NorthWest Biotherapeutics Inc (NWBO)

[Lykiri]

So, did

1. NICE violate their own rules?

or

2. Did the UK already grant regulatory approval, and the price is known?

or

3. Other?


Flipper44,

IMO 3. Other!

If the timeline is still tracking there is a first Appraisal Committee meeting on 08 November 2018. IMO that is a hugely important date!

DCVax-L for treating newly diagnosed glioblastoma multiforme [ID836]
In development [GID-TA10143] Expected publication date: 01 May 2019

Provisional Schedule
Committee meeting: 1 08 November 2018

https://www.nice.org.uk/guidance/indevelopment/gid-ta10143
Guide to the processes of technology appraisal

2.5.24 An appraisal may begin before UK regulatory approval for the technology has been granted.

2.5.25 For an STA, NICE aims to hold the first Appraisal Committee meeting as soon as possible after the technology gains a positive opinion from the Committee for Medicinal Products for Human Use of the European Medicines Agency, or equivalent from the Medicines and Healthcare Products Regulatory Agency. It is therefore essential that the company informs NICE of all developments in the regulatory approval process. This ensures that NICE publishes guidance on the use of the new technology as soon as possible after receipt of the marketing authorisation and/or its introduction into the UK.

https://www.nice.org.uk/Media/Default/About/what-we-do/NICE-guidance/NICE-technology-appraisals/technology-appraisal-processes-guide-sept-2014.pdf

EMA
25 January 2018 EMA/630043/2008

Procedural advice on the evaluation of advanced therapy medicinal product in accordance with Article 8 of Regulation (EC) No 1394/2007

1. Introduction

As provided for in the ATMP Regulation (EC) No 1394/2007, the scientific evaluation of Marketing Authorisation Applications (MAAs) for ATMPs is primarily performed by the Committee for Advanced Therapies (CAT).[The CAT prepares a draft opinion on the quality, safety and efficacy of each ATMP subject to marketing authorisation application (MAA) which is sent for final approval to the Committee for Medicinal Products for Human Use (CHMP). The CHMP recommendation is then sent to the European Commission, which adopts a decision binding in all Member States./b

6. Timetable of the assessment:

Once the initial MAA is validated, the EMA starts the procedure at the monthly start date published on the EMA website. The submission deadlines and detailed procedural timetables are published on the EMA website (see, 'submission deadlines and full procedural timetables').

6.1 Standard timetable for the evaluation of an Advanced Therapy Medicinal Product (ATMP) for initial marketing authorisation application under the centralised application

6.2. Accelerated Assessment: The principles for the accelerated assessment procedure in accordance with Article 14(9) of Regulation (EC) 726/2004 also apply for ATMPs as per the “guideline on the Procedure for Accelerated Assessment Pursuant to Article 14 (9) of Regulation (EC) No 726/2004”.

In case of Advanced Therapy Medicinal Product (ATMP)s, the CAT decides on the accelerated assessment request. The timetable will be arranged to include the review by the Committee for Advanced Therapies.
The initial assessment phase will last 120 days similarly to the standard marketing authorisation procedure; the second phase of assessment will last 30 days - the timetable therefore is 120 + 30 days.
5.9 Role of the EMA The EMA ensures that the draft opinion of the CAT is given within 200 days (not including any clockstops for the applicant to provide answers to questions from the CAT and/or CHMP).
In case a medicinal product for human use contains or consists of GMO, the EMA manages the coordination of the consultation with the GMO competent authority during the assessment procedure.

In the case of advanced therapy medicinal products which incorporate medical devices or active implantable medical devices, (“combined ATMPs”), the EMA manages the coordination of the consultation with the Notified Body at the relevant time points of the procedure.
The EMA ensures that the opinion of the CHMP is given within 210 days (not including any clock-stops for the applicant to provide answers to questions from the CAT and/or CHMP).

The EMA Product Team prepares:
• The Committees assessment report on the basis of the CAT (Co-)Rapporteur(s)’ assessment reports ensuring scientific and regulatory consistency;
• The draft and final opinions for transmission by the CAT and final approval by the CHMP, respectively.
The EMA prepares and communicates with the CAT (Co-)Rapporteurs and CHMP coordinators any relevant public information related to the outcome of the assessment of ATMPs and the withdrawal of an application submitted to the EMA.
The EMA transmits the CHMP Opinion to the Commission.

ATMP Advanced therapy medicinal product
CAT Committee for Advanced Therapies
CHMP Committee for Medicinal Products for Human Use
CP Centralized procedure
EMA European Medicines Agency
EU European Union
GMO Genetically modified organisms
MAA Marketing authorization application
NICE National Institute for Health and Care Excellence
STA Single Technology Appraisal

http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline/2018/02/WC500242957.pdf

Handbook about Regulatory Guidelines and Procedures for the Preclinical and Clinical Stages of Advanced Therapy Medicinal Products (ATMPs)

Section IV. – Marketing Authorization.

As stipulated in the ATMP Regulation, marketing authorization for advanced therapies has to be applied for via the centralized procedure (CP) at the EMA. In general, the standard CP practices and requirements apply and that data as outlined in Annex I of Directive 2001/83/EC needs to be submitted in the marketing authorization application (MAA). However, again a few specific modifications/amendments had to be set down to account for the unique nature of ATMPs. For instance, it is not the Committee for Medicinal Products for Human Use (CHMP), but the Committee for Advanced Therapies (CAT) that is primarily responsible for the scientific evaluation of the application for an ATMP. Furthermore, some specific requirements exist for the summary of product characteristics (SmPC) and a modified assessment timetable applies.
It should be pointed out, however, that obtaining marketing authorization does not guarantee immediate access to the European markets. In most European countries, licensed medicinal products also need to undergo national pricing and reimbursement (P&R) procedures (the so-called “fourth hurdle” to market access). In contrast to the harmonized marketing authorization procedures, pricing and reimbursement systems in the EU are purely national responsibilities and requirements can be vastly different. While a detailed description of P&R systems in EU Member States goes beyond the scope of this handbook, it should be pointed out that the first centrally authorized ATMPs have experienced significant difficulties in obtaining reimbursement agreements in a broad range of countries. Further post-authorization clinical trials are often necessary, not only to fulfil obligations of regulatory approvals (e.g. as part of a conditional marketing authorization), but also to guarantee access to the targeted patients across the EU. Therefore, these efforts need to be considered in the investment needed to develop an ATMP.

1. MAA procedure for ATMPs (incl. assessment timetable).

The details of the MAA evaluation procedure specific to ATMPs, the roles of the various agency stakeholders as well as guidance to the applicant are outlined in a procedural guidance document (Procedural advice on the evaluation of ATMPs 2009).

In summary, the MAA of an ATMP is primarily evaluated by two groups – the CAT Rapporteur’s and the CAT Co-Rapporteur’s assessment teams. Each group includes matter experts for the various disciplines (e.g. quality, safety, efficacy, Pharmacovigilance etc.) as well as a CHMP coordinator. The teams are appointed based on the best available expertise for the ATMP under assessment. The CAT (Co-)Rapporteurs and CHMP coordinators jointly draft the assessment reports (AR) to be circulated to all committee members as well as the applicant.
The CAT is responsible for overall scientific assessment of the application, including the drafting of the milestone ARs (e.g. D120 List of Questions, D180 List of Outstanding Issues); however, critical aspects of the MAA are being discussed at the CHMP, and comments from CHMP as well as other involved working parties are being taken into consideration. The outcome of the CAT’s assessment is a draft opinion on the ATMP’s quality, safety and efficacy, which is forwarded to the CHMP. Based on the CAT opinion, the CHMP adopts a final scientific opinion on the granting, variation, suspension or revocation of a marketing authorization. This recommendation is then sent to the European Commission (EC) for a decision binding in all Member States. In case the CHMP disagrees with the CAT’s draft opinion, a clarification meeting will be organized by the EMA in order to resolve any divergences prior to the adoption of the final opinion by CHMP.
Importantly, the assessment of an ATMP marketing authorization application is being assessed according to a specific timetable. Adherence to these preset procedural timelines allows for the proper coordination of the multiple stakeholders and their tasks that are required during the MAA assessment. The timetables including the monthly deadlines for submission, start of procedure as well as other milestones of the CP are published on the EMA’s website under www.ema.europa.eu > Human regulatoy > Pre-authorisation > Submission dates > Timetables > Timetable: Advanced therapy medicinal products – Full application.

http://p-bio.org/wp-content/uploads/2016/06/ATMP-Handbook-Drug-Development-and-Regulation-Bioreg-Project.pdf