UK: Exceptions To The Usual Routes For Applying For Marketing Authorisations

Last Updated: 2 November 2006
Article by Anna Wray


There are four procedures for applying for a marketing authorisation for a medicinal product. These are a national procedure, a centralised procedure, the mutual recognition procedure and the decentralised procedure. Under these procedures there are a number of different types of application that can be made. There are also a number of exceptions to the usual routes and legislation/ guidance has recently been published on three exceptions, namely, the conditional marketing authorisation, compassionate use and accelerated assessment.

This article considers the scope of the legislation/ guidance and discusses the impact that these will have on industry.


Under Directive 2001/83/EC (as amended) ("the Medicinal Code") and Regulation (EC) 726/2004 ("the Regulation"), before a medicinal product can be placed on the market it must obtain an MA. This means that that the quality, safety and efficiency has to be demonstrated by submitting the results of extensive pre-clinical and clinical trials and tests.

However, certain categories and types of medicinal products justify the submission of less data in support of the use of the medicinal product, and in certain limited circumstances, the manufacturer may be authorised to place their medicinal product on the market. Further, in certain circumstances it may be appropriate to expedite the entry of a medicinal product onto the market.

In order to harmonise the situation across the EU, the Commission has recently published legislation/ guidance on three such situations.

Scope Of The Legislation/ Guidance

Regulation (EC) 507/2006 on the Conditional Marketing Authorisation

Regulation (EC) 507/2006 is published under Article 14(7)1 of the Regulation. A conditional marketing authorisation ("cMA") will be granted based on an incomplete dossier in respect of the clinical section. The cMA will be granted subject to specific obligations. A cMA is to be used as a way to meet unmet needs of patients and in the interests of public health and is intended to be used for three types of medicinal products:

  • those that aim at the treatment, prevention of diagnosis of seriously debilitating or life threatening diseases; or
  • those to be used in emergency situations as recognised by the WHO or the EU; or
  • orphan medicinal products.

In order to be granted a cMA, four requirements must be met (two positive and two negative). The four requirements are:

  • the risk-benefit balance of the medicinal product is positive;
  • it is likely that the applicant will be in a position to provide the comprehensive data set;
  • unmet clinical needs will be fulfilled2; and
  • the benefit to public health by the availability of the medicinal product outweigh the risks caused by the missing data3.

The cMA holder will be required to complete ongoing studies or to conduct new studies in order to confirm that the risk-benefit balance remains positive and to provide the additional data. In addition, obligations in relation to pharmacovigilance data may also be imposed. The status of the cMA must be clearly outlined in the summary of product characteristics and the package leaflet.

A cMA will be granted for a period of one year and can be renewed annually. If the specific obligations are met, the cMA should be converted to a full marketing authorisation.

Further guidelines concerning the application and the practical arrangements for obtaining a cMA are due to be drawn up.

Guideline On Compassionate Use

There are a number of national programmes for compassionate use available in the different Member States, either on a named patient basis (such as in the UK, where "specials" are available, for example, a liquid formulation of an analgesic usually available in tablet form) or for a group of patients. The guideline has been published under Article 83(1)4 of the Regulation so that Member States can request an opinion from the CMHP regarding the conditions for compassionate use of a medicinal product, although it is without prejudice to Member States’ competence on this matter and in fact, Article 83 is intended to be complementary to national legislations.

Under the guideline, a compassionate use programme is intended for EU patients who suffer from a disease for which no satisfactory remedy exists and/ or who cannot enter a clinical trial. The objectives are threefold:

  • to improve access for EU patients to compassionate use programmes;
  • to follow a common approach for the conditions of use, distribution and types of patients; and
  • to increase transparency between the Member States regarding available treatment.

In order to set up a compassionate use programme, there are several requirements that must be met:

  • the product must be unauthorised in the EU;
  • the patient has a serious/ chronic illness (e.g. cancer/ HIV/ Aids);
  • the use of the product will be for a group of patients (i.e. use of a product on a named patient basis is outside the scope of the guidelines);
  • the product must be the subject of an application for a centralised marketing authorisation or be undergoing clinical trials.

There are several points that should be noted about a compassionate use programme:

  1. although safety data may be collected during a compassionate use programme, it is not a substitute for clinical trials;
  2. the compassionate use programme cannot be used for an authorised product which is used for an unauthorised indication (i.e. off-label use).

Notification of the EMEA is mandatory for products which would have to be authorised via the centralised procedure. For products for which the centralised procedure is optional, the Member States may notify the EMEA, although the CHMP can also decide to give an opinion. The applicant should not contact the EMEA of their own volition, but may inform the EMEA of compassionate use programmes in the Member States.

Guideline On Accelerated Procedure

The guideline has been published under Article 14(9)5 of the Regulation and sets out the procedure for applying for an accelerated assessment for a marketing authorisation whereby the time period of 210 days to give an opinion would be reduced to 150 days.

Applicants requesting an accelerated assessment will be required to justify that the product is expected to be of a major public health interest and innovative in terms of the therapy. The CHMP opinion will be based on the material submitted to justify the request and will not be based on the dossier submitted for the marketing authorisation. At any time during the accelerated assessment procedure, the CHMP may decide that an accelerated assessment is no longer appropriate and revert to the normal procedure.

The applicant is expected to justify on a case-by-case basis that the product is of "a major public health interest" as there is no definition of what this constitutes. It is recommended that to facilitate the accelerated assessment, the applicant pre-notify the EMEA prior to the submission of the request. In any event, the applicant is required to agree the accelerated assessment with the EMEA, prior to the submission of the marketing authorisation dossier. The applicant is also able to request pre-submission guidance from the EMEA.

The request should be a short, comprehensive document, typically 5-10 pages in length. The guidelines also lists the key items that should be addressed in the request:

  • the unmet needs and the available methods of prevention, diagnosis or treatment;
  • the extent to which the medicinal product is expected to have major impact on medical practice, its major added value, and/or how it addresses the greater unmet needs; and
  • a brief outline of the main available evidence (e.g. number of clinical trials, key results) on which the applicant bases its claim of major public health interest.

Impact On Industry

The three situations outlined above will facilitate the entry of medicinal products to the market on an expedited basis. However, for the procedures to be used, there are stringent criteria that must be met.

The cMA Regulation and the compassionate use guidelines are likely to be restricted in the number of products that are able to utilise these procedures, whereas, the accelerated assessment procedure is likely to be of more universal application. In order to use the procedures, forethought is required in the R & D and planning stages for marketing authorisations.

The pharmaceutical industry should carefully consider whether the procedures might be appropriate for a medicinal product, as there are benefits in making such applications. For blockbuster drugs, the accelerated assessment procedure is likely to enable the product to reach the market quicker, which will overall benefit patients.


1. "Following consultation with the applicant, an authorisation may be granted subject to certain specific obligations, to be reviewed annually by the Agency. The list of these obligations shall be made publicly accessible.

By way of derogation from paragraph 1, such authorisation shall be valid for one year, on a renewable basis.

The provisions for granting such authorisation shall be laid down in a Commission Regulation adopted in accordance with the procedure referred to in Article 87(2)."

2. This is defined as "a condition for which there exists no satisfactory method of diagnosis, prevention or treatment authorised in the Community or, even if such a method exists, in relation to which the medicinal product concerned will be of major therapeutic advantage to those affected."

3. It should be noted that in emergency situations a cMA may be granted based on incomplete pre-clinical or pharmaceutical data and that it is not necessary to meet the second and third requirements (outlined above).

4. "By way of exemption from Article 6 of Directive 2001/83/EC Member States may make a medicinal product for human use belonging to the categories referred to in Article 3(1) and (2) of this Regulation available for compassionate use."

5. "When an application is submitted for a marketing authorisation in respect of medicinal products for human use which are of major interest from the point of view of public health and in particular from the viewpoint of therapeutic innovation, the applicant may request an accelerated assessment procedure. The request shall be duly substantiated.

If the Committee for Medicinal Products for Human Use accepts the request, the time-limit laid down in Article 6(3), first subparagraph, shall be reduced to 150 days."

The content of this article is intended to provide a general guideto the subject matter. Specialist advice should be sought about your specific circumstances.

To print this article, all you need is to be registered on

Click to Login as an existing user or Register so you can print this article.

In association with
Related Video
Up-coming Events Search
Font Size:
Mondaq on Twitter
Register for Access and our Free Biweekly Alert for
This service is completely free. Access 250,000 archived articles from 100+ countries and get a personalised email twice a week covering developments (and yes, our lawyers like to think you’ve read our Disclaimer).
Email Address
Company Name
Confirm Password
Mondaq Topics -- Select your Interests
 Law Performance
 Law Practice
 Media & IT
 Real Estate
 Wealth Mgt
Asia Pacific
European Union
Latin America
Middle East
United States
Worldwide Updates
Check to state you have read and
agree to our Terms and Conditions

Terms & Conditions and Privacy Statement (the Website) is owned and managed by Mondaq Ltd and as a user you are granted a non-exclusive, revocable license to access the Website under its terms and conditions of use. Your use of the Website constitutes your agreement to the following terms and conditions of use. Mondaq Ltd may terminate your use of the Website if you are in breach of these terms and conditions or if Mondaq Ltd decides to terminate your license of use for whatever reason.

Use of

You may use the Website but are required to register as a user if you wish to read the full text of the content and articles available (the Content). You may not modify, publish, transmit, transfer or sell, reproduce, create derivative works from, distribute, perform, link, display, or in any way exploit any of the Content, in whole or in part, except as expressly permitted in these terms & conditions or with the prior written consent of Mondaq Ltd. You may not use electronic or other means to extract details or information about’s content, users or contributors in order to offer them any services or products which compete directly or indirectly with Mondaq Ltd’s services and products.


Mondaq Ltd and/or its respective suppliers make no representations about the suitability of the information contained in the documents and related graphics published on this server for any purpose. All such documents and related graphics are provided "as is" without warranty of any kind. Mondaq Ltd and/or its respective suppliers hereby disclaim all warranties and conditions with regard to this information, including all implied warranties and conditions of merchantability, fitness for a particular purpose, title and non-infringement. In no event shall Mondaq Ltd and/or its respective suppliers be liable for any special, indirect or consequential damages or any damages whatsoever resulting from loss of use, data or profits, whether in an action of contract, negligence or other tortious action, arising out of or in connection with the use or performance of information available from this server.

The documents and related graphics published on this server could include technical inaccuracies or typographical errors. Changes are periodically added to the information herein. Mondaq Ltd and/or its respective suppliers may make improvements and/or changes in the product(s) and/or the program(s) described herein at any time.


Mondaq Ltd requires you to register and provide information that personally identifies you, including what sort of information you are interested in, for three primary purposes:

  • To allow you to personalize the Mondaq websites you are visiting.
  • To enable features such as password reminder, newsletter alerts, email a colleague, and linking from Mondaq (and its affiliate sites) to your website.
  • To produce demographic feedback for our information providers who provide information free for your use.

Mondaq (and its affiliate sites) do not sell or provide your details to third parties other than information providers. The reason we provide our information providers with this information is so that they can measure the response their articles are receiving and provide you with information about their products and services.

If you do not want us to provide your name and email address you may opt out by clicking here .

If you do not wish to receive any future announcements of products and services offered by Mondaq by clicking here .

Information Collection and Use

We require site users to register with Mondaq (and its affiliate sites) to view the free information on the site. We also collect information from our users at several different points on the websites: this is so that we can customise the sites according to individual usage, provide 'session-aware' functionality, and ensure that content is acquired and developed appropriately. This gives us an overall picture of our user profiles, which in turn shows to our Editorial Contributors the type of person they are reaching by posting articles on Mondaq (and its affiliate sites) – meaning more free content for registered users.

We are only able to provide the material on the Mondaq (and its affiliate sites) site free to site visitors because we can pass on information about the pages that users are viewing and the personal information users provide to us (e.g. email addresses) to reputable contributing firms such as law firms who author those pages. We do not sell or rent information to anyone else other than the authors of those pages, who may change from time to time. Should you wish us not to disclose your details to any of these parties, please tick the box above or tick the box marked "Opt out of Registration Information Disclosure" on the Your Profile page. We and our author organisations may only contact you via email or other means if you allow us to do so. Users can opt out of contact when they register on the site, or send an email to with “no disclosure” in the subject heading

Mondaq News Alerts

In order to receive Mondaq News Alerts, users have to complete a separate registration form. This is a personalised service where users choose regions and topics of interest and we send it only to those users who have requested it. Users can stop receiving these Alerts by going to the Mondaq News Alerts page and deselecting all interest areas. In the same way users can amend their personal preferences to add or remove subject areas.


A cookie is a small text file written to a user’s hard drive that contains an identifying user number. The cookies do not contain any personal information about users. We use the cookie so users do not have to log in every time they use the service and the cookie will automatically expire if you do not visit the Mondaq website (or its affiliate sites) for 12 months. We also use the cookie to personalise a user's experience of the site (for example to show information specific to a user's region). As the Mondaq sites are fully personalised and cookies are essential to its core technology the site will function unpredictably with browsers that do not support cookies - or where cookies are disabled (in these circumstances we advise you to attempt to locate the information you require elsewhere on the web). However if you are concerned about the presence of a Mondaq cookie on your machine you can also choose to expire the cookie immediately (remove it) by selecting the 'Log Off' menu option as the last thing you do when you use the site.

Some of our business partners may use cookies on our site (for example, advertisers). However, we have no access to or control over these cookies and we are not aware of any at present that do so.

Log Files

We use IP addresses to analyse trends, administer the site, track movement, and gather broad demographic information for aggregate use. IP addresses are not linked to personally identifiable information.


This web site contains links to other sites. Please be aware that Mondaq (or its affiliate sites) are not responsible for the privacy practices of such other sites. We encourage our users to be aware when they leave our site and to read the privacy statements of these third party sites. This privacy statement applies solely to information collected by this Web site.

Surveys & Contests

From time-to-time our site requests information from users via surveys or contests. Participation in these surveys or contests is completely voluntary and the user therefore has a choice whether or not to disclose any information requested. Information requested may include contact information (such as name and delivery address), and demographic information (such as postcode, age level). Contact information will be used to notify the winners and award prizes. Survey information will be used for purposes of monitoring or improving the functionality of the site.


If a user elects to use our referral service for informing a friend about our site, we ask them for the friend’s name and email address. Mondaq stores this information and may contact the friend to invite them to register with Mondaq, but they will not be contacted more than once. The friend may contact Mondaq to request the removal of this information from our database.


This website takes every reasonable precaution to protect our users’ information. When users submit sensitive information via the website, your information is protected using firewalls and other security technology. If you have any questions about the security at our website, you can send an email to

Correcting/Updating Personal Information

If a user’s personally identifiable information changes (such as postcode), or if a user no longer desires our service, we will endeavour to provide a way to correct, update or remove that user’s personal data provided to us. This can usually be done at the “Your Profile” page or by sending an email to

Notification of Changes

If we decide to change our Terms & Conditions or Privacy Policy, we will post those changes on our site so our users are always aware of what information we collect, how we use it, and under what circumstances, if any, we disclose it. If at any point we decide to use personally identifiable information in a manner different from that stated at the time it was collected, we will notify users by way of an email. Users will have a choice as to whether or not we use their information in this different manner. We will use information in accordance with the privacy policy under which the information was collected.

How to contact Mondaq

You can contact us with comments or queries at

If for some reason you believe Mondaq Ltd. has not adhered to these principles, please notify us by e-mail at and we will use commercially reasonable efforts to determine and correct the problem promptly.