Orphan medicinal products - Major developments

 28/03/2014

Guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to anotherpdf(85 KB), March 2014

  • Annex to Guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another,date [doc fprmatmsw8(117 KB)] - [pdf formatpdf(103 KB)]

25/10/2013
Responses to the public consultation on the guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another

5/07/2013
Public consultation on the guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another
Today, the unit in charge of pharmaceuticals launches a public consultation on guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another

Guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another (12 June 2013)pdf(76 KB) - Word version with track changesmsw8(133 KB)

Annex: Application form for orphan medicinal product designation (Rev. 4, 22 April 2013)pdf(102 KB) - Word version with track changesmsw8(124 KB)

Comments and suggestions are invited by 30 September 2013 and should be sent by email to:
sanco-pharmaceuticals-d5@ec.europa.eu

21/1/2009

Implementation of the Regulation of Orphan Medicinal Products: Public call for expressions of interest for Commission appointees to the Committee for Orphan Medicinal Products of the European Medicines Agency.
The Commission publishes today a public call for expressions of interest related to the appointment by the European Commission of members representing patients' organisations at the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMEA). The public call is available herepdf .
Expressions of interest should be notified to the European Commission by e-mail to : entr-pharmaceuticals@ec.europa.eu .
The deadline for receipt of the notifications is Friday 13 March 2009

19/9/2008

Guideline on aspects of the application of Article 8(1) and (3) of Regulation (EC) No 141/2000: Assessing similarity of medicinal products versus authorised orphan medicinal products benefiting from market exclusivity and applying derogations from that market exclusivitypdf(109 KB)

This guideline concerns the granting of market exclusivity for authorised orphan medicinal products according to Article 8(1) of Regulation (EC) No 141/2000, in particular the assessment of the "similarity" criterion - and the granting of derogations from that market exclusivity under Article 8(3) of that regulation.

17/9/2008

Guideline on aspects of the application of Article 8(2) of Regulation (EC) No 141/2000: Review of the period of market exclusivity of orphan medicinal productspdf(40 KB) This guideline concerns the review of market exclusivity for authorised orphan medicinal product according to Article 8(2) of Regulation (EC) No 141/2000. It sets out the general principles and procedures by which the period of market exclusivity of orphan medicinal products is reviewed and may be reduced from ten to six years.

11/7/2007

Update of the "guideline on format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another"pdf(65 KB)

The "guideline on format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another" has been updated: sponsors will from now on be required to provide only ONE paper version of their application (instead of three paper versions); instead, an additional electronic version (CD Rom) will be requested.

07/03/2007

Consultation on a draft "Guideline on aspects of the application of Article 8(2) of Regulation (EC) No 141/2000: Review of the period of market exclusivity of orphan medicinal products"pdf(44 KB)

Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products lays down a Community procedure for the designation of medicinal products as orphan medicinal products and provides incentives for the research, development and placing on the market of such products. In particular, Article 8(1) provides for a period of ten years of market exclusivity where a marketing authorisation in respect of an orphan medicinal product is authorised throughout the Community. In turn, Article 8(2) provides that this period may be reduced to six years if it is established that the designation criteria are no longer met. In this respect, this draft guideline sets out the general principles and procedures by which the period of market exclusivity of orphan medicinal products is reviewed and may be reduced to six years.

We invite comments on the draft guideline, to be sent to claire.scharf-kroener@ec.europa.eu until 7 May 2007.

24/10/06

Update of the annex to Guideline on format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to anotherpdf(243 KB)

In preparation of the coming enlargement, the annex to Guideline on format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another has been updated
Annex (word format)msw8(121 KB) revision October 2006

7/09/06

Inventory of Community and Member States' incentive measures to aid the research, marketing, development and availability of orphan medicinal products. Revision 2005pdf(262 KB)

The Regulation on Orphan Medicinal Products adopted in 2000 requires Member States to communicate to the Commission detailed information concerning any measure that they have enacted to support research into, and the development and availability of, orphan medicinal products or medicinal products that may be designated as such. On the basis of this information, the Commission prepared and published on 1 January 2001 a first detailed inventory of all incentives made available by the Community and the Member States to support research into and availability of orphan medicinal products, thus meeting its obligations according to the regulation. This inventory has been updated in 2006 for the second time.

27/06/06

Commission Staff working document on the experience acquired as a result of the application of Regulation (EC) No 141/2000 on orphan medicinal products and account of the public health benefits obtained (Document on the basis of Article 10 of Regulation (EC) No 141/2000)pdf(555 KB)

3/04/06

Orphan Medicinal Products: Outcome of consultation on a general report on the experience acquired as a result of the application of Regulation (EC) 141/2000 on orphan medicinal products and account of the public health benefits obtained.

As required by Article 10 of Regulation (EC) 141/2000 on orphan medicinal products, the European Commission has drafted a Commission Staff Working Document on the experience acquired as a result of the application of Regulation (EC) 141/2000 on orphan medicinal products and account of the public health benefits obtained.
All interested parties were invited to comment on the draft Commission Staff Working Document until 8 March. The outcome of the consultation is now published until publication of final documentpdf(19 KB)

8/02/06

Consultation on a general report on the experience acquired as a result of the application of Regulation (EC) No 141/2000 on orphan medicinal products and account of the public health benefits obtained

As required by Article 10 of Regulation (EC) No 141/2000 on orphan medicinal products, the European Commission has drafted a Commission report on the experience acquired as a result of the application of Regulation (EC) No 141/2000 on orphan medicinal products and account of the public health benefits obtained. All interested parties are invited to comment on the attached draft Commission Staff Working Documentpdf(414 KB) .Comments should be sent to Elena Prats, European Commission, BREY 10/130, 1049 Brussels, Belgium.

E-mail submissions are preferred and should be sent to elena.prats@ec.europa.eu The consultation will be closed on 8 March 2006.

20/12/04

Study on the price of orphan drugs (see 16/11/04) ERRATUM: Etude relative au prix des médicaments orphelins - Phase I (Français)pdf(12 KB)ERRATUM: Study on the price of orphan drugs - Phase I (English)pdf(12 KB)

16/12/04

Draft guideline on similarity of medicinal products and "clinical superiority" related to the orphan regulation

The Commission invites comments on a draft guideline on similarity of medicinal products and "clinical superiority" related to the orphan regulation (Regulation (EC) No 141/2000 of the European Parliament and of the Council on orphan medicinal products). Comments should be sent by 4 February 2005 to Dr Peter Arlett, DG Enterprise and Industry. Electronic submissions are preferred and should be sent before the deadline to entr-pharmaceuticals@ec.europa.eu
Please note that the guideline does not cover the application of Article 8 paragraph 2 of the Regulation relating to the reduction of the period of market exclusivity.
The draft guideline can be found by clicking here.pdf(119 KB)

16/11/04

Study on the price of orphan drugsThe Commission has published an independent study on the price of orphan drugs. The study, conducted by Alcimed, looks at the price of orphan drugs authorised in the EU and how these prices were arrived at. In addition, the study discusses how sufficient profitability might be assessed and judged. This latter aspect relates to the operation of Article 8 (market exclusivity) of the Orphan Regulation (Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products).The study will inform discussions on the development of both guidelines relating to the operation of Article 8 and the 2006 Commission report foreseen in Article 10 of the Orphan Regulation.Any opinions in this report are those of the authors and do not necessarily reflect those of the European Commission
Please note that the French version is the original one. The English version is a translation.

26/08/04

Revised Commission guideline on applications for orphan medicinal productspdf(143 KB)
The Commission guideline on the "format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another" has been updated. The revisions to the guideline include reference to Icelandic and Norwegian participation in the designation process, EU Enlargement, and cross-reference to the Commission Communication (2003/C 178/02).The Commission conducted a three-month public consultation on the revisions. Two responses to the consultation were received, from the Bioindustry Associationpdf(7 KB) and the Emerging Biopharmaceutical Enterprises grouppdf(31 KB) within the European Federation of Pharmaceutical Industry Associations. The responses were carefully considered by the Commission in consultation with the EMEA Committee for Orphan Medicinal products. Where possible, the changes suggested in the responses have been included. Some of the suggestions were considered outside the scope of the current revision and will be taken into consideration in the overall review of the orphan regulation foreseen for 2005.
Annex (word format)

01/08/03

Commission Communication on Regulation (EC) No 141/2000 on orphan medicinal products

Following the first three years of application of the Regulation on orphan medicinal products, the Commission has issued a Communication which sets out a position on certain matters relating to the implementation of the designation and market exclusivity provisions. This has been done in response to requests for interpretation and clarification.
The Communication is intended to provide guidance to the European Medicines Evaluation Agency, the Member States, the pharmaceutical industry and other interested parties.
The Communication considers points in relation to:

  • Article 3 (criteria for designation)
  • Article 5 (procedure for designation and removal from the Community register on orphan medicinal products)
  • Article 7 ( Community marketing authorisation)

In addition, the Commission is obliged to draw up detailed guidelines on the application of Article 8 of Regulation (EC) No 141/2000. This obligation is met in part by section D on market exclusivity (Article 8) in the Communication.
The Communicationpdf is published in the Official Journal of the European Union C 178, 29.07.2003, p. 2.

14/10/02

First revision and update of guideline on format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another.pdf(156 KB)

Following the end of the comment period, and in accordance with Article 5. 3 of Regulation 141/2000 on orphan medicinal products a first revision and update of the format and contents guideline is now available.
[ Word Formatmsw8(117 KB) ] - .pdf Formatpdf(156 KB) ]

27/08/02

DG Enterprise publishes an update of the inventory of national and community measures supporting research into and availability of orphan medicinal products.pdf(230 KB)

The Regulation on Orphan Medicinal Products adopted in 2000 requires Member States to communicate to the Commission detailed information concerning any measure that have enacted to support research into, and the development and availability of, orphan medicinal products or medicinal products that may be designated as such. On the basis of this information, the Commission prepared and published on 1 January 2001 a first detailed inventory of all incentives made available by the Community and the Member States to support research into and availability of orphan medicinal products, thus meeting its obligations according to the regulation. This inventory has been updated for the first time.

12/04/02

Final version of guideline on format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another.pdf(181 KB)
Following the end of the comment period, and in accordance with Article 5. 3 of Regulation 141/2000 on orphan medicinal products a final version of the format and contents guideline is now available.
The annex to this guideline is available in a downloadable form. [ PDF Formatpdf(217 KB) ] [ Word formatmsw8(85 KB) ]

12/02/02

The Committee for Orphan Medicinal Products Following changes to the Committee an updated list of memberspdf(133 KB) is provided.

01/06/01

DG Enterprise publishes its first inventory of national and community measures supporting research into and availability of orphan medicinal products inventorypdf(179 KB)
The Regulation on Orphan Medicinal Products adopted last year requires Member States to communicate to the Commission detailed information concerning any measure that have enacted to support research into, and the development and availability of, orphan medicinal products or medicinal products that may be designated as such. On the basis of this information, the Commission has prepared a first detailed inventory of all incentives made available by the Community and the Member States to support research into and availability of orphan medicinal products, thus meeting its obligations according to the regulation. This inventorypdf(179 KB) shall be updated regularly.

04/09/00

8th August 2000 Commission designates first three Orphan Medicinal Products

Following the entry into force of the regulation on Orphan Medicinal Products, the Commission, on 8th August 2000, adopted the first three Community decisions designating potential new compounds as Orphan medicinal products. These medicinal products have now been entered into the newly created Community Register for Orphan medicinal products. It should however be noted that the safety, efficacy and quality of these products have not yet been assessed through the Community marketing authorisation procedures and therefore they cannot at this time be marketed in the European Community. ®

28/04/00

Rules on Orphan Medicinal Products become applicable.

On 27 April 2000, the Commission adopted a regulationpdf laying down implementing rules and setting out definitions essential for the application of the regulation on Orphan Medicinal Products. This means that companies may, as from 28 April submit applications for designation as Orphan Medicinal Products to the EMEA for consideration by the newly created Committee of Orphan Medicinal Products. Submitted applications must be in accordance with the criteria laid down in this regulation draft guidelines on format and contentpdf, application formspdf and will be validated by the EMEA to ensure that all required documentation has been submitted before being provided in summary form to the Committee.

14/04/00

Commission nominates members of the Committee for Orphan Medicinal Products.

The recently adopted regulation on Orphan Medicinal Products creates a new Committee, the Committee for Orphan Medicinal Products, responsible for providing opinions on whether or not a new medicinal product should be designated as an Orphan Medicinal Product. Designation allows the medicinal product concerned access to a series of incentives including market exclusivity, fee waivers and assistance with the protocols for clinical trial investigations. This Committee marks an important step in the increasing co-operation between regulatory authorities and patients, being the first European Scientific Committee to have representatives from patient organisations as full members. Based on an internal selection procedure the Committee has nominated the following members to represent patient organisations, Yann Le Cam, Mois"?©s Abascal Alonso, representing the European Association for Orphan Diseases, Eurordis and Alastair Kent representing the European Alliance of Genetic Support Groups. The Commission also nominated Professor Jean-Michel Alexandre, Dr. Mary Teeling, current president and vice president of the CPMP and Dr. Gianmartino Benzi who is on the management board of the EMEA to facilitate close liaison and interaction with the work of the EMEA.
The list of members from the EU Member States is provided.pdf

31/03/00

COMMISSION REGULATION laying down the provisions for implementation of the criteria for designation of a medicinal product as an orphan medicinal product and definitions of the concepts "similar medicinal product" and "clinical superiority"

Standing Committee gave a positive opinion on 23 March 2000. The agreed text is attachedpdf. This text is now subject to linguistic verification by Member States before it will be finally adopted by the Commission.

24/01/00

Regulation on Orphan Medicinal Products enters into force

On 22/1/2000 Regulation (EC) No.141 /2000pdf of the European Parliament and of the Council of on Orphan Medicinal Products was published in Official Journal of the European Communities L18. According to Article 10 of this regulation, the regulation is now in force. Applications will be accepted from the date of adoption of its implementing regulations. The Commission is currently preparing the first draft of an implementing regulation as foreseen in Article 3(2) and Article 8(4).

15/12/99

Regulation on Orphan Medicinal Products, European Parliament, Strasbourg, 15th December 1999

The European parliament accepted the common position of the Council on the proposal for a regulation on Orphan medicinal products without amendment - an example of the increasing co-operation between the European institutions. This regulation, which was proposed by the Commission in July 1998, offers incentives to encourage the research, development and marketing of medicines to treat rare diseases, thus providing patients with these diseases with new possibilities for treatment. The regulation will enter into force on the date of its publication in the Official Journal, and will apply from the date of adoption by the Commission of two implementing regulations, which are currently being drafted by the Commission, foreseen for early March 2000.

20/10/99

European Parliament adopts timetable for second reading of Regulation on Orphan Medicinal productspdf(74 KB).
Mme Grosstête has been apppointed as rapporteur.

27/09/99

Council reaches common position on amended Orphan Medicinal Products regulation
Formal agreement on a common position on a draft regulationpdf on Orphan Medicinal products was reached in Strasbourg. The text will now be forwarded to the European Parliament for its second reading.

23/06/99

Council reaches political agreement on amended Orphan Medicinal Products regulation

Luxembourg, 21 June 1998. The Internal market Council reached a unanimous political agreement on a common position on a draft regulation on Orphan Medicinal products (see news of 15 June). The common position will be formally adopted at the next Council meeting after linguistic and legal verifications.

15/06/99

The Commission adopted its amended proposalpdf for a European Parliament and Council Regulation on Orphan Medicinal Products on 15th June. The amended proposal takes account of the amendments proposed by the European Parliament and preliminary discussions in Council. The proposal aims to establish a centralised procedure for the designation of orphan medicinal products and to put in place incentives for the research, marketing and development of orphan medicinal products, in particular by the granting of a 10 year market exclusivity period post authorisation for designated product

27/07/98

The European Commission has adopted a Proposal for a European Parliament and Council Regulation on Orphan medicinal products. The aim of this proposal is to establish a Community procedure for designating orphan medicinal products and to introduce incentives for the research, development and marketing of orphan medicinal products, by providing : protocol assistance, direct access to the Community centralised procedure, fee waivers, and exclusive marketing rights for a ten year period.

Proceedings of the Workshop on "Rare diseases and Orphan Drugs -European Perspective"pdf(288 KB) convened by the European Foundation for the Advancement of Medicine in cooperation with the European Commission - Brussels - 5 May 1998

Proposal for a European Parliament and Council Regulation (EC) on orphan medicinal productspdf