Lambda Research Accelerated

EMA unveils the new features to enhance the PRIority MEdicines scheme

18 Apr 2023

To improve its PRIority MEdicines (PRIME) programme, which aims to hasten the development of medicines for unmet needs, the European Medicines Agency (EMA) has taken recommendations from a recent report. The modifications include making sure that drug sponsors and regulators agree on the process and deadlines for getting the medicine through review.
To give businesses creating medications for unmet medical needs more opportunity to interact with regulators, EMA introduced the pathway in 2016. The roadmap and tracker will take the role of the PRIME yearly update beginning in March 2023 as a pilot programme for any items that have not previously been covered in a Kick-off meeting.
The regulatory roadmap and development tracker, which contains details on anticipated regulatory filings and contacts with regulators, must be kept up to date and maintained by applicants as per EMA requirements
The revised guidance now permits submission preparedness meetings, which will be set up about a year before the applicant files their application for a marketing authorisation.
Potential applicants would also be required to provide developed strategies for the generation of post-marketing proof, where appropriate.
To arrange a submission readiness meeting with the PRIME Rapporteur and the assessment team, pertinent national experts, and the EMA product team, applicants are urged to get in touch with their PRIME scientific coordinator about 15 months prior to when they anticipate submitting their applications. The framework of the meeting will be the same as the kick-off meeting, but candidates will have an earlier opportunity to discuss what needs to be done before they submit their applications.
The guidance indicates that in this situation, applicants should consider that, to the extent feasible, the [submission] package should include all necessary data needed to support the intended [marketing application authorization (MAA)]. This should take into account the possibility that applications for conditional marketing authorizations or marketing authorizations under unusual circumstances may not require as much evidence. At this time, applicants would also be required to demonstrate developed strategies for the creation of post-marketing evidence, as appropriate.

Decision Tree :

PRIME: area of unmet medical need :

Oncology

Multiple myeloma
Diffuse large B-cell lymphoma
B-cell acute lymphoblastic leukaemia
Mantel cell lymphoma
Locally advanced or metastatic solid tumour
Primary mediastinal large B-cell lymphoma
Metastatic synovial sarcoma
Glioblastoma
Rituximab refractory post-transplant lymphoproliferative disorder
Hodgkin lymphoma
Myelodysplastic syndromes
Sezary syndrome
Urgent allogeneic haematopoietic stem cell transplantations

Endocrinology/Gynaecology / Fertility / Metabolism

Acute hepatic porphyria
Obesity and control hunger caused by genetics
Primary hyperoxaluria type 1
Mucopolysaccharidosis
Acid sphingomyelinase deficiency
X-linked myotubular myopathy
Thymidine kinase 2 deficiency
Type 1 diabetes
C3 glomerulopathy

Haematology / Hemostaseology

Transfusion-dependant B-thalassemia
Haemophilia B
Sickle cell disease
Epstein-Bar Virus-associated Post Transplant Lymphoproliferative Disorder
Haemophilia A
Paroxysmal nocturnal haemoglobinuria
Fanconi anaemia Type A
Myelofibrosis

Neurology

Spinal muscular atrophy
Early cerebral adrenoleukodystrophy
Huntington's disease
Variant late infantile neuronal ceroid lipofuscinosis 6
Friedreich's ataxia

Vaccines

Ebola
Chikungunya
Tuberculosis
Zika virus
Lower respiratory tract disease (LRTD) caused by RSV

Infectious diseases

Hepatitis D virus infection
Septic shock
Respiratory syncytial virus
BK virus, cytomegalovirus, human herpes virus-6, Epstein Barr virus, and/or adenovirus in allogeneic HSCT recipients

Immunology / Rheumatology / Transplantation

Prevention of graft rejection following solid organ transplantation
X-linked severe combined immunodeficiency
Dermatomyositis
Leukocyte Adhesion Deficiency-I

Ophthalmology

X-linked Retinitis Pigmentosa owing to defects in Retinitis Pigmentosa GTPase Regulator
Achromatopsia associated with defects in CNGB3
Leber's congenital amaurosis due to the p.Cys998X mutation in the CEP290 Gene

Gastroenterology / Hepatology

Progressive familial intrahepatic cholestasis
Non-alcoholic steatohepatitis
Primary biliary cholangitis

Cardiovascular Diseases

Reversal of antiplatelet effects of ticagrelor in patients with uncontrolled major or life-threatening bleeding or requiring urgent surgery or invasive procedure
Pulmonary arterial hypertension