WL-Guidance document Relating to the fast-track authorisation procedure

Transcription

1 List of contents 1 Definitions, terms, abbreviations Abbrevations Introduction and Purpose Scope Legal basis Other applicable documents Part 1: Application for a fast-track authorisation procedure Criteria that must be fulfilled for an FTP application Formal aspects and the documentation to be submitted for the application for the fast-track authorisation procedure Processing the request for a fast-track authorisation procedure Combination of an FTP application with request for a procedure with prior notification (PPN) Application fee Planning the application submission after approval of the FTP application Part 2: Submission of the authorisation application using the fast-track authorisation procedure Formal aspects and the documentation to be submitted Evaluation phase Processing time limits Labelling phase between preliminary decision and official authorisation decision Sample testing Post-authorisation phase Application fee for the authorisation application Appendix Listing of Clinical Studies Graphical representation of the fast-track application procedure... 8 Change history Version Valid and binding as of: Modified without version change Description, comments (by author) Author s initials Chapter 6.1: Criteria specified in greater detail Chapter 6.2: Tabular overview of studies now required for the application Chapter 6.3: Time limit specified for the reply to the preliminary decision on the FTP application New chapter 6.4: Combination of FTP application with a request for a procedure with prior notification (PPN) Chapter 7.5: Sample testing simplified vof, rc, apk, lod QM-Ident: ZL104_00_001e_WL / V06 / vof, rc, apk, lod, cis / fua / / 8

2 In order to standardise document names, the published "Information sheet " (MB) ZL104_00_001e_MB Explanations relating to the fast-track authorisation procedure is being replaced by "Guidance documents" (WL). The function and effects of the document are not changed as a result of the name change. cis Section 6.5: Fee for Presubmission Meeting added. rc Appendix: Translation of some missing words sel / Section 5: links revised Modification of the description of the criteria in Section 6.1; Information regarding early communication of the submission date Modification of an inconsistency regarding time limits prior to submission; clarification of content. Conversion of the Guideline into an information sheet, clarifications to content without any substantive changes cis er, wag zeg, zro er, sh, apk 1 Definitions, terms, abbreviations 1.1 Abbrevations FTP HGebV TPA LoQ NAS VAM VAZV PPN Fast-track authorisation procedure Ordinance on Fees levied by the Swiss Agency for Therapeutic Products of 22 June 2006 (SR ) Federal Act of 15 December 2000 on Medicinal Products and Medical Devices (Therapeutic Products Act; SR ) List of Questions Medicinal product with new active pharmaceutical ingredient Ordinance of 17 October 2001 of the Swiss Agency for Therapeutic Products on Medicinal Products (SR ) Ordinance of 22 June 2006 of the Swiss Agency for Therapeutic Products on the simplified authorisation of medicinal products and the authorisation of medicinal products with notification procedure (SR ) Procedure with Prior Notification 2 Introduction and Purpose This guidance document specifies the requirements for an application and for an authorisation for a medicinal product in the fast-track procedure, and is intended primarily for administrative bodies. Swissmedic uses this guidance document first and foremost as a resource for applying the legal provisions in a uniform and equitable manner. For applicants, the document is intended to clarify the specific requirements that must be fulfilled so that corresponding authorisation applications can be processed as quickly and efficiently as possible. A fast-track procedure is possible for the authorisation of a medicinal product, provided that the criteria according to Art. 5 of the VAM are cumulatively fulfilled. This faster evaluation procedure is possible thanks to the targeted advance planning of resources. QM-Ident: ZL104_00_001e_WL / V06 / vof, rc, apk, lod, cis / fua / / 8

3 A fast-track authorisation procedure has the same documentation requirements and authorisation procedure as a normal procedure. In contrast with the normal procedure, an application for a fasttrack procedure (FTP) must be submitted to Swissmedic and approved beforehand. Part 1 of the information sheet describes the conditions and criteria that must be met for a positive outcome from the application for fast-track authorisation procedure, as well as the procedure for applying for a fast-track procedure. Formal and content requirements for an application for authorisation of a medicinal product through a fast-track authorisation procedure, as well as the evaluation procedure itself, are described in part 2. 3 Scope This guidance document applies to applications for authorisation of human medicinal products at Swissmedic 4 Legal basis Art. 5 of the VAM (SR ) Fast-track authorisation procedure 5 Other applicable documents Document identification ZL000_00_006e_WL Guidance document Time limits for authorisation applications ZL000_00_001e_WL Guidance document Formal requirements ZL000_00_002e_VZ Overview documents to submitted 6 Part 1: Application for a fast-track authorisation procedure 6.1 Criteria that must be fulfilled for an FTP application The following conditions must all be met in accordance with Art. 5 of the VAM in order for a human medicinal product, or any major variations thereto, to be evaluated by means of a fast-track authorisation procedure: a) It is a promising therapy for a severe, debilitating or life-threatening disease. Promising is considered to mean that it can be assumed that treatment of the disease in this way will reduce the risk of debilitation or loss of life. This must be demonstrated in clinical trials with one or more primary endpoints. All of the following criteria must be fulfilled in this respect: The trial end point(s) must be clinically relevant The occurrences attributed to the trial endpoints(s) must be sufficiently numerous in order to permit an assessment of the effect size. There must be a clear causal link between the treatment and the clinical effect. The target population on which the application is based must have been investigated in correspondingly designed clinical studies. The requested indication should be defined according to the implemented study and should not be framed in broader terms. Moreover, a fast-track procedure for a much more broadly framed indication should not be requested for a small subgroup of the investigated target population unless an extrapolation is scientifically justified. The assessment of the clinical relevance depends on the individual clinical presentation and the clinical and scientific practice related thereto. The target population according to the requested indication should include patients with the corresponding established and clearly defined disease QM-Ident: ZL104_00_001e_WL / V06 / vof, rc, apk, lod, cis / fua / / 8

4 entity. Only in cases where clinical hard endpoints such as overall survival cannot be investigated at reasonable expense, surrogate parameters that are clinically established, scientifically validated and recognised by international guidelines may be appropriate in order to demonstrate clinical relevance. In the relevant clinical context, such surrogate parameters could be functional capacity in daily life, or the progression of a disease. b) Treatment using currently approved medicinal products is either unavailable or unsatisfactory. Lack of treatment options: Applies only to diseases for which no treatment options with authorised medicinal products exist, or where non-medicinal treatments (such as surgery) are not curative. Unsatisfactory treatment options: The available treatment options with authorised medicinal products may be unsatisfactory in several ways, such as insufficient effect, unsatisfactory safety, or no established standard treatment with authorised medicinal products. The proposed new treatment should improve the treatment options based on new evidence. c) A high therapeutic benefit is expected from using this new medicinal product. High therapeutic benefit: The therapeutic benefits are clinically superior to those from treatments authorised to date or from standard treatments with medicinal products authorised to date. This must be demonstrated as probable on the basis of the clinical documentation submitted, even without an assessment of the detailed data. In cases where respect for planning is of particular importance, it is possible to request a Scientific Advice Meeting Formal aspects and the documentation to be submitted for the application for the fast-track authorisation procedure The FTP application must be submitted sufficiently in advance of the authorisation application to enable realistic planning of the application timeline. A request for the implementation of a PPN can be submitted at the same time as the FTP application (see chapter 6.4). The application must be made in writing by the authorisation holder or one of their legal representatives/companies to Swissmedic with the notice: Application for a fast-track procedure (FTP). The application should be scientifically justified and backed up by documentation. The following documents should be submitted: a) Covering letter describing the indication(s) scheduled for Switzerland as precisely as possible. The wording of the scheduled indication should be based on the investigated study population and substantiated by study results. If applicable, the covering letter must also refer to authorisation applications or the existence of questions or decisions from other authorities. b) Rationale stating the extent to which the medicinal product to be notified for authorisation satisfies all the criteria for an FTP. The rationale should be substantiated by existing data and by references (e.g. summary of the pivotal study) and should not usually exceed 15 pages. c) Relevant top-line 2 results of ongoing studies, if available, should also be submitted, although these will not be scrutinised in detail for the assessment of the FTP application but merely reviewed as supporting information. d) An overview of the data scheduled for the future authorisation application: tabular listing with a brief description of the pivotal studies, number of patients for efficacy and safety results, and stating whether interim or final study reports are involved. The table from CTD module 5.1 "Table 1 Further information on this subject ZL105_00_001e_WL Guidance document Meetings for applicants held with the Authorisation sector 2 as yet incomplete study or interim reports available according to ICH-E3 QM-Ident: ZL104_00_001e_WL / V06 / vof, rc, apk, lod, cis / fua / / 8

5 of All Clinical Studies" can be used as a template for this listing (e.g. see Appendix 8.1). In this case, the pivotal studies should be presented in the ICH E3 format when the authorisation application is submitted. e) Draft version of the Information for healthcare professionals or the Summary of Product Characteristics 6.3 Processing the request for a fast-track authorisation procedure The applicant will receive a confirmation of the receipt of the application and the documentation will be evaluated. Whether an FTP is approved will be decided on the basis of the selected indication, the scientific justification and the submitted documentation. Swissmedic will decide within 30 days whether the criteria for a fast-track authorisation procedure are met or not. The applicant will be informed of the decision in a preliminary decision or, in the case of an approval, directly with an official approval decision. The applicant has the option to take a stance regarding the preliminary decision within 30 days. A time limit of CD, depending on the respective review workload and the volume of submitted documentation, is usually required before the preliminary decision can be made by Swissmedic. The actual time limit can be viewed 10 days after receipt in the egov Portal (the internet-based egov service for electronic legal transactions and for information and data sharing). 6.4 Combination of an FTP application with request for a procedure with prior notification (PPN). It is now possible to submit a request for the implementation of a PPN (see Procedure with prior notification guidance document) at the same time as an FTP application. To this end, all the required information and documentation for the FTP application and the request for a PPN must be submitted concurrently. This means that, if the FTP application were to be rejected, the applicant does not need to wait for this rejection before submitting a request to implement a PPN, but can confirm the possible implementation of the desired PPN as a response to the preliminary decision. If the request to switch to the PPN is received together with the statement in response to the FTP application, this will be processed as a standalone new request for the implementation of a PPN with the corresponding time limits. 6.5 Application fee The application will be invoiced in accordance with the Ordinance on Fees Levied by the Swiss Agency for Therapeutic Products (HGebV; SR ). In the event of a rejection of the FTP application with simultaneous request for a PPN, the applicant will be charged for the costs of the FTP application. 6.6 Planning the application submission after approval of the FTP application If the application for a fast-track authorisation procedure is approved, the authorisation application can be submitted at the earliest two months and at the latest six months following the official decision to approve the application for the fast-track authorisation procedure. Applicants must inform Swissmedic in writing of the date on which they want to submit the authorisation application (exact date) as soon as possible but at least one month before submitting the authorisation application. The applicant may, if required, apply for a preliminary discussion of the dossier with Swissmedic at a pre-submission meeting. A pre-submission meeting is recommended in order to avoid a formal objection being raised to the documentation. This meeting must take place at least one month before the authorisation application is submitted, and the applicant will be charged a fee for the time involved at a rate of CHF 200 an hour (Art. 4, para. 1, HGebV). Formal questions relating to the authorisation application that will be submitted will be discussed at this meeting, especially relating to: Completed formal control check list Index of scientific and administrative documentation Manufacturers involved, if applicable QM-Ident: ZL104_00_001e_WL / V06 / vof, rc, apk, lod, cis / fua / / 8

6 etcd or paper submission Scope and expected submission date of any documentation to be submitted at a later date (dates given are binding) The aim of this discussion is to ensure that the authorisation application is complete upon submission and will not lead to any formal objections. 7 Part 2: Submission of the authorisation application using the fasttrack authorisation procedure 7.1 Formal aspects and the documentation to be submitted In the fast-track authorisation procedure, the applicant must submit the authorisation application together with all documentation required according to the application type to Swissmedic by the agreed date. If it is planned to make the submission in ectd format, it is recommended that applicants with limited or no experience with ectd submit a test sequence in good time (at least 3 weeks before submitting the application), in order to avoid exceeding the time limits as a result of technical problems. 7.2 Evaluation phase The application is reviewed according to the process for a first authorisation (ZL 101 First authorisation of NAS and miscellaneous, incl. major variations). 7.3 Processing time limits The time limits are those stipulated in the Guidance document Time limits for authorisation applications. 7.4 Labelling phase between preliminary decision and official authorisation decision The applicant accepts the texts in the product information and on the packaging elements in the state following revision and/or correction by Swissmedic. Discussions regarding the corrections to the medicinal product information texts made by Swissmedic can lead to a delay in the process. 7.5 Sample testing Experimental sample testing by Swissmedic does not usually take place during the authorisation process. In the preliminary decision, however, the applicant is informed whether sample testing takes place after authorisation in connection with market surveillance. 7.6 Post-authorisation phase The applicant shall ensure that the authorised medicinal product is placed on the market as soon as possible following the granting of the authorisation. The applicant must fulfil the conditions stated in the official decision within the time limit specified. 7.7 Application fee for the authorisation application The application will be invoiced in accordance with the Ordinance on Fees Levied by the Swiss Agency for Therapeutic Products (HGebV; SR ). QM-Ident: ZL104_00_001e_WL / V06 / vof, rc, apk, lod, cis / fua / / 8

7 8 Appendix 8.1 Listing of Clinical Studies Type of Identifier Location of Report Objective (s) of the Design and Type of Control Test Product(s ); Dosage Regimen; Route of Administr ation Number of Subjects Healthy Subjects or Diagnosis of Patients Duration of Treatmen t Status; Source: R1 Efficacy/M4E R1_.pdf see Table 5.1 Listing of Clinical Studies, p. 47 QM-Ident: ZL104_00_001e_WL / V06 / vof, rc, apk, lod, cis / fua / / 8

8 Product info/ Labelling Scheduling for the application maximum 6 months Answer to prelim. dec. Authorisation process Evaluation II Examination by Swissmedic Application for fast-track procedure Answer to LoQ Answer to prelim. dec. Evaluation I Swissmedic examination Improvemts. by firm FC WL-Guidance document 8.2 Graphical representation of the fast-track application procedure Fast-track autorisation procedure Months prior to submission After reception of application Swissmedic time after reception Scientific Advice Meeting, if requested 0 5 CD Application received 5 CD Formal control completed 30 CD max. 120 CD 5 CD Doc OK Preliminary decision: acceptance / rejection 65 CD 30 CD 70 CD List of Questions Answer to preliminary decision: if accepted, date for Presubmission meeting And for planned submission max. 90 CD Communication re. date of answers to LoQ (14 CD) 20 CD - 2 mths Official decision: acceptance / rejection: if accepted, if applicable, submission of a request for a Presubmission Meeting 50 CD Answers to LoQ received - 1 mth Communication of the calendar week of the submission of the authorisation application as early as possible Written communication of the date for the submission of the authorisation application and, if applicable, the Presubmission Meeting 120 CD max. 90 CD Preliminary decision Communication re. date of answers to preliminary decision (14 CD) Answers to Preliminary decision received 0 Submission of application 20 CD Legend: Milestone / actions by applicant 140 CD Official decision Texts published to platform / sample testing if necessary (60 CD) Milestone / actions by Swissmedic QM-Ident: ZL104_00_001e_WL / V06 / vof, rc, apk, lod, cis / fua / / 8