Authorisation Procedures for medicinal products
Procedures for evaluating medicinal products and granting marketing authorisation
The EMA and the authorisation procedure
What is a Marketing Authorisation needed for?
- Cover letter
- Application form and annexes (e.g. manufacturing licences, proof of payment of fees, letters of access)
- Product Information including the Summary of Product Characteristics (SmPC), labelling, patient leaflet, braille, results of readability testing
- Information about the experts
- Specific requirements for different types of applications
- Environmental Risk Assessment
- Pharmacovigilance system description
- Risk Management Plan
Are there specified requirements for formatting such as filenames, margins, font type and size, hyperlinking, etc?
Are there different National administrative requirements (i.e. delivery addresses, number of copies, paper/electronic, etc)?
Mutual Recognition Procedure
- Derived from biotechnology processes, such as genetic engineering
- Intended for the treatment of HIV/AIDs, cancer, diabetes, neurodegenerative disorders or autoimmune diseases and other immune dysfunctions
- Orphan medicinal products
|Centralised Procedure||Decentralised and Mutual Recognition Procedures||National Procedure|
|Less flexible procedure as only one tradename and one MA holder can be used||More flexible procedure as different tradenames and MA holders can be used||One tradename and oneMAH|
|The EMA chooses the Rapporteur / Co-Rapporteur for the assessment||The applicant chooses theRMS with the most relevant expertise||The applicant chooses the country|
|All or nothing procedure as the MAA is either accepted or rejected in all EU Member States||Can withdraw MAA from Member States that are causing difficulties, whilst continuing with applications in other states||Can withdraw the MAA at anytime|
|One application and one assessment||Multiple assessments and complex administration procedures||One application and one assessment|
|Well defined timescale throughout the procedure||Although the main assessment has a defined timescale, the national phase can be long and undefined||Undefined assessment timeline|
- according to Article 8(3) of Directive 2001/83/EC, relating to full applications containing all appropriate quality, nonclinical and clinical data;
- according to Article 10 of Directive 2001/83/EC, relating to generic medicinal products and similar biological medicinal products;
- according to Article 10a of Directive 2001/83/EC, relating to applications relying on well-established medicinal use supported by bibliographic literature;
- according to Article 10b of Directive 2001/83/EC, relating to applications for new fixed combination products;
- according to Article 10c of Directive 2001/83/EC, relating to informed consent from a marketing authorisation holder for an authorised medicinal product.
Orphan Medicinal Product Designation in the EU
What are the criteria for orphan designation?
- It is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting no more than 5 in 10,000 people in the EU at the time of submission of the application.
- It is intended for the diagnosis, prevention or treatment of a life-threatening, seriously debilitating or serious and chronic condition and, without incentives, it is unlikely that the revenue after marketing of the medicinal product would cover the investment in its development.
- No satisfactory method of diagnosis, prevention or treatment of the condition concerned is authorised, or;
- If a satisfactory method exists, the medicine in question must be of significant benefit to individuals affected by the condition.
When can an orphan designation be applied for?
How much does an application for OMPD cost?
- EMA Scientific Advice procedure, designed to help sponsors optimise development of their orphan medicinal product within the scope of the orphan indication
- Protocol assistance, also enables sponsors to ask questions related to:
- significant benefit over other satisfactory methods of treatment, diagnosis, or prevention of the indication in question
- development of medicinal products for rare conditions (e.g. whether two products are deemed ‘similar medicinal products’)
- study design related to clinical superiority over similar products in the same indication
Points to consider in gaining orphan designation
- In theory, regulators require the same level of proof of efficacy and safety for orphan drugs as for other drugs. There are examples of pivotal studies being accepted with reduced patient numbers; however, this is condition or drug-specific. Any development plans should be discussed with the EMA through Protocol Assistance procedures.
- Orphan designation will not automatically allow accelerated review for marketing authorisation, conditional approval or approval under exceptional circumstances. Applicants would still need to apply for such procedures.
What is the procedure for gaining orphan designation?
- Adopt a positive opinion, or
- If a negative opinion is being considered, COMP will forward a list of questions to the sponsor. The sponsor will be advised as to whether written responses or an oral explanation will be required in time for the next COMP meeting
At Day 90 the COMP will either:
- Adopt a positive opinion to be forwarded to the European Commission, or
- In the absence of sufficient justification in the written response or oral explanation, adopt a negative opinion
On receipt of a negative opinion a sponsor can appeal. However, the intent to do so should be notified without delay. Negative opinions will be published on the EMA website, but the EMA offer the possibility of withdrawing applications likely to receive a negative opinion before adoption takes place at the COMP meeting.
What information is required for OMPD applications?
- Cover Letter – identifying sponsor, medicinal product, proposed orphan indication
- Application form – available on EMA website
The common FDA/EMA application form can be used. However, it is important to note that there are key differences in the required supportive documentation for application in the two regions. Additionally, applications for designation will be reviewed separately by the respective Authorities.
- Core dossier – Parts A-F in accordance with guidance document ENTR/6283/00
Part A: Description of the Condition
Part B: Prevalence of the Condition
Part C: Potential for return on Investment
Part D: Existence of other methods of diagnosis, prevention or treatment
Part E: Description of stage of development
Part F: Bibliography
- Proof of Establishment in the EU
- Letter of Authorisation (if applicable)
- Translations of product name and proposed orphan indication into all official EU languages, plus Icelandic and Norwegian
Key components of application
- the progress of development since the initial application
- updated details of the current regulatory status
- any incentives received for development of the product
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