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Creating A “Speak Up Culture” For Patient Safety

Keeping your patients informed and enabling them to be involved in their own care is an essential step to create a person-centred care.

What are the best strategies for engaging patients and creating appealing and safe clinical trials?

Pharmaceutical development has to demonstrate the value of the new health technology relative to the outcomes experienced by patients. This patient experienced value of the new health technology is a requirement and precondition for regulatory and payer approval. How and when to engage patients? If patient engagement works, the involvement needs to start already during the early stages of development. HTA bodies and other stakeholders can benefit from the established involvement afterwards. Patient engagement should not be a HTA problem. It is a drug development problem. The drug developer should recognise the value of involving patients from the very beginning, even before the product enters clinical development and should continue across the entire life cycle and work to adapt present perceptions and practices throughout their organisations. The appropriate patient-reported outcomes (PROs), which provide self-reported patient assessments of their quality of life, daily functioning, and symptom severity, are an essential element within pivotal clinical trials to capture the patient’s perspective and to support regulatory and payer approval.

How to efficiently use patient insights in early drug development?

Successful patient participations enlighten patients’ experiences, needs, obstacles and preferences. Patient participation influence recommendations about the appropriate use of the new health technologies. Patient preferences for some health technologies already entered clinical practice guidelines academic research and regulatory decision-making.

How can companies create and maintain patient-centric clinical studies? What should be the first step?

The appraisement of benefits and tolerable risks or harm looks different, if you are the real patient or the treating physician who should give consideration of a just approved health technology.

To generate appropriate insights out of clinical trials for or primary stakeholders – our patients should be the first step for a sustainable drug development.

Many health systems take person-centered care and shared decision-making between patient and healthcare provider for granted, therefore it is inadequate to involve only very informed and organised patients who have the personal qualifications and capabilities to participate. Health systems have to develop robust processes to enable all patients, independent of their socioeconomic status and cultural heritage to participate and be actively involved into their own care. Patients should be informed and ask questions and develop a “speak up culture” for patient safety.

Join Jost during our 15th Annual Optimising Clinical Trials Summit: Site Selection, Feasibility and Patient Recruitment in October where he will be sharing his insights on Understanding Patients: Needs During Drug Development – The Patient Safety Perspective.

Here is a short abstract of his presentation:

Trust of the patient into its physician is the key factor for prescribing a new health technology and adherence to treatment. In this context, the safety profile is often more important than efficacy. Subsequent to the treatment decision, the patient has to take the medicine as planned. If reimbursement is not a problem, safety is one of the key drivers to stop treatment. Patient-reported outcomes are the patient’s voice within pivotal clinical trials for regulatory and payer approval. The journey should not end after the approval. Medical insights generated within the pivotal clinical trials have to reach the most important stakeholders in healthcare: our patients. However, there is no agreement about how best to use patients’ perspectives in clinical development as well as during the market access journey which follows after the regulatory approval. The first step is to understand the patients’ needs from a patient perspective as early as possible. Later, this patient experience is incorporated in the regulatory approval and in the calculation of the cost of new treatments as the outcome of the benefit assessment. We should not forget to communicate all these medical insights out of the clinical trials to enable normal patients and normal physicians for knowledge-based treatment decisions of innovative healthcare solutions.


Jost LEEMHUIS is currently the Head of Safety Science Germany at Roche Pharma AG. Before that he was a Principal Investigator and Group Head at the University of Freiburg

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