Combining data from expanded access programs and conventional clinical trials: a statistical application to vemurafenib

Lead Investigator: Tobias Polak, Erasmus Medical Center
Title of Proposal Research: Combining data from expanded access programs and conventional clinical trials: a statistical application to vemurafenib
Vivli Data Request: 6475
Funding Source: Health~Holland grant EMCLSH20012 (Grant from the Dutch Government)
Potential Conflicts of Interest: None

Summary of the Proposed Research:

Patients suffering from seriously debilitating or life-threatening conditions who are not eligible for further treatments or any clinical trials, may resort to ‘expanded access’: pre-approval access to investigational treatments. These programs are increasingly used to generate data in patients that are older, frailer, and therefore not suited for clinical trials. As these programs are non-randomized, several selection bias issues need to be accounted for.

This research investigates statistical methods to incorporate data from expanded access programs in the analyses of (randomized) controlled trials. In this work, we will illustrate novel techniques on the basis of trial data and expanded access data for vemurafenib. Although expanded access is not primarily seen as research-oriented, we sought to improve the useability of data from all patients that are treated in this setting with experimental medicine. This project will yield direct insights, via scientific publications and statistical methodology, that are directly beneficial to regulators, drug developers and (bio)statisticians. This project is different from proposal 6476, as we will cover a different drug under investigation (with various differing data sources).

Requested Studies:

Data Contributor: Roche
Study ID: NCT01248936
Sponsor ID: ML25597

An Open-label Multicenter Study on the Efficacy of Continuous Oral Dosing of Vemurafenib on Tumour Response in Previously Treated Patients With Metastatic Melanoma
Data Contributor: Roche
Study ID: NCT00949702
Sponsor ID: NP22657

BRIM 3: A Randomized, Open-Label, Controlled, Multicenter, Phase III Study in Previously Untreated Patients With Unresectable Stage IIIC or Stage IV Melanoma With V600E BRAF Mutation Receiving Vemurafenib (RO5185426) or Dacarbazine
Data Contributor: Roche
Study ID: NCT01006980
Sponsor ID: NO25026

An Open-label, Single-arm, Phase II, Multicenter Study, to Evaluate the Efficacy of Vemurafenib in Metastatic Melanoma Patients With Brain Metastases
Data Contributor: Roche
Study ID: NCT01378975
Sponsor ID: MO25743

Dutch Melanoma Treatment Registry – a real-world registry maintained by Dutch physicians looking at the real-world treatment regimens, safety, effectiveness and costs of treatment regimens for melanoma.
Data Contributor: I WILL BRING MY OWN
Sponsor ID: Dutch Melanoma Treatment Registry