steps to prevent the disease from happening in the first place (see Preventing Cancer From Developing). Just as we are using precision medicine to determine which patients are likely to respond to which drugs, we are also becoming more sophisticated in using individual and population-wide data to predict the risk of a particular person developing cancer.

One of the best-known examples of using an individual’s genetic information to predict his or her chances of developing cancer is the connection between certain mutations in the BRCA1 and BRCA2 genes and hereditary breast and ovarian cancers. Behavioral and environmental data can also provide a wealth of information that can be used in cancer prevention and control. In addition, in those cases where the cause of a particular cancer is well understood, precise strategies aimed at reducing cancer risk can result in preventing cancer before it even starts. A good example of this is the HPV vaccine, which has been directly linked to reducing the risk of developing cervical cancer (see sidebar on How Do the Three FDA-approved HPV Vaccines Differ?​).

One of the biggest and most well-studied risk factors for cancer is tobacco use, and thanks to the wealth of research demonstrating the health consequences of tobacco use, we have seen dramatic progress in the area of tobacco control through the implementation of policies and educational initiatives aimed at preventing use and facilitating cessation (see Figure 9​). One relatively recent development is the increasing popularity of e-cigarettes and other electronic nicotine delivery systems (ENDS). Researchers are still working to understand the full impact of e-cigarettes on health (see sidebar on E-cigarettes: What We Know and What We Need to Know​​). However, a significant concern is that ENDS may be harmful, particularly to youth, if they increase the likelihood that nonsmokers or former smokers will use combustible tobacco products, or if they discourage smokers from quitting (41).

The prevention of skin cancer also remains a critical issue where significant additional progress can be made (see Protect Skin From UV Exposure​). The majority of skin cancers, including as many as 90 percent of melanomas, are caused by exposure to UV light (69). Despite the evidence implicating sun exposure and indoor ta​nning as risk factors for skin cancer, the incidence of melanoma in the United States continues to increase (6). To reverse this trend, policymakers must work on common-sense policies and educational campaigns that will help reduce an individual’s exposure to UV light, especially for those under 18 years of age, who are at the greatest risk for developing melanoma as a result of this exposure.

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