The current lack of standards makes it difficult for life sciences sponsors to demonstrate the safety and efficacy of their Cell and Gene Therapy products (CGTP) and impedes regulatory reviews.
By establishing CDISC standards which are required by the FDA, sponsors will be able to accelerate their clinical development and regulatory reviews by having clear expectations from regulators as regards to safety and efficacy evidence generation.
The standards will cover 3 areas:
Once released, the standards will be freely available via the CDISC website.
We are looking to partner with a limited number of biotechs and pharmas to provide us with use cases as input to the standards. Partners will benefit by having their experimental assays and bioinformatics protocols aligned with FDA’s requirements and the future standards.
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Cell and Gene Therapies are a new transformational category of drugs which uses genetically modified organisms to cure or prevent diseases which often have no cure.
Because research and development for these new drugs are very recent, there are no detailed standards for producing efficacy and safety evidence. Furthermore, because cell and gene therapies are genomically modified live organisms, monitoring their activity in the patient is much more complex than for small molecule drugs. The absence of standards and inherent complexity lead to long regulatory approval cycles as sponsors struggle to meet unpredictable requirements from the regulators and delay the availability of new cures to patients.
The Clinical Data Interchange Standards Consortium (CDISC) and Embleema are partnering to co-develop standards for cell and gene therapy monitoring. Developing such standards will facilitate the dialog between regulators and sponsors, accelerate drug development and approvals and ultimately benefit patients as new cures are available sooner.
CDISC standards are required by the United States Food and DrugAdministration (FDA) and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA),recommended by the China National Medical Products Administration (NMPA) and adopted bythe world’s leading research organizations. Embleema’s HIVE software is the only tool authorized at the FDA for regulatory reviews of data and analytics evidence for CGTP and has deep expertise in building regulatory-grade experimental assays and bioinformatics pipelines.
CDISC and Embleema will involve biopharma, regulators, academia and scientific organizations in the processing of developing the new standards.
It is our intent to partner with the FDA in developing the new standards. Both CDISC and Embleema have a track record of working with the FDA, which require CDISC standards for regulatory submissions.