In today’s medicines development landscape, patient experience data are a pre-requisite for a regulatory submission [21]. As the experts on their own disease, patients bring an invaluable perspective to drug development, which cannot be obtained by other means. The present communication describes to our best knowledge the first systematic guidance framework for development of a patient-focused, standardized TPP with crucial aspects reviewed and validated with the patient community. With appropriate modifications to meet different conditions, we believe this guidance can be leveraged across the medicines development community.

The TPP development process we describe embeds patient experiences, preferences and recommendations from the patient community starting early in development. This is important in order to ensure that full development uses patient-relevant endpoints and label claims. “Sanity checks” during the exploratory and lead-optimization phases may help weed out unsuitable candidate molecules early. In practice, resources are a limiting factor for all R&D organizations and also for patients. A guiding principle is to incorporate patient perspectives in a systematic process starting as early as possible.

This TPP development process emphasizes the value of patient perspectives on five topics: target population, unmet need, dosage frequency and route of administration, efficacy endpoints, and acceptability of benefit/​risk profile trade-off considerations for new therapies. Table 4 provides examples of the added value from including patient perspectives on these five areas in a TPP. Other ultimate benefits from patient engagement in R&D include enhanced clinical study recruitment and retention, better adherence, and a stronger focus on health equity [22,23,24].

Arguably, efficacy endpoints are the most important of the five topics. If a TPP includes patient-relevant endpoints (accepted by regulators) in registration trials, a drug which has gone through successful clinical development can be launched with patient-relevant label claims. This beneficial outcome may be complicated to achieve. To incorporate patients’ perspective on the relevance and burden of specific symptoms is likely to be fairly straightforward. The identification of appropriate endpoints to measure such symptoms will need discussions involving many different disciplines and stakeholders including regulatory bodies, yet, difficulties should not diminish the importance of this topic. The growing use of Core Outcome Sets offers an encouraging path forward. A core outcome set is a minimum set of outcomes that should be measured and reported in all clinical trials undertaken in a specific health condition [25]. Core Outcome Sets are developed with patients and carers included in the process to identify patient-relevant outcomes [26].

The framework presented here relies on close multidisciplinary collaboration and will require multiple stakeholders to consider how to implement its components into existing processes. R&D organizations will need to ensure that adequate human and financial resources are allocated to the task of successfully integrating patient input into an updated TPP before the next development milestone. For commercial R&D organizations, sufficient time and resources must also be invested in translating a company TPP with its internal jargon into a patient-friendly version. Internal, medical, and scientific vocabulary ensures rapid and specific communication among and between researchers and regulators. Yet patients’ views will only be valuable if advisers have a good understanding of the characteristics and goals formalized in a TPP. Conversely, patients’ views, expressed in lay language, need to translate back carefully into developer terminology for internal processes to properly reflect patients' perspectives. This two-way information exchange will require strong internal and external communication skills.

For a commercial R&D organization, information about patient experiences, needs and priorities is acquired through many channels. There is a risk that resources are spent unnecessarily on developing new engagement with patients when relevant, up-to-date information is already available from existing sources. It is helpful to research such data and identify remaining questions before engaging with patients, to ensure relevant questions are asked and relevant information is provided by patient representatives.

Information relevant to patient engagement is to a large extent qualitative and unstructured. There is great interest in developing new or repurposing existing methods such as natural-language programming to expand the capacity of qualitative researchers to analyze larger amounts and more diverse data types. These methods can increase sample size, reduce project time, and significantly reduce costs. Standardization of methods will allow new data to be analyzed using the same interpretation and identification [27]. Such developments could further enhance the quality and reliability of patient-derived insights in the future.

The implementation of patient-focused, standardized TPPs with key aspects validated with the patient community brings a number of potential benefits: increased patient-focused relevance, closer alignment with regulatory expectations, and labels which include patient-relevant endpoints. In our experience, the patient community has been appreciative of their participation in these processes, reporting an improved understanding of their lived experience and a potential for their input to have greater influence on the aspirational profile of a drug in development. In a wider perspective, patient engagement can lead to enhanced clinical study recruitment and retention, and a stronger focus on diversity, health equity and access to treatments. In addition, clinical adoption of new treatments may be increased if claims and associated communications are tailored closer to those needs that matter most to patients.

There have been doubts about the degree of awareness of the importance of a more evolved patient voice among rank-and-file drug reviewers and pharmaceutical decision makers [28]. The development of patient-focused, standardized TPPs represents a route towards more widespread awareness among those closely involved in the development processes.

With time, the process we describe will provide the patient community with greater experience of the full process from providing patient input into the early TPP to seeing the drug come to market. The formalization of “what does success look like” for a specific drug in a TPP and the focus on five areas of relevance may provide clarity on the information R&D organizations use in drug development. Such knowledge would help patient organizations focus their communications to enhance their influence with R&D organizations.

There have recently been calls for the development of “Patient experience data dossiers” which would be owned by patient organizations and would consolidate evidence from various sources, including diagnosis experiences, clinical and economic aspects of the disease, and perspectives on available treatments and unmet needs [29, 30]. Such dossiers would be aids to specific and meaningful discussions between patients and numerous healthcare stakeholders, and could be a useful resource for any organization seeking to develop a patient-informed TPP.

In conclusion, the push to integrate patient lived experiences and needs into the TPP and to do this as early as possible has the potential to enhance the medicine development process and provide additional value to all stakeholders. Over the coming years we hope a greater experience with the process in our and other R&D organizations will provide useful tweaks and adaptations to optimize the TPP guidance framework.