The European Commission's Expert Group on Safe and Timely Access to Medicines for Patients (STAMP) has published a further series of discussion papers on European pharmaceutical regulation, as well as data related to the early performance of the PRIority MEdicines (PRIME) designation scheme of the European Medicines Agency (EMA), which is intended accelerate assessment procedures for early-stage innovative medicines.
Implications | The latest STAMP expert group meeting held on the 28 June presented discussion papers on the off-label use of medicinal products, and raised Dutch proposals to "strengthen the balance in the pharmaceutical system" across the European Union (EU) and among individual member states. Participants also provided an update on EMA activities linked to the PRIME accelerated assessment and adaptive pathways programmes. |
Outlook | The analysis conducted by the STAMP expert groups is aimed at stimulating high-level discussion among European and national-level pharmaceutical policymakers and regulators. The expert group is not responsible for formulating policy action, but provides a forum for exchanges of views that could potentially feature in future proposals for EU regulatory changes. |
The fifth meeting of the European Commission's Expert Group on Safe and Timely Access to Medicines for Patients (STAMP) expert group was held in Brussels (Belgium) on the 28 June, with minutes from the discussions being published today (19 July). The meeting published a high-level overview of the latest European Union (EU) and national-level initiatives in pharmaceutical regulation (available here). The expert group is responsible for providing analysis to the European Commission regarding the implementation of pharmaceutical regulation across the EU. The group also holds responsibility for examining future policy options to improve regulatory and support systems related to safe and timely access to innovative medicines for European patient populations.
The most significant of the documents to be published on this occasion provides an in-depth analysis of the PRIority MEdicines (PRIME) scheme for unmet medical needs (available to view here). A separate analysis that utilised some of the same material was previously published on the European Medicines Agency (EMA) website (available here) on 29 June. The STAMP report on the response of pharmaceutical companies to the PRIME designation relates only to early applications that were made during the one-month period from May to June 2016. In May, the EMA reported that it had received 18 applications for inclusion in a new early-stage innovative-medicine scheme (as of mid-April 2016; see Europe: 6 May 2016: EU priority-medicine initiative attracts 18 applications ahead of CHMP meeting in May). The latest figures show that the number of applications received fell to eight (as of mid-May) and nine (as of mid-June). There were also two applications (one each in May and June) that were received but not started, as these fell outside of PRIME's intended scope. Of the 37 applications that were received between mid-April and mid-June, approximately 20 were from small and medium-sized enterprises (SMEs), one was from an academic grouping and 16 were submitted by "others". No further details regarding this designation for "others" was provided by the STAMP committee, although it is likely to simply mean that these are companies that do not qualify or are not registered as SMEs. The small number of academic submissions will be a cause of concern, since the PRIME designation was intended to be primarily open to academia in addition to the SME sector.
PRIME is essentially a breakthrough status designation, and since its inception in March 2016 was intended to primarily focus on rare cancer medicines and neurodegenerative-disease areas, as well as therapeutic indications for Alzheimer's disease and antimicrobial resistance; the figures provided by the STAMP expert group on the therapeutic area of applications confirms that this is the case. Considering the 35 applications received from April to June (excluding two that were outside PRIME's scope), the STAMP group concluded that the top therapeutic areas were oncology (12), infectious diseases (five), pneumology-allergology (four), vaccines (three), haematology-haemostaseology (three), gastroenterology-hepatology (two), immunology-rheumatology-transplantation (two), neurology (two), cardiovascular diseases (one), and ophthalmology (one). Furthermore, the committee noted that 16 of the applications received were for orphan-designated products, compared with 19 non-orphan drugs.
A cumulative overview shows that six medicines have been recommended as being eligible to received a PRIME designation (as of May 2016). These include CCX-168, an orphan-designated drug under development by ChemoCentryx (US) for the treatment of patients with active ANCA-associated vasculitis. Additional information on companies and products that have successfully submitted applications include Kite Pharma's (US) CAR-T candidate KTE-C19 for the treatment diffuse large B-cell lymphoma (DLBCL) and patients with primary mediastinal B-cell lymphoma (PMBCL) or transformed follicular lymphoma (TFL). Furthermore, PRIME status has been granted to Novartis's (Switzerland) chimeric antigen receptor T-cell (CART) programme CTL019 for paediatric patients with relapsed or refractory B-cell acute lymphoblastic leukaemia. Another notable inclusions was Biogen's (US) Alzheimer candidate aducanumab, which is to undergo Phase III development.
Outlook and implications
The first group of medicines to receive the PRIME designation include – as expected – a high number of oncology drug candidates (two). Other therapeutic areas that have benefited (as of May 2016) are within the areas of vaccine development (one candidate), immunology-rheumatology-transplantation (one), neurology (one), and haematology-haemostaseology (one). The EMA has previously forecast that it expects to receive an average of 100 applications for PRIME status per annum. On a previous occasion, EMA representatives also predicted that eventually about one-third of successful applications could receive eventual market authorisation in the EU for the given indication. At present, the PRIME medicine scheme appears on course to achieve its target of 100 applications per year: the number of applications will probably average less than 10 per month going forward.
The performance of the PRIME medicine designation has been satisfactory to date, and has been well-received by the pharmaceutical industry in general; there has been a high level of interest in the scheme from industry (particularly the SME sector) according to STAMP, and this is likely to continue. Successful applicants benefit from early scientific advice on drug-development plans from the EMA, fee-waivers for SMEs and the academic sector for scientific advice requests, and shorter timeframes for scientific assessment reviews (see Europe: 28 October 2015: European Commission publishes draft proposals on safe and timely access to new medicines). The scheme will support drug development for early and mid-stage candidates, and is likely to accelerate the development of new therapies targeting unmet patient needs, based on preliminary clinical data.