Post marketing surveillance (PMS) is part of the drug development process (also known as Phase IV) and is mandated by the pharmaceutical regulators (FDA, EMA and other national bodies). This is to allow for the collection of safety and efficacy data once a drug has received (conditional) marketing approval and to assess how it performs in 'the clinical setting'. PMS data has to be collected on every patient receiving the drug, or an agreed number of these patients, and is usually part of long-term data collection that can run for a period of about 10-15 years. Its important to bear in mind that a PMS registry has to fulfil certain requirements:
- Regulatory compliant data capture, storage and processing in a certified database system
- Accurate and reliable measures of patient health and well being
- Technical solution needs to be accessible, sustainable, and resource-sparing to patients and families
The regulators have expressed a preference for the development of disease specific PMS systems over drug-specific ones, especially for rare diseases. Also, there is an increasing recognition by industry that a shared post marketing infrastructure for a specific disease group would facilitate and expedite PMS, while conserving resources.
Setting up a disease specific PMS system is however a very complex issue with many stakeholders to be considered, including patients, patient organisations, patient registries, clinicians, regulators and the relevant pharmaceutical companies. The TREAT-NMD Executive Committee has been in active consultation with representatives of all of these stakeholders over the past year to ascertain the feasibility of developing a disease-specific PMS platform.
As a result of the consultation, it is clear that having a PMS platform integrated into the resources of our neuromuscular community has several advantages:
- Data oversight (involving patient representatives and academics)
- Transparency of data collection, access and usage
- Quicker access to add new drugs
- Showing clinical benefit (efficacy) over the long term will aid in the justification for reimbursement by health agencies and insurance companies.
- Reduces fragmentation of data
- Possibility to also include Natural History data collected at the same participating sites into the same platform
- Possibility to exchange data with national registries
- Conservation of resources (patient, family, physician, clinic), as an post marketing infrastructure shared by many/most drugs would enable coordination and consistency of data collection
Disease group specific (shared) PMS platforms do not yet exist and traditionally pharmaceutical companies have set up their own drug-specific databases for the mandatory data collection. Translarna is the first drug to receive conditional marketing authorization for DMD, and is already available in some countries. Other drugs are in the pipeline for DMD and also for SMA. Therefore, there is an urgent need for a PMS system, since for Translarna, PTC will have to start collecting PMS data on their drug shortly.
We feel this poses an exciting opportunity for the neuromuscular community as a whole to build on the existing registry initiatives to develop a PMS platform that can not only be used for Translarna but for other drugs in the future. The consultation which has been performed indicates that TREAT-NMD has the opportunity to act as an independent third party that can bring together a public-private partnership of this kind and deliver a disease-specific PMS platform for the neuromuscular community on a national and international level.
This is not an exhaustive list of FAQs but are there mainly to highlight the main area around the subject. If you do have any additional questions that have not been answered satisfactorily in this section then please feel free to contact us.