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Suggested Citation:"Summary." National Academies of Sciences, Engineering, and Medicine. 2017. Preparing for Future Products of Biotechnology. Washington, DC: The National Academies Press. doi: 10.17226/24605.
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Summary

In July 2015, the Office of Science and Technology Policy in the Executive Office of the President initiated an effort to modernize the U.S. regulatory system for biotechnology products consisting of three primary activities:

  1. Development of an update to the Coordinated Framework for Regulation of Biotechnology (referred to hereafter as the Coordinated Framework) to clarify the roles and responsibilities of the agencies that regulate the products of biotechnology;
  2. Formulation of a long-term strategy to ensure that the federal regulatory system is equipped to efficiently assess the risks, if any, associated with future products of biotechnology while supporting innovation, protecting health and the environment, promoting public confidence in the regulatory process, increasing transparency and predictability, and reducing unnecessary costs and burdens; and
  3. Commission of an external, independent analysis of the future landscape of biotechnology products with a primary focus on potential new risks and risk-assessment frameworks.

With regard to the third item, the U.S. Environmental Protection Agency (EPA), the U.S. Food and Drug Administration (FDA), and the U.S. Department of Agriculture (USDA) were charged to

Commission an external, independent analysis of the future landscape of biotechnology products that will identify (1) potential new risks and frameworks for risk assessment and (2) areas in which the risks or lack of risks relating to the products of biotechnology are well understood. The intent of this review is to help inform future policy making. It is also anticipated that due to the rapid pace of change in this arena, an external analysis would be completed at least every 5 years.1

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1 Executive Office of the President. 2015. Memorandum for Heads of Food and Drug Administration, Environmental Protection Agency and Department of Agriculture. July 2. Available at https://obamawhitehouse.archives.gov/sites/default/files/microsites/ostp/modernizing_the_reg_system_for_biotech_products_memo_final.pdf. Accessed January 31, 2017.

Suggested Citation:"Summary." National Academies of Sciences, Engineering, and Medicine. 2017. Preparing for Future Products of Biotechnology. Washington, DC: The National Academies Press. doi: 10.17226/24605.
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To accomplish this directive, the three regulatory agencies asked the National Academies of Sciences, Engineering, and Medicine to convene a committee of experts to conduct the study “Future Biotechnology Products and Opportunities to Enhance Capabilities of the Biotechnology Regulatory System.” Committee members were selected because of the relevance of their experience and knowledge to the study’s specific statement of task (Box S-1), and their appointments were approved by the President of the National Academy of Sciences in early 2016.

THE COMMITTEE’S PROCESS

To address its statement of task, the Committee on Future Biotechnology Products and Opportunities to Enhance Capabilities of the Biotechnology Regulatory System spent several months gathering information from a number of sources. It heard from 74 speakers over the course of three in-person meetings and eight webinars and received responses to a request for information from a dozen federal agencies. It also solicited statements from members of the public at its in-person meetings and accepted written comments through the duration of the study. The committee also made use of several recent National Academies studies related to future products of biotechnology, particularly Industrialization of Biology: A Roadmap to Accelerate the Advanced Manufacturing of Chemicals,2Gene Drives on the Horizon: Advancing Science, Navigating Uncertainty, and Aligning Research with Public Values,3 and Genetically Engineered Crops: Experiences and

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2 NRC (National Research Council). 2015. Industrialization of Biology: A Roadmap to Accelerate the Advanced Manufacturing of Chemicals. Washington, DC: The National Academies Press.

3 NASEM (National Academies of Sciences, Engineering, and Medicine). 2016. Gene Drives on the Horizon: Advancing Science, Navigating Uncertainty, and Aligning Research with Public Values. Washington, DC: The National Academies Press.

Suggested Citation:"Summary." National Academies of Sciences, Engineering, and Medicine. 2017. Preparing for Future Products of Biotechnology. Washington, DC: The National Academies Press. doi: 10.17226/24605.
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Prospects.4 The committee reviewed these reports and reflected on their recommendations related to the Coordinated Framework, with the aim of understanding how those prior recommendations fit with the broader view of biotechnology products in this report and the opportunities to enhance the capabilities of the biotechnology regulatory system. For its purposes, the committee defined biotechnology products as products developed through genetic engineering or genome engineering (including products where the engineered DNA molecule is itself the “product,” as in an engineered molecule used as a DNA information-storage medium) or the targeted or in vitro manipulation of genetic information of organisms, including plants, animals, and microbes. The term also covers some products produced by such plants, animals, microbes, and cell-free systems or products derived from all of the above.

FUTURE BIOTECHNOLOGY PRODUCTS

The committee was charged to describe biotechnology products likely to emerge in the next 5–10 years. The committee scanned the horizon for new products by inviting product developers to speak at the various meetings; reviewing submitted public comments; reading scientific literature, popular press reports, and patents; consulting previous reports by the National Academies; searching publicly available projects developed by international Genetically Engineered Machine teams;5 and checking information available on regulatory agencies’ websites and crowdfunding websites. It also made use of the Synthetic Biology Database6 curated by the Woodrow Wilson Center. Based on this exercise, the committee anticipates that the scope, scale (number of products and variants thereof), and complexity of future biotechnology products may be substantially different from products developed as of 2016.

The committee grouped future products into three major classes: open-release products, contained products, and platforms. Table S-1 summarizes types of open-release products that the committee saw on the horizon, that is, plants, animals, microbes, and synthetic organisms that have been engineered for deliberate release in an open environment. The ability to sustain existence in the environment with little or no human intervention is a key change between existing products of biotechnology and some of the future ones anticipated in this class. Furthermore, the types of environments in which a product may persist are likely to become more diverse. Plants and insects may be designed to continue in low-management systems such as forests, pastures, and cityscapes; microbes may be developed to persist in those environments as well as in mines, waterways, and animal guts. The committee thought that future open-release products would be developed for familiar uses, such as agricultural crops, but would also likely be developed for uses such as cleaning up contaminated sites with engineered microbes, replacing animal-derived meat with meat cultured from animal cells, and controlling invasive species through gene drives.7

On the basis of its information-gathering efforts, the committee concluded that future biotechnology products that are produced in contained environments are more likely to be microbial based or synthetically based rather than based on an animal or plant host (Table S-2). Organisms of many genera are used in fermenters to produce commodity chemicals, fuels, specialty chemicals

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4 NASEM. 2016. Genetically Engineered Crops: Experiences and Prospects. Washington, DC: The National Academies Press.

5 See team list for iGEM championship. Available at http://igem.org/Team_List?year=2016&name=Championship&division=igem. Accessed February 12, 2017.

6 Synthetic Biology Products and Applications Inventory. Available at http://www.synbioproject.org/cpi. Accessed October 11, 2016.

7 A gene drive is a system of biased inheritance in which the ability of a genetic element to pass from a parent to its offspring through sexual reproduction is enhanced. Thus, the result of a gene drive is the preferential increase of a specific genotype, the genetic makeup of an organism that determines a specific phenotype (trait), from one generation to the next, and potentially throughout the population.

Suggested Citation:"Summary." National Academies of Sciences, Engineering, and Medicine. 2017. Preparing for Future Products of Biotechnology. Washington, DC: The National Academies Press. doi: 10.17226/24605.
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TABLE S-1 Market Status of Products Designed for Open Release in the Environmenta

Product Description On Marketb Under Developmentc Early-Stage Concept
Plants and Plant Products Bt crops with recombinant DNAd (rDNA)
Herbicide-resistant crops with rDNA
Disease-resistant crops with rDNA
RNAie modified crops ✓✓✓ ✓✓✓
Fragrant moss
Do-it-yourself glowing plants
Genome-editedf crops ✓✓✓ ✓✓✓
Crops with CRISPRg knockouts ✓✓✓ ✓✓✓
Grasses for phytoremediation
Plants as sentinels
Crops with increased photosynthesis efficiency
Ever-blooming plants
Nitrogen-fixing nonleguminous plants
Bioluminescent trees
Plants with gene drives for conservation purposes
Plants with gene drives for agricultural purposes
Animals and Animal Products Fluorescent zebra fish
Sterile insects
Genome-edited animals (e.g., polled cattle)
Reduced-allergen goat’s milk
Landmine-detecting mice
Animals revived from near extinction or extinction
Animals with gene drives for control of invasive mammals
Animals with gene drives for control of insect pests
Microbes and Microbial Products Biosensors/bioreporters
Bioremediation
Engineered algal strains ✓✓✓
Nitrogen-fixing symbionts
Probiotics
Genomically engineered microbial communities ✓✓✓
Biomining/bioleaching ✓✓✓
Synthetic Organisms/Nucleic Acids Cell-free products
DNA barcodes to track products
RNA-based spray for insect-pest control
Genomically recoded organisms
Biological/mechanical hybrid biosensors

✓✓✓ = an area the committee has identified as having high growth potential.

aThe table reflects the market status of products at the time the committee was writing the report.

b“On Market” is equivalent to “in use”; thus, products that have received regulatory approval but are not in use were not considered by the committee to be “On Market.”

c“Under development” spans products from the prototype stage to field trials.

dRecombinant DNA is a novel DNA sequence created by joining DNA molecules that are not found together in nature.

eRNAi or RNA interference is a natural mechanism found in nearly all organisms in which the levels of transcripts are reduced or suppressed and can be exploited with biotechnology to modify an organism.

fGenome editing is a specific modification of the DNA of an organism to create mutations or introduce new alleles or new genes.

gCRISPR or clustered regularly interspaced short palindromic repeat is a naturally occurring mechanism of immunity to viruses found in bacteria that involves identification and degradation of foreign DNA. This natural mechanism has been manipulated by researchers to develop genome-editing techniques.

Suggested Citation:"Summary." National Academies of Sciences, Engineering, and Medicine. 2017. Preparing for Future Products of Biotechnology. Washington, DC: The National Academies Press. doi: 10.17226/24605.
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TABLE S-2 Market Status of Contained Productsa

Product Description On Marketb Under Developmentc Early-Stage Concept
Animals/Plants and Animal/Plant Products Transgenic laboratory animals (mini-swine, mice, rats, dogs) ✓✓✓
Genetically engineered salmon grown in land-based facilities
Animal cell culture–derived products (e.g., cowless leather and cowless meat) ✓✓✓ ✓✓✓
Polymers produced by plants for industrial use
Greenhouse crops with CRISPR knockouts
Microbes and Microbial Products Industrial enzymes
Biobased chemicals to replace fossil fuel feedstocks
Bioluminescent microbes for home and landscape uses
Yeast-derived molecules to create products (e.g., vanillin, stevia, saffron, egg whites, milk protein, gelatin) ✓✓✓ ✓✓✓
Synthetic silk
Bacterium-derived antimicrobials
Genomically engineered bacterial strains for fermentation-based products ✓✓✓
Gas-phase microbial systems
Algae-derived products (e.g., substitute for shark fins and shrimp, biofuels, ethylene) ✓✓✓ ✓✓✓
Probiotics
Leaching/metal recycling organisms
Synthetic Organisms/Nucleic Acids Organ-on-a-chip
V. natriegens platform
Genomically recoded organisms ✓✓✓ ✓✓✓
Cell-free ex ression s stems ✓✓✓ ✓✓✓
Biological–mechanical hybrid biosensor
Implantable biosensors

✓✓✓ = an area the committee has identified as having high growth potential.

aThe table reflects the market status of products at the time the committee was writing the report.

b“On Market” is equivalent to “in use”; thus, products that have received regulatory approval but are not in use were not considered by the committee to be “On Market.”

c“Under development” spans products from the prototype stage to field trials.

or intermediates, enzymes, polymers, food additives, and flavors. When considering the laboratory as a contained environment, many examples of transgenic animals from vendors are widely used today for research and development. Because performing biotechnology in contained environments allows higher control over the choice of host organism, systems with advanced molecular toolboxes are already in high use.

Biotechnology platforms are tools that are used in the creation of other biotechnology products. They include products that are traditionally characterized as “wet lab,” such as DNA/RNA, enzymes, vectors, cloning kits, cells, library prep kits, and sequencing prep kits, and products that are “dry lab,” such as vector drawing software, computer-aided design software, primer calculation software, and informatics tools. These two categories continue to meld as newer approaches are published or commercialized.

There are a variety of technical, economic, and social trends that are driving and that will con-

Suggested Citation:"Summary." National Academies of Sciences, Engineering, and Medicine. 2017. Preparing for Future Products of Biotechnology. Washington, DC: The National Academies Press. doi: 10.17226/24605.
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tinue to drive the types of biotechnology products developed in the next decade. Technical and economic trends in the biological sciences and biological engineering are accelerating the rate at which new product ideas are formulated and the number of actors who are involved in product development. With regard to social trends, it was evident to the committee through its information-gathering activities and the mechanisms for public comment that there are many competing interests, risks, and benefits regarding future biotechnology products; it was also clear that the United States and international regulatory systems will need to achieve a balance among these competing aspects when considering how to manage the development and use of new biotechnology products. Many sectors of society have concerns over the safety and ethics of various biotechnologies, whereas others see prospects for biotechnology to address challenging social and environmental issues. Biotechnology products that are on the horizon are likely to generate substantial public debate. For example, gene-drive technology, for which there have already been numerous studies and reports regarding its use, is a technological advance that will increase the amount of public debate and for which society will have to take a balanced approach among the interested and affected parties, developers, and scientists.

THE BIOTECHNOLOGY REGULATORY PROCESS AND THE COORDINATED FRAMEWORK

The committee was asked to describe the existing risk-analysis system for biotechnology products and to describe each agency’s authorities as they pertain to the products of biotechnology. In order to carry out these portions of its statement of task, the committee reviewed the regulatory authorities that apply to biotechnology products.

The committee found that the Coordinated Framework appears to have considerable flexibility in statutory authority to cover a wide range of biotechnology products. In some cases, however, the jurisdictions of EPA, FDA, and USDA are defined in ways that may leave gaps or redundancies in regulatory oversight. Even when jurisdiction exists, the available legal authorities may not be ideally tailored to new and emerging biotechnology products. Furthermore, agencies other than EPA, FDA, and USDA will likely have responsibilities to regulate some future biotechnology products, and their roles are not well specified in the Coordinated Framework.

Despite the flexibility of the Coordinated Framework to cover a wide range of biotechnology products, the committee also found that the existing biotechnology regulatory system is complex and could be considered to appear fragmented, resulting in a system that is difficult for product developers—including individuals, nontraditional organizations, and small enterprises—as well as consumers, product users, and interested members of the public to navigate. This complexity can cause uncertainty and a lack of predictability for developers of future biotechnology products and creates the potential for loss of public confidence in oversight of future biotechnology products.

The increased rate of new product ideas means that the types and number of biotechnology products in the next 5–10 years may be significantly larger than the current rate of product introduction. EPA, FDA, USDA, and other relevant agencies will need to be prepared for this potential increase, including finding effective means of evaluation that maintains public safety, protects the environment, and satisfies the statutory requirements appropriate for each agency. The increased number of actors who are involved in product development means that the regulatory agencies will need to be prepared to provide information regarding the regulatory process to groups that may have little familiarity with the Coordinated Framework. This group of actors may include small- and medium-sized enterprises, do-it-yourself (DIY) bioengineers, or developers supported by crowdfunded activities with direct-to-consumer distribution models and the potential for domestic manufacturing.

Suggested Citation:"Summary." National Academies of Sciences, Engineering, and Medicine. 2017. Preparing for Future Products of Biotechnology. Washington, DC: The National Academies Press. doi: 10.17226/24605.
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UNDERSTANDING RISKS RELATED TO FUTURE BIOTECHNOLOGY PRODUCTS

The committee was asked to determine whether future products could pose different types of risks relative to existing products and organisms. In all the types of products summarized above, advances in biotechnology are leading to products that involve the transformation of less familiar host organisms, have multiple engineered pathways, are comprised of DNA from multiple organisms, or are made from entirely synthetic DNA. Such products may have few or no comparators8 to existing nonbiotechnology products, which function as the baseline of comparison in current regulatory risk assessments of biotechnology products. Figure S-1 summarizes the progression in terms of complexity and novelty that the committee thought was likely in future biotechnology products over the next 5–10 years. Products that fit in column A are those similar to existing biotechnology products evaluated under the existing Coordinated Framework and for which current methods of risk assessment can be applied. Examples include new genetically engineered crops and fermentation-based production of small molecules, enzymes, or other biochemicals. Products described by column B are those that represent an expansion of the familiar set of organismal hosts and genetic pathways, for which there are few comparators but nonetheless well-established approaches to assessing risk. Examples include animal cell culture–derived products (such as cowless meat or leather) and plants for bioremediation, decoration, or other environmental or consumer use. Products in column C are those that are currently at the forefront of research activities, where the use of rapid design-build-test-learn cycles allows much more complex designs of genetic pathways in a wider variety of host organisms, but which also represent more sophisticated uses of products, such as open release into the environment of organisms intended to modify populations of natural organisms. Examples include genetically engineered mosquitoes for fighting malaria or the Zika virus, genomically engineered microorganisms, and implantable biosensors. Such products are on the horizon, but at the time the committee was writing its report, most had not yet entered the biotechnology regulatory system. The few that had entered the system had few or no nonbiotechnology products to which they could be compared, and, as they were first-of-their-kind products, no previous biotechnology product had established a path to follow through the regulatory system. Finally, products in column D represent those in which multiple organisms may be used in complex microbial communities, such as microbiome engineering and synthetic consortia for bioremediation or biomining applications. These products also have no comparators (or the relevance of potential comparators is ambiguous) and no established regulatory path.

For future biotechnology products in all degrees of complexity and novelty, the committee considered the risk-assessment endpoints related to human health or environmental outcomes, such as illness, injury, death, or loss of ecosystem function. It concluded that the endpoints are not new compared with those that have been identified for existing biotechnology products, but the intermediate steps along the paths to those endpoints have the potential to be more complex, more ambiguous, and less well characterized. In addition, the committee found that the scope, scale, complexity, and tempo of biotechnology products that are likely to enter the regulatory system in the next 5–10 years have the potential to critically stress the regulatory agencies, both in terms of capacity and expertise. Furthermore, many early-stage developers of biotechnology products or biological technology that may lead to products do not currently consider regulatory perspectives or future requirements during technology (and sometimes product) development, which has the potential to complicate the evaluation of risks associated with the release of future biotechnology products. It will clearly be important for EPA, FDA, USDA, and other agencies relevant to the future regulation of biotechnology products to maintain an assessment of the scope of these products and be prepared to evaluate them as they are submitted for regulatory assessment.

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8 The term comparator refers to a known nonbiotechnology organism that is similar to the engineered organism except for the engineered trait.

Suggested Citation:"Summary." National Academies of Sciences, Engineering, and Medicine. 2017. Preparing for Future Products of Biotechnology. Washington, DC: The National Academies Press. doi: 10.17226/24605.
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FIGURE S-1 Characteristics of future biotechnology products, organized by similar levels of complexity in terms of types and number of organisms, genes and traits, and comparators involved.
NOTE: Products of biotechnology can be conceptualized as fitting into the depicted columns with the indicated characteristics, moving toward column D as a product increases in complexity and likelihood of providing new challenges for risk assessment.

OPPORTUNITIES FOR ENHANCEMENT OF THE BIOTECHNOLOGY REGULATORY SYSTEM

A major task of the committee was to indicate what scientific capabilities, tools, and expertise may be useful to the regulatory agencies to support oversight of future products of biotechnology. The committee requested information from federal agencies regarding current investments in regulatory science.9

At a high level, the committee found that there are existing frameworks, tools, and processes for risk analyses and public engagement that can be used to address the many issues that are likely to arise in future biotechnology products in a way that balances competing issues and concerns. However, given the profusion of biotechnology products that are on the horizon, there is a risk that the capacity of the regulatory agencies may not be able to efficiently provide the quantity and quality of risk assessments that will be needed. An important approach for dealing with an increase in the products of biotechnology will be the increased use of stratified approaches to regulation, where new and potentially more complex risk-analysis methods will need to be developed for some

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9 As discussed in Chapter 4, on the basis of definitions provided by FDA and the Society for Risk Analysis, the committee understood regulatory science to involve developing and implementing risk-analysis methods and maximizing the utility of risk analyses to inform regulatory decisions for biotechnology products, consistent with human health and environmental risk–benefit standards provided in relevant statutes.

Suggested Citation:"Summary." National Academies of Sciences, Engineering, and Medicine. 2017. Preparing for Future Products of Biotechnology. Washington, DC: The National Academies Press. doi: 10.17226/24605.
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products, while established risk-analysis methods can be applied or modified to address products that are familiar or that require less complex risk analysis. With this approach, new risk-analysis methods are focused on products with less familiar characteristics and/or more complex risk pathways. Multiple criteria are usually embedded within risk analyses to ascertain if an estimated level of risk is consistent with the risk-management goals established during the problem-formulation phase of a risk assessment. In some cases, additional risk analyses may be needed to refine risk estimates, to evaluate risk-mitigation measures, or both. In order to implement the appropriate rigor of risk analyses for new biotechnology products, it will be necessary to establish scientifically rigorous criteria based on factors affecting the perception of risk, the degree of uncertainty, and the magnitude of risk and nature of potential risks.

To help articulate what capabilities, tools, and expertise might be useful to meet these objectives, the committee created a conceptual map for decision making aimed to assess and manage product risk, streamline regulation requirements, and increase transparency, as shown in Figure S-2.

As envisioned by the committee, a single point of entry (illustrated in Figure S-2) could be used by a product developer to evaluate whether the intended use of the product is regulated under

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FIGURE S-2 Providing access to the U.S. regulatory system through a single point of entry.
NOTES: Potential product developers and interested parties would begin by going to an entry point and providing characteristics of the intended product and its use pattern. If the product does not fall under a federal statute, the developer would be notified that the product is not federally regulated. If the product is regulated, the appropriate agency or agencies would be identified for the developer. An evaluation of the product’s familiarity to regulatory agencies and its complexity in terms of risk analyses as compared to existing biotechnology products would be ascertained (red bins). Depending on the product’s familiarity and the complexity of its risk analysis, a different set of risk-analysis processes would be employed (blue boxes). For products that are familiar to the regulatory agencies and are not complex, a more expedited process could be used under the assumption that relevant risk-analysis processes are well established. For products that are less familiar, more complex, or less familiar and more complex, increasingly unique risk-analysis processes (that incorporate additional external input) may need to be established.
Suggested Citation:"Summary." National Academies of Sciences, Engineering, and Medicine. 2017. Preparing for Future Products of Biotechnology. Washington, DC: The National Academies Press. doi: 10.17226/24605.
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a given statute and provide a determination of whether the product is familiar and not complex, is unfamiliar or complex, or is unfamiliar and complex compared to existing biotechnology products. Once a determination has been made, the appropriate processes within the relevant agency (or agencies) would be used to provide the necessary risk analysis to support a regulatory decision. For products that are familiar and noncomplex, an expedited process might be used (for example, a notification process). For products that are determined to be unfamiliar or complex or unfamiliar and complex, new human health and ecological risk-analysis methods might be needed to inform a regulatory decision. A desirable feature of an integrated, stratified approach to regulatory oversight is that over time product types originally placed in the unfamiliar or complex bin or the unfamiliar and complex bin would “move” to a bin of less complexity or more familiarity based on experience gained in evaluating additional products in a category.

SUMMARY CONCLUSIONS

On the basis of its assessment of the trends in biotechnology, the likely products of biotechnology in the next 5–10 years, and the current authorities and capabilities of the regulatory agencies, the committee identified a set of broad themes regarding future opportunities for enhancement of the U.S. biotechnology regulatory system.

The bioeconomy is growing rapidly and the U.S. regulatory system needs to provide a balanced approach for consideration of the many competing interests in the face of this expansion. The competing interests and concerns articulated by the Executive Office of the President include supporting innovation, protecting human health, preserving biodiversity, reducing negative environment effects, promoting public confidence in the regulatory process, increasing transparency and predictability in the regulatory process, reducing unnecessary costs and burdens, making use of new tools from a broad range of disciplines, and interacting with the global economy. The pipeline of biotechnology products likely to emerge over the next decade probably will result in disruptive innovations and significant societal impacts; a carefully balanced, coordinated approach toward future biotechnology products that incorporates input from stakeholders—including interested and affected parties, relevant federal agencies, and nontraditional product developers—will be required.

The profusion of biotechnology products over the next 5–10 years has the potential to overwhelm the U.S. regulatory system, which may be exacerbated by a disconnect between research in regulatory science and expected uses of future biotechnology products. The number and complexity of products, new pathways to risk-assessment endpoints, large range of types of products (for example, those for open release in the environment or marketed as direct-to-consumer), new actors (including DIY bioengineers, small- and medium-sized enterprises, and crowdfunders), and complex alignment of potential future products with agency authorities are likely to change rapidly as biotechnology advances. A disconnect between research in regulatory science and its use in biotechnology research and product development creates a situation in which new products may be conceived and designed without sufficient consideration of regulatory requirements, which can lead to surprises and delays late in the development cycle. The update to the Coordinated Framework10 and the National Strategy for Modernizing the Regulatory System for Biotechnology Products,11 recently released by the Executive Office of the President, provide

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10 Executive Office of the President. 2017. Modernizing the Regulatory System for Biotechnology Products: An Update to the Coordinated Framework for the Regulation of Biotechnology. Available at https://obamawhitehouse.archives.gov/sites/default/files/microsites/ostp/2017_coordinated_framework_update.pdf. Accessed January 30, 2017.

11National Strategy for Modernizing the Regulatory System for Biotechnology Products is available at https://obamawhitehouse.archives.gov/sites/default/files/microsites/ostp/biotech_national_strategy_final.pdf. Accessed January 31, 2017.

Suggested Citation:"Summary." National Academies of Sciences, Engineering, and Medicine. 2017. Preparing for Future Products of Biotechnology. Washington, DC: The National Academies Press. doi: 10.17226/24605.
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an excellent starting point for addressing the products that will appear in the next 5–10 years. But additional investments are needed to be prepared for the subsequent generation of products that are on the horizon and to ensure that there is a consistent, efficient, and effective decision-making framework that continues to balance innovation and safety.

Regulators will face difficult challenges as they grapple with a broad array of new types of biotechnology productsfor example, cosmetics, toys, pets, and office suppliesthat go beyond contained industrial uses and traditional environmental release (for example, Bt or herbicide-resistant crops). The diversity of biotechnology products anticipated over the next decade confronts consumer- and occupational-safety regulators with two related challenges:

  1. To find jurisdiction under existing statutes to regulate all the products that may pose risks to consumers and
  2. To utilize the best available risk-analysis tools consistent with agency authorities to provide nuanced oversight that protects consumers while fostering beneficial innovation.

Existing statutes offer promising pathways to meet these challenges, although there may be cases when a novel product falls outside the jurisdiction of EPA, FDA, or USDA and is either in a jurisdictional gap (where no regulator has authority to address potential safety concerns) or under the jurisdiction of another agency, such as the Consumer Product Safety Commission, that has fewer statutory authorities and capabilities to conduct rigorous and timely risk analysis. For this reason, EPA, FDA, and USDA may at times need to make use of the flexibility available under their statutes to minimize gaps in jurisdiction and to position novel products under the statutory framework most suited to each product’s characteristics and level of risk.

The safe use of new biotechnology products requires rigorous, predictable, and transparent risk-analysis processes whose comprehensiveness, depth, and throughput mirror the scope, scale, complexity, and tempo of future biotechnology applications. Regulatory oversight that is unnecessarily complex runs the risk of driving an “imitate not innovate” mentality and may not scale to match the pace of biotechnology innovation. Building on the approach outlined in the National Strategy, the committee believes that the advancement of existing risk-analysis methodologies within an easily accessible, participatory governance framework can establish an oversight process that matches the scope, scale, complexity, and tempo of future technological developments and increases public confidence in the safety of products entering the marketplace.

In addition to the conclusions and recommendations from this report, EPA, FDA, USDA, and other agencies involved in regulation of future biotechnology products would benefit from adopting recommendations made by previous National Academies’ committees related to future products of biotechnology, which are consistent with the findings and recommendations in this report. Given the assessments of some future biotechnology products and the role of the regulatory system, many of the recommendations of previous National Academies’ committees are directly relevant and should be considered when taking actions to enhance the capabilities of the U.S. biotechnology regulatory system.

SUMMARY RECOMMENDATIONS

On the basis of its conclusions, the committee developed a number of detailed recommendations regarding actions that can be taken to enhance the capabilities of the biotechnology regulatory system in order to be prepared for anticipated future products of biotechnology.

Suggested Citation:"Summary." National Academies of Sciences, Engineering, and Medicine. 2017. Preparing for Future Products of Biotechnology. Washington, DC: The National Academies Press. doi: 10.17226/24605.
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Recommendation 1: EPA, FDA, USDA, and other agencies involved in regulation of future biotechnology products should increase scientific capabilities, tools, expertise, and horizon scanning in key areas of expected growth of biotechnology, including natural, regulatory, and social sciences.

The information gathered by the committee indicates a substantial new set of technologies that are being brought to bear in future products and the agencies should continue to maintain their scientific capabilities across a broad range of disciplines. Example priority areas, discussed in more detail in the body of the report, including areas such as comparators, off-target gene effects, and phenotypic characterization; genetic fitness, genetic stability, and horizontal gene transfer; impacts on nontarget organisms; control of organismal traits; modeling (including risk-analysis approaches under uncertainty) and life-cycle analyses; monitoring and surveillance; and economic and social costs and benefits.

  • Recommendation 1-1: Regulatory agencies should build and maintain the capacity to rapidly triage products entering the regulatory system that resemble existing products with a history of characterization and use, thus reducing the time and effort required for regulatory decision making, and they should be prepared to focus questions on identifying new pathways to risk-assessment endpoints associated with products that are unfamiliar and that require more complex risk assessments.
  • Recommendation 1-2: In order to inform the regulatory process, federal agencies should build capacity to scan the horizon continuously for new products and processes that could present novel risk pathways, develop new approaches to assess and address more complex risk pathways, and implement mechanisms for keeping regulators aware of the emerging technologies they have to deal with.
  • Recommendation 1-3: EPA, FDA, USDA, and other relevant federal agencies should work together to (a) pilot new approaches for problem formulation and uncertainty characterization in ecological risk assessments, with peer review and public participation, on open-release products expected during the next 5 years; (b) formulate risk–benefit assessment approaches for future products, with particular emphasis on future biotechnology products with unfamiliar functions and open-release biotechnology products; and (c) pool skills and expertise across the government as needed on first-of-a-kind risk–benefit cases.
  • Recommendation 1-4: EPA, FDA, USDA, and other relevant federal agencies should create a precompetitive or preregulatory review “data commons” that provides data, scientific evidence, and scientific and market experience for product developers.
  • Recommendation 1-5: Consistent with the goals and guidance stated by the Office of Science and Technology Policy in the Executive Office of the President in its July 2015 memo, the Biotechnology Working Group should implement a more permanent, coordinated mechanism to measure progress against and periodically review federal agencies’ scientific capabilities, tools, expertise, and horizon scanning as they apply to the profusion of future biotechnology products.

Recommendation 2: EPA, FDA, and USDA should increase their use of pilot projects to advance understanding and use of ecological risk assessments and benefit analyses for future biotechnology products that are unfamiliar and complex and to prototype new approaches for iterative risk analyses that incorporate external peer review and public participation.

Suggested Citation:"Summary." National Academies of Sciences, Engineering, and Medicine. 2017. Preparing for Future Products of Biotechnology. Washington, DC: The National Academies Press. doi: 10.17226/24605.
×

The rate of technology development in the biological sciences and engineering will create a situation in which many new types of products will be developed in the next 5–10 years. In order to handle the scope and complexity of future biotechnology applications, the regulatory agencies should make use of pilot products to identify ways to improve the comprehensiveness, effectiveness, and throughput of the regulatory process.

  • Recommendation 2-1: Regulatory agencies should create pilot projects for more iterative processes for risk assessments that span development cycles for future biotechnology products as they move from laboratory scale to field or prototype scale to full-scale operation.
  • Recommendation 2-2: Government agencies should pilot advances in ecological risk assessments and benefit analyses for open-release products expected in the next 5–10 years, with external, independent peer review and public participation.
  • Recommendation 2-3: Government agencies should initiate pilot projects to develop probabilistic estimates of risks for current products as a means to compare the likelihood of adverse effects of future biotechnology products to existing biotechnology and nonbiotechnology alternatives.
  • Recommendation 2-4: Regulatory agencies should make use of pilot projects to explore new methods of outreach to the public and developer community as a means of horizon scanning, assessing need areas for capability growth, and improving understanding of the regulatory process.
  • Recommendation 2-5: EPA, FDA, and USDA should engage with federal and state consumer- and occupational-safety regulators that may confront new biotechnology products in the next 5–10 years and make use of pilot projects, interagency collaborations, shared data resources, and scientific tools to pilot new approaches for risk assessment that ensure consumer and occupational safety of new biotechnology products, particularly those that may involve novel financing mechanisms, means of production, or distribution pathways.

Recommendation 3: The National Science Foundation, the U.S. Department of Defense, the U.S. Department of Energy, the National Institute of Standards and Technology, and other agencies that fund biotechnology research with the potential to lead to new biotechnology products should increase their investments in regulatory science and link research and education activities to regulatory-science activities.

Increased investments in regulatory science will be needed to align desired science advancements with existing and anticipated regulatory requirements. It will be valuable for developers of biotechnology to incorporate regulatory perspectives earlier in the product and technology development process, and the research funding agencies can help enhance the regulatory system by increasing the awareness of regulatory science at an early stage.

  • Recommendation 3-1: The federal government should develop and implement a long-term strategy for risk analysis of future biotechnology products, focused on identifying and prioritizing key risks for unfamiliar and more complex biotechnology products, and work to establish appropriate federal funding levels for sustained, multiyear research to develop the necessary advances in regulatory science.
  • Recommendation 3-2: Federal agencies that fund early-stage biotechnology-related research and regulatory agencies should provide support to academic, industry, and government researchers to close gaps and provide linkages to market-path requirements for regulatory success.
Suggested Citation:"Summary." National Academies of Sciences, Engineering, and Medicine. 2017. Preparing for Future Products of Biotechnology. Washington, DC: The National Academies Press. doi: 10.17226/24605.
×
  • Recommendation 3-3: Government agencies that fund biotechnology development, working together with regulatory agencies and each other, should also invest in new methods of understanding the ethical, legal, and social implications associated with future biotechnology products.
  • Recommendation 3-4: Government agencies with an educational mission, including those that support scientific training, should identify and fund activities that increase awareness and knowledge of the regulatory system in courses and educational materials for students whose research will lead to advances in biotechnology products.
Suggested Citation:"Summary." National Academies of Sciences, Engineering, and Medicine. 2017. Preparing for Future Products of Biotechnology. Washington, DC: The National Academies Press. doi: 10.17226/24605.
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Between 1973 and 2016, the ways to manipulate DNA to endow new characteristics in an organism (that is, biotechnology) have advanced, enabling the development of products that were not previously possible. What will the likely future products of biotechnology be over the next 5–10 years? What scientific capabilities, tools, and/or expertise may be needed by the regulatory agencies to ensure they make efficient and sound evaluations of the likely future products of biotechnology?

Preparing for Future Products of Biotechnology analyzes the future landscape of biotechnology products and seeks to inform forthcoming policy making. This report identifies potential new risks and frameworks for risk assessment and areas in which the risks or lack of risks relating to the products of biotechnology are well understood.

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