PROMs: Giving Patients a Voice

Why PROMs should be embraced by pharma.

Katarina Halling, PRO Group Director, AstraZeneca

April 2009 saw patient-reported outcome measures became a requirement within the NHS for four elective surgical procedures. They have since become a key tool in aiding patient-centricity within healthcare. 

In order to effectively determine the value of healthcare and improve health outcomes, we need to find out how the patient, who receives such healthcare, is feeling. How are they adapting to their treatment? What symptoms are they suffering from? How is their condition limiting daily life? In other words, what is important to the patient and what are their expectations from the treatment?

Benefits of treatment from patient perspective

The best way to access this information is to simply ask the patient. This is where patient-reported outcomes measures (PROMs) come in. PROMs are questionnaires that measure and provide information about the benefits of treatment from the perspective of the patient. PROMs essentially reflect the “voice of the patient,” free from the input and interpretation of physicians, carers and medical experts. Patient-reported outcomes (PROs) are those that cannot easily be evaluated using clinical and laboratory means, such as severity of pain and patient satisfaction with treatment.

There are different types of PROMs: generic instruments can be used over a wide range of patient populations and treatments; disease-specific measures focus on symptoms and limitations that are related to a certain disease; treatment-specific instruments focus on the symptoms and side-effects of particular treatment plans; and, patient-reported experience measures (PREMs) assess the quality of care and treatment experience of the patient.

Why do PROMs matter to pharma?

When pharma has access to data from PROMs, they can extract key lessons to help them implement changes and interventions that future patients can appreciate, such as reducing the burden of disease, preventing illness and injury, and bridging the communication gap between patient, physician, and the pharmaceutical industry.

Despite the benefits of PROMs, there is an underutilization of PROMs across the healthcare industry. Some healthcare systems don’t welcome the incorporation of PROMs into their current clinical set-up, due to the costs and resources that can be used up during systems integration.

PROMs fundamental for pharma

A spokesman for Boehringer Ingelheim says, “PROMs are an important factor to pharma in assessing a patient’s quality of life. Quality of life measures/tools such as the EQ-5D are used by companies like ourselves to assess a patient’s quality of life.”

PROMs are no longer ‘nice to have,’ but critical for successful regulatory approval, reimbursement and credibility to patients".

Katarina Halling, PRO Group Director, Global Medicines Development at AstraZeneca agrees, “PROMs are fundamental for the pharmaceutical industry. PROMs add value from guiding early development efforts through to post-approval communication to payers, patients and prescribers. At a time when regulators are defining treatment benefit as ‘how patients feel and function,’ payers ask why they should pay for a certain outcome, and patients’ voices are getting stronger. PROMs are no longer ‘nice to have,’ but critical for successful regulatory approval, reimbursement and credibility to patients.” She adds, “By applying scientific methods to listening to patients and developing or selecting PRO instruments and endpoints based on what is important and relevant to patients, we generate patient-centric claims and messages of relevance for patients, regulators, payers and prescribers.”

The results from Boehringer Ingelheim’s questionnaires are used to weigh the length of time a patient is alive and what their quality of life is throughout their lifetime. “We can, therefore, take account of not only their length of life gain, but also their quality of life changes,” they explain.

How can pharma use PROMs?

Below are three specific ways pharma can utilize PROMs to improve quality of care for the benefit of the patient:

1. To facilitate in providing care that is patient-centered

“PROMs help us not only to deliver treatments that are meaningful for patients, but they can also assist in changing the treatment paradigm,” says Halling. “They also help us facilitate open discussions between clinicians and patients about the issues that are at the center of patients’ concerns, and to develop diagnostic tools that can better tailor treatments for patients. For example, having a short scientifically-developed PRO tool in the waiting room can make the dialogue more focussed between the patient and clinician.”

The information gathered from patients can be longitudinal, meaning it can be used to track improvements in health status and outcomes of a particular patient over time, from before treatment commences through to the end of treatment. Treatment plans can be restructured based on the changing needs and desires of the patient. In other words, the care plan is adjusted to the patient, and not the other way around. Treatment-specific PROMs and PREMs are most useful for determining these factors and helping pharma identify where patient-centricity can be improved in treatment plans and medications.

2. To provide more effective clinical trials and inform the licensing of new drugs

Halling believes, “Any company wanting to be at the forefront of pharma must have a solid PRO strategy in place in early development that can be efficiently implemented in clinical trials as well as real-world evidence studies. At AstraZeneca, we have long recognized the importance of PROMs and how giving patients a greater voice can have a real impact on the success of our trials. In recent years we have, for example, started to recruit experts solely focused on PROs to help us focus more on patients and their needs as well as implement the measures in clinical trials so that the outcome can support regulatory approval, reimbursement, as well as provide patients with the information they are looking for.”

PROMs are a fundamental part of our phase III trial design, and we try to be as transparent as possible in the way they are recorded by using the methods widely accepted as the gold standard".

Boehringer Ingelheim’s spokesperson explains, “In recent Oncology and Respiratory phase III trials we used the EQ-5D to record patient’s quality of life throughout the trial. These trials were conducted with patients experiencing life-threatening conditions. The EQ-5D is considered the gold standard by NICE when recording quality of life and we have been complimented on using this within our trials.” He concludes, “In summary, PROMs are a fundamental part of our phase III trial design, and we try to be as transparent as possible in the way they are recorded by using the methods widely accepted as the gold standard.”

PROMs ensure pharmaceutical developers and patients travel along the same journey during the treatment process, with the same outcomes in mind, which are the outcomes important to the patient.When a patient feels that they are being heard and they can observe changes based on the surveys they complete, they become more invested in their own treatment and the results of clinical trials. When patients are highly engaged, adherence to treatment is better, which leads to improved health outcomes as desired by the patient, as well as more accurate clinical trial results.

PROMs are also used in clinical trials for drug licensing and verification of label claims. These are important when determining the cost-effectiveness of a drug when viewed in light of its impact on a patient’s quality of life.

3. To aid in the research and development of new treatments applicable to a population with similar needs and desires

Very often, patients tell us things that we haven’t heard from their treating physicians. This is the foundation for the selected PROMs and it enables us to develop our R&D programmes to become patient-centric and to deliver more credible, scientifically valid outcomes".

Halling says, “By listening to patients from an early stage, we are able to get a much broader picture of the things that matter to them and to understand what endpoints are most meaningful. Very often, patients tell us things that we haven’t heard from their treating physicians. This is the foundation for the selected PROMs and it enables us to develop our R&D programmes to become patient-centric and to deliver more credible, scientifically valid outcomes.”

By including disease-specific and treatment-specific PROMs that address patients’ disease symptoms, drug tolerability symptoms, function, and health-related quality of life, pharma can form a comprehensive picture of how a new treatment impacts patients for good and bad. Halling continues, “These measures can also directly translate into how we quantify the value of our products and can help to differentiate our products from the competition - which is often a critical requirement in reimbursement and in getting the medicines to patients as quickly as possible.”

“To better serve patients, we need to continue to collaborate between pharma, regulators, academia, payers and - not least – directly with patients, as this kind of cooperation can help spur more innovation in the industry and, ultimately, develop life-changing medicines for patients,” emphasizes Halling.

The Future of PROMs

The rich information that can be extracted from patient-reported outcome measures has numerous applications, all of which are directed towards meeting the patient’s needs and desired outcomes. Pharma can make use of PROMs and PREMs to provide customized care plans for individual patients, aggregate health improvement plans for populations, and program improvements for health care providers and organizations.

According to Halling, “Although pharma companies have shifted to collect more information directly from patients, there is still room for improvement. For example, we are driving for patient insight work to be done with scientific methods so that the resulting information can be used directly to inform the PRO strategy, instead of having separate pieces of market research and scientific patient insight.” She concludes, “We need to shift some endpoints from clinician-reported to patient-reported and apply the most efficient way of collecting the information.” This can be achieved through electronic diaries or mobile phones.

It is the challenge of pharma to study and conduct the adequate design and implementation of PRO instruments. This is a challenging task, but it is critical for the future of healthcare. With appropriate PROM designs, accurate data interpretation, and patient-centered data collection, the benefits for individual patients, whole populations and care providers look to be vast.

Soren Skovlund, Global Director, Patient Research and Engagement, Novo Nordisk will be presenting on "Patient Reported Outcomes as a Means to a More Patient-Centric Trial" at Patient Centric Clinical Trials Europe. Click here for more information.

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