1. Regulatory Responses (n=11) Company Responses (n=20)
Benefit-risk framework:
purpose and involvement
Regulatory Responses (n=11)
Company Responses (n=20)
Percentage %
Indifferent
Agree
Strongly agree
Strongly disagree
Disagree
100 75 50 25
CIRS survey: Industry overview
Statement
The purpose of establishing an appropriate BR framework is to improve: The transparency of decision making (Q9.6b)
The purpose of establishing an appropriate BR framework is to improve: Communication of the decision (Q9.6c)
The purpose of establishing an appropriate BR framework is to improve: The consistency of decision making (Q9.6a)
It is important that all stakeholders (agencies, companies, doctors and patients) are part of the development and validation of an appropriate benefit-risk framework (Q 9.12)
In order to clearly convey an opinion of the benefits and risks that a medicine offers, an overarching benefit-risk framework provides the elements of a common discussion language. This slide shows the views of regulatory agencies and industry on statements related to the importance of such a framework. The respondents had the opportunity to rate their agreement with the statements as strongly disagree or disagree (orange), indifferent, agree or strongly agree (green).
This was part of a survey undertaken by CIRS in 2011 in which 11 major regulatory agencies and 20 pharmaceutical companies identified the current state of utilisation of benefit-risk methodologies and perceptions of the barriers and value of developing a benefit-risk framework. The biggest barrier to implementing a formal framework within companies and agencies was simply the lack of a scientifically accepted or recognised framework. This is against a background where there was generally good agreement on the need and function of such a tool for communication, structured discussion and the enhancement of transparency and accountability. Respondents indicated that rather than looking for one single benefit-risk methodology, a toolbox of methodologies that are flexible and adaptable for different situations is required, although taking this concept forward would necessitate the development of a consensus amongst the major stakeholders on a general scientifically accepted, overarching framework.
As the framework for benefit-risk assessment is developing, it has become increasingly apparent that the one of the stakeholders, the patient, should play an important role in informing regulatory decisions. However, eliciting a perspective of benefit and harms that is based on evidence obtained from patients being studied during clinical development and determining how that perspective should be used in regulatory decision making is a complex area that requires much additional study. Many regulatory agency and company decision makers recognise that patient input could be invaluable in informing their thinking, and it is generally agreed that going forward, it will be of utmost importance to include the patient’s perspective early in the development and assessment of medicine.
In April 2012, a CIRS Workshop will explore these issues by gaining a perspective from various stakeholders in the development and review of new medicines, with a particular emphasis on the opportunities and barriers to including patients’ perspectives.