JCA preparation for a new therapy for a rare haemato-oncology disease​

• The client was investigating a new therapy for an aggressive haemato-oncology indication ​
• An assessment was required regarding the heterogeneity of the patient population in the literature, the current treatment pathway for patient, key outcomes and predicted PICOs for the JCA​
• The client also wanted to gain insights on the feasibility of using a retrospective external control arm for their clinical trial, based on the literature base​

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• Complete a targeted literature review (TLR) for the specific indication to provide an understanding of the current clinical landscape, outcomes with available treatments and variation in clinical practice​
• Provide insight on patient characteristics and current treatment pathways for the target indication​
• Define draft PICOs to address predicted evidence requirements across priority European member states to inform risk identification/mitigation strategy development and clinical evidence planning ​

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• A literature review was completed, in line with Cochrane guidance​
• Insights on the current/evolving clinical landscape, patient stratification, outcomes with current treatments and variation between countries were reviewed in a workshop with the client team​
• Draft PICOs covering predicted variation in European market requirements were developed to inform strategic decisions regarding the product positioning and clinical development strategy ​

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• The client was provided with a detailed and up-to-date review of the clinical landscape for the target patient population and predicted implications for the JCA scope to address potential heterogeneity across member states ​
• This directly informed prioritised requirements for clinical development and key risks requiring further investigation/exploration (e.g., through early scientific advice)​

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