Skip to main content

Table 3 Summary of how-to guide for PE in the early discovery and preclinical phases

From: Co-creation of practical “how-to guides” for patient engagement in key phases of medicines development—from theory to implementation

Section Description and purpose/rationale
STEP 1: Preparations for setting up partnership and collaboration Focuses on the importance of preparations to ensure that long-term partnerships are created and nurtured. This sets the stage for future collaborations and aims to prevent tokenistic and one-off patient engagement.
Understand patients’ views and abilities to contribute to initial steps of drug development for the condition
Prepare patients for participation in the program by educating patients on what preclinical research is
Ensure that research-active organizations (e.g., academia and the pharmaceutical industry), regulators, governments, and other health system entities recognize and accept the importance of patient input in early medicines development
Prepare the research team for a meaningful, effective, and respectful interaction with patients and give them tools to build a mutually beneficial long-term partnership
STEP 2: Understanding the condition profile and therapy area Reveals to researchers and scientists the patients’ experience and perspective on what it is like to live with the condition under study and what might be the unmet needs
Gather patient input to educate preclinical scientists/researchers about: the patients’ symptom burden and impact on daily life; what therapies patients use and their associated efficacies (or lack); patients’ views on an ideal therapy; patients’ views on risk-benefit trade-offs they will accept with new treatments
Uncover patients’ unmet needs
Engage and help patients to understand what preclinical research is, why it is needed, and where it sits in the overall medicines development continuum
Act as an initial dialogue between patients, research teams, and other stakeholders toward creating a long-term relationship across the span of the medicine’s development
STEP 3: Developing research methodology Focuses on working with patients to evaluate and identify the optimal tools and approaches to address research objectives
Describes how to
Work with patients to evaluate optimal tools and approaches to address research objectives in laboratory-based and virtual (in silico) studies
Work with patients to evaluate possible studies to address clinical questions
Define a range of outcome measures to be used in upcoming clinical studies ensuring these are patient-centered, and understand how outcome measures in laboratory-based and preclinical studies will translate into clinical outcomes that are meaningful for patients
Consider public health implications and real-world anecdotal evidence in the research
STEP 4: Target product profiles and target value profiles Brings together outcomes from previous sections to develop two profiles that represent (1) the patients’ perspective on the product and (2) researcher/pharma-oriented documents that support research and development (R&D)
A target value profile (TVP) is a consolidated set of “expected and minimally acceptable characteristics” of a chemical molecule, biological product, or medical device, which are used as treatments and are valuable and meaningful for patients because they address areas of unmet needs. Along with other insights, the TVP informs the target product profile (TPP)—an updatable guidance for the pharma industry/drug developers with targeted characteristics of a potential product. The TVP—as a main element of TPP that encompasses core values and addresses unmet patient needs—should be co-developed with patients
The guide provides examples of how to gain patient input and incorporate patients’ value in the TVP