Guest Column | August 25, 2022

How Can We Improve Patient Centricity In Benefit-Risk Assessments?

By Jeff Parke


The goal of patient centricity and including patients’ voices is now a top priority across our entire ecosystem. This includes regulators, academia, and pharma. Pharma-patient partnerships have thrived because of the autonomy and confidence patients have acquired. Patients who have experienced the disease firsthand are an invaluable resource. They can clarify their priorities and needs well before we begin to discover and develop new therapies.

However, the industry approach to patient engagement has been fragmented. Some still consider patient involvement as a nonessential activity in medical product development. Patient involvement can be perceived as difficult and time-consuming. There is also a lack of valid methods and guidance from regulators, so it is uncertain whether data about patients will be accepted for regulatory decision-making. Patients may not be motivated to participate in premarket benefit-risk assessments, as they could make it more difficult for some patients.

Many initiatives, including those of the Clinical Trial Transformation Initiative (CTTI), as well as others, like the Patient-Centered Outcomes Research Institute (PCORI), have given us valuable insights into patient engagement. These initiatives give us a look into the types of metrics and operating models that work best, as well as the skills and experience that are most effective. 

Is Finding Common Ground Mission Impossible For Risk-Benefit Assessments?

Patients' evaluations of the benefits and risks of treatment will greatly be influenced by how they feel the treatment affects them. The assessment of the benefits and risks of a treatment depends on many factors,  including the degree of uncertainty about the benefits and/or risks, severity, stage, and available support for emotional and physical management.

Industry, regulators, and patient groups increasingly recognize the value of assessing patient preferences and incorporating them in benefit-risk assessments and are exploring how to do this, but there is no consensus about the extent and nature of patient involvement.

The FDA's regulatory review is based on the benefit-risk analysis. The European Medicines Agency (EMA), however, has also taken steps to incorporate patient preferences and values into regulatory decision-making by revising its approach to interacting with consumers and patients. Regulators are open to the possibility of reviewing and using preferences data from patients, but they remain cautious. Their view is that patient-centered value measures can only be adopted if scientifically supported methods are able to reliably quantify patient preferences. Contrary to clinical trials, any regulatory decision must consider the population-level benefits and risks rather than those of a particular group or individual. Common concerns include how patient preference measures can capture the diversity of populations at risk.

Stakeholders are beginning to realize that patient perspectives can make us more confident about product development, regulatory review, post-market surveillance, and other decisions.

Becoming Partners  

To truly integrate patients into healthcare product development and life cycle management, consensus will be required from all parties to ensure that they work together to find common solutions and share a common path. It is vital to promote dialogue, especially among people who may approach the issue from different perspectives. Only then can we overcome the current barriers.

Although cultural shifts are necessary among sponsors, patients, and regulators, some first initiatives have demonstrated that it is possible. Take, for example, Patient Preferences in Benefit Risk Assessments During the Drug Life Cycle (PREFER), a public-private collaborative research project under the Innovative Medicines Initiative (IMI), aimed to determine when and how patients' preferences regarding benefits and risks should be considered when making decisions about medicinal products.

We must recognize that each approach is unique and that we must share our knowledge and experience to make consistent progress in understanding the best ways that patients, regulators, and research sponsors can work together to improve medical development.

We need to work with all stakeholders to develop scientifically sound metrics that can reliably assess the preferences of patients, create frameworks to incorporate that information into product development, as well as examine how data from patient experiences can be used to inform structured benefit-risk assessments in regulatory decision-making.

Our industry is built on the support of patients and caregivers, as well as their voices. They are the driving force behind all life sciences organizations, whether it's developing new scientific mechanisms at a lab bench, making clinical studies less difficult, creating oncology therapies that make cancer manageable, or engineering technologies that can cure a life-threatening rare condition in a child. Our industry was built on patients, and they will always be the core of what we do. So, why are patients misinformed that the pharmaceutical companies truly understand their needs?

The Role Of Patient Advocacy Groups

A Medical Device Innovation Consortium survey found that patients trust and have better experiences with patient advocacy groups (PAGs). Patients can reach out to PAGs via phone, social media, or in person to get information about everything from treatment information to education. Patients feel supported emotionally and educationally through PAGs. They are more personal and have more frequent interactions than their pharma counterparts.

Our industry claims to be "patient-centric." We use the phrase "mission-driven" often to describe our passion and ourselves. To help show the unmet needs, we embrace and share patient stories. Yet, we must improve the way we serve the patients who are at the heart of our industry.

We are an industry that values connection and can't exist apart from each other. Effective patient engagement begins with building strong relationships with PAG leaders, bridging our differences to provide patients with the support they require and deserve.

It is imperative that companies increase awareness among all stakeholders about the importance of patients' views in benefit-risk assessments, to share existing approaches and identify challenges, as well as to recommend next steps to bridge those gaps.

Notes For Additional Reading

  • Lamberti MJ, Mathias A, Myles JE, et al., “Evaluating the impact of patient recruitment and retention practices,” Therap Innov Regul Sci. 2012;46(5):573–80.
  • Sung N, Crowley W, Genel M, et al. “Central challenges facing the national clinical research enterprise,” JAMA 2003;289:1278–87.
  • Challenges in Patient Recruitment, Implementation, and Fidelity in a Mobile Telehealth Study, Justine Baron, Shashivadan Hirani, and Stanton Newman.
  • A Report of the Science of Patient Input Program of the Medical Device Innovation Consortium (MDIC), Matthew McCarty and Michael Otlewski, 2019.

About The Author:

Jeff Parke is a clinical operations professional with more than 17 years of experience in roles in consulting, project management, study management, and site activation, spanning the therapeutic areas of oncology, neurology, and rare diseases. In addition to executing clinical development programs, he also has expertise with vendor selection and oversight, site selection, and patient recruitment.