Authorisation of ATMPs in EU: routes to facilitate prompt availability for patients.

Transcription

1 Authorisation of ATMPs in EU: routes to facilitate prompt availability for patients. This presentation only reflects the views of its author and does not necessarily reflect the opinion of the Commission

2 Content I. Standard procedure to obtain a MA in the EU. II. Possibilities to shorten deadlines: A. Reduction of timelines of decisionmaking procedure. B. Reduced data requirements: - MA under exceptional circumstances. - Conditional MA. - Application of risk-based approach for ATMPs. - Remarks. III. The future for ATMPs.

3 I. Standard procedure to obtain a MA 1. Submission of a valid MAA MAA is submitted to the EMA. Application must contain (among others): results of preclinical and clinical trials, environmental risk assessment, description of manufacturing process and control methods, manufacturing license, description of pharmacovigilance system and risk management plan, and compliance with paediatric requirements: waiver, deferral or results of studies in the agreed PIP.

4 I. Standard procedure to obtain a MA (cont.) 2. Scientific assessment Scientific assessment to be performed within 210 days (clock stops may apply). Main scientific assessment carried out by CAT and CHMP. However, a marketing authorisation procedure may involve up to five committees (PRAC, PDCO, COMP). 3. Granting of authorisation Marketing authorisation issued by COM after consulting a committee of Member States. Typically the adoption procedure takes 2 months.

5 I. Typical timeline to obtain a MA Product development, including clinical trials and preparation of dossier Submission of valid MAA Scientific assessment MA months * 8-15 weeks* *Range for authorised ATMPs.

6 II. Possibilities to shorten deadlines.

7 A. Reduction of timelines of decisionmaking procedure Scientific assessment Accelerated assessment procedure for medicines having major interest for public health: 150 days (clock stops may apply). Granting of authorisation No fast-track procedure specifically foreseen. COM may shorten deadline for consultation of MS committee (very exceptional).

8 B. Possibilities to reduce data requirements Paediatric requirements: deferrals/waivers can be granted unless a classwaiver applies, a request to this effect should be submitted to EMA. It's important to think to paediatric requirements well in advance the submission of the MAA. Results of preclinical and clinical trials: marketing authorisation under exceptional circumstances conditional MA risk-based approach for ATMPs

9 B. MA under exceptional circumstances MA can be granted in the absence of comprehensive data on efficacy and safety. Available if: product intended to treat rare disease, scientific knowledge does not permit having full data, or unethical to collect full data. MA is subject to obligations, e.g.: obligation to conduct post-authorisation studies, strengthened pharmacovigilance, MA is subject to annual reassessment.

10 B. Conditional Marketing Authorisation MA granted in the absence of full clinical data.* Available for limited category of products: product intended to treat a life-threatening disease or seriously debilitating condition, products that have obtained orphan designation, or products to be used in a situation of emergency, provided that unmet medical need, and benefit of immediate marketing outweighs risks inherent in having insufficient data. MA is subject to obligations, e.g.: obligation to conduct post-authorisation studies (always), strengthened pharmacovigilance (optional), MA is valid for one year it should be renewed yearly.

11 B. Risk-based approach for ATMPs COM Directive 2009/120 provides that the amount of data to apply for a marketing authorisation can be determined according to a risk-based approach. However, respondents to COM's public consultation claimed that no sufficient flexibility is applied. Requests for additional simplification were particularly strong in connection with autologous products.

12 B. Remarks Conditional MA can be perceived as more burdensome than MA under exceptional circumstances: Renewal application must be submitted six months before the yearly expiry date. However, MA under exceptional circumstances remains an "exceptional" route to market medicines in EU only possible if it is expected that there is not possibility to collect missing data.

13 B. Remarks (cont.) Application of "risk based approach" could be the most advantageous route for ATMPs (if exploited to full potential): Not subject to annual reassessment/renewal. Post-marketing obligations can be costly. Post-marketing studies to be submitted as Type II variation.

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15 III. The future for ATMPs

16 III. ATMPs in EU: current situation Four marketing authorisations have been granted. Much larger number of products are being used under the hospital exemption (or other derogatory provisions of Directive 2001/83). Research in ATMPs is significant and the majority is carried out by SMEs, hospitals or academia. 250 clinical trials on ATMPs (EudraCT data ). 87 requests for classification (November 2007-June 2013). 93 requests for scientific advice relating to 65 products (November 2007-June 2013).

17 III. The future for ATMPs The regulation of ATMPs is important to protect patients from unsound therapies. ATMP Regulation has been a positive step to protect patients. Main principles of the Regulation remain valid. The marketing authorisation process ensures a robust assessment of quality, efficacy and safety of ATMPs that are marketed in the EU.

18 III. The future for ATMPs (cont.) The regulatory framework must facilitate that research translates into medicines available for patients. The main obstacle to (early) access to patients to ATMPs is not the length of the decision-making process but the (high) requirements to submit a valid MAA and HTAs. However, there is margin to streamline current marketing authorisation procedure for ATMPs.

19 III. The future for ATMPs(cont.) Requirements for MAA must be proportionate and sufficiently adapted to the specific characteristics of this type of products, particularly for autologous products. Risk-based approach could be further exploited. However, under current legal framework, the risk-based approach does not apply to certain quality requirements (e.g. manufacturing license per site, batch release requirements). Additional incentives could be considered to facilitate translation of research from hospitals/academia/smes into medicines.

20 Thank you for your attention.