By Supriya Desai M.D, Director of Medical Safety Operations, Sciformix Corporation
Today, there is greater awareness surrounding the benefit and risk of pharmaceutical products, amongst health care professionals (HCPs) and consumers alike. Safety and efficacy data from clinical trials are no longer sufficient and there is a demand for more evidence including effectiveness of treatments as well as comparative effectiveness, relative to other therapies. This requires real world data based on longer follow-up, which is closer to and more representative of the patient population than what is typically obtained via clinical trials. Patient reported outcomes (PROs) can help fulfill this need.
PROs are data elements directly reported by patients or their surrogates about experiences with care, including symptoms, functional status or quality of life. They have gained increasing importance in healthcare and are commonly evaluated in clinical trials for drug and medical device development (1). Studies have shown that patient reports are more valuable compared to HCP reports as patients often report earlier, perceive the impact and severity of reactions differently and give more detailed descriptions. Companies, who successfully incorporate PROs in their safety and pharmacovigilance (PV) operations would likely gain a competitive advantage through direct patient involvement and higher quality of data.
The PROSPER Consortium
One way to improve the quantity and quality of safety information is to encourage patient-reported outcomes of adverse events (PRO-AEs), based on a more patient-centric approach, that incorporates less of formal data collection processes, reliant on HCP input. The Patient-Reported Outcomes Safety Event Reporting (PROSPER) Consortium was convened to improve safety reporting by better incorporating the perspective of the patient.
The PROSPER consortium has developed guidance on PRO-AE data, which aims to ensure that the patient ‘voice’ and perspective feed appropriately into the collection of safety data, so that safety and benefit-risk proﬁles of medicines is better deﬁned. The guidance covers a minimum core dataset for use by industry or regulators to structure PRO-AEs and how data can be evaluated to drive safe and effective use of medicinal products (2).
PRO-AEs: Tools, Benefits and Challenges
Engaging patients as partners in the data collection enterprise can enable systematic collection of PRO-AEs and provide a more comprehensive picture within a population. Effective involvement of patients in the AE reporting process is possible by use of novel Adverse Drug Reaction (ADR) reporting tools and processes like ADR online forms, mobile applications and through local pharmacies. Developing guidelines for the follow-up procedure for reports in consultation with patients’ and HCP organizations enables the collection of high-quality data, conveys feedback to patients on how the information will be used and provides patients with further information or where information is available.
The beneﬁts of PRO-AEs are applicable across various stakeholders. Patients can know what to expect in terms of potential adverse events due to prescribed medications and also medication preferences, based on the prior experiences of other patients. Drug developers can understand how well patients will tolerate a product, particularly for oral therapies when compliance is strongly associated with symptomatic side effects. PRO-AEs can provide the HCPs with information of value of an individual patient’s experience which, when combined with the physician perspective based on experience and training, provides a more accurate understanding of the patient’s symptoms and thereby helps improve measurement of symptoms in both clinical trials and practice (2).
There are, however, potential public health downsides due to PRO-AEs being disseminated without the appropriate beneﬁt-risk context, causing unnecessary alarm or leading to discontinuation of effective therapies. Other perceived disadvantages of PRO-AEs include costs, possibility of swamping safety signal with noise, exacerbation of the placebo effect at pre-approval phases and a signiﬁcant risk of having duplicates in the database for cases received from multiple sources.
Patient-Centric Regulations and Technological Advances
Technological advances offer unprecedented access for multidirectional communication, making it easier for patients to be an active participant. Simpliﬁed internet search capabilities, digital mobile devices and patient online communities are just a few examples of tools that allow patients to gain increased knowledge about conditions, symptoms, treatment options and side effects. Social media, for example, is a promising source for new safety data obtained directly from patients. It has the potential to become an added new-age tool to monitor data in real-time, making it an early indicator of potential safety issues for further investigation. However, social media can result in challenging safety data that is incomplete, incorrect or duplicated.
PRO-AE-enabled websites and online patient forums, initiated and managed by companies allow a more structured approach, where the dataset can be designed pre-emptively, and so are far likelier to provide higher quality of data.
Despite the increased attention on the perceived value of PRO-AEs, their full potential is yet to be realized in PV. Current methods for safety reporting and risk assessment still rely heavily on HCPs despite known limitations such as under-reporting and discordant perspectives between patient and HCP reports. Overall, there seems to be a reluctance to adopt an approach that includes PRO-AEs due to multiple challenges, such as concerns regarding feasibility and validity.
PV teams, who address these challenges, would be able to integrate PRO-AEs into their PV strategies towards incorporation of the patients’ “voice” in the safety process. Such PV organizations are better positioned over the long term to ensure patient safety and add real value back to the patients by enhancing product benefit-risk profiles.
- Basch E. Annu Rev Med. 2014; 65:307-17, Nov 20 2013. New frontiers in patient-reported outcomes: adverse event reporting, comparative effectiveness, and quality assessment.
- Anjan K. Banerjee, Sally Okun, I. Ralph Edwards et al. Drug Saf (2013) 36:1129–114. Patient-Reported Outcome Measures in Safety Event Reporting: PROSPER Consortium Guidance.
About the Author:
Supriya Desai MD, PGDBM is a Clinical Research and Pharmacovigilance (PV) Executive with over 16 years of experience in clinical practice and in various leadership roles in the healthcare industry including clinical research, drug safety and PV. In her current role as Medical Director and Practice Head at Sciformix, she provides scientific and operational leadership to a global medical team (across India, Philippines and US); involved in medical review activities across pharmacovigilance, safety writing, signal management and allied safety surveillance activities, spanning diverse therapeutic areas. Email: firstname.lastname@example.org