Each month, we gather a few partner organisations from the BEAMER project to discuss one of the six dimensions of quality care—safety, effectiveness, patient-centredness, timeliness, efficiency and equity—as it relates to adherence to treatments. Today, we’ll speak with representatives from patient organisation World Duchenne Organisation, biopharmaceutical company Pfizer and research institution IDIAPJGol.
You’ll learn more about who they are and why they care so much about improving adherence across Europe. You’ll also gain insights about how their specific roles within the BEAMER project can help prove critical to achieving the quality care that comes when we work together towards better health outcomes.
My name is Elizabeth Vroom, and I’m the chair and co-founder of the World Duchenne Organization (WDO).
I started advocating for Duchenne muscular dystrophy when my son was diagnosed at 2 years old. He is now 32.
WDO is a global umbrella organisation of 50 national patient organisations across 38 countries and an eligible member of the European Medicines Agency. WDO is dedicated to finding viable treatments and a cure for Duchenne and Becker Muscular Dystrophy, promoting good standards of care and informing parents and people living with the condition.
Only people with disabilities and diseases know what it means to have this condition. This means they can bring a different perspective to caregivers, researchers and policymakers. Their questions and needs are based on their own experiences, interests and vision.
By working together at an international level, we can achieve more. And this is the starting point for our strategy at the World Duchenne Organization.
My name is Claire Everitt, and I run the Engineering Team for home-use medical devices at Pfizer.
We aim to develop products that are easy to use and can address patients’ needs. Our research with patients has helped us to identify potential patterns of unmet need that, when resolved, could enable these individuals to take better control of their conditions and improve their health outcomes and quality of life.
We are excited to connect with patients and healthcare industry professionals through the BEAMER project as we work to tackle these longstanding problems and advance healthcare support.
My name is Carles Vilaplana, and I am a data analyst and researcher at the Institut Universitari d’Investigació en Atenció Primària (IDIAP Jordi Gol). My PhD centred on adherence to cardiovascular medications.
Within this organisation, I work in the Drug Study Unit where we study the effectiveness and safety of drugs in the Catalan Primary Care (PC) Public Health System. The research undertaken consists of studying the effectiveness and safety of treatments with real-world data. We accomplish those objectives via pharmacoepidemiologic and pharmacovigilance studies with observational data (Electronic Health Care records-SIDIAP) and prospective studies (independent clinical trials). One of our main lines of work and study is the evaluation of drug effects based on the level of adherence.
Quality care is safe, personalised and patient-centred care based on an individual’s needs and preferences. Quality in care means having access to the Standards of Care (SoC), a care plan based on patient preferences, and treatment options whenever available.
In addition, quality care is evidence-based. This means that it is documented properly, so the impact and follow-up of certain interventions are monitored accordingly, revisited and continuously improved.
An aspect of quality care I am focused on is ensuring treatment is safe, effective and designed to address patients’ needs. In addition to developing medications that meet high-quality standards, biopharmaceutical companies design devices and accessories to help patients take and look after their medication, and administer support programmes concerning their broader needs. For me, quality care requires understanding the needs and priorities of the patient in the context of their routine and daily life, to ensure that we can design products which could positively impact quality of life.
Quality care means offering high-standard care that responds always to patients’ needs and preferences whilst ensuring safety and well-being. For us, this requires supporting patients and clinicians with evidence about all relevant treatments and core aspects like safety, efficacy and efficiency. By doing this, we support these individuals during treatment decision-making processes and guarantee the best quality care we, and our system, can offer.
Ensuring the safety and efficacy of our medicines and vaccines is deeply embedded in the development process for any new products. Clinical trials are designed to ensure any new product meets these criteria, which are clear prerequisites for approval.
However, safe and efficacious medicines can still be ineffective in the real world if they are not taken as intended, which can negatively impact patients’ health. Patients have different reasons for not taking prescribed medication, which can be very personal, but in practice, people often miss treatments unintentionally or due to lack of necessary support rather than through choice.
Our goal through BEAMER is to ensure patients have full knowledge of the potential consequences of non-adherence, and to explore how providing increased support—tailored to specific patients’ needs and preferences—could be a benefit to patients. Participating in BEAMER provides an opportunity for us to better understand what these individuals require and to embed these insights into the design of our future products and support programmes.
WDO represents the global Duchenne and Becker MD patient community.
The DMD community is one of the oldest and most impactful communities in the field of rare diseases. Our goal is to bring the perspectives of patients with rare diseases to the consortium and leverage our regulatory experience in paediatric and rare disease medicine development. We want to share our everyday, true-life experiences as patients and carers with this project.
In collaboration with the rest of the partners and their expertise, we strongly believe that the project can deliver tools and methodologies capable of addressing the challenges in treatment adherence and help invite better outcomes that improve patients’ quality of life.
The benefit of working in this type of consortium is that it brings together colleagues with a diverse range of skills and insights which, although complex at times, is the best recipe for success. To solve such a complex problem, we need insights from all stakeholders, starting with patients and patient organisations and including other industry professionals. Incorporating the voices of patients and healthcare professionals will help to ensure that we design a model that is relevant, practical and easy to use. And, including the biopharmaceutical perspective is significant, because it will help guarantee that the model is simple to implement when developing future patient support programmes.
From IDIAPJGOL, we work every day to study adherence behaviour of the most prevalent diseases, especially as it relates to efficacy, effectiveness and safety. IDIAPJGOL will provide the BEAMER project with the scientific expertise and knowledge about adherence, drug safety and evaluations of drug combination adherence. We also have a lot of experience in analysing data from primary care electronic health records.
Our goal in this consortium is to support methodologically and with up-to-date know-how as it concerns treatment adherence. Like our colleagues, we truly believe in the aim of this project.