The prestigious academic journal, Lancet Respiratory Medicine, has published a spotlight feature on the UK Cystic Fibrosis Registry’s framework for working with industry to make medicines safer. The programme, which began in 2012, has been a model for CF registries across the world, and a gold standard for other conditions.
This publication will help other charities and research organisations create similarly successful programmes, which enable medicines regulators to monitor drug safety using the best methods possible.
The Registry’s main goal is to improve patient outcomes; and engaging in studies of newly licensed drugs, monitoring safety and effectiveness, helps achieve this aim.
After clinical trials are completed, during the licensing process for the new drug, regulators like the European Medicines Agency decide on whether a long-term study is required. The main reason is that additional monitoring may be needed, ‘in the real world’ to get a more comprehensive and long-term understanding of safety and/or effectiveness.
These studies usually last over five years so that longer term benefits and any problems can be identified.
How you are helping the Registry lead the way
The UK CF Registry has been a pioneer in drug safety studies, thanks to the high engagement of people with CF (99% of people with CF consent to participating), and the hard work of clinical teams, enabling the Registry to be a world-leader in data-driven research and quality improvement.
The drug safety programme involves providing reports of non-identifiable data to the European Medicines Agency (EMA). The EMA requires pharmaceutical companies that have approval to market a medicine to provide it with reports on long-term safety. By directing pharmaceutical companies to existing Registries like the UK CF Registry, the EMA is able to guarantee the use of high quality data provided by an independent third party.
The pharmaceutical company covers the costs of this work, which in turn supports the Registry infrastructure, enriching the data resource for the CF community. This means no extra burden on patients or clinical teams, and the income from providing this service to the EMA can reimburse CF centres for the cost of collecting Registry data, through special UK CF Registry support grants.
Bigger is better
When drug safety studies are run without a Registry, this often means that only those taking the company’s medicine are monitored. This can make it difficult to tell whether any potential effects of the medication, good or bad, are actually to do with the medicine, or just part and parcel of the existing medical condition. Using a Registry, with ‘all comers’ data, a specially matched comparison group can be used to assess whether changes seen in the population taking the medicine are associated with the medicine itself, or not.
Non Registry-based drug safety studies can also mean that medical teams have to spend time entering data that is collected elsewhere. Using a Registry means that routinely collected information can be used retrospectively, while maintaining the confidentiality of patients who have consented to have their data stored in the Registry.
Listen to a podcast featuring Professor Di Bilton, of Imperial College London and the Royal Brompton Hospital, accompanying the Lancet spotlight.
Read more about how the CF Registry team works with industry.
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