Though recipients of medicines, input from patients has historically been limited within healthcare systems, and by extension health research. This has resulted in their experiences, views, and decisions regarding medicine use sometimes being downplayed or overlooked. Over the past few decades, however, patients have become increasingly involved in all aspects of their care. So it is not surprising that this patient-centric approach has extended to drug development and post-marketing surveillance activities, including the evaluation of harm. As such, patients are being involved in the direct spontaneous reporting of suspected adverse drug reactions (ADRs), and in issues relating to signal detection and evaluation, risk management, medication error assessment, benefit–risk assessment and risk communication (Smith 2016).

Patient representatives are also now more often involved in the planning, operation, interpretation and reporting of clinical trials, although there is undoubtedly more work to be done (Sacristan JA 2015, EMA 2013). More work is needed, for instance, to ensure safety datasets generated during trials are not only collected appropriately, in valid, standardised ways where possible, but that they also closely represent the participants’ experiences. This is a methodologically challenging area due to relatively higher number of (expected or unexpected) safety end points compared to efficacy end points. Recent guidance as to how to incorporate patient-reported adverse event (AE) data is welcome, though clarity is needed as to how these will be handled (assessed and reported) in relation to traditional AE data, whereby a clinician interprets patients’ reports which are then categorised through dictionaries, and summarised (when mentioned) in reports and publications (Banerjee 2013).

A significant step forward is the adaptation of the NCI Common Terminology Criteria for Adverse Events (CTCAE) into a patient-reported outcome tool, and a study in a clinic setting that showed cancer patients who reported their symptoms to their cancer care providers using a web-based survey lived five months longer than those patients who used standard reporting methods (Blinder 2017). Exploration of whether this measurement approach translates to use in clinical trials and/or for other diseases or geographical settings is needed, as is methodological work to investigate the factors influencing reporting by participants/patients. Even if the latter are given sophisticated tools to monitor symptoms, these may not be feasible for use in low-resource settings, and there may be context-specific barriers to reporting to be overcome, relating to AE data but also the other variables (such as medical histories and concomitant medications) required for AE interpretation (Allen 2013). Reflecting on the growing body of health research community engagement activities in low and middle income countries may help with progress in this area (

Aside from clinical trials, post-marketing spontaneous reporting of potential ADRs is also evolving to ensure the patient ‘voice’ is heard, incorporated and represented. Some countries have had systems in place for decades for the public to directly report suspected ADRs, though this is a relatively recently development in others, including Europe. There are indications that allowing patients to self-report provides additional useful information to supplement health workers’ reports and helps identify new potential ADRs (Berrewaerts 2016).

Another important contribution is the analysis of online reports, stories or narratives about people’s experiences of illnesses, including side effects of medicines. Aside from methodological research to try to make sense of references to potential side-effects of medicines in general web traffic, there are organisations who actively solicit such data (Berrewaerts 2016):

  •; a website where people share their health data online to track progress of their illness, ultimately to help others with the same condition or experience. An integral post-marketing surveillance component aggregates data obtained to reveal new insights about living with particular conditions and their treatments, providing a much-needed personal context. Adverse event date collected in this way are now being shared with the US regulatory authority and best practice guidance for online research with patients, written by patient representatives, is also available (
  • uses rigorous qualitative methods, conducted by social scientists with training in sociology, anthropology, health policy, psychology, discourse analysis and history, to tell the stories of people’s experiences of various health conditions, some of which relate to adverse effects.


Allen EN, Mushi AK, Massawe IS, et al. How experiences become data: the process of eliciting adverse event, medical history and concomitant medication reports in antimalarial and antiretroviral interaction trials. BMC Medical Research Methodology. 2013;13:140.

Banerjee AK, Okun S, Edwards IR, et al. Patient-Reported Outcome Measures in Safety Event Reporting: PROSPER Consortium Guidance. Drug Safety. 2013;36(12):1129-1149.        

Blinder VS, Eberle CE, Aviki E, Schleicher SM, Patil S, Basch EM, Sloan JA, Bennett AV. Overall survival results of a randomized trial assessing patient-reported outcomes for symptom monitoring during routine cancer treatment. Abstract ASCO Annual meeting 2017 (

Berrewaerts J, Delbecque L, Orban P, Desseilles M. Patient Participation and the Use of Ehealth Tools for Pharmacoviligance. Frontiers in Pharmacology. 2016;7:90.

European Medicines Agency. The patient's voice in the evaluation of medicines; how patients can contribute to assessment of benefit and risk. EMA/607864/2013.

Sacristán JA, Aguarón A, Avendaño-Solá C, et al. Patient involvement in clinical research: why, when, and how. Patient preference and adherence. 2016;10:631-640.

Smith MY, Benattia I. The Patient’s Voice in Pharmacovigilance: Pragmatic Approaches to Building a Patient-Centric Drug Safety Organization. Drug Safety. 2016;39:779-785.


Additional useful links

Clinical Trials Transformation Initiative: Patient groups & clinical trials. Evidence-based Recommendations & Tools to Maximize Effective Engagement Between Patient Groups & Sponsors of Research.

DIA Insights: patient engagement. Considerations guide and study results.

EUPATI Guidance documents on patient involvement in R&D for Pharmaceutical industry-led medicines R&D, ethics committees, regulatory processes and health technology assessments (HTA).

European Medicines Agency (EMA): patients and consumers.

Innovative Medicines Initiative (IMI) and patients - a partnership.

NIH Division of Cancer Control and Population Science. Patient-reported version of the common terminology criteria for adverse events (PRO-CTCAETM)

Patient-Centered Outcomes Research Institute (PCORI)


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