Facultades Particulares

One reviewer suggested combining the identification of benefits and risks with the exercise of assigning weights and values to avoid repetition. However, this suggestion could be accommodated by auto-populating the benefits and risks in Section 3 into Section 6. More guidance could be given on listing the reasons for inclusion or exclusion of benefits, like local disease burden (medical need), available alternatives, strength of evidence, clinical relevance and convenience to patients. For completeness, one reviewer recommended adding another section to indicate if the benefit-risk balance is positive or negative, before being asked to provide reasons for a negative benefit-risk balance. While this study was conducted for new active substances, one reviewer recommended that the template could be amended to accommodate clinical variations.

DISCUSSION

The findings of this study showed that the successful implementation of a new process or tool in an established regulatory agency is dependent on the fundamental understanding of the principles behind the template. The concept of weighting or assigning relative importance and valuing is a technique that is relatively new to both

0 20 40 60 80 100

Healthcare professionals Health technologies assessment

agencies (HTA)

Patients/ patient advocacy groups Other regulatory agencies Media/ public domain

Percentage of responders

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HSA and other regulatory agencies (Chapter 3). However, weighting and valuing is seen as an explicit presentation of the subjective interpretation of a set of clinical information. This exercise aimed to enhance the transparency of decision-making by making it clear that the priorities placed on a set of benefits and risks ultimately affect the resulting benefit-risk balance. Without an understanding of the rationale behind weighting and valuing, some reviewers could not appreciate its contribution to effective documentation and communication.

As for all new initiatives, an implementation strategy or change management programme should be drawn up. This would consist of dialogues with senior management, a dedicated training plan and the use of training tools. It is expected that senior management should be made aware of the potential advantages of the BR Summary Template and are agreeable to implementing this across the relevant departments in the agency. A top-down approach might be required to ensure the appropriate implementation of this template, as this may be helpful in situations where reviewers are unable to comprehend its role and advantages in the entire process. However, the end-users or reviewers should also clearly understand the usefulness of the template, its role in the current processes and the impact on existing workflow so as to ensure maximum compliance. This could be achieved through a standard training programme which would include a driver from senior management. In addition, it should include leaders among the users who would be trained as pioneers for the successful implementation of the BR Summary Template.

As is evident from this study, the User Manual proved to be a valuable tool, however amendments would be required to enhance its effectiveness.

The current BR Summary Template would require a revision to the technical capabilities and an improvement for the documentation of safety information and adverse events. The User Manual should be revised to include examples and case studies to better illustrate the use of the template. It appears that the capacity of the BR Summary Template to effectively communicate a benefit-risk decision has been clearly exhibited, as supported by the reviewers who were willing to share this template with stakeholders. However, this should go hand–in–hand with the legal framework to give the agency the mandate to implement it. Without the assurance of

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legal protection and support of management, reviewers would be unlikely to release such reports, especially to stakeholders who are lay persons.

The reviewers in this study indicated their willingness to share the completed BR Summary Template for a specific product with other regulatory agencies where there is a memorandum of understanding. It is also appropriate to examine the utility of this template as a means of transferring knowledge and communicating the basis of a decision. For major regulatory agencies it may be a requirement to provide details of the evaluation to achieve a level of transparency stipulated by the jurisdiction.

However, this study, even in the absence of these details, has demonstrated that the BR Summary Template is an effective tool to communicate benefit-risk decisions.

Therefore it may be considered as a basic report template for agencies that are in transition to build up their evaluation capabilities. Thus this would be an ideal tool for communicating benefit-risk decisions to emerging regulatory agencies, since the components of the template address the basic needs of a sound and scientific discussion.

From another perspective, established agencies may find that the BR Summary Template replicates existing publicly available reports and is thus judged by some to be redundant. Attempts to use IT to auto-populate existing information from current reports should be undertaken to improve on this aspect. Through this study and Chapter 3, it can be seen that weighting and valuing are not consistently applied but the relevance of such an exercise in effective communication is accepted. Again, it is important to educate regulators on the use of weights and values as they form a key component with regard to communicating decisions in a transparent manner. It is only through a global understanding of the need for a common template that consistency in evaluating benefits and risks can be achieved.

The outcome of this case study, involving reviewers within the Health Sciences Authority as representative of the emerging markets in the region, has demonstrated that the principles of the BR Summary Template are applicable to other jurisdictions or similar agencies. This is indeed encouraging in the current climate, where the debate surrounding the benefit-risk assessment of medicines is on the top of many

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regulatory agencies’ agenda. Thus the promising features of the BR Summary Template will, no doubt, contribute to such on-going discussion.

SUMMARY

• The BR Summary Template is adequate to document benefits, risks, relevant summaries and conclusions

• A revision of the BR Summary Template should include technical improvements and more details for safety information

• The User Manual and navigation functions are useful to guide the reviewer in completing the template

• More guidance should be provided for weighting and valuing, as well as the use of examples and case studies, in the User Manual

• The BR Summary Template can be a useful tool for communicating benefit-risk decisions to a variety of stakeholders

• The principles behind the template may be useful for guiding the benefit-risk assessment of medicines

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CHAPTER 7

Evaluation of regulatory agencies’ strategies