(This article is now part of the 'Long Reads' series of our Drug Safety Matters podcast. Listen to the article and a discussion with the author here.)
In recent years, patient engagement has emerged as a transformative concept in healthcare. No longer mere recipients of medical care, patients are now recognised as valuable partners in the development, regulation, and safe use of medicines. This shift marks a fundamental change in the healthcare landscape, acknowledging the importance of incorporating patient perspectives and experiences. This article explores the concept of patient engagement, its importance, and the compelling case of valproate to illustrate the need for active patient involvement in healthcare decision-making.
The development, regulation, and safe use of medicines are complex processes that involve numerous stakeholders, including patients, healthcare providers, regulators, and pharmaceutical companies. Patient engagement in these processes is essential to ensuring that the interests and needs of patients are considered at every stage of the process.
Patient engagement involves actively involving patients and their representatives in healthcare decision-making processes. It recognises that patients possess unique insights into their conditions, treatment outcomes, and quality of life. By actively including patients in research, drug development, clinical trials, regulatory decisions, and post-marketing surveillance, healthcare stakeholders can harness this wealth of knowledge to improve patient outcomes.
In clinical trials, patient involvement helps ensure that trials are designed in a way that is relevant and meaningful to them. Patients can also provide input on the development of patient-reported outcomes measures, which are used to assess the impact of treatments on their quality of life.
Patient engagement in the regulation of medicines is also critical to ensuring that the process is transparent and that patients’ needs are considered. They may further provide input on the labelling and packaging of medicines to make medical information easier to understand for other users.
Their value even extends to the safe use of medicines, where they may be involved in developing educational materials about medications to provide patients access to accurate and up-to-date information about their treatments. Patients may also be involved in the development of adverse event reporting systems, helping to ensure that adverse events are reported in a timely and accurate manner.
By involving patients in these key processes, healthcare interventions address the needs, preferences, and priorities of the individuals they are intended to serve. Patients bring a personal perspective and insights that traditional research may overlook. By involving patients in decision-making, healthcare systems become more patient-centred and foster a sense of ownership and empowerment among patients. Patient engagement enhances transparency and trust between patients, healthcare providers, regulators, and pharmaceutical companies. When patients are engaged, they feel heard and respected, leading to increased adherence to treatment plans and better health outcomes.
One notable example of patient engagement's impact is the case of valproate, a medication used primarily for the treatment of epilepsy and bipolar disorder. In the past, valproate has been associated with severe birth defects if taken during pregnancy. Unfortunately, many women were not adequately informed of this risk.
However, through patient engagement initiatives, exemplified by patient testimonies at a public hearing on the drug, the voices of affected individuals and advocacy groups were heard and considered by regulatory authorities. The information and testimonies shared during the public hearing influenced the European Medicines Agency's (EMA) assessment of the risk-benefit profile of valproate, as well as their recommendations regarding the use, labelling, and risk minimisation measures for the drug.
Patient advocacy groups, such as the Independent Medicines and Medical Devices Safety Review in the United Kingdom, played a pivotal role in drawing attention to this issue. They raised awareness about the risks associated with valproate during pregnancy and provided further advice and support to affected women and their families. The involvement of these patient groups prompted regulatory bodies and pharmaceutical companies to act, leading to improved warnings, strengthened regulations, and enhanced patient safety measures in the use of valproate.
The Valproate Toolkit, developed by the Medicines and Healthcare Products Regulatory Agency (MHRA) in collaboration with patient groups, is another example of patient engagement in action. This resource provides comprehensive information and tools to support healthcare professionals in advising women of childbearing age about the risks and benefits of valproate therapy.
The EMA and MHRA have also engaged with patient advocacy groups and organisations to gather patient experiences and perspectives. They have conducted surveys and consultations to better understand the impact of valproate on patients' lives and to identify areas where additional support and information are needed.
These efforts have helped increase awareness among patients and healthcare professionals about the risks associated with valproate. Patients are now better informed about the need to use effective contraception while taking valproate and the importance of regular reviews of treatment options. Furthermore, the EMA and MHRA have actively promoted shared decision-making between patients and healthcare professionals so that patients may make informed choices about their treatment.
Patient engagement has emerged as a crucial driver of progress in healthcare. Recognizing patients as partners in decision-making processes has the potential to transform the way medicines are developed, regulated, and used. The case of valproate highlights the importance of patient engagement in addressing healthcare challenges and improving patient safety. The active involvement of patients, healthcare providers, regulators, and pharmaceutical companies fosters a more patient-centred approach, builds trust, and ultimately enhances patient outcomes. Embracing patient engagement is not only an ethical imperative but a practical necessity to shape a healthcare system that truly meets the needs and aspirations of those it serves.
