By Published On: December 11th, 2019Categories: News

Good Registry Practice

Registries are a great source of real world data (RWD), but uncertainties about the quality of the data collected undermines confidence in the validity and reliability of the evidence generated, which is obviously a problem if you need the data to support regulatory submissions/commitments or reimbursement discussions.

The European Medicines Agency (EMA) and the European Network for Health Technology Assessment (EUnetHTA) have provided recommendations (Good Registry Practice) and tools (Registry Evaluation and Quality Standards Tool – REQueST®) to address these quality concerns and to support the use of patient registries for regulatory purposes and health technology assessment.

These best practice recommendations and clarifications are particularly important (and timely) in the context of patient disease registries used to develop treatments for rare diseases and to address regulatory commitments e.g., imposed post- authorisation safety studies (PASS) and long-term follow-up of patients treated with advanced therapy medicinal products (ATMPs), such as gene therapy for haemophilia and CAR-T cell therapy for cancer(s).

Too little, too late? Definitely not! As noted by China’s National Medical Products Administration (NMPA) earlier this year:

“At present, there are no mature and relevant regulations worldwide. Without sufficient experience, how to formulate guidelines that fit the reality of China’s [+ rest of world] pharmaceutical industry requires active exploration and innovation.”

We often forget that globally harmonised standards for clinical research are relatively new. We agreed on a harmonised ‘global’ clinical trials ethical and quality standard (ICH E6, also known as ICH GCP) in 1996, and in Europe, we implemented these standards through the clinical trials directive (Directive 2001/20/EC) at a national level in 2004. That’s only 15 years ago! It’s therefore not a surprise that the regulation and governance of the lower risk clinical data sources (real world data sources), such as Patient Registries is still in its infancy.

It’s early days yet, with a lot of work needed in the near future, but the good news is that registry holders and pharmaceutical companies now have tangible considerations and recommendations from European regulators that can be acted on to improve confidence in the validity and reliability of RWE generated from Patient Registry data.

Is this a step closer to the Registry equivalent of GCP?

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