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

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