Regulatory Focus™ > News Articles > EMA Looks to Formalize Involvement of Patients Within the Regulatory Process

EMA Looks to Formalize Involvement of Patients Within the Regulatory Process

Posted 29 October 2013 | By Alexander Gaffney, RAC

There is perhaps no bigger trend among established regulatory regimes around the world at this time than increasing the involvement of patients in the regulatory process.

The US Precedent

In the US, this involvement has been growing since the early 1990s, when the AIDS crisis energized a movement among patient groups to press the US Food and Drug Administration (FDA) for faster and less risk-averse approvals.

In recent years, this involvement has grown, with most-if not all-advisory committees now reserving a seat for a patient representative and allowing public comment at committee hearings.

Moreover, under the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA), FDA is tasked with setting up patient-focused drug development paradigms for more than a dozen disease areas, taking into account the actual and perceived needs and wants of patients for treatments. The hope is that this work might facilitate approvals for certain subsets of patients who may be considerably less risk-averse than other patients.

Elsewhere, FDA has launched a "Patient Initiative" and "Patient Preference Initiative" aimed at better communicating FDA's regulatory processes to patients, thereby allowing a more constructive and productive dialogue between regulators and patients. The agency has also recently launched a patient-focused adverse event reporting form, the 3500B.

Meanwhile, in Europe…

Now the European Medicines Agency (EMA) seems poised to expand its own regulatory constructs similarly.

In a 29 October 2013 announcement posted to its website, EMA explained that it is interested in bringing more patient voices into the regulatory dialogue, and particularly in the assessment of the risks and benefits of a product under consideration.

"[EMA] believes that incorporating the knowledge, views and preferences of patients throughout the lifecycle of medicines, from early stages of development through to authorisation and monitoring of safety and efficacy, adds value to the scientific assessment," it wrote.

However, the agency noted that this process is not without its challenges. As FDA has found through its own efforts, patients-like experts-can occupy different sides of a benefit-risk spectrum. Some favor lax approvals with little attention paid to potential risks, especially if a condition has few or no approved treatments, while others are more risk-averse and willing to hold out for treatments they view as more refined or worthwhile. And with more than 500 million citizens in the EU, there is surely no shortage of opinions on either side of the benefit-risk equation.

"Transferrable" Conclusions

In a report also issued on 29 October, EMA cites the influence of the HIV/AIDS community, calling historical interactions between those patient groups and regulators a model-albeit one that has evolved and matured over time-for future dialogue between EMA and other patient groups. In addition, EMA said knowledge gained from one group of patients can potentially benefit others as well, such as attitudes toward releasing clinical data or general norms for industry-patient interactions.

"Much of this knowledge is valuable and transferrable, and ways of sharing this knowledge (not only externally but in terms of training new patient representatives within the group) are currently being considered, including the development of a 'school of excellence,'" EMA explained.

EMA, like FDA, seems to be most interested in what it called "new ways to capture patient preference and represent benefit-risk" in specific diseases, noting that each disease area has its own unique challenges that may alter perceptions of both benefit and risk.

At present, EMA said there is no patient involvement in its scientific advisory groups such as the Committee for Human Medicinal Products (CHMP), which is primarily responsible for evaluating pharmaceutical and biological products for the agency. While the groups regularly convene Scientific Advisory Groups (SAGs) of their own, EMA said it was assessing whether long-term "expert patients" might be appointed to the SAGs and other groups.

Already, EMA has formalized a patient position within its newly created Pharmacovigilance Risk Assessment Committee (PRAC), the report noted.

For now, EMA is in the midst of "drawing up a proposed framework" to formalize more patient-regulator interactions in the hopes that it can add value to the regulatory process in the future.


EMA Report

Press Statement


Tags: patients, EU

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