Post Marketing Surveillance Frequently Asked Questions
What is postmarketing surveillance?
Post-marketing is the period in the drug development pathway after a therapy receives marketing authorisation (sometimes referred to as phase IV). Post-marketing surveillance refers to the collection of data on the safety, including unexpected/adverse side effects to a particular therapy, and in some cases, data on the effectiveness of the therapy to treat the condition (efficacy) in the clinical setting.
Why does post-marketing surveillance data need to be collected?
During clinical trials a drug is usually tested in a selected group of patients under agreed protocols, detailed methods and intense scrutiny to the trial study design and objectives. The aim of post-marketing surveillance data is to see how the drug performs once the therapy moves into the ‘normal clinical setting’. This includes measures of safety (side effects), as well as efficacy (improvements in patient quality of life).
What is the role of EMA or FDA?
New therapies are only available after pharmaceutical companies have received marketing authorization (or conditional marketing authorization) from regulatory authorities. In Europe this is the European Medicines Agency (EMA) and in the United States this is the Food and Drug Administration (FDA). In other countries there are similar regulatory agencies. These regulatory authorities mandate post-marketing surveillance of authorized drugs.
How is post-marketing surveillance data collected?
Traditionally pharmaceutical companies will engage a contract research organisation (CRO) to help with establishing a registry for data collection. Data reporting will normally be done by clinicians, and in some cases, the patients themselves through patient reported outcomes (PRO). This data is collected in a similar way to clinical trials when patients see their clinicians at their regular appointments. Any clinical site that sees a patient receiving a new therapy will normally enter into a contract to agree to the reporting of data for PMS purposes. Generally, clinicians are compensated for their time to enter data into a database.
What are the criteria for a post-marketing surveillance database?
All PMS data has to be entered into and stored in a regulatory compliant certified database. Similar to a clinical trial, this most often is a password-protected web-based database, which fulfils all international legal requirements for privacy and safety of data transfer.
What are the advantages of having a disease-specific (shared) PMS platform?
There has been a change in the mind-set around collecting PMS data in the community, including the regulators, academics and some patient organisations. The community would prefer a disease-specific PMS platform, collecting information on a range of drugs for one disease. This would provide greater transparency, faster reporting, reduced fragmentation of data and greater efficiency for the pharmaceutical companies by utilising an existing platform, amongst other benefits. This model would also reduce the burden on patients and clinicians, harmonising the whole data collection process. A disease specific platform could also include additional information from pre-existing patient registries and natural history datasets. It could record the occurrence of adverse events in the affected population who are not taking a drug, making the information collected on such events from the different drugs more meaningful, something which is not possible in traditional drug-specific PMS databases.
How does TREAT-NMD plan to deliver the post-marketing surveillance platform?
This is a complex issue with many stakeholders to be considered, including patients, patient organisations, patient registries, clinicians and the relevant pharmaceutical companies. The TREAT-NMD Executive Committee (including patient representatives) has been in active consultation with the stakeholders to ascertain the feasibility of developing a disease-specific PMS platform.
In response to stakeholders’ preference for a disease-specific post-marketing platform, the TREAT-NMD Executive recommendation is as follows: “A new regulatory compliant IT platform for PMS purposes will need to be developed as neither the currently existing TREAT-NMD associated registries nor the aggregate global database is set up to these standards. In collaboration with a CRO and/or an academic research organisation, such as the CINRG group (which has much of the needed infrastructure for natural history studies and clinical data for regulatory requirements), core PMS modules can be developed for the approved drugs. Customised datasets for the drug-specific modules will be developed in conjunction with the relevant pharmaceutical companies, based on regulatory requirements.
PMS will require multiple individual contracts to be set up between pharmaceutical companies’, data collection sites (e.g. hospitals) and national patient registries. TREAT-NMD has been advised that the creation of a new company/organisation (NewCo) would be the best mechanism to oversee such an effort as it would ensure rapid decision making and clear contract management. In case such a company generates income this would be used to expand and develop activities across the TREAT-NMD registries.”
How would the national patient registries be involved in the TREAT-NMD model of a disease specific post-marketing surveillance platform?
National registries will contribute to the overall post-marketing platform in a couple of different ways.
- Patient identification and recruitment
In the first instance, the organisations that take care of the national registries in countries where the drug will be made available will be engaged. At this stage the registries would be used for patient identification and recruitment. Each national registry involved will have an individual contract with NewCo which outlines the activities required and the appropriate financial reimbursement directly to the registries. The negotiation of contracts will be facilitated by NewCo\
- Prospective data collection
The prospectively collected natural history data will benefit both the PMS platform and the national registries. The data collected for the PMS will (initially) only involve a subset of patients and a subset of countries but these can be extended. The PMS data will overlap with the data collected by the national registries, but are not necessarily the same and additional data will have to be added by clinicians to fulfil the regulatory requirements. It will, however, provide means to extend or adapt the data collection by the national registries. This could facilitate the collection of more extensive prospective natural history data on larger cohorts of patients.
What are the next steps?
The TGDOC will be heavily involved and a steering committee (including patient representatives and clinicians) will be established. Meanwhile we hope to begin to set in place the mechanisms for this kind of platform to work while exploring the models of collaboration and remuneration through NewCo. The work will progress country by country as the first drug (Translarna) is rolled out. Updates will be provided regularly via the TGDOC and TREAT-NMD newsletters.