Challenge:

Alacrita was approached by a company developing an enzyme approved by the FDA for the treatment of toxic plasma chemotherapy concentrations in patients with delayed clearance due to impaired renal function. The company was planning to investigate repeated administration of the drug as an adjunct to high dose chemotherapy for patients with central nervous system (CNS) cancers.

The patient’s subjective experience of the condition and its treatment is a key element of the information stakeholders require to inform decision making. This perspective is particularly important in conditions such as cancer that are associated with significant morbidity and treatment-related toxicity. Alacrita’s consultants led the development and refinement of a patient-reported outcome (PRO) strategy to demonstrate the benefit of routine administration of the drug in cancer patients also receiving high dose chemotherapy therapy.

Solution:

Alacrita’s consultants reviewed the current FDA-approved product label for the drug, Biologics License Application (BLA) documents, internal client documents and investigator-led study protocols. A targeted literature review was undertaken to identify patient relevant endpoints in which treatment with the drug alongside high-dose chemotherapy have demonstrated treatment benefit, which identified potential benefit in terms of cognitive function, physical function and HRQL.

The PRO strategy was refined through discussions between Alacrita consultants and KOLs. The PRO strategy was further refined through regular interactions with the client’s team during weekly team meetings, alongside preparation of an FDA briefing document for a type C meeting. A report was generated providing recommendations for the PRO strategy for measuring patient-relevant outcomes for treatment with the drug alongside high dose chemotherapy in patients with CNS cancer.

The recommendations were in line with the Center for Medical Technology Policy (CMTP) recommendations for the incorporation of PROs into clinical comparative effectiveness research in adult oncology in order to capture the patients’ subjective experience and provide complete information for decision making. The recommended PRO strategy represented a robust and comprehensive approach for capturing the patient voice, in order to demonstrate the benefit of repeated administration of the drug as an adjunct to high dose chemotherapy for patients with CNS cancers. The proposed approach was designed to generate data suitable for publication in peer-reviewed journals and address the requirements of multiple stakeholders.

The recommendations set out in the report were incorporated in the Phase 2 study protocol synopsis for further refinement and discussions with KOLs, patient interviews and discussions with the FDA.

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