drug licensing Flashcards
(35 cards)
Who is responsible for processing drug regulations in Europe?
European Medicines Agency.
Who formulates the standards required for drug licensing to be achieved?
European Commission.
What is the role of the EMA in processing drug licensing requests?
The EMA’s primary role is to protect the health and safety of humans and animals via the evaluation of medicines intended for such use. They scientifically evaluate all applications and co-ordinate a safety monitoring system.
What are National Health Authorities?
These are smaller authorities which exist in different nations which are responsible for drug regulation in that place. They liaise with the EMA on EU-wide regulatory issues, but are responsible for the advice and assessment of applications within their own place, e.g. the UK has the MHRA.
What are Marketing Authorisation Applications?
This is the application a pharmaceutical company may make before receiving approval to license to the public. These can be submitted via a centralised route, decentralised route, mutual recognition route, or a national procedure route.
What is the centralised procedure/route?
This is a single application with the assessment co-ordinated by the EMA where the CHMP will likely carry out a scientific evaluation. The European Commission will ultimately make the decision based on EMA recommendation. Once approved, the authorisation is valid in all EU Member States. It is mandatory for advanced therapies (orphan drugs) and certain disease areas, e.g. cancer, virology, neurodegenerative diseases.
What is the decentralised procedure/route?
This is a simultaneous submission to reference member states (RMS) and concerned member states (CMS). The application is received by the RMS, who then may pass it on to CMS.
What is the Mutual Recognition Procedure?
This is used when a drug already has Marketing Authorisation in a member-state (e.g. not new products). It is therefore used to expand the use of nationally approved products to more CMS.
What is the National Procedure?
This is an application for marketing authorisation to the national health authority of a particular state.
What would be included in a Marketing Authorisation Application?
The labelling information (e.g. patient information leaflet, the packaging), any information from additional follow-up studies, the risk management plan, and the paediatric investigation plan (compliance, waivers).
What do the CHMP review in the evaluation of MAA’s?
They look at the benefit:risk ratio, the risk management plan, the product information, and assess whether there is any need to carry out any further safety/efficacy studies before approval.
What are the key responsibilities of the MHRA?
The MHRA ensure that the safety, quality, and efficacy of all medicines is maintained. They also carry out inspections (GCP, pharmacovigilance), as well as governs clinical trials and completes MAAs when received. Also enforce laws, e.g. detect illegal medicine supply.
What does legislation of authorisation of medicinal products include?
Their manufacture, import, distribution, sale, supply, advertising, labelling, pharmacovigilance.
Give examples of drugs which do not require a marketing authorisation.
Drugs may be known as ‘specials’ when they are ordered by a doctor/physician/nurse for treatment of a particular individual. For example, vitamin D supplements, midodrine tablets (used for orthostatic hypertension when fludrocordisone is not effective). However there must be sufficient evidence that there will be efficacy/safety.
Who is the regulatory body governing drug approval in the US?
The Food and Drug Administration (FDA), in particular the Centre for Drug Evaluation and Research (CDER).
How are the FDA involved in the clinical trial/drug licensing process
The FDA has mandatory consultation during each phase of development, including meetings or documentation. They may put clinical trials on hold until Sponsor’s respond to questions. After a successful phase 3 trial, the sponsor may then submit a MAA to the FDA.
How is the FDA licensing process different to the EMA process?
Although similar, the FDA hold advisory committee meetings which members of the public can attend, as well as competing pharmaceutical companies. This allows external expertise to be noted, as well as the FDA’s own receive. This helps with the opinion of if a drug should be licensed or not. A review is undertaken after this committee meeting regarding the labelling and inspection of manufacture, before action is taken.
What is accelerated approval?
Accelerated approval may be given in situations where the treatment has demonstrated a huge therapeutic benefit over standard, or in the absence of a standard treatment for life-threatening diseases (e.g. advanced metastatic cancer, Alzheimer’s disease). However, if confirmatory studies do not exhibit the same benefit, then the drug may be withdrawn.
What is Orphan Drug Legislation?
Orphan Drugs refer to treatments used in rare diseases. Separate legislation has been produced to encourage the research, development, and approval of drugs for rare diseases.
What is defined as a rare disease?
In the EU, this is classed as less than 5 in 10,000 patients. In the US, this is classed as a disease affecting less than 200,000 patients.
Why might researchers be encouraged to look at rare diseases?
The EU offers incentives to drug companies/researchers for development of orphan drugs, e.g. scientific advice on protocols, fee reductions, access to grants, and 10-year marketing protection. These drugs will also be monitored by the Committee for Orphan Medicinal Products.
What is the required structure used for MAAs or NDAs?
Applications should produce a Common Technical Document (CTD) consisting of modules 2,3,4,5 which concerns the table of contents, the introduction, the clinical overview and summary, the non-clinical overview and summary, the quality overall summary, quality, the non clinical study reports and the clinical study reports.
What happens in the MAA is successful?
After MAA authorisation, there must be approval for the SmPC and USPI regarding the labelling and detailed description of the intended use (including PK/PD effects, adverse events, precautions, dosage).
Why is a risk management plan necessary within an MAA? What does it aim to do?
Risk management plans are required to ensure there are effective means to reduce/prevent the risks associated with the medicinal product. Remember, all medicinal products have risks. It should characterise the safety profile of the drug, state how it would be characterised further after approval and licensing to public, and how these risks may be minimised, etc.
