The landscape of healthcare is constantly evolving. But today it’s all obvious that incorporating the patient’s perspective into value strategies has become essential.
Health agencies such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) are willing to approve treatments that the patient really needs. Healthcare agencies and, more generally, healthcare systems around the world want to provide care that is not only effective, but also patient centered. To achieve this goal, they need to understand and integrate patients’ needs, preferences and experiences into all phases of new treatment development.

This article explores practical ways of integrating the patient perspective into value strategy, to ensure that future patient healthcare solutions are not only clinically effective, but also fully meet patients’ deepest needs. To answer the question “How to integrate the patient perspective into your value strategy?”, we’ll start with possibilities on how to integrate the patient’s perspective right from the clinical development phases, moving on to possibilities during the market access phase and finally ending with suggestions once the product is on the market, including with certain subtleties depending on the therapeutic area.

Clinical development
Engaging patients in early decision-making processes is possible and advantageous. Choosing a patient-centric approach, as early as possible can be beneficial in order to select the soonest the solution that best meets the patient’s needs. Engaging patients from the outset can be realistic by involving them in the endpoint optimization process, for example. This can be very useful in ensuring that the primary and secondary endpoints match patient expectations.

Moreover, when assessing the feasibility of a protocol, it is also possible to integrate the patient’s point of view. Indeed, testing a protocol by asking patients directly to thank the Carenity community in order to optimize patient recruitment and retention is also a way of ensuring that the majority of future patients will be willing to take part in the study.
Involving patients in early decision-making processes can also help anticipate possible blockages in the target product profile as early as possible, such as improving the acceptability and adherence of a new drug.

Market access
Now regarding market access, understanding the burden of disease and everyday challenges in terms of quality of life for patients is essential. Indeed, it has become a key factor in the construction of applications for market authorization, price negotiation and reimbursement.
And to obtain this type of information on the burden of disease or quality of life, it’s obvious that asking patients directly about their needs, preferences and perspectives is the way to go.

Preference studies are a means of identifying which treatments are preferred (in terms of route of administration, for example) in order to justify to the regulatory agencies that the choices are validated by the patients who will be the users of the treatments.
The perspective of patients and their caregivers is now essential for regulatory agencies who want to authorize treatments on the market that will be useful and that patients really need.

Rare disease
Beyond the clinical development and market access phases, it is also important to collect patient data throughout the life of the treatment, including when it is on the market.
Integrating the patient’s point of view can also prove beneficial in therapeutic areas where there is relatively little literature, such as rare diseases. Indeed, reaching patients directly in this type of context, where data is hard to come by, is extremely useful in creating value where it is needed for the patient.

Conclusion
Integrating patients’ needs, preferences and experiences into all phases of new treatment development is possible, from clinical phases to end of product life cycle. Carenity, with its community of over 500,000 members worldwide, can help you to integrate the patient perspective into your value strategy.

Today, medical research and medicine in general are seeking to develop an approach more patient-centered. There is a trend in concepts such as collaborative decision-making, precision medicine or patients having the ability to access their medical records and even to play an active role in their own health.

Indeed, patients can generate data (Patient-Generated Health Data (PGHD)) and can even contribute themselves to better health outcomes. Patients can also improve communication between them and healthcare providers through self-reported data.

1. Patient generated data

Patient-Generated Health Data (PGHD) is characterized as information produced by patients themselves. It is “health-related data created, recorded, or gathered by or from patients (or family members or other caregivers) to help address a health concern”. The notion of Patient-Generated Health Data was introduced by the Office of the National Coordinator for Health Information in the USA.

The primary origin of PGHD is the use of mobile health applications and internet of things such as connected objects, medical and/or wearable devices. The benefits of PGHD are diverse, including improved monitoring of chronic health conditions outside clinical environments. PGHD represents a variety of information, including medical history, treatment records, biometric data, symptoms, and lifestyle choices. Examples include monitoring blood glucose or blood pressure using home health equipment and tracking exercise and diet through mobile apps or wearable devices (watches).

2. Self-reported data

Self-reporting is a method used for data collection in epidemiological and medical research. This approach is based on participants answering the researcher’s questions without the researcher intervening directly. Examples of self-reporting methods encompass questionnaires, surveys, and interviews.

Self-reported data may originate from various sources, including social networks or online studies. For instance, platforms like Carenity can conduct surveys directly with patients thanks to their large community of over 500,000 members.

One of the best-known forms of self-reported data are patient-reported outcomes (PROs). PROs are defined by the FDA as “any report of the status of a patient’s health condition that originates directly from the patient, without interpretation by a clinician or any other party.” PRO instruments are validated questionnaires or scales that ask patients to report their symptoms, physical functioning, emotional well-being, preferences and health-related quality of life. (For more information on PROs and how Carenity can help you choosing one, see our article).

3. Benefits of patient-generated health data and self-reported data

A patient-centric approach using patient-generated health data and self-reported health data is beneficial because, as the patient is responsible for capturing and recording the data, they decide whether or not to share their health data. It is a prospective approach based on patient explicit consent meaning less complex regulatory requirements and faster access to data (compared to secondary databases), as well as less complex data linkage methods.

Plus, Carenity will soon offer the ability to collect Personal Health Records (PHR) from patients, encompassing a wide range of data including images of prescriptions, medical reports, clinical data, and medication information. These diverse datasets will be consolidated to create a more comprehensive patient profile, integrating complementary information for enhanced understanding and management of individual health needs.

4. Real word data

Patient generated and Self-reported data participate to the contribution of real-world data (RWDs). RWDs are data collected on an observational basis rather than as part of a traditional randomized controlled trial.

According to the FDA “Real-World Data (RWD) are the data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources”. There are various forms of RWDs such as texts, imaging, and networks from electronic health records, medical claims, data extracted from product and disease registries.

Figure 1: RWD potential sources

Real-world data (RWD) serve a variety of purposes, including acting as historical controls and reference groups in controlled trials. They constitute a valuable and extensive source of data, offering a cost-effective alternative to conventional clinical trials for data collection.

When appropriately utilized and analyzed, RWD have the potential to generate reliable and unbiased real-world evidence (RWE). This evidence encompasses clinical insights into the usage, as well as the potential benefits or risks, of medical products. It is derived through thorough analysis of RWD, providing valuable insights into real-world healthcare scenarios.

Conclusion

Patient-Generated Health Data (PGHD) holds significant potential for facilitating the delivery of an efficient and patient centered care according to the National Academy of Medicine, in addition to the possibility to increase patient awareness. Patient reported outcomes (PROs) also permit to improve patient care through assessing the real-life experiences of the patients.

Real-world evidence (RWE), as clinical evidence, can be generated and is useful throughout the full drug development cycle. RWE can play a role during the drug development phase, to enhance the comprehension of diseases by integrating the patients’ perspective. Obviously, during the Health Technology Assessment phase and the access phase, RWE can permit to have a deeper perspective about the management of a disease and various treatments. And, even in the post-access phase, to evaluate whether and how a new treatment, once integrated into a healthcare system, fulfills its objectives.

Sources:

https://www.ncbi.nlm.nih.gov/books/NBK580630/
https://pubmed.ncbi.nlm.nih.gov/34799255/
https://www.ncbi.nlm.nih.gov/books/NBK568438/
https://www.oecd-ilibrary.org/sites/d4751fff-en/index.html?itemId=/content/component/d4751fff-en#:~:text=How%20individuals%20assess%20their%20own,indicators%20that%20only%20measure%20survival.
https://bmcmedresmethodol.biomedcentral.com/articles/10.1186/s12874-022-01768-6
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8323556/
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4456898/
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4862344/#:~:text=Self%2Dreporting%20is%20a%20common,questionnaires%2C%20surveys%2C%20or%20interviews
https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence
https://www.ema.europa.eu/en/documents/other/executive-summary-patient-experience-data-eu-medicines-development-and-regulatory-decision-making-workshop_en.pdf
https://www.argenx.com/sites/default/files/media-documents/argenx_Policy_Event_Consensus_Paper.pdf

The integration of patient perspectives and experiences is becoming increasingly important in today’s clinical research, especially during the development of health products or services. Indeed, regulatory authorities such as FDA or EMA are increasingly demanding this type of data coming directly from patients in order to approve products that meet a real need for patients.

This type of data coming directly from patients can be generated thanks to patient-reported outcome (PROs).  PROs are defined by the FDA as “any report of the status of a patient’s health condition that originates directly from the patient, without interpretation by a clinician or any other party.” PRO instruments are validated questionnaires or scales that ask patients to report their symptoms, physical functioning, emotional well-being, preferences and overall quality of life.

Progressively, longitudinal observational and experimental studies have incorporated more and more data coming directly from patient’s experience, especially thanks to PROs.

PROs complete traditional clinical and biological data in assessing not only the safety or effectiveness of a product but also the real-life experiences of the patients. Traditional clinical endpoints such as survival or tumor shrinkage are still essential, but it is also important to consider the impact of the disease and treatment on patients’ health-related quality of life.   Using PROs in healthcare is pivotal to support shared decision-making, compare interventions, improve patient care, influence regulatory decisions and optimize health economic evaluation, amongst others.

How to choose the right PRO?

Because of the growing need for data coming directly from the patient for the development of healthcare products and services development, choosing the right PRO has become a critical step in the process and it raises the question of “How to choose the right PRO?”   

Patients provide insights in a variety of domains like symptoms (such as pain, fatigue), functional status (like sexual, bowel, or urinary functioning), wellness (physical, mental, social), quality of life, or satisfaction with care or treatment. To improve decision-making in patient-centered outcomes research (PCOR), it is essential to measure these PROs in a standardized manner but always adapted to the context (pathology, product or service concerned, patient population, profile) using validated questionnaires that demonstrate specific measurement properties.

Due to the wide variety of instruments available, it can be complex to identify the most appropriate and suitable PROs to use in a specific context. There is no consensus on a standardized method for selecting PROs, considering the scientific background and the regulatory recommendations.

How Carenity can help you?

According to Carenity, the selection of the most effective PROs should take into account patients’ needs in addition to scientific and regulatory expectations. This is why Carenity has developed a method named Patient-Driven Outcome Selection (PDOS): A patient-focused approach for selecting outcomes using patient-generated data that are representing meaningful domains for patients as best as possible.

This patient-centric, structured, replicable and generalizable method for PROs selection and development using patient-generated data was developed and tested as pilots in 3 therapeutic areas: lung cancer, breast cancer and diabetes.

PDOS methods 

The PDOS method is a four-step methodology:

Firstly, it is important to start with a literature review of patient-centered researches and clinical trials to determine which PROs are frequently used in a specific therapeutic area.

Secondly, outcomes and sub-domains identified are put into perspective with medical experts’ perceptions available health guidelines, and HTA bodies’ expectations.

Then, in the third stage, an extensive direct-to-patient survey aims to rank the domains by order of importance from the patient perspective and highlight potential additional domains to be assessed.

Finally, a gap analysis is performed to identify the most suitable PROs to use.

Application example of PDOS method in breast cancer

Regarding breast cancer, the literature review showed that some outcomes were well covered by existing PROs: Quality of life (QoL), survival vs QoL considerations, satisfaction with breast(s) and cancer healthcare pathway. On the other hand, some outcomes were partially or poorly covered (copying strategies, satisfaction and confidence in decision making). Moreover, experts’ groups recommended to consider several profile attributes QoL in breast cancer context (cancer stage, treatment strategy, complementary care, etc.).

The direct-to-patient online survey launched in several countries among the breast cancer Carenity patient communities contained socio-demographic and medical questions, as well as questions aiming to rank  the previous outcomes and identifying new ones. Needs and expectations appeared to be slightly different between domains covered by PROs and domains really valued by patients with breast cancer.

Indeed, for instance coping strategies (attitudes and behaviors used to maintain emotional well-being and to adjust to the stresses caused by cancer) and burden of the disease (lack of energy, ability to feel like a woman, pain, weight loss) were only considered as intermediate domains for current PROs whereas it was a major domain for patients. And on the opposite, satisfaction with cancer healthcare pathway was classify as a major domain in current PROs whereas it is just a minor domain from patients’ perspectives.

This first example highlights the gap between existing PROs and real patients ‘perspectives bringing to light the fact that always having in mind patient centricity is key to answer patients needs.

Application example of PDOS method in lung cancer  

Regarding lung cancer, the literature review showed that in current PROs major domains are daily life, physical functioning and well-being, emotional well-being, medical care and treatment care. Medical care and Physical functioning and well-being were also considered as major domains for patients and caregivers in the online questionnaire, but end-of-life care and caregivers’ perspectives were major domains for patients.

This second example with a different disease highlights the same point as the first example.

Testing this methodology in multiple therapeutic areas showed that this approach allows us to highlight differences according to patients’ profile and disease, and to identify domains that are under-assessed in frequently used PROMs.

Conclusion

The increasing importance of Health-Related Quality of Life (HR-QoL) in healthcare decision-making reinforces the need to choose relevant PRO measures.

The PDOS method developed by Carenity is a patient-centric and structured approach to identify the most appropriate instrument according to patients, physicians and HTA bodies. This method can support Evidence Generation Plans, optimize the selection of study endpoints, facilitate HTA discussions by backing-up the measured outcomes and encourage shared decision-making when used accros the care pathway.

Figure 1: Key steps of the PDOS method (Patient-Driven Outcome Selection)

Sources:

Enhancing-the-Patients-Voice-Standards-in-the-Design-and-Selection-of-Patient-Reported-Outcomes-Measures-for-Use-in-Patient-Centered-Outcomes-Research.pdf (pcori.org)

https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence

https://www.ema.europa.eu/en/documents/other/executive-summary-patient-experience-data-eu-medicines-development-and-regulatory-decision-making-workshop_en.pdf

Table 4, Primary criteria for evaluating and selecting patient-reported outcome measures (PROMs) for use in performance measurement – Patient-Reported Outcomes in Performance Measurement – NCBI Bookshelf (nih.gov)

Curious about Patient Experience Data (PED) and its role in regulatory approvals? As highlighted in a recent scientific publication and industry survey, Real World Data (RWD), and Patient Experience Data (PED), in addition to classic clinical trial data, are sometimes necessary to complete medical registration files.

However, they can pose challenges for companies seeking approvals. Authorities like the Food and Drug Administration (FDA) in the US or the European Medicines Agency (EMA) in Europe are increasingly demanding these types of data to approve products or confirm conditional approval, emphasizing the importance of addressing patients’ needs. 

1. From Patient Experience Data (PED) to Real-World Evidence (RWE)

A. What is Patient Experience Data (PED)? 

Patient Experience Data (PED) refers to information gathered from patients. Various methodologies collect these data, such as capturing patients’ insights into their health status, symptoms, disease progression, treatment preferences, quality of life, or the impact of healthcare through validated patient-reported outcomes that are quantitative data. Qualitative information can also be obtained through focus groups, surveys, or interviews to gain a broader understanding of patients’ experiences. 

 B. What is Patient Reported Outcomes (PRO)?  

Patient Reported Outcomes (PRO) refer to measures based on a report directly from patients, without interpretation by a clinician or anyone else, about the patient’s health condition. 

C. What is Real-World Data (RWD)? 

Real-World Data (RWD) comprises information about patient health from everyday medical practice. Various sources, such as electronic health records, medical claims, information from product or disease registries, and data acquired through digital health technologies, contribute to RWD. All these sources share the commonality of being obtained in real-life situations. 

D. What is Real Word Evidence (RWE)?  

Real-World Evidence (RWE) refers to clinical proof obtained outside conventional clinical trials through the analysis of RWD regarding the utilization, advantages, or risks of a medical product. 

From a regulatory decision-making point of view, those data about patient health from everyday life are very important because they allow to gain more insights about the patient’s perspective and point of view but also it can be interesting in terms of safety information for instance.    

Today, health authorities’ communication on the need of those patient data is improving but not everything is yet clear. 

2. Insights from “Current Practices and Challenges When Submitting Patient Experience Data for Regulatory Decisions by the US Food and Drug Administration: An Industry Survey” methodology, results, and perspectives.   

A study, published in December 2023, involved 50 participants from the industry (84%) and contract research organizations (16%) to understand practices and challenges related to the submission of patient experience data. 

 A. Study methodology

Conducted by a pharmaceutical industry research team, the study underwent pretesting with ten industry and Contract Research Organization (CRO) members to assess readability and comprehension. It was then distributed to large professional special interest groups. 

The study consisted of an online survey with two parts: one focused on the gathering, submission, and utilization of PED by the FDA in regulatory decision-making, and the second part was a best-worst exercise to identify potential PED initiatives. Eligibility criteria for participants were based on their experience with PED, requiring more than 2 years of experience. 

 B. Study results

The study indicates that to enhance the gathering and submission of high-quality PED to the FDA for regulatory decision-making, improving dialogue between the FDA and the industry is essential.   

It remains unclear to applicants for approval what information is precisely expected at the regulatory level. As not all PED are significantly important in regulatory decision-making, it is the industry’s role to emphasize the significance of their data, such as patient preferences. Improving dialogue with the FDA is deemed useful for achieving this.   

Meeting the FDA more frequently and the availability of expanded FDA guidance on the development of PED or Clinical Outcome Assessments would also be beneficial. 

 C. Main challenges highlighted 

The challenges mostly cited in PED collection was:  

• The difficulty of justifying the effort involved in collecting that data without being sure of the agency’s utilization of such data in regulatory decision-making.  

• The lack of harmonized process to understand what is needed by the FDA or EMA for instance is also a challenge, a sort of PED evidentiary standards would be useful.  

Participants of the survey also considered that the agency should justify the reasons for not using PED data when they are provided by the applicant. The participants recommend to the authorities to provide a sort of template with categories such as “data not fit for purpose”, “data incomplete” or “not addressing the regulatory decision”, “lack of data quality”, to be aware of the expectancies of the agencies.  

D. Future priorities and goals  

Developing guidance on the development of PED and a dedicated meeting pathway are among the highest priorities according to the study results, because being able to create and consequently utilize more helpful PED is important. Plus, according to the study participants PED should be considered as an integral component such as efficacy or safety data which is in line with authorities’ expectations. 

3. How to answer the agencies’ requests of gathering and submitting high-quality PED or RWD ?  

The role of patient-centered data is crucial for regulatory agencies, despite some improvements needed in terms of the use of PED and RWE information. 

 A. Solutions for obtaining patients data 

Various solutions exist for acquiring patient data as requested by health authorities. Companies, such as CROs specialized in generating such data through studies conducted directly with patients, offer viable options. Carenity, with over a decade of expertise, is one of these companies dedicated to meeting agencies’ requests for gathering and submitting high-quality PED or RWD.   

In collaboration with the industry, in high-prevalence and rare diseases, Carenity conducts studies to provide significant data, such as patient preferences or treatment preferences. Many results from Carenity’s studies, part of various day-to-day projects, are regularly submitted to health authorities and can prove valuable in completing submission dossiers. 

 B. Practical use of PRO  

The utilization of validated Patient Reported Outcomes (PRO), a current practice at Carenity, is also noteworthy for regulatory agencies. PRO reveals patients’ perspectives without biases from healthcare professionals. Additionally, studies can be conducted to define value in health, identify disease burden and unmet needs, or gain patient perspectives through discrete choice experiment studies. Carenity offers real-world studies that complement pivotal clinical data for submission for reimbursement. Beyond quantitative data, Carenity can provide qualitative data through interviews and their analysis. 

4. Conclusion  

Regulatory agencies express a desire to enhance the utilization of Patient Experience Data (PED), despite existing obstacles in transparency within regulatory decision-making. It is crucial to persist in efforts and consistently provide agencies with both PED and Real-World Data (RWD). Companies possessing the capability to generate such data, such as Carenity, emerge as dependable partners for the industry in meeting the regulatory agencies’ demand for high-quality data. 

Sources:  

https://www.ema.europa.eu/en/documents/other/executive-summary-patient-experience-data-eu-medicines-development-and-regulatory-decision-making-workshop_en.pdf 

https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence 

https://link.springer.com/article/10.1007/s40271-023-00653-8#Sec2  

https://www.fda.gov/media/124063/download

Carenity (EvidentIQ Group) has been selected as one of the Top 10 Patient Monitoring Solution Providers 2023 by Medhealth Outlook.

Medhealth Outlook serves as the backdoor to the latest technologies and trends in the healthcare, pharma, and medical industries.

We are proud for this recognition who reflects our capacity to play a pivotal role as a Patient Monitoring Solution.

Discover Lise’s rich interview conducted by Medhealth Outlook below:

Vendredi 8 octobre 2021, Michael Chekroun, fondateur de Carenity a été reçu par Jean-Marc Sylvestre dans l’émission ENJEUX & PRIORITÉS sur B SMART, pour évoquer l’actualité de Carenity. Visualiser l’interview en cliquant ci-dessous :