The Cure Parkinson's Trust (CPT) has joined Parkinson's UK and others in a consortium project - the new “Critical Path for Parkinson’s (CPP) Consortium”. The consortium, involving industry regulators and other interested parties from academia to charities, is focused on data sharing and regulatory science relevant to Parkinson's (PD) across the globe. One of the CPP's key objectives is to agree a common standard of data collection and CDISC is mentioned as the preferred format and a collaborator. This is an area that is of particular interest to CPT as we wish the data from the trials that we support to be used in the best way possible for the advancement of the understanding of the condition and the improvement of clinical trial design, therefore the format of data collection is crucial. The first work plan for the consortium is as follows:

“The primary objective of this international consortium is to collect, standardize, and aggregate clinical trial data in order to create a database that will enable robust description of the disease progression of PD in defined subsets of patients. This database will then be used to support the development of quantitative drug development platforms for PD, which will be put through a formal review process at the FDA and EMA for regulatory endorsement. This process is a key element that will facilitate the development and registration of future potential new medicines to treat PD. C-Path data management will  use PD specific CDISC clinical data standards, the format recommended by regulatory agencies." 

The consortium has access to data from several large PD studies and have committed to contribute it to the consortium for analysis. It will be brought into a data set compliant with the CDISC format. We assume that future additions to this valuable database will ideally need to be in CDISC format - another reason for CPT to consider how best to ensure that its clinical trials align with streamlined regulatory review of new drugs.

Please see the CPP webpage for further information.

Update November 2016:

Further evidence of the benefits of sharing data and CPT’s involvement in the CPP Initiative – 'The regulators issue letters of support for the use of imaging biomarker for dopamine uptake in clinical trials' - read the full article here.

Interestingly in the UK, NICE are also proposing positive support for the use of this imaging process in the revision of its current guidelines for PD – CPT have been involved in the consultation and have submitted comments in line with patients views.

Update February 2017- CPP Statement:

 First Year Successes:

Clinical Trials are the most costly and lengthy part of the development of new drugs to treat Parkinson’s. Critical Path for Parkinson’s is working with researchers, drug companies and regulators to change clinical trials so they're smarter, more likely to succeed and meet regulatory approvals. What have we achieved in the first year?

Global community working together:

Nine major pharmaceutical companies have joined the project during this first year together with academic organizations, government agencies, patient advocacy organizations and experts at the forefront of Parkinson’s research. CPP provides an international platform for these members to work together in gaining support from worldwide regulatory agencies for new ways of designing clinical trials in Parkinson’s to ensure that new drugs are tested in the best way possible. This cooperation and unification in regulatory interaction allows companies to prioritize their own time and resources towards Parkinson’s disease drug development programs.

Another important benefit of CPP is that it provides a platform for charities to work together and alongside industry towards a united aim with people living with Parkinson’s at the core. Five Parkinson’s charities from the UK and USA are working together in CPP: (Parkinson’s UK, Parkinson ’s Disease Foundation, Davis Phinney Foundation, Michael J Fox Foundation and The Cure Parkinson’s Trust).  This important initiative is strengthening the global voice of people with the condition and has encouraged us to seek additional ways we can work together. We can better help people affected by Parkinson’s by maximizing our combined resources and work in a collaborative way.

Sharing data

CPP is using data in the most efficient way to improve outcomes for people affected by Parkinson’s. Critical Path Institute has been given access to data from high quality clinical research past and present and has combined these data into a single database. So far, the database of Parkinson’s disease studies includes information from over 4,300 subjects who have agreed that their data, which was collected in a clinical study, can be shared.

Improving Clinical Trials

Critical Path for Parkinson’s consortium received a ‘letter of support’ from the European Medicines Agency (EMA) in October 2016 which confirms that the EMA is officially supporting the use of a specific brain scan, dopamine transporter imaging, as a diagnostic for people in the earliest stages of Parkinson's to participate in clinical trials. This is great news and follows similar support from the FDA given in 2015. Diagnosis of Parkinson’s is more difficult in the early stages. Support from the EMA and FDA for dopamine transporter imaging helps to ensure that each person who participates in a clinical trial of a new drug for Parkinson’s actually has Parkinson’s disease, so trial participants are not incorrectly treated. Dopamine transporter imaging may increase the efficiency of clinical trials of new treatments.

Bringing together knowledge and patients’ experiences allows researchers in companies and regulatory agencies, together with other experts in Parkinson’s to better understand the condition, with the potential for earlier detection and comprehensive assessment of progression. This will help us to find out how new drugs could work most efficiently.