Prioritisation List (EPL) – Pharmaceuticals
EUnetHTA prioritises topics for Joint Assessments
EUnetHTA has published the second iteration of 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 at the national level. This is one of the key elements of Joint Action 3.
The EPL is published as part of the overarching Topic Identification, Selection and Prioritisation (TISP) task group. The topics identified and prioritised in the first EPL remain relevant and valid.
Topics have been identified through horizon scanning activities and via feedback from EMA and EUnetHTA partners. Selection and prioritisation are based on four criteria:
- Whether the topic is of national/sub/national interest;
- Uptake;
- Whether the partners are interested in being part of the authoring team for a specific compound;
- Feasibility in relation to timelines.
Attaining the EPL listing demonstrates a significant degree of interest on the part of HTA bodies in receiving a Joint Relative Efficacy Assessment (REA) on the specific topic. EUnetHTA will, therefore, engage with all companies on the EPL in order to establish further dialogue to increase the opportunities for collaboration on prioritised topics.
* EUnetHTA JA3 will not be accepting any further Letters of Intent with regard to new pharma Joint Assessments through the end of Joint Action 3 on 31st May, 2021.
For further information please contact:
International non-proprietary name (INN) | Product name (if available) | MAH/ pMAH | Anticipated indication, including age and sex if applicable | Application type EMA | Estimated submission date to EMA/JA start | Status and prioritisation source |
---|---|---|---|---|---|---|
Follicular lymphoma (FL), relapsed / refractory. | EoI | Q3 2019 | ||||
Mantle cell lymphoma (MCL), 1L. | EoI | Q3 2020 | ||||
Marginal zone lymphoma (MZL), relapsed / refractory. | EoI | Q3 2021 | ||||
Non-germinal center B-cell-like (non-GCB) diffuse large B-cell lymphoma (DLBCL), 1L. | EoI | Unknown | ||||
2/3L Triple negative metastatic breast cancer | EoI | Q1 2020 | ||||
Triple Negative Breast Cancer – Neoadjuvant | EoI | Q1 2020 | ||||
1L advanced gastric cancer | EoI | Q1 2020 | ||||
1L Niercelcarcinoom stadium IV (in combinatie met axitinib) | EoI | Q1 2020 | ||||
Unresectable or metastatic solid tumours with a microsatellite instability high (MSI-H) or mismatch repair deficient (dMMR) biomarker. | EoI | Q1 2020 | ||||
2L advanced esophageal cancer, squamous | EoI | Q1 2020 | ||||
Acute steroid-refractory graft versus host disease (GvHD) in adults and children – second-line following allogeneic bone marrow transplant (BMT) | IMA | Q2 2020 | ||||
Treatment relapsed or refractory multiple myeloma | EoI | Unknown | ||||
Apabetalone | Resverlogix | Reducing adverse cardiac events in people with cardiovascular disease, type 2 diabetes and low HDL | IMA | Unknown | EPL 2.0 (July 2019) | |
Arimoclomol citrat | Orphazyme | Niemann-Pick disease, type C (NPC) | OD | Q2 2020 | EPL 2.0 (July 2019) | |
Autologous CD34+ haematopoietic stem cells (HSCs) transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA | Lenti-D | BlueBird Bio | Cerebral adrenoleukodystrophy | IMA | EPL 1.0 (November 2018). In dialogue with company concerning participation | |
Avacopan | Vifor Pharma UK or ChemoCentryx | Treatment of anti-neutrophil cytoplasmic antibody-associated vasculitis | IMA | Q2 2020 | EPL 2.0 (July 2019) | |
Brolucizumab | Novartis | Neovascular age-related macular degeneration | IMA | 2019 | EPL 1.0 (November 2018). Letter of Intent received | |
Cefiderocol | Shionogi | Severe gram-negative infections | IMA | 2019 | EPL 1.0 (November 2018). Letter of Intent received | |
Cenicriviroc | Allergan | Liver Fibrosis in Adults with Non-Alcoholic Steatohepatitis (NASH). | IMA | Q4 2019 | EPL 2.0 (July 2019) | |
Crizanlizumab | Novartis | Prevention of vaso-occlusive crises in sickle cell disease patients aged 16 years and over | IMA | 2019 | EPL 1.0 (November 2018). Company will submit Letter of Intent. Compound has replaced originally listed EPL compound.** | |
Cysteamine bitartrate | Lynovex | NovaBiotics Ltd | Treating exacerbations of cystic fibrosis | Unknown | Unknown | EPL 2.0 (July 2019) |
Darolutamide | Bayer | Non-metastatic, castration resistant prostate cancer | IMA | EPL 1.0 (November 2018). Company refused to participate | ||
Durvalumab | Imfinzi | AstraZeneca | Advanced or metastatic non-small cell lung cancer (EGFR and ALK wild type) – first line | EoI | EPL 1.0 (November 2018). In dialogue with company concerning participation | |
ECCS-50 (Cytori stem cell therapy) | Cytori Therapeutics | Moderate to severe hand dysfunction due to scleroderma | IMA | EPL 1.0 (November 2018). Establishing contact | ||
Enasidenib | Celgene | Relapsed or refractory acute myeloid leukaemia (AML) with an isocitrate dehydrogenase 2 (IDH2) mutation | IMA | 2019 | EPL 1.0 (November 2018). Assessment ongoing | |
Esketamine | Spravato | Janssen | Major Depressive Disorder with imminent risk of suicide | EoI | Q4 2019 | EPL 2.0 (July 2019) |
Fedratinib | Inrebic | Celgene | Myelofibrosis | IMA | Q4 2019 | EPL 2.0 (July 2019) |
Givosiran | Alnylam | Prevention of acute attacks of hepatic porphyria | Prime, OD | Unknown | EPL 2.0 (July 2019) | |
Ibrutinib | Imbruvica | Janssen | 1L Chronic Lymphatic Leukemia young and fit patients | EoI | Q3 2019 | EPL 2.0 (July 2019) |
Inclisiran | The Medicines Company | Hypercholesterolaemia, elevated LDL-cholesterol (LDL-C) despite maximum tolerated doses of LDL-C lowering therapies. | IMA | Unknown | EPL 2.0 (July 2019) | |
Insuline for inhalation | Afrezza | MannKind | Type 1 and 2 diabetes mellitus | IMA | Unknown | EPL 2.0 (July 2019) |
Lifileucel | Contego | Iovance | Maliganant melanoma, advanced melanoma | Unknown | Q1 2020 | EPL 2.0 (July 2019) |
Lurbinectedin | Zepsyre | Pharma Mar | Small cell lung cancer (SCLC) | Unknown | Q2 2020 | EPL 2.0 (July 2019) |
Naxitamab | Y-mAbs Therapeutics | High risk neuroblastoma | OD | Unknown | EPL 2.0 (July 2019) | |
Nolasiban | ObsEva | Improving pregnancy rate following in vitro fertilization/intracytoplasmic sperm injection (IVF/ICSI) procedures. | IMA | Q1 2020 | EPL 2.0 (July 2019) | |
Olaparib | Lynparza | AstraZeneca and MSD | Olaparib is indicated as monotherapy maintenance treatment for gBRCAm metastatic pancreatic cancer patients whose disease has not progressed on first line platinum based chemotherapy Adult population | EoI | Q1 2020 | EPL 2.0 (July 2019) |
Pembrolizumab | Keytruda | MSD | Breast cancer, metastatic, triple negative, 1L. | EoI | Q1 2020 | EPL 2.0 (July 2019) |
Remestemcel-L | Prochymal | Mesoblast | Crohn’s disease | EoI | Q2 2020 | EPL 2.0 (July 2019) |
Rexmyelocel T | Rexgenero | Critical limb ischemia in patients with diabetes mellitus | IMA | EPL 1.0 (November 2018). Company has expressed willingness to participate in this Joint Assessment at the time of EMA submission | ||
Rivaroxaban | Xarelto | Bayer | In combination with acetylsalicylic acid, for the prevention of atherothrombotic complications in adult patients with stable coronary arterial disease (CAV) or symptomatic peripheral arterial disease (PAV) with an increased risk of ischemic events. | EoI | Unknown | EPL 2.0 (July 2019) |
Rocapuldencel-T | Argos | Metastatic renal cell cancer | IMA | Unknown | EPL 2.0 (July 2019) | |
Rovalpituzumab tesirine | Abbvie | Small-cell lung cancer (SCLC), 1L. | IMA | Unknown | EPL 2.0 (July 2019) | |
Roxadustat | Astellas | Treatment of anemia due to chronic kidney disease (CKD). | IMA | Q4 2019 | EPL 2.0 (July 2019) | |
Sapacitabine | Cyclacel | Acute myeloid leukemia | IMA | Q4 2019 | EPL 2.0 (July 2019) | |
Satralizumab | Roche | Neuromyelitis optica and neuromyelitis optica spectrum disorders | IMA | EPL 1.0 (November 2018). In dialogue with company concerning participation | ||
Selinexor | Karyopharm Therapeutics | Treatment of patients with relapsed refractory multiple myeloma (RRMM) | IMA, OD, AC | Unknown | EPL 2.0 (July 2019) | |
Selonsertib | Gilead | Non-alcoholic steatohepatitis (NASH) | IMA | EPL 1.0 (November 2018). In dialogue with company concerning participation | ||
Selumetinib | AstraZeneca | Paediatric patients with inoperable NF1 plexiform neurofibromas type-1 (NF1) | IMA | Unknown | EPL 2.0 (July 2019) | |
Setmelanotide | Rhythm Pharmaceuticals | Pro-opiomelanocortin deficiency obesity | IMA | 2019 | EPL 1.0 (November 2018). Establishing contact | |
Siponimod | Novartis | Secondary Progressive Multiple Sclerosis | IMA | 2019 | EPL 1.0 (November 2018). Letter of Intent received. Prioritization of different compound compared to orginal EPL listing.* | |
Valoctocogene roxaparvovec (BMN 270) | Biomarin | Treatment of haemophilia A | PRIME, ATMP, AC, OD | Unknown | EPL 2.0 (July 2019) | |
Veliparib with carboplatin and paclitaxel | AbbVie | Untreated epithelial ovarian, fallopian tube or primary peritoneal cancer | OD, IMA | Unknown | EPL 2.0 (July 2019) | |
Venetoclax in combination with a hypomethylating agent or low-dose cytarabine | Venclyxto | AbbVie | Adults with newly-diagnosed acute myeloid leukaemia (AML) who are ineligible for intensive chemotherapy | EoI | Unknown | EPL 2.0 (July 2019) |
Voxelotor | Global Blood Therapeutics | Sickle cell disease | IMA | 2019 | EPL 1.0 (November 2018). Establishing contact |
Acronym List for EPL
IMA – Initial Market Application
EoI – Extension of Indication
AC – Accelerated Access
OD – Orphan Designation
PRIME – Priority Medicine
ATMP – Advanced Therapy Medicinal Product
*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.