Prioritisation List (EPL) – Pharmaceuticals

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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.

For further information please contact:

wp4_pharmaceuticals@zinl.nl

International non-proprietary name (INN)Product  name (if available)MAH/ pMAHAnticipated indication, including age and sex if applicable Application type EMA Estimated submission date to EMA/JA startStatus and prioritisation source
ApabetaloneResverlogixReducing adverse cardiac events in people with cardiovascular disease, type 2 diabetes and low HDLIMAUnknownEPL 2.0 (July 2019)
Arimoclomol citratOrphazymeNiemann-Pick disease, type C (NPC)ODQ2 2020EPL 2.0 (July 2019)
AvacopanVifor Pharma UK or ChemoCentryxTreatment of anti-neutrophil cytoplasmic antibody-associated vasculitis
IMAQ2 2020EPL 2.0 (July 2019)
Autologous CD34+ haematopoietic stem cells (HSCs) transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNALenti-DBlueBird BioCerebral adrenoleukodystrophyIMAEPL 1.0 (November 2018). In dialogue with company concerning participation
BrolucizumabNovartisNeovascular age-related macular degenerationIMA2019EPL 1.0 (November 2018). Letter of Intent received
CefiderocolShionogiSevere gram-negative infectionsIMA2019EPL 1.0 (November 2018). In dialogue with company concerning participation
CenicrivirocAllerganLiver Fibrosis in Adults with Non-Alcoholic Steatohepatitis (NASH).IMAQ4 2019EPL 2.0 (July 2019)
CrizanlizumabNovartisPrevention of vaso-occlusive crises in sickle cell disease patients aged 16 years and overIMA2019EPL 1.0 (November 2018). Company will submit Letter of Intent. Compound has replaced originally listed EPL compound.**
Cysteamine bitartrateLynovexNovaBiotics LtdTreating exacerbations of cystic fibrosisUnknownUnknownEPL 2.0 (July 2019)
DarolutamideBayerNon-metastatic, castration resistant prostate cancerIMAEPL 1.0 (November 2018). Company refused to participate
DurvalumabImfinziAstraZenecaAdvanced or metastatic non-small cell lung cancer (EGFR and ALK wild type) – first lineEoIEPL 1.0 (November 2018). In dialogue with company concerning participation
ECCS-50 (Cytori stem cell therapy)Cytori TherapeuticsModerate to severe hand dysfunction due to sclerodermaIMAEPL 1.0 (November 2018). Establishing contact
EnasidenibCelgeneRelapsed or refractory acute myeloid leukaemia (AML) with an isocitrate dehydrogenase 2 (IDH2) mutationIMA2019EPL 1.0 (November 2018). Assessment ongoing
EsketamineSpravatoJanssenMajor Depressive Disorder with imminent risk of suicideEoIQ4 2019EPL 2.0 (July 2019)
FedratinibInrebicCelgeneMyelofibrosisIMAQ4 2019EPL 2.0 (July 2019)
GivosiranAlnylamPrevention of acute attacks of hepatic porphyriaPrime, ODUnknownEPL 2.0 (July 2019)
IbrutinibImbruvicaJanssen1L Chronic Lymphatic Leukemia young and fit patientsEoIQ3 2019EPL 2.0 (July 2019)
Follicular lymphoma (FL), relapsed / refractory.EoIQ3 2019
Mantle cell lymphoma (MCL), 1L.EoIQ3 2020
Marginal zone lymphoma (MZL), relapsed / refractory.EoIQ3 2021
Non-germinal center B-cell-like (non-GCB) diffuse large B-cell lymphoma (DLBCL), 1L.EoIUnknown
InclisiranThe Medicines CompanyHypercholesterolaemia, elevated LDL-cholesterol (LDL-C) despite maximum tolerated doses of LDL-C lowering therapies.IMAUnknownEPL 2.0 (July 2019)
Insuline for inhalationAfrezzaMannKindType 1 and 2 diabetes mellitusIMAUnknownEPL 2.0 (July 2019)
LifileucelContegoIovanceMaliganant melanoma, advanced melanomaUnknownQ1 2020EPL 2.0 (July 2019)
LurbinectedinZepsyrePharma MarSmall cell lung cancer (SCLC)UnknownQ2 2020EPL 2.0 (July 2019)
NaxitamabY-mAbs TherapeuticsHigh risk neuroblastomaODUnknownEPL 2.0 (July 2019)
NolasibanObsEvaImproving pregnancy rate following in vitro fertilization/intracytoplasmic sperm injection (IVF/ICSI) procedures.IMAQ1 2020EPL 2.0 (July 2019)
OlaparibLynparzaAstraZeneca and MSDOlaparib 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
EoIQ1 2020EPL 2.0 (July 2019)
PembrolizumabKeytrudaMSDBreast cancer, metastatic, triple negative, 1L.EoIQ1 2020EPL 2.0 (July 2019)
2/3L Triple negative metastatic breast cancerEoIQ1 2020
Triple Negative Breast Cancer - NeoadjuvantEoIQ1 2020
1L advanced gastric cancerEoIQ1 2020
1L Niercelcarcinoom stadium IV (in combinatie met axitinib)EoIQ1 2020
Unresectable or metastatic solid tumours with a microsatellite instability high (MSI-H) or mismatch repair deficient (dMMR) biomarker.EoIQ1 2020
2L advanced esophageal cancer, squamousEoIQ1 2020
Remestemcel-LProchymalMesoblastCrohn's diseaseEoIQ2 2020EPL 2.0 (July 2019)
Acute steroid-refractory graft versus host disease (GvHD) in adults and children - second-line following allogeneic bone marrow transplant (BMT)IMAQ2 2020
Rexmyelocel TRexgeneroCritical limb ischemia in patients with diabetes mellitusIMAEPL 1.0 (November 2018). Company has expressed willingness to participate in this Joint Assessment at the time of EMA submission
RivaroxabanXareltoBayerIn 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.EoIUnknownEPL 2.0 (July 2019)
Rocapuldencel-TArgosMetastatic renal cell cancerIMAUnknownEPL 2.0 (July 2019)
Rovalpituzumab tesirineAbbvieSmall-cell lung cancer (SCLC), 1L.IMAUnknownEPL 2.0 (July 2019)
RoxadustatAstellasTreatment of anemia due to chronic kidney disease (CKD).IMAQ4 2019EPL 2.0 (July 2019)
SapacitabineCyclacelAcute myeloid leukemiaIMAQ4 2019EPL 2.0 (July 2019)
SatralizumabRocheNeuromyelitis optica and neuromyelitis optica spectrum disordersIMAEPL 1.0 (November 2018). In dialogue with company concerning participation
SelonsertibGileadNon-alcoholic steatohepatitis (NASH)IMAEPL 1.0 (November 2018). In dialogue with company concerning participation
SelinexorKaryopharm Therapeuticstreatment of patients with relapsed refractory multiple myeloma (RRMM)IMA, OD, ACUnknownEPL 2.0 (July 2019)
SelumetinibAstraZenecaPaediatric patients with inoperable NF1 plexiform neurofibromas type-1 (NF1)IMAUnknownEPL 2.0 (July 2019)
SetmelanotideRhythm PharmaceuticalsPro-opiomelanocortin deficiency obesityIMA2019EPL 1.0 (November 2018). Establishing contact
SiponimodNovartisSecondary Progressive Multiple SclerosisIMA2019EPL 1.0 (November 2018). Letter of Intent received. Prioritization of different compound compared to orginal EPL listing.*
Valoctocogene roxaparvovec (BMN 270)BiomarinTreatment of haemophilia APRIME, ATMP, AC, ODUnknownEPL 2.0 (July 2019)
Veliparib with carboplatin and paclitaxelAbbVie Untreated epithelial ovarian, fallopian tube or primary peritoneal cancerOD, IMAUnknownEPL 2.0 (July 2019)
Venetoclax and Venetoclax with bortezomib and dexamethasoneVenclyxtoAbbVieIn combination for untreated acute myeloid leukaemia unsuitable for intensive chemotherapyEoIUnknownEPL 2.0 (July 2019)
Treatment relapsed or refractory multiple myelomaEoIUnknown
VoxelotorGlobal Blood TherapeuticsSickle cell diseaseIMA2019EPL 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.

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