EUnetHTA Prioritisation List (EPL) Pharmaceuticals
EUnetHTA prioritises topics for Joint Assessments
EUnetHTA publishes the EUnetHTA Prioritisation List (EPL). The EPL expresses the identified significant interest of national HTA bodies in relation to individual compounds as presented in the list. By proactively selecting those topics in direct collaboration with national bodies, EUnetHTA strives to increase the implementation of Joint Assessments qualitatively and quantitatively at the national level. This is one of the key elements of Joint Action 3.
The Heads of HTA Agencies of the countries represented in the EUnetHTA Executive Board (ExB) have agreed to take a more proactive approach in performing Joint Assessments. There is a strong consensus within EUnetHTA that this approach will strengthen the methods and procedures of the pharmaceutical Joint Assessments to increase the benefits of EU cooperation on HTA and to shape the future model of collaboration. This facilitates national implementation and uptake of the Joint Assessments, and therefore the EUnetHTA ExB has agreed upon the creation of the EPL.
The EPL is based on the Innovation Observatory. The list was cross-checked with the open access Dutch Horizon Scan and the publicly available EMA list of medicines under evaluation to estimate regulatory timelines. In compiling the list, EUnetHTA partners were asked to indicate their interest, including using the assessment at the national level. The final prioritisation was based on this feedback and combined with the anticipated feasibility of the compatibility of timelines of the EUnetHTA assessment and estimated regulatory timelines. Therefore, prioritisation does not discriminate between pharmaceutical companies, compounds or indication. Of note, the EPL is an ad-hoc approach. As such, it will be evaluated and, in the future, may lead to a systematic approach.
The EPL stands complementary to the already well-established mechanism for voluntary submission of new compounds, which will continue in the future to run in parallel to the EPL.
EUnetHTA will interface with all companies on the EPL in order to establish further dialogue and increase cooperation on prioritised topics.
Drug | Company | Indication | Type of Submission | Expected Date of Positivel CHMP Opinion | Status as of May 2019 |
|---|---|---|---|---|---|
Brolucizumab | Novartis | Neovascular age-related macular degeneration (nAMD) | NCE | 2019 | Letter of Intent received |
Cefiderocol | Shionogi | Severe gram-negative infections | NCE | 2019 | Letter of Intent received |
Crizanlizumab | Novartis | Prevention of vaso-occlusive crises in sickle cell disease patients aged 16 years and over | NCE | 2020 | Company will submit Letter of Intent. Compound has replaced originally listed EPL compound.** |
Durvalumab | AstraZeneca | Advanced or metastatic non-small cell lung cancer (EGFR and ALK wild type) – first line | Type II variation | 2020 | In dialogue with company concerning participation |
ECCS-50 (Cytori Cell Therapy) | Cytori Therapeutics | Moderate to severe hand dysfunction due to scleroderma | NCE | 2019 | Establishing contact |
Enasidenib | Celgene | Relapsed or refractory acute myeloid leukaemia (AML) with an isocitrate dehydrogenase 2 (IDH2) mutation | NCE | 2019 | Assessment ongoing |
Lenti-D | BlueBird Bio | Cerebral adrenoleukodystrophy | NCE | 2020 | In dialogue with company concerning participation |
Rexmyelocel T | Rexgenero | Critical limb ischemia in patients with diabetes mellitus | NCE | 2020 | Company has expressed willingness to participate in this Joint Assessment at the time of EMA submission |
Satralizumab | Chugai Pharma | Neuromyelitis optica and neuromyelitis optica spectrum disorders | NCE | 2019 | In dialogue with company concerning participation |
Selonsertib | Gilead | Non-alcoholic steatohepatitis (NASH) | NCE | 2019 | In dialogue with company concerning participation |
Setmelanotide | Rhythm Pharmaceuticals | Pro-opiomelanocortin deficiency obesity | NCE | 2019 | Establishing contact |
Siponimod | Novartis | Secondary Progressive Multiple Sclerosis | NCE | 2019 | Letter of Intent received. Compound has replaced originally listed EPL compound.* |
Voxelotor | Global Blood Therapeutics | Sickle cell disease | NCE | 2020 | In dialogue with company concerning participation |
*Originally prioritised compound: ribociclib+fulvestrant for advanced HR-positive, HER2-negative breast cancer in postmenopausal women – first or second line.
**Originally prioritised compound: ribociclib+alpelisib+fulverstrant for HR-positive, HER2-negative postmenopausal breast cancer – second or third line.
