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Therapeutic Access and Emerging Treatments

Opinion
Video

Explore the facilitation of novel therapy access for desmoid tumors and discover emerging treatments reshaping the landscape.

This is a video synopsis/summary of a Peer Exchange involving Ryan Haumschild, PharmD, MS, MBA; Tony Phillip, MD; Mrinal Gounder, MD; Ravin Ratan, MD, MEd; and Derek Van Amerongen, MD, MS.

Haumschild discusses access to novel desmoid tumor therapies with Gounder. Gounder highlights the importance of patient education and clinical trials for accessing novel therapies. He emphasizes collaboration with patient advocacy groups and the role of patient navigators in directing individuals to specialized care. The discussion delves into newly approved therapies, notably γ-secretase inhibitors such as nirogacestat, which offer hope for patients. Haumschild emphasizes the significance of clinical trials, patient advocacy, and newer FDA approvals in transforming the treatment landscape.

Van Amerongen provides insights into the factors that plan sponsors should consider when evaluating the cost-effectiveness of desmoid tumor treatments. He clarifies the misnomer of the term cost-effective in health services literature, stressing the importance of efficacy and understanding offsets. Van Amerongen defines value as the combination of clinical benefit and cost, emphasizing the need for transparency and supporting standard of care while avoiding unnecessary interventions. The conversation underscores the multidimensional considerations involved in evaluating treatment options for desmoid tumors.

Video synopsis is AI generated and reviewed by AJMC® editorial staff.

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