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Hi @charlton, Pragmatic trials are interventional in nature, as they involve random assignment to the treatment groups. Given this fact, it would be inaccurate to refer to them as ‘observational’ studies. However, they differ in important ways from randomized control trials (RCTs). RCTs are designed in a manner that prioritizes a highly controlled environment. While this comes at the cost of an artificial environment that can differ from the actual surrounding environment, it reduces the potential for biases that could impair the validity of causal inferences. While their results should be generalized cautiously to the real-world, they have high internal validity when designed, executed, analyzed, and reported properly. Pragmatic trials are designed to prioritize reflecting the surrounding environment. This is why they are said to provide insights about “effectiveness”, which is the benefit that the treatment produces in routine clinical practice. For example, pragmatic trials tend to include patients with comorbidities, the methodology aims to reflect the lower adherence levels that occur in clinical practice, blinding is rarely utilized (as it does not reflect real-life practice), and investigators have more clinical freedom to make therapeutic decisions (i.e., less protocol standardization than in RCTs). Due to the above, PAAB considers pragmatic trials to fall within the realm of RWEs. Please see our RWE guidance document on parameters for acceptability and presentation format.

Hey @kshulist The guidance does apply in this scenario. In addition to the considerations identified in your question, section 4.2 of the guidance relating to emphasis is also applicable. There should be no emphasis on burdens upon which the product has not demonstrated an effect and it should be part of a balanced presentation that includes burdens upon which the product has demonstrated an effect. Clear and prominent disclosure as per section 5.2 of the guidance will be required and this may include clarification of the indication even if it has already been included elsewhere. As a reminder, PAAB will be starting to review under the new guidance starting February 19th.

Hey @palanski Great question. PAAB’s strategic plan for 2023-2025 includes a focus on increasing the extent to which HCPs value health product advertising and industry-generated patient information. This is the primary motivation for the shift outlined in the disease burden document. This shift is one element of a multi-pronged approach towards that aim. The standards outlined in the document were generated in consideration of our global benchmarking and stakeholder consultation activities. While health product manufacturers’ have expressed a long-standing desire to provide more detailed presentations of disease states, it took some time to establish a set of standards that effectively aligned that desire with the interests of patients and HCPs. The objective of PAAB is to ensure that disease information (including burden of disease) does not suggest use and efficacy for outcomes which the brand has not been demonstrated to address. This objective remains however PAAB has looked to explore ways that we can meet this objective while allowing more latitude on disease presentations. In short, the document seeks to outline principles that will mitigate the risk that the disease burdens be interpreted as outcomes that the drug can improve. The document also reiterates the requirement that the disease information and descriptions of disease burden must pertain to the promoted product’s indication. Our hope is that manufactures will view the guidance document as a collaborative means to providing greater flexibility in relation to disease information presentations in HCP advertising, disease information presentations in patient information, and disease burdens conveyed in creative imagery.