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Clinical studies

Our aim is to conduct high-caliber clinical research that is in compliance with applicable laws and regulations. We set Group-wide requirements that aim to ensure that high ethical and scientific standards are met when conducting clinical trials.

We only conduct clinical studies to investigate issues relevant to patients, healthcare professionals or society, and only when our established methodology finds the given medicines show significant therapeutic promise and a positive benefit-risk ratio. Accordingly, to ensure patient safety and avoid interrupting the development of promising products, we carefully select patients based on known risk factors. These include age and comorbidities, which we reflect in the design of our clinical studies. Notably, we only enroll the specific number of patients needed to answer the posed scientific and medical questions. We reconcile and review the safety reports from our clinical studies and marketed products and immediately address any unforeseen risks. Senior boards such as the Pharmacovigilance Advisory Board and the Medical Safety and Ethics Board maintain oversight of any emerging safety concerns. In addition, cross-functional Benefit Risk Assessment teams adapt the benefit-risk assessment and development strategy of each product to ensure it delivers maximum safety and efficacy to our patients. In addition, a sound, established scientific methodology must be available to investigate these scientific or medical 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 participants to undue risk or irreversible harm. Data privacy is also very important to us, and we maintain a strong focus on data protection and confidentiality in compliance with statutory regulations.

Diversity, equity and inclusion in clinical trials

Based on our Standard on Human Research, we aim to conduct clinical studies that adequately represent the diverse patient populations expected to use our products once they are approved. To ensure fair, balanced and scientifically justified study representation, we cemented our commitment to Diversity, Equity and Inclusion in clinical trials by collaborating with healthcare providers and community advocates to eliminate common barriers to clinical trial participation.

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. At every level of our organization and based on the function, we are additionally either offering or mandating to educate staff about the value of a close, more consistent patient interaction and the requirements to protect our patients’ independence and privacy.

Roles and responsibilities

Clinical development, including clinical studies and their related governance processes, are the responsibility of our Global Development unit. The Head of Global Research & 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 Integrated Protocol Review Committee is responsible for the studies performed by the company on products that are under clinical development, while the Global Medical Decision Board is responsible for our own studies with approved products 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 product 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 (MSEB) oversees the safety of the participants in our clinical studies and, as necessary, reviews the benefit-risk profiles of investigational products.

Our commitment: International guidelines and requirements

Our Quality Policy defines the strategic framework that ensures our products, services and systems deliver high quality, safety and efficacy to our patients. It details the most relevant laws and codes, criteria and guidance (e.g. for product development and manufacturing), and our senior management’s responsibility to ensure quality is embedded in everything we do.

Our Standard on Human Research provides the framework for conducting clinical studies and helps ensure we adhere to all applicable legal, ethical and scientific standards. Further quality documents detail for instance the strategic direction of all quality related activities or disclose our position on data privacy. In addition to the relevant national laws and regulations, these documents 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 and Risk Management (RDQRM) 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 (e.g. investigator sites and vendor audits). We respond immediately to observations made 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. As planned, in 2023, RDQRM concluded most of the audits of the Annual Audit Plan.

Conducting clinical studies responsibly

Every clinical study follows defined procedures to ensure it is conducted to high 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. As in the previous year, in 2023, none of the regulatory inspections conducted on our clinical research activities resulted in regulatory action.

Disclosure of clinical studies and publication of results

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

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 products. 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 products 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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