When Innovation and Commercialization Collide: A Patient Advocate View in Neuroblastoma

Bird, Nick; Knox, Leona; Palmer, Antonia; Heenen, Delphine; Blanc, P.; Scobie, Nicole; Ludwinski, Donna

doi:10.1200/jco.21.01916

Cited by 9 publications

(16 citation statements)

References 31 publications

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“…All of this work needs to be conducted with an eye toward subsequent regulatory file and access of novel agents for our patients. 93 With increasing understanding of the molecular basis of neuroblastoma, there are numerous rationally targeted agents in development. The challenges moving forward remain how best to identify, prioritize, and combine novel agents to improve the treatment of HRNB.…”

Section: Discussionmentioning

confidence: 99%

High-Risk and Relapsed Neuroblastoma: Toward More Cures and Better Outcomes

DuBois

Macy

Henderson

2022

American Society of Clinical Oncology Educational Book

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Approximately half of the patients diagnosed with neuroblastoma are classified as having high-risk disease. This group continues to have inadequate cure rates despite multiagent chemotherapy, surgery, high-dose chemotherapy with autologous stem cell rescue, and immunotherapy directed against GD2. We review current efforts to try to improve outcomes in patients with newly diagnosed disease by integrating novel targeted therapies earlier in the course of the disease. We further examine a growing list of options available for patients with relapsed or refractory high-risk disease, with an eye toward graduating successful strategies from a relapsed/refractory setting to the frontline setting. Last, we review efforts to study and potentially mitigate the array of late effects faced by survivors of high-risk neuroblastoma.

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Section: Discussionmentioning

confidence: 99%

High-Risk and Relapsed Neuroblastoma: Toward More Cures and Better Outcomes

DuBois

Macy

Henderson

2022

American Society of Clinical Oncology Educational Book

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“…Where drug approvals are necessary to change standard of care, the need to satisfy scientific, regulatory, and reimbursement requirements must also be a key consideration. 32 Questions over EFS as the best trial endpoint have persisted as both CCG-3891 and COG-ANBL0532 reported significant differences in EFS but not OS. Supporting the notion of discordant EFS and OS, survival after relapse, and lack of survival advantage with transplant in the immunotherapy era, a retrospective analysis of 170 patients treated at MSK between 2003 and 2013, 60 of whom had received prior transplant, reported five-year EFS favoring the transplant group (65% vs 51%) but near identical five-year OS (76% vs 75%).…”

Section: Efs Versus Osmentioning

confidence: 99%

“…End‐of‐induction response as an even earlier endpoint for efficacy in future studies is currently the subject of some discussion given its well‐established prognostic association with improved survival. Where drug approvals are necessary to change standard of care, the need to satisfy scientific, regulatory, and reimbursement requirements must also be a key consideration 32 …”

Section: Exploring the Controversiesmentioning

confidence: 99%

To transplant, or not to transplant? That is the question. A patient advocate evaluation of autologous stem cell transplant in neuroblastoma

Bird

Scobie²,

Palmer³

et al. 2022

Pediatric Blood & Cancer

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High-dose chemotherapy with autologous stem cell transplant (ASCT) has been a mainstay of high-risk neuroblastoma treatment for several decades, demonstrating improvements in event-free survival but with risks of serious or even life-threatening acute toxicities, severe long-term adverse health effects for survivors, and ongoing contention regarding overall survival benefit. The merits of ASCT in the modern era of immunotherapy are a source of debate among parents, advocates, and some physicians. Here we examine evidence for and against ASCT, explore parent attitudes and their turmoil over decision-making, and strongly encourage international research consortia to develop a coordinated strategy to accelerate progress toward a future that avoids the routine use of ASCT in high-risk neuroblastoma.

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“…Patient advocates can play many roles in research, for example, championing accessing stored tissue and aiding the recruitment of trial participants. Their early engagement in study concept discussion could lead to valuable insight for industry to better understand the treatment pathway, patient community and commercialisation impact [62]. 9.2.…”

Section: Patient Advocates' Perspectivementioning

confidence: 99%