Patient Voice in RWE: generating evidence that will impact payer and HTA decision-making

Patient Voice in RWE: generating evidence that will impact payer and HTA decision-making

With the rise of patient-centered medicine and accelerated approvals, health technology assessment (HTA) bodies and payers are looking to real-world evidence (RWE) to strengthen evidence packages and reduce uncertainties at the time of launch.

Increasingly, stakeholders are recognizing the importance of RWE that incorporates patients’ real-world experiences in understanding what treatments work, and for whom, in clinical practice.

Real-world evidence is generated through applying data science to real-world data (RWD), or data “relating to patient health status or the delivery of healthcare routinely collected from EHRs, claims, registries, patient-reported outcomes, devices/applications, etc.” Decision-makers are exploring how this evidence can supplement clinical trials and provide additional context on the effectiveness of therapies.

There is some overlap between patient experience data, which provides information about a patient’s experience with a disease or condition, and real-world data (RWD), however, they are not one and the same. For example, patient-reported outcomes (PROs) collected in an electronic health record (EHR) would be considered both RWD and patient experience data, however, lab test results in the medical record would be considered only RWD. As personalized medicine continues to gain traction, decision-makers like ICER, FDA, NPC, EMA, EUNetHTA, and EHDEN are calling for patients’ voices to be included in the drug development and assessment process. Such information – direct input from patients on how they feel and function is often lacking from RWD sources, and FDA and EMA identified such missing data as problematic for interpretation.

We spoke with Ashley Jaksa MPH, Scientific Partnerships Lead, Aetion and Chung Yen Looi, DPhil Partnerships Director, Monsenso, about the evolving landscape of patient experience data and how biopharma organizations can incorporate the patient voice into their RWD and RWE programs.

Q: Ashley, can you tell our readers a little more about what Aetion does? 

AJ: Aetion is a healthcare technology company that uses routinely collected health care data to generate RWE on the safety and effectiveness of medical treatments and technologies. We were founded by Harvard epidemiologists to develop a way to conduct scalable and transparent RWD analyses at the highest level of scientific rigour. Our RWE analytics platform helps biopharma organizations, regulators, HTAs, payers, and other researchers generate RWE to inform decisions on the safety, effectiveness, and value of medical interventions across the product lifecycle. Our platform is data fluent, meaning it can analyze almost any type of RWD, and we often work with our customers and partners to identify the most fit-for-purpose data set for their research question.

Q: Why is the patient experience important for biopharma to understand, and where in the drug development lifecycle is it most important to integrate patients’ voices? 

CL: I think we can all agree that pharma and its stakeholders want to develop safe and effective drugs and bring them to market, to improve patients’ lives. As such, asking patients directly about how a drug impacts their lives is critical. Without the patient voice, we will never know how patients experience therapies in the real world, and how medical interventions could make a meaningful difference to their lives. Capturing and incorporating the patient voice across the product lifecycle can help biopharma companies to better understand the real-world impact of their drug, treatment responders, and meaningful endpoints based on a wider, representative population, in a quick and cost-effective way. 

AJ: Similar to RWE, patient experience data is important in almost all stages of the product lifecycle—not just as a post-marketing exercise. For example, understanding the treatment pathways and patient experiences on first-line therapies can help shape what comparators and outcomes should be captured in future randomized controlled trials to differentiate the drug in the market.

Q: How can incorporating the patient voice improve market access?

CL: Patient voice is a powerful way to demonstrate the impact of a drug on patients’ quality of life to payers and HTA bodies, particularly for cost-effectiveness evaluations. Some biopharma organizations have used PRO measures on daily functioning and health-related quality of life as their primary and secondary endpoints in clinical and real-world studies, while others have directly linked PROs to their drug reimbursement contracts. Patient voice contributions can help biopharma companies differentiate their product from similar drugs for coverage, especially for payers who are tying reimbursement to the value to patients based on outcomes and have longer-term responsibilities for their populations.  Some of the common myths are that “payers don’t care about PRO evidence, PROs only matter if they make it to the label, and that PROs should only be collected in Phase IV.” The facts are that not all payers are alike. Some pay a lot of attention to PRO evidence. Payers will consider all data from pivotal trials, whether or not it is on the package insert, and coverage decisions will be made on pivotal trials, so waiting until Phase IV to collect PROs would be too late.

Q: What are the common barriers in generating high-quality, patient-centric RWE?

AJ: I think the common barriers to generating RWE and patient-centric RWE are very similar; researchers must ensure that the data exists, that the data is capturing the relevant underlying medical concept, and that it is generated in a way that limits bias and confounding. Traditional RWD sources, like claims and EHRs, often have limited patient-centric data. This is a challenge for researchers attempting to incorporate this information into their studies. However, groups like Friends of Cancer Research and data providers like Monsenso are working to ensure this information is being collected. Once it is collected, it is essential to validate that these data and algorithms are actually measuring what we are intending to measure. Finally, researchers must be cognizant of bias and confounding and control for this as much as possible through study design and analytic methods. 

CL: Adding to Ashley’s points, I think data privacy and security, patients’ burden of contributing to research, and standardized methodology to analysis and interpretation are common barriers. It may seem like a no-brainer to ensure that patient data is managed in a secure way, but a recent study showed that almost a third of health apps out there collect patient data without privacy policy, and about one in five data transmissions occurred on insecure communication protocols. Furthermore, as patients are already suffering from their disease or conditions, contributing to research could add to their burden, which is why data capture platforms should be designed in a way that is easy-to-use and that provides valuable insights to patients. Finally, there is a lack of standardization in analysis and interpretation—which is why companies like Aetion are important to ensure that critical questions are answered, data sets are relevant, and standards are followed to derive and present meaningful insights that will inform decision-making.

Q: How can biopharma generate high-quality, patient-centric RWE for decision-makers? 

AJ: In addition to addressing the common barriers mentioned above, we think it is important to follow a principled approach to study design and execution. The main reasons why decision-makers like regulators and HTAs/payers dismiss RWE are due to deficient data selection and insufficient methodology. Following principled epidemiologic methods of selecting fit-for-purpose data, avoiding common methodological mistakes, and ensuring the protocol and results are clearly communicated are essential to ensuring the decision-makers trust the evidence. This planning and execution of RWE studies is not easy; it takes forethought. Biopharma manufacturers should be considering RWE at every stage of the product lifecycle, and proactively planning for how RWE studies will be incorporated into each product’s launch. 

Q. What is the cost-benefit for biopharma of generating patient-centric RWE? 

CL: Relative to the costs of bringing a new drug to market – which could go up to billions of dollars and take over a decade, with limited success ratesthe costs associated with collecting patient-centric RWE is insignificant. In return, patient-centric evidence could provide significant savings and valuable insights as early as possible to ensure that new drugs are truly driven by patients’ unmet needs, that they impact outcomes that matter to patients, and, ultimately, that they have the greatest chance of reaching the market and making a meaningful difference to patients. 

AJ: RWE can provide value for biopharma organizations across the product lifecycle—from eliminating costs typically incurred during clinical trials to accelerating time to market. Using a validated RWE platform can take this a step further, allowing companies to run more efficient, credible studies and reach insights faster than they could with traditional line programming. By ensuring patient-centric outcomes are collected in clinical data sources, then incorporating patient-centric RWE across drug development and commercialization programs, biopharma organizations can unlock insights that deliver value not only for their company but for patients as well. 

Q: Lastly, for our readers who are not familiar with Monsenso, Looi can you kindly tell a little more about what Monsenso does? 

CL: of course, Monsenso is a digital health company that enables patient-centric care and research. We’re a spinout from the IT University of Copenhagen based on the groundbreaking research of Professor Lars Kessing and Dr. Mads Frost on bipolar disorder and depression. We were founded in 2013 and have been listed on Nasdaq First North in Denmark since 2020. Our solution enables pharma companies to collect self-reported and device-generated data directly from patients in a simple, scalable, secure, and cost-effective way. Our cloud-based platform connects patients and investigators via an app and a web portal, respectively, to collect data in real-time across clinical development, post-market studies, and clinical practice to inform decision-making. Our platform can integrate with wearable devices and sensors, and combine with other RWD sources to provide a more holistic view of the impact of a drug on patients’ lives.

Identifying postnatal depression with mHealth technology

Identifying postnatal depression with mHealth technology

Baby Blues occur within the first three days after birth and may last until a few weeks later. It is expected that between 60 to 80% of new mothers experience baby blues [1].

However, is estimated that 10 to 15% of new adult mothers develop Postpartum Depression (PPD) within the first year after giving birth, and the percentage increases up to 26% in adolescent mothers [1].

Furthermore, a recent study made with women suffering from bipolar disorder, indicated that 67% of them had a depressive episode after the first child, and they all experienced a relapse with their next birth [1].

According to Dr. Shoshanna Bennett, there are two main ways to differentiate Baby Blues from PPD. Firstly, Baby Blues always begin in the first few days following delivery and should be gone two weeks after childbirth. Besides, the symptoms are mild – frequent tearfulness, feelings of dependence and stress [2].

Unlike Baby Blues, the severity of the postpartum depression disrupts a woman’s ability to function. Therefore, if the symptoms are severe enough and last more than two weeks, it can be considered PPD and she should seek help [2].

While PPD is a major health issue for many women, it often remains undiagnosed. Although several measures have been created to detect depressive symptomatology in women who have recently given birth, the development of a postpartum depression screening program requires careful consideration [3].

Evidence-based decisions need to be made regarding two aspects. Firstly, healthcare systems need to develop an effective screening test that not only has good sensitivity and specificity, but is also quick, easy to interpret, readily incorporated into practice, and culturally sensitive. Second, health care systems should use these tools as a cost-effective, potential harm, and policies for a referral [3].

Fortunately, preliminary research suggests postpartum depression is responsive to treatment interventions thus providing a rationale for the development of a screening program [3].

The Monsenso mHealth solution can help healthcare professionals identify PPD at an early stage. New mothers can be asked to download the app and fill in self-assessments on their smartphones on a daily basis. With the self-assessments, new mothers can rate themselves on relevant parameters such as their levels of stress, anxiety, and tearfulness, as keeping track of the number of hours they slept within a 24-hour period. The system can also collect sensor data such as their level of physical activity, social activity and mobility.

Healthcare professionals can monitor new mothers on a regular basis and contact them if the system indicates any triggers or early warning signs that their Baby Blues is evolving into PPD. It is important to take into consideration that the early intervention of PPD reduces the long-term disadvantages that PPD causes for mother and child.

Current research suggests that postpartum depression has salient but selective effects on the mother/infant relationship, and child growth and development. Young children of mothers with postpartum depression have greater cognitive, behavioural, and interpersonal problems than children of nondepressed mothers. Overall, it is exposure to prolonged episodes of postpartum depression or to recurrent episodes of maternal depression that are most likely to have long-term effects on the child [3].


  1. The Environmental, Cultural, Relational and Physiological Aspects of Postpartum Depression. The Faculty of the Adler Graduate School. Joanne E. Campbell. 2010.
  2. Do you have Baby Blues or Postpartum Depression? PBS.Org. Dr. Shoshanna Bennett.
  3. Postpartum Depression: Literature review of risk factors and interventions. Toronto Public Health. 2003.
Improving the treatment of postpartum depression in teenage mothers

Improving the treatment of postpartum depression in teenage mothers

Postpartum depression (PPD) is a mood disorder that can affect women after giving birth, and the symptoms are similar to the ones presented in major depressive disorder. It is estimated that 10 to 15% of new adult mothers develop Postpartum Depression (PPD) within the first year after giving birth -and the percentage increases up to 26% in adolescent mothers. [1]

According to a survey published in 2012 by the Paediatrics journal, the highest incidence of postpartum depression occurs amongst girls aged 15 to 19, which is twice as high as their older counterparts. [2]

Why is that?

Adolescent mothers have to face more challenges than adult mothers. A teenage mother will suddenly face multiple changes in her life: she might have to quit school and be home-schooled instead, she might also need to get a job, and her social life will change drastically. Besides all of these changes, she will also become affected by a hormonal imbalance, which can lead to depression.

Due to all the hardship adolescent mothers have to cope with, they tend to rate their child’s health and their own as suboptimal. Besides, many young mothers are already under the stress of becoming new parents, which can also be linked to postpartum depression. [2]

Unfortunately, in most cases, PPD remains undiagnosed. The main problem is that it’s not always easy to distinguish baby blues from postpartum depression. Unlike baby blues, postpartum depression is a more severe problem that shouldn’t be ignored. While mothers who experience baby blues feel sorrow for the first two weeks after delivery, mothers who suffer from postpartum depression feel an overwhelming sadness or irritability a month after childbirth. Symptoms also include trouble eating and sleeping, difficulty concentrating, fatigue, loss of pleasure, reduced sex drive, and thoughts of suicide. [3]

The best treatment available for PPD is early intervention, since this condition is extremely responsive to treatment. Once the baby is born, family members can show their support towards young mothers by helping take care of the baby to help prevent postpartum depression. Besides, family members can also encourage her to seek help if she displays long periods of sadness and tearfulness.

A study at Women & Infants Hospital of Rhode Island found that an intervention program cut the incidence of postpartum depression in teenage mothers by 50%. [2]

The Monsenso mHealth solution can help identify PPD in young mothers at an early stage. Care providers could encourage new mothers to download the Monsenso app on their smartphones. The app will enable them to complete daily self-assessments in which they can rate themselves on relevant parameters such as their levels of stress, anxiety, tearfulness and the number of hours they slept. The Monsenso app makes it easier for adolescent mothers to reveal and share the current status of their mental health without feeling uncomfortable.

Furthermore, the Monsenso solution also enables healthcare professionals to monitor patients on a daily basis, and intervene when the system displays a patient presenting triggers or early warning signs. The clinical web portal also works as a two-way encrypted communication channel enabling patients and clinicians to always stay in touch.


[1] The Environmental, Cultural, Relational and Physiological Aspects of Postpartum Depression. The Faculty of the Adler Graduate School. Joanne E. Campbell. 2010.

[2] The Mental Health of Teen Moms Matters. Diana Reese.

[3] About Teenage Mothers & Depression. Michelle Bolyn. 2015.