PreambleBIO represents more than 1,000 biotechnology companies, academic institutions, state biotechnology centers and related organizations in all 50 U.S. states and 33 other nations. BIO members are involved in the research and development of health-care, agricultural, industrial and environmental biotechnology products.Research involving animals has been critical to understanding the fundamental processes of human biology that are so integral to modern medicine. Biotechnology companies have depended on this research to develop more than 160 drugs and vaccines approved by the U.S. Food and Drug Administration, helping 325 million people worldwide and preventing incalculable human suffering. There are now a number of research techniques involving animals, which hold great promise for aiding humanity. However, many serious diseases such as AIDS, Alzheimer's Disease, Parkinson's Disease, hepatitis, cancer, cardiovascular diseases, and diabetes, are still poorly understood and treated. Therefore, new and more effective therapies and diagnostics are desperately needed to improve the lives of patients.BIO members are compelled by ethical and legal concerns to evaluate the safety and efficacy of potential medicines and food products before they are given to humans and animals; the use of animals in research is a requirement for many such products. The appropriate and responsible use of animals is therefore an indispensable part of biomedical and agricultural research. BIO members are committed to act ethically and to apply high standards of care when using animals in scientific procedures.BIO members are committed to reducing the number of animals used for research when it is possible to develop, validate and use alternative methodologies consistent with regulatory requirements for testing, while maintaining the scientific integrity of the research. BIO affirms and upholds the science-based regulation and oversight of animal research by the U.S. Food and Drug…

Reaffirming and Broadening Our Commitment to Transparency and Clinical Trial Data SharingThe member companies of the Biotechnology Industry Organization (BIO) are committed to improving human health through the development of innovative therapies. We strongly support research that aims to improve human health through better drug development and recognize that responsibly sharing our clinical trial data can help to advance such research, while reinforcing public confidence in the safety and efficacy of our medicines.Drug development is a highly complex, costly, lengthy, and competitive endeavor. The interests of all participating stakeholders must, therefore, be carefully balanced when considering increased access to clinical trial data. Foremost, study participants themselves must have confidence that their personal medical information and privacy will be respected in accordance with the terms of their informed consent and in compliance with relevant laws and regulations. Additionally, in order for innovative biotechnology companies to successfully attract the investment necessary to fund a drug or biologic program over the decade or more required for its development, it is imperative that data not be disclosed prematurely or in a manner that does not protect confidential and proprietary information. Moreover, most BIO member companies are small, pre-revenue enterprises that operate with limited resources. While evaluation of requests for additional data will divert resources from their core mission of directly developing innovative therapies, BIO members recognize the value of supporting, to the extent possible, qualified external medical and scientific research.We are, therefore, committed to building upon our routine publication of clinical research results and ongoing collaborations with academic and government researchers in order to support additional efforts to improve public health.How Do We Currently Promote Transparency and Access to Clinical Trial Data…

September 2017Genome editing is an emerging technology that allows for the targeted modification of DNA for a multitude of applications. In human health, researchers are exploring ways to use genome editing of somatic cells to treat or prevent genetically defined human diseases.Clinical use of genome editing in somatic cells, or non-heritable cells, is currently subject to oversight similar to that of other advanced biotherapeutic technologies such as gene therapy. Specifically, the combined activities and responsibilities of the US Food and Drug Administration (FDA), through its statutory role as the regulator of drug development, and the NIH/Recombinant DNA Advisory Committee (RAC), as the forum for public discussion, serve in this oversight capacity to protect patients while ensuring that important research moves ahead.In February 2017, the National Academy of Sciences and the National Academy of Medicine Committee on Human Gene Editing, after careful review of scientific, ethical and public views on human genome editing, released a report supporting the current regulatory and oversight mechanism for somatic cell gene editing for the potential treatment of disease. The report also concluded that genome editing in humans for purposes other than treating or preventing a disease or disability should not proceed at this time. BIO fully agrees with these two conclusions.Regarding the clinical use of genome editing in germline cells (where the DNA edit is passed on to future generations), the 2017 authors noted that “there is a need for caution in any move toward germline editing, but that caution does not mean prohibition.” The Committee concluded that under a very strict set of 10 criteria, including when 1) no other reasonable alternatives exist, 2) government restrictions have expired, and 3) research on risk/benefit standards has advanced in the field, certain germline editing could be permissible for the treatment of disease.BIO views the science of germline…