Core Outcome Measures in Effectiveness Trials

Achieving consensus for clinical trials: the REiNS International Collaboration

General Information

Abstract:
The neurofibromatoses (NF)--including neurofibromatosis 1 (NF1), neurofibromatosis 2 (NF2), and schwannomatosis--are related tumor-suppressor syndromes characterized by a predisposition to multiple tumor types and other disease manifestations, which often result in functional disability, reduced quality of life, pain, and, in some cases, malignancy. With increasing knowledge of the biology and pathogenesis of NF, clinical trials with targeted agents directed at NF tumors have become available. Most clinical trials for patients with NF have used designs and endpoints similar to oncology trials. However, differences in the disease manifestations and natural history of NF (compared to cancers) require the development of new designs and endpoints to perform meaningful NF clinical trials. The Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration was established in 2011 at the Children's Tumor Foundation meeting to achieve consensus within the NF community about the design of future clinical trials, with a specific emphasis on endpoints. The REiNS Collaboration includes 7 working groups that focus on imaging of tumor response; functional, visual, patient-reported, and neurocognitive outcomes; whole-body MRI; and disease biomarkers. This supplement includes the first series of recommendations by the REiNS Collaboration. The hope is that these recommendations will be used by members of the group and by researchers outside of the REiNS International Collaboration to standardize the measurement of outcomes and thus improve clinical trials for patients with NF. Ultimately, we plan to engage industry partners and national regulatory agencies in this process to facilitate the approval of drugs for patients with NF.

Aim:
The goal of the group’s efforts is to recommend outcome measures and methodologies that will be used by REiNS members and by other researchers to standardize endpoints for NF clinical trials.

Authors:
Plotkin, Scott R. Blakeley, Jaishri O. Dombi, Eva Fisher, Michael J. Hanemann, C. Oliver Walsh, Karin S. Wolters, Pamela L. Widemann, Brigitte C.

Publication

Journal:
Neurology
Volume:
81
Issue:
21 Suppl 1
Pages:
S1 - 5
Year:
2013
DOI:
Further Study Information

Date:
Not applicable
Funding source(s):
Supported by the Children’s Tumor Foundation.

Health Area

Disease Category
Genetic disorders

Disease Name
Neurofibromatoses

Target Population

Age Range
0 - 100

Sex
Either


Nature / type of Intervention
Any

Method(s)

Literature review
Semi structured discussion
Teleconferences

The Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration was established in 2011 at the Children’s Tumor Foundation meeting to achieve consensus within the NF community about future clinical trials and to accelerate the identification of agents that will benefit individuals with NF. The REiNS Collaboration is organized around 7 working groups, with leaders of the groups identified based on their expertise at the initial meeting in 2011. Each REiNS group establishes a meeting schedule; the majority of meetings are held by teleconference. In-person meetings of the entire group are held at least twice per year to coordinate efforts among the working groups and to achieve consensus for recommendations. The leadership team held a preliminary meeting with the US Food and Drug Administration (FDA) to discuss endpoints for NF trials. At this meeting, the FDA encouraged the REiNS Collaboration to use validated functional and patient-reported outcomes for trials intended for drug approval. The authors provided a summary of published clinical trials for Neurofibromatosis.


Stakeholders Involved

None

Study Type

Systematic review of outcomes measured in trials

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