Annual Report 2021

Clinical studies

We conduct high-caliber clinical research that always complies with applicable laws and regulations. When performing clinical studies, we adhere to the highest ethical and scientific standards worldwide.

We only conduct clinical studies to investigate issues that are relevant to patients, healthcare professionals, or society, and only when the medicines being tested show significant therapeutic promise and have a positive benefit-risk ratio. In addition, a sound, established scientific methodology must be available to investigate these scientific or medical questions. We only enroll the specific number of participants required to answer each of these questions.

Protecting the safety, well-being, dignity, and rights of the patients and healthy volunteers participating in our clinical studies is of utmost importance to us. We do not intentionally expose study subjects to undue risk or irreversible harm. Personal data privacy is also very important to us, and we maintain a strong focus on data protection and confidentiality in compliance with statutory regulations.

We assure that no subject enrolling in a clinical study is discriminated against on the basis of ethnic origin, gender or socio-economic status.

Patient-focused drug development

We are improving our approach to research and development by committing to patient-focused drug development that more actively involves patients, caregivers, and their advocates in our work. Their valuable insights into disease and treatment management will help us make more informed decisions at each stage of the medicine development process. We aim to make our studies easy for patients to understand while ensuring all participants have positive experiences as they contribute to our understanding of the particular disease and its treatment. We are also working to further develop the way in which our research work is communicated and how it can improve the healthcare people receive. At every level of our organization, we are additionally educating staff about the value of closer, more consistent patient interaction and the requirements to protect our patients’ independence and privacy.

Clinical studies in low- and middle-income countries

We conduct all our clinical studies in accordance with local laws and regulations, and we adhere to all relevant international scientific and ethical standards, irrespective of the region or country.

Roles and responsibilities

Clinical drug development, including clinical studies and the related governance process, are the responsibility of the Global Development unit. The Head of Global Development reports to the CEO Healthcare, who is a member of the Executive Board.

We have established two internal committees to oversee our clinical studies. The Development Studies Committee is responsible for the studies performed by the company on medicines that are under clinical development, while the Global Medical Decision Board is responsible for our own studies with approved medicines, as well as for all studies performed by independent investigators and supported by us (so-called investigator-sponsored studies). Both bodies consist of medical-scientific experts and executives with long-standing experience in clinical research.

Before administering a new drug to humans, there must be sufficient evidence that it offers a potential therapeutic benefit, is sufficiently safe for use in humans, and has a positive benefit-risk ratio. We only take the critical step of a first-in-human clinical trial after diligently conducting extensive preclinical testing. The decision lies with a separate committee, the Human Exposure Group, chaired by our Global Chief Medical Officer.

We continuously analyze potential risks for study participants before and during our clinical studies. Our Medical Safety and Ethics Board oversees the safety of subjects participating in our clinical studies and, as necessary, reviews the benefit-risk profiles of investigational drugs.

Our commitment: International guidelines and requirements

Our Human Subjects Research and Development Policy provides the framework for conducting clinical studies and helps ensure that we adhere to all applicable legal, ethical and scientific standards. In addition to the relevant national laws and regulations, these standards also include:

Regular supervision of clinical studies

Our clinical study processes and procedures are regularly inspected by relevant regulatory authorities to verify their compliance with applicable laws and guidelines.

The Research & Development Quality unit applies a risk-based identification strategy to determine areas that need to be audited. Quality assurance audits are performed internally within Healthcare R&D (for example, process audits) and externally (for example, at vendors’ sites and investigational sites). We respond immediately to observations during audits by investigating their root causes and, according to their criticality, defining and implementing corrective and preventive actions to improve processes, prevent reoccurrence of irregularities and ensure compliance.

Due to the Covid-19 pandemic, we postponed some audits from 2020 to 2021. However, for all audit types we successfully implemented a remote audit approach. As a result, we were able to largely implement the audit plan for 2021, shifting only a small number of audits to 2022.

Conducting clinical studies responsibly

Every clinical study follows defined procedures to ensure it is conducted to the highest quality standards in line with good working practices (GxP) for the development and manufacturing of drugs, the ethical principles of the Declaration of Helsinki and other international guidelines and regulations. In 2021, regulatory authority inspections did not unveil significant issues which had any impact on patient rights, patient safety, or the data integrity of a study.

Disclosure of clinical studies and publication of results

We are obligated to disclose findings from our clinical studies. We do this publicly in a complete, accurate, balanced, transparent, and timely manner as laid out in our Clinical Trial Disclosure Policy. We publish results from our clinical studies in medical journals in line with applicable laws and industry codes. In this way, we adhere in particular to the current version of the Good Publication Practice (GPP3) and follow the recommendations of the International Committee of Medical Journal Editors (ICMJE). Our Standard on Clinical Trial Data Transparency underscores our strong commitment in this matter.

Enabling early access to new medicines

Not all patients have the opportunity to take part in a clinical study and must therefore wait for a new pharmaceutical product to be approved. Through our Early Access Program, we can, under specific circumstances, enable patients to gain early access to new, potentially life-saving medicines. The offer is aimed at people with serious conditions who have already received all available therapies without success. It allows them to be treated with medicines that have already been clinically tested but have not yet been approved. Furthermore, we offer patients who participated in one of our clinical studies post-study access to the investigational product, provided that certain conditions are met. Here, too, we meet stringent statutory, ethical, and scientific standards. By performing a thorough assessment of all available data, we ensure that the potential benefits outweigh the potential risks for patients.

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