Changes from plenary report to adopted text

Added2. The application of the orphan medicine sponsor shall be accompanied by the following particulars and documentation:

Added(a) name or corporate name and permanent address of the orphan medicine sponsor;

Added(b) active substances of the medicinal product;

Added(c) proposed condition for which it is intended or the proposed therapeutic indication;

Added(d) justification that the criteria laid down in Article 63(1) or in the relevant delegated acts adopted in accordance with Article 63(2) are fulfilled and a description of the stage of development, including the expected therapeutic indication. [Am. 189]

AddedThe orphan medicine sponsor shall be responsible for the accuracy of the particulars and documentation.

Added3. The Agency shall, in consultation with the Member States, the Commission and interested parties, draw up detailed guidelines on the required procedure, format and content of applications for designation and for the transfer of the orphan designation pursuant to Article 65.

Added4. The Agency shall adopt a decision granting or refusing the orphan designation based on the criteria referred to in Article 63(1) or in the relevant delegated acts adopted in accordance with Article 63(2) within 90 days of the receipt of a valid application. The application is considered valid if it includes all the particulars and documentation referred to in paragraph 2. [Am. 190]

AddedFor the purpose of establishing whether the orphan designation criteria are fulfilled, the Agency may consult the Committee for Medicinal Products for Human Use or one of its working parties referred to in Article 150(2), first subparagraph. The outcome of such consultations shall be annexed to the decision, as part of the scientific conclusions of the Agency which justify the decision.

AddedThe decision together with the Annexes referred to in this paragraph shall be notified to the applicant.

Added5. Decisions of the Agency on granting or refusing the orphan designation shall be made public after deletion of any information of a commercially confidential nature.

AddedArticle 65 Transfer of orphan designation

Added1. The orphan designation may be transferred from a current orphan medicine sponsor to a new orphan medicine sponsor. The transfer shall be subject to prior approval by the Agency, following the submission of an application for the transfer to the Agency.

Added2. The application of the current orphan medicine sponsor shall be accompanied by the following particulars and documentation:

Added(a) name or corporate name and permanent address of the current and new orphan medicine sponsor;

Added(b) decision on granting an orphan designation as referred to in Article 64(4);

Added(c) designation number as referred to in Article 67(3), point (e).

Added(ca) reasons for the transfer of the orphan designation. [Am. 191]

Added3. The Agency shall adopt a decision granting or refusing the transfer of the orphan designation within 30 days of the receipt of a valid application by the current orphan medicine sponsor. The application is considered valid if it includes all the particulars and documentation referred to in paragraph 2. The Agency shall address its decision to the current and new orphan medicine sponsor.

AddedArticle 66 Validity of orphan designation

Added1. An orphan designation shall be valid for seven years. During this period, the orphan medicine sponsor shall be eligible for incentives referred to in Article 68.

Added2. By way of derogation from paragraph 1, on the basis of a justified request of the orphan medicine sponsor, the Agency may extend the validity, where the orphan medicine sponsor can provide evidence that the relevant studies supporting the use of the designated orphan medicinal product in the applied conditions are ongoing and promising with regard to the filing of a future application. Such an extension shall be limited in time, taking into account the expected remaining time needed to file an application for marketing authorisation.

Added3. By way of derogation from paragraph 1, where an orphan designation is valid at the time when a marketing authorisation for an orphan medicinal product has been submitted in accordance with Article 5, the orphan designation shall remain valid until a decision is adopted by the Commission in accordance with Article 13(2).

Added4. An orphan designation ceases to be valid once an orphan medicine sponsor has obtained a marketing authorisation for the relevant medicinal product in accordance with Article 13(2).

Added5. At any time, an orphan designation may be withdrawn at the request of the orphan medicine sponsor. The orphan medicine sponsor may provide a reasoned justification for the withdrawal request, which shall be made publicly available. [Am. 192]

AddedArticle 67 Register of designated orphan medicinal products

Added1. The register of designated orphan medicinal products shall list all designated orphan medicinal products. It shall be set up and managed by the Agency and be publicly available.

Added2. Where an orphan designation ceases to be valid or is withdrawn pursuant to Article 66, the Agency shall make an entry in the register of designated orphan medicinal products.

Added3. The information on the designated orphan medicinal product entered in the register of designated orphan medicinal products shall include at least the following:

Added(a) the information on the active substance;

Added(b) the name and address of the orphan medicine sponsor;

Added(c) the condition for which it is intended or the proposed therapeutic indication;

Added(d) the designation date;

Added(e) the designation number;

Added(f) the decision on granting the orphan designation.

Added(fa) where applicable, any request made in accordance with Article 66(2) and any decisions taken in that respect. [Am. 193]

Added4. The Commission shall be empowered to adopt delegated acts in accordance with Article 175 in order to amend the information to be included in the register of designated orphan medicinal products referred to in paragraph 3 to ensure appropriate information of the users of that register.

AddedArticle 68 Protocol assistance and research support for orphan medicinal products

Added1. The orphan medicine sponsor mayshall, prior to the submission of an application for marketing authorisation, request advice from the Agency on the following: [Am. 194]

Added(a) the conduct of the various tests and trials necessary to demonstrate the quality, safety and, efficacy and environmental impact of the medicinal product, as referred to Article 138(1), second subparagraph, point (p); [Am. 195]

Added(b) the demonstration of significant benefit within the scope of the designated orphan indication;

Added(c) the demonstration of similarity to or clinical superiority over other medicinal products, which have market exclusivity for the same indication.

Added2. Medicinal products designated as orphan medicinal products under the provisions of this Regulation shall be eligible for incentives made available by the Union and by the Member States to support research into, and the development and availability of, orphan medicinal products and in particular aid for research for small- and medium-sized undertakings and entities not engaged in economic activity provided for in framework programmes for research and technological development. [Am. 196]

AddedArticle 69 Orphan marketing authorisation

Added1. Applications for an orphan marketing authorisation shall be submitted in accordance with Articles 5 and 6 and the related marketing authorisation shall be obtained in accordance with Articles 13(2).

Added2. In addition, the applicant shall demonstrate that the medicinal product has been granted an orphan designation and that the criteria set out in Article 63(1) or in the relevant delegated acts adopted in accordance with Article 63(2) are fulfilled for the therapeutic indication sought. [Am. 197]

AddedWhere appropriate, the applicant shall provide relevant evidence to demonstrate that the medicinal product addresses a high unmet medical need as specified in Article 70(1).

Added3. The Committee for Medicinal Products for Human Use shall assess whether the medicinal product fulfils the requirements set out in Article 63(1) or in the relevant delegated acts adopted in accordance with Article 63(2). In the situation referred in paragraph 2, subparagraph 2, that Committee shall also assess whether the medicinal product addresses a high unmet medical need as specified in Article 70(1). [Am. 198]

AddedSuch assessment shall be subject to the same timelines as the application for the marketing authorisation itself and detailed conclusions of such assessment shall be part of the scientific opinion of the Committee for Medicinal Products for Human Use in accordance with Article 12(1).

AddedThe assessment and its conclusions shall be part of the opinion referred to in Article 12(1) and, where relevant, the opinion referred to in Article 12(3).

Added4. The orphan marketing authorisation shall cover only those therapeutic indications, which fulfil the requirements set out in Article 63(1) or in the relevant delegated acts adopted in accordance with Article 63(2) at the time when the orphan marketing authorisation is granted. [Am. 199]

Added5. If after the submission of an application for the orphan marketing authorisation and prior to the opinion of the Committee for Medicinal Products for Human Use the orphan designation is withdrawn in accordance with Article 66(5), the application for the orphan marketing authorisation shall be treated as the application for a marketing authorisation in accordance with Article 6.

Added6. An applicant may submit an application for a separate marketing authorisation for other indications which do not fulfil the requirements set out in Article 63(1) or in the relevant delegated acts adopted in accordance with Article 63(2). [Am. 200]

AddedArticle 70 Orphan medicinal products addressing a high unmet medical need

Added1. An orphan medicinal product shall be considered as addressing a high unmet medical need where it fulfils the following requirements:

Added(a) there is no medicinal product authorised in the Union for such condition; or orwhere, despite medicinal products being authorised for such condition in the Union, the applicant demonstrates that the orphan medicinal product, in addition to having a significant benefit, will bring exceptional therapeutic advancement; [Am. 201]

Added(b) where a medicinal product is authorised for such condition, in addition to having a significant benefit, it will bring exceptional therapeutic advancement and the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or mortality for the relevant patient population. [Am. 202]

Added2. A medicinal product for which an application has been submitted in accordance with Article 13 of [revised Directive 2001/83/EC] shall not be considered as addressing a high unmet medical need.

Added3. Where the Agency adopts scientific guidelines for the application of this Article, it shall consult the Commission and, the authorities or bodies and other relevant stakeholders referred to in Article 162. [Am. 203]

AddedArticle 71 Market exclusivity