Research & Development
Research & Development
Inventing is our passion. We embrace our responsibility to address the health needs of patients and society through world-class science.
We are committed to addressing unmet medical needs through innovative research and development (R&D). R&D expenses were $10.1 billion in 2016, $6.7 billion in 2015 and $7.2 billion in 2014 (which included restructuring costs and acquisition- and divestiture-related costs in all years). The talent of our scientists, combined with recent scientific and technological advances, is leading to an exciting period for research as we seek new and more effective ways to treat diseases.
Our R&D model is designed to increase productivity and improve the probability of success by prioritizing resources based on medical need, scientific opportunity and commercial potential.
We are committed to advancing our most promising research and clinical development to bring forward new medicines and vaccines that will make a meaningful difference in patients’ lives.
Faced with the complex challenges of bringing important new therapies to patients while simultaneously controlling the rising costs of R&D, we are using important new preclinical, clinical and quantitative tools to help us rapidly differentiate between developmental candidates that will clearly meet patient needs and those that will not. A focus on genetics, genomics and translational medicine is also critical to these efforts.
For instance, we are able to develop biomarkers—those characteristics that can be objectively measured and evaluated as indicators (or markers) of normal biologic processes, disease processes or responses to therapy. Since biomarkers provide critical information in the drug discovery and development processes, our intent is to apply them very early in the development of novel therapeutic candidates to provide preliminary evidence of their potential benefit before proceeding with further development.
In addition, we are using novel quantitative approaches that harness the power of mathematical modeling to analyze preclinical experiments to inform our clinical trial designs and to develop models based on published literature. We now have the capability to first simulate a trial thousands of times, exploring the impact of different factors that influence a specific disease and/or patient population, as well as the related efficacy and safety of responses.
With this integrated approach, we can optimize the next phase of clinical trials and, importantly, make pivotal decisions earlier and more confidently, increasing productivity and the efficient use of our resources. By eliminating likely failures sooner and focusing on those mechanisms that appear more promising, we believe we can bring innovative products to patients faster and more efficiently, while still maintaining a rigorous focus on scientific excellence and patient safety.
We recognize that real-world evidence has an increasing role in how medicines and vaccines are developed and evaluated. Through the contributions of epidemiologists, health economists, outcome researchers, data specialists and other health-policy researchers across our company, we strive to comprehensively assess the best available information on the value of our medicines and vaccines.
We are including pediatric development programs in the company’s new drug and vaccine development strategies worldwide, where relevant, in response to unmet clinical needs.
When appropriate, we will develop and seek approval for pediatric indications and develop age-specific formulations. We rely on an internal Pediatric Development Committee to review and provide input into all pediatric development strategies across various therapeutic areas. The Committee serves as a Center of Excellence within our company to consult on pediatric development issues and key pediatric policy questions. For a listing of all clinical trials, including pediatric clinical trials, click here.
We prioritize our R&D efforts on advancing candidates that we believe represent the next areas of breakthrough science that will make a difference and deliver value for patients, physicians and payers.
Our company is prominently positioned at the intersection of invention and the burden of disease. Our products and research priorities are aligned with the current and projected global burden of disease as defined by the World Health Organization (WHO), as well as with the increasing need for new therapies targeted at diseases such as chronic hepatitis C and antibiotic-resistant infections.
Our research pipeline illustrates the productivity of our R&D efforts. We currently have a number of candidates under regulatory review in the U.S. and internationally. An update on our R&D activities can be found in our Form 10K or on our corporate website.
We support academic and community-based physicians and researchers in expanding clinical and scientific knowledge, and in improving understanding of the appropriate use of our products.
Our Investigator Studies Program is an example of our efforts to advance science and improve patient care by supporting, through the provision of drugs and vaccines and/or funding, high-quality research that is initiated, designed, implemented and sponsored by external investigators. This program encourages research in emerging scientific areas of interest, and has an established track record of presentations and the publication of results in peer-reviewed journals.
We are a member of and support numerous professional associations, including the American Association for the Advancement of Science (AAAS), the U.S. National Institutes of Health (NIH), the U.S. National Science Foundation (NSF), the World Medical Association (WMA) and the Council for International Organizations of Medical Societies (CIOMS). In addition to promoting dialogue and the exchange of ideas in research, we sponsor research conferences—such as selected Gordon Research Conferences, an international forum in which researchers discuss advances in biologic, chemical and physical science—that cover areas in which our company is conducting research.
As an early supporter of the Physician Payments Sunshine Act (PPSA), we believe in broad disclosure of financial relationships between physicians and the pharmaceutical industry. In October 2009, our company began voluntarily disclosing all payments to U.S.-based health care professionals who speak on behalf of our company, about our products and other health care issues.
We comply with the PPSA provisions of the U.S. Affordable Care Act, which requires pharmaceutical manufacturers to annually disclose information on payments and other transfers of value furnished to U.S.-licensed physicians and U.S. teaching hospitals to the Department of Health and Human Services (HHS). Learn more.
We also collaborate with external researchers and other members of the pharmaceutical industry by participating in selected scientific consortia. Consortia are an important mechanism by which researchers can work together in a precompetitive manner to address complex scientific challenges common to all parties. These consortia are typically in the form of public-private partnerships.
PUBLIC-PRIVATE RESEARCH PARTNERSHIPS
We recognize that individuals or companies cannot successfully develop drugs single-handedly. Most cases of true innovation come from robust and honest collaboration among individuals and organizations with diverse backgrounds and capabilities, brought together by the idea of changing the course of human health.
As part of our R&D strategy, we pursue opportunities to establish external alliances to complement our substantial internal research capabilities, including research collaborations, as well as licensing agreements for preclinical and clinical compounds that have the potential to drive both near- and long-term growth. Our research laboratories establish significant external alliances to advance drug discovery and development, improve R&D productivity, and successfully commercialize novel therapeutics and vaccines.
Drug Discovery Collaborations
Innovative Medicines Initiative – Accelerating Research
Within Europe, we participate in a number of Innovative Medicines Initiative (IMI) projects. IMI, Europe’s largest public-private initiative aiming to speed drug development and improve safety, supports collaborative research projects and builds networks of industrial and academic experts to boost pharmaceutical innovation.
One ongoing IMI project centers on collaboration with the European Patients’ Academy on Therapeutic Innovation (EUPATI) by developing standards and training for patient advocacy group leaders. EUPATI launched the in-depth Patient Expert Training Course in 2014 and, in 2016, launched a web-based educational toolbox, hosting educational material in English, Italian, Spanish, Polish, German, French and Russian aiming to reach 12,000 patient advocates across Europe.
Accelerating Medicines Partnership – New Diagnostics & Therapies
We are a member of the Accelerating Medicines Partnership, a venture among the National Institutes of Health, 10 biopharmaceutical companies and several nonprofit organizations to transform the current model for developing new diagnostics and treatments by jointly identifying and validating promising biological targets of disease. The ultimate goal is to increase the number of new diagnostics and therapies for patients and reduce the time and cost of developing them.
Biomarkers Consortium – Biomarker-Based Technologies
The Biomarkers Consortium, in which we participate, is a public-private biomedical research partnership managed by the Foundation for the National Institutes of Health (FNIH). Its goal is to combine the forces of the public and private sectors to accelerate development of biomarker-based technologies, medicines and therapies for the prevention, early detection, diagnosis and treatment of disease. Working together, the members of the Biomarkers Consortium are building uniquely powerful collaborations that are accelerating the development of biomarker-based technologies, medicines and therapies.
To date, the Biomarkers Consortium has launched 10 projects in areas as diverse as Alzheimer’s disease, cardiovascular disease, osteoporosis and breast cancer. A number of other promising projects are also moving toward implementation. Our company contributes to a number of FNIH projects within this consortium.
Multi-Regional Clinical Trials Center of Brigham and Women’s Hospital and Harvard
We are a participating member of the Harvard Multi-Regional Clinical Trials (MRCT) Center. The MRCT Center develops guidance, training resources and tools that promote safe and ethical clinical trials. We perform our work by convening representatives from industry, not-for-profit organizations and academia, as well as investigators, patients and patient advocacy groups, to create practical resources for the ethical design and conduct of multiregional clinical trials.
Current areas of focus include working with global leaders to promote regulatory convergence and internationally accepted best practices; developing guidance and tools to promote safe and ethical trials; and training clinical trial professionals with a particular focus on low- and middle-income countries.
National Institutes of Health Alzheimer’s Disease Neuroimaging Initiative
We continue to participate in the National Institutes of Health Alzheimer’s Disease Neuroimaging Initiative (ADNI), the largest public-private partnership in Alzheimer’s disease research. This study, which is designed to gain new insights into the onset and progression of Alzheimer’s disease, has now expanded to ADNI3, with the goal of improving clinical trial design and aiding drug development. ADNI3 will seek to identify and track early changes in the brain before the onset of Alzheimer’s symptoms by using imaging techniques, including the newly developed tau PET ligands, and biomarker measures in blood and cerebrospinal fluid.
Predictive Safety Testing Consortium – Safety-Testing Methods
We are a member of the Predictive Safety Testing Consortium (PSTC), a unique public-private partnership led by the nonprofit Critical Path Institute (C-Path). The PSTC brings together pharmaceutical companies to share and validate their safety-testing methods under the advisement of the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
The 18 corporate members of the consortium share internal experiences with preclinical and clinical safety biomarkers in kidney, liver, skeletal muscle, testicular toxicity, vascular injury and cardiac hypertrophy. All biomarker research programs have a strong translational focus to select new safety tools that are applicable across the drug-development spectrum. Advancing the science and use of biomarkers in drug development is a critical area of focus for our company.
The following are notable PSTC achievements:
- The FDA and EMA qualified seven new urine tests that signal kidney injury
- The PSTC opened a biomarker-qualification process with the FDA for new biomarkers of drug-induced liver and skeletal muscle injury
- The Japanese Pharmaceuticals and Medical Devices Agency (PMDA) qualified new biomarker laboratory tests that signal kidney injury
Clinical Trials Transformation Initiative/Duke University – FDA
We continue to focus efforts on improving the way in which clinical trials are recruited and conducted. To further those efforts, we are an active member of the Clinical Trials Transformation Initiative (CTTI), a public-private partnership led by FDA and the Duke Clinical Research Institute. CTTI works with industry and academic partners to identify and promote practices that will increase the quality and efficiency of clinical trials, and will foster a high-quality clinical trial system that is patient-centered and efficient, enabling reliable and timely access to evidence-based prevention and treatment options. Current working groups focus on patient-informed consent processes and standards, Investigational New Drug safety reporting and on best practices for Data Monitoring Committees.
TransCelerate BioPharma Inc.
We are a participating member of TransCelerate BioPharma Inc., a nonprofit organization focused on improving the health of people around the world by simplifying and accelerating the research and development of innovative new therapies. We support the philosophy that collaboration on topics across the biopharmaceutical research and development community is the best way to address our industry’s most complex challenges and drive positive change.
We actively participate in all five of the strategic priority areas: (1) Improve the Site Investigator Experience; (2) Facilitate Information Sharing; (3) Enable Harmonization of Clinical Trial Processes; (4) Enhance Sponsor Efficiencies; and (5) Improve the Patient Experience. Additionally, we participate in the newly launched Pharmacovigilance initiative. Our colleagues contribute across a broad range of responsibilities. These include serving on the Board of Directors and fulfilling Executive Committee, Sponsor, Workstream lead and Workstream member roles.
We remain committed to developing partnerships and ideas to help improve care and access to medicine around the world. Read more about Business Development & Licensing.
Our governance structure is as vital to our success as the life-changing products we bring to market.
Our Research Leadership Team develops the divisional strategy, allocates resources and manages the R&D portfolio. The team comprises the heads of functional areas within our company’s research laboratories, and each area provides expert, efficient support of our drug candidates—ushering them from drug discovery through product life-cycle management.
CODE OF CONDUCT
All of our employees must abide by our Code of Conduct, which applies to the way we work with external researchers, doctors and academics. According to our Guiding Principles for Business Practices Involving the Medical and Scientific Community, all activities involving the medical and scientific community that are sponsored or supported by our company, including our subsidiaries, are to have a legitimate, well-articulated business purpose. In addition, all activities are to be implemented in accordance with the highest standards of ethics and integrity, having the utmost regard for patient health and safety.
In accordance with our company policy, we do not tolerate fraud or misconduct in our research activities—whether by an employee or by an external business partner. We deal promptly, directly and appropriately with all reported cases. MRL policy is aligned with our company’s Corporate Policy on Reporting and Responding to Misconduct.
To help ensure compliance, we have clear policies in place to provide guidance to employees on ethical and lawful conduct. It is the responsibility of all employees to conduct themselves ethically and lawfully.
Our company’s compliance program is based on chapter 8 of the U.S. Federal Sentencing Guidelines, “Sentencing of Organizations,” as amended, which sets forth the elements of an effective compliance program, as well as more specific guidance for the pharmaceutical industry issued by the Office of the Inspector General in 2001. Learn more about our company’s compliance program.
The company has allocated responsibility and accountability for compliance to the divisional level. Therefore, each division has established its own compliance committee to tackle specific divisional issues and requirements.
The stated objective of the Compliance Committee Charter within our research laboratories is to ensure ongoing compliance with applicable laws and requirements in all business areas through appropriate management structure, processes and training. In order to manage compliance, the Committee is composed of members of the Research Leadership Team. As a result, compliance efforts encompass the entire division and go beyond simply addressing the conduct of clinical trials.
The Committee also promotes ethical science and provides guidance to MRL employees on our company’s standards and corporate policies, as well as necessary education related to specific requirements applicable to the research community.
|Research and Development expenses (in billions)1||$8.2||$7.5||$7.2||$6.7||$10.1|
|Employees involved in research activities||13,600||12,300||11,400||11,900||12,300|
|New products approved2||3||0||7||2||3|
|Products in the pipeline and under regulatory review2||41||35||33||31||39|
|Top 20 global burdens of illness addressed by our products and pipeline3||55%||88%||88%||88%||88%|
|Established significant external licenses and collaborations4||61||40||35||64||57|
|Filed U.S. patent applications||192||159||125||185||195|
|1. In 2016, the increase was driven primarily by higher acquired in-process research and development (IPR&D) impairment charges, increased clinical development spending, higher restructuring and licensing costs, partially offset by a reduction in expenses associated with a decrease in the estimated fair value measurement of liabilities for contingent consideration, as well as by the favorable effects of foreign exchange.|
2. Candidates in our company’s research pipeline or under regulatory review as reported in the United States Securities and Exchange Commission Form 10-K, page 16, filed on February 28, 2017. This includes candidates in Phase II, Phase III, or under regulatory review as of February 24, 2017. When candidates attain regulatory approval they are removed from this pipeline view.
3. As defined by the Institute for Health Metrics and Evaluation (IHME) using GBD2015 data.
4. Starting in 2014, we no longer capture select early licenses and research collaborations that were included in the metric for previous years.