Parkinson’s Clinical Trial Simulators Win Support of Regulators in EU

EMA issues letter to industry backing model-based simulators

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The words clinical trials anchor an interlocking word puzzle of related terms.

The European Medicines Agency (EMA) has issued a letter in support of model-based clinical trial simulators for Parkinson’s disease that are being developed by the Critical Path for Parkinson’s (CPP) consortium.

In its letter, the European regulatory agency called on industry sponsors to share with the consortium data from previous clinical trials, which will be used to help inform the model.

The two-part goal of the EMA’s missive is to help improve the simulation platform’s methodology while getting the model into the hands of researchers.

“This letter of support represents a major milestone for the consortium, and we want to recognize the outstanding work of all C-Path programs that support CPP, as well as the generous contribution of all collaborators worldwide for their role in this achievement,” Diane Stephenson, PhD, executive director of CPP at the  Critical Path Institute (C-Path), said in an institute press release.

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Clinical trials are the gold standard for determining whether or not an experimental treatment is safe and effective. These in-human studies usually go through months or even years of rigorous work, before the first patient is ever enrolled, to best design the trial.

In addition to smoothing out any logistical issues, this work aims to ensure that a trial enrolls the right set of patients — those most likely to respond to treatment — and that the best measures are being used to detect a potential effect of treatment.

The EMA’s letter of support covers two clinical trial simulators that both seek to optimize the design of trials aiming to assess changes in patients’ motor function through the standard Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Parts II and III.

Part II assesses the severity of motor symptoms, while Part III measures the impact of such symptoms on daily life.

Using the platform, researchers will feed in the most comprehensive evaluation of MDS-UPDRS Parts II and III score progression in early motor Parkinson’s to date. The simulation platform then is expected to help determine optimal trial features, such as the number of participants, patient inclusion criteria, and duration.

“The EMA supports the primary objectives of the applicant and agrees to issue a letter of support to the CPP Consortium to encourage industry sponsors to share with CPP the patient-level data from completed Phase [2] and [3] clinical trials in the intended target population as defined in the context of use statement,” the EMA wrote in its letter.

The agency added that expanding the model’s data “will allow the CPP team to continue to enhance quantitative novel methodology in drug development, while also encouraging the CPP team to disseminate and provide access to the current version of the model for implementation by sponsors actively designing clinical trials in early motor [Parkinson’s disease].”

These are the second and third clinical trial simulators for Parkinson’s from the CPP consortium to win EMA support. The first was based on the use of neuroimaging biomarkers to optimize patient selection for trials focused on early Parkinson’s stages. It received a similar letter of support in 2018.

The CPP consortium was founded in 2015 through a partnership between C-Path and Parkinson’s UK. One of the largest charity funders of Parkinson’s research in the world, the consortium works to facilitate international collaboration between members of industry, academia, government, and patient advocacy groups, with the aim of improving care for people with Parkinson’s.

“This is an example of C-Path’s role as a neutral convener for collaboration between regulators, industry and researchers, in the public and private sectors, working together to accelerate delivery of Parkinson’s disease therapies into the hands of patients,” Stephenson said.

David Dexter, PhD, associate research director at Parkinson’s UK, added: “On behalf of all of those affected by Parkinson’s, we acknowledge the need for global collaborations that aim to assure that all clinical trials, whether successful or not, can be used to develop innovative tools that accelerate the path to new treatments.”