Driving assessment

How can we improve patient orientation in benefit-risk assessments

By Jeff Parker

The goal of being patient-centric and including the voice of patients is now a top priority across our entire ecosystem. This includes regulators, academia and the pharmaceutical industry. Drug-patient partnerships have flourished because of the empowerment and trust that patients have gained. Patients who have experienced the disease first hand are an invaluable resource. They can clarify their priorities and needs long before we begin to discover and develop new therapies.

However, the industry approach to patient engagement has been fragmented. Some still view patient involvement as a non-essential activity in the development of medical products. Patient engagement 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 patient data will be accepted for regulatory decision-making. Patients may not be motivated to participate in pre-market benefit-risk assessments, as this may make it difficult for some patients.

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

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

Patients’ assessments of the benefits and risks of treatment will be greatly influenced by how they think the treatment affects them. Assessing 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 the support available for emotional and physical management.

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

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

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

Become Partners

To truly integrate patients into health 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 essential to promote dialogue, especially between people who may approach the issue from different angles. Only then can we overcome the current barriers.

Although cultural shifts are needed among sponsors, patients and regulators, some early initiatives have shown that it is possible. Take for example, Patient Preferences in Benefit-Risk Assessments During the Drug Lifecycle (PREFER)a collaborative public-private research project under the Innovative Medicines Initiative (IMI), aimed at determining when and how patient preferences regarding benefits and risks should be taken into account when making decisions about medications.

We must recognize that each approach is unique and that we must share our knowledge and experience to make steady progress in understanding the best ways 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 patient preferences, create frameworks to incorporate this information into product development, as well as examine how data from patient experiences can be used to inform structured assessments of benefits and risks. in regulatory decision-making.

Our industry relies on the support of patients and caregivers, and their voices. They are the driving force behind every life science organization, whether developing new scientific mechanisms on a lab bench, making clinical studies less difficult, creating oncology therapies that make cancer manageable or design technologies that can cure a life-threatening rare disease in a child. Our industry was built on patients, and they will always be at the heart of what we do. So why are patients misinformed that pharmaceutical companies really 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 contact PAGs by phone, social media, or in person for information on everything from treatment information to education. Patients feel emotionally and educationally supported through PAGs. They are more personal and have more frequent interactions than their pharmaceutical counterparts.

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

We are an industry that values ​​connection and cannot exist separately from each other. Effective patient engagement begins with building strong relationships with PAG leaders, bridging our differences to provide patients with the support they need and deserve.

It is imperative that companies raise awareness among all stakeholders of the importance of the patient perspective in benefit-risk assessments, in order to share existing approaches and identify challenges, as well as recommend next steps to address these shortcomings.

Notes for further 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 Recruitment, Implementation, and Patient Loyalty in a Mobile Telehealth StudyJustine Baron, Shashivadan Hirani and Stanton Newman.
  • A report from 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 over 17 years of experience in consulting, project management, study management and site activation roles spanning the therapeutic areas of oncology, neurology and rare diseases. In addition to running clinical development programs, he also has expertise in vendor selection and oversight, site selection, and patient recruitment.