scholarly journals The Evolution of AIFA Registries to Support Managed Entry Agreements for Orphan Medicinal Products in Italy

2021 ◽  
Vol 12 ◽  
Author(s):  
Entela Xoxi ◽  
Karen M Facey ◽  
Americo Cicchetti
Keyword(s):  
Pharmaceutical Companies ◽  
Medicinal Products ◽  
Value For Money ◽  
Orphan Medicinal Product ◽  
Collection System ◽  
Web Based ◽  
Best Value ◽  
Orphan Medicinal Products ◽  
Managed Entry Agreements ◽  
Equal Split

Italy has a well-established prominent system of national registries to support managed entry agreements (MEAs), monitoring innovative medicinal products (MPs) with clinical as well as economic uncertainties to ensure appropriate use and best value for money. The technological architecture of the registries is funded by pharmaceutical companies, but fully governed by the national medicines agency (AIFA). A desktop analysis was undertaken of data over a 15-year timeframe of all AIFA indication-based registries and associated EMA information. The characteristics of registries were evaluated, comparing orphan MPs vs. all MPs exploring cancer and non-cancer indications. OMP (orphan medicinal product) registries’ type vs. AIFA innovation status and EMA approval was reviewed. Of the 283 registries, 182 are appropriateness registries (35.2% relate to OMPs, with an almost equal split of cancer vs. non-cancer for OMPs and MPs), 35 include financial-based agreements [20% OMPs (2 non-cancer, 5 cancer)], and 60 registries are payment by result agreements [23.3% OMPs (4 non-cancer, 10 cancer)]. Most OMPs (53/88) came through the normal regulatory route. With the strengthening of the system for evaluation of innovation, fewer outcomes-based registries have been instigated. AIFA has overcome many of the challenges experienced with MEA through developing an integrated national web-based data collection system: the challenge that remains for AIFA is to move from using the system for individual patient decisions about treatment to reviewing the wealth of data it now holds to optimize healthcare.

scholarly journals Orphan Dugs in EU / Lieky na zriedkavé choroby v EÚ

2013 ◽  
Vol 60 (Supplementum-VIII) ◽  
pp. 26-31 ◽  
Author(s):  
T. Foltánová ◽  
M. Mazág
Keyword(s):  
Medicinal Product ◽  
Small And Medium Enterprises ◽  
Orphan Drugs ◽  
Added Value ◽  
Medicinal Products ◽  
Orphan Medicinal Product ◽  
Whole Process ◽  
Orphan Medicinal Products ◽  
Commission Regulation ◽  
Medium Enterprises

Medical and scientific knowledge about rare diseases is minimal or lacking, thus making research difficulties for pharmaceutical industry. Orphan drugs in EU are under supervision of European Commission, European medical agency (EMA) and Committee for orphan medicinal products (COMP). The presentation provides a brief review of all supportive incentives in the field of orphan medicinal products as: the European orphan medicinal product (OMP) regulation, Guideline on Clinical Trials in Small Populations and Commission Regulation (EC) No 2049/2005 / support of small and medium enterprises (SMEs). It also introduces the concept of Clinical added value of orphan medicinal products, as one of the key instruments to increase the availability of orphan medicinal products in the member states. Separately it stresses the necessity of Health technology assessment implementation in whole process of orphan medicinal product development as well as the implementation of the Europlan indicators into the Slovak National plan

scholarly journals European and Italian regulation on orphan medicinal products

2013 ◽  
Vol 14 (2) ◽  
pp. 89-98
Author(s):  
Roberta Joppi
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Potential Benefit ◽  
Orphan Drugs ◽  
Pharmaceutical Companies ◽  
Drug Status ◽  
Medicinal Products ◽  
Eu Legislation ◽  
Orphan Medicinal Products ◽  
The Eu

The paper presents an overview of the European and Italian Regulation on Orphan Medicinal Products (OMPs), along with some data on the OMPs licensed in the EU from 2000 to 2012. The EU legislation encourages pharmaceutical companies to develop drugs for rare diseases, so-called “orphan drugs”. The European Medicine Agency recognizes orphan drug status mainly on the basis of the prevalence of the disease (≤ 5/10,000), and potential benefit. Orphan status implies incentives for pharmaceutical companies. From 2000 up to 2012 890 candidate orphan drug designations received a positive opinion and the marketing authorization was granted to 72 OMPs corresponding to 80 different indications. Currently, 59 OMPs are available to Italian patients either because licensed to the market by the AIFA or included in the list of the L. 648/96. Despite of an encouraging regulation nearly all the currently estimated rare diseases still await treatments.

scholarly journals What principles should govern the use of managed entry agreements?

2011 ◽  
Vol 27 (1) ◽  
pp. 77-83 ◽  
Author(s):  
Marianne Klemp ◽  
Katrine B. Frønsdal ◽  
Karen Facey
Keyword(s):  
Budget Impact ◽  
New Technologies ◽  
Patient Access ◽  
Value For Money ◽  
Health Technologies ◽  
Advantages And Disadvantages ◽  
Best Value ◽  
Managed Entry Agreements ◽  
Policy Forum ◽  
Management Of Uncertainty

Background: To ensure rapid access to new potentially beneficial health technologies, obtain best value for money, and ensure affordability, healthcare payers are adopting a range of innovative reimbursement approaches that may be called Managed Entry Agreements (MEAs).Methods: The Health Technology Assessment International (HTAi) Policy Forum sought to identify why MEAs might be used, issues associated with implementation and develop principles for their use. A 2-day deliberative workshop discussed key papers, members' experiences, and collectively addressed four policy questions that resulted in this study.Results: MEAs are used to give access to new technologies where traditional reimbursement is deemed inappropriate. Three different forms of MEAs have been identified: management of budget impact, management of uncertainty relating to clinical and/or cost-effectiveness, and management of utilization to optimize performance. The rationale for using these approaches and their advantages and disadvantages differ. However, all forms of MEA should take the form of a formal written agreement among stakeholders, clearly identifying the rationale for the agreement, aspects to be assessed, methods of data collection and review, and the criteria for ending the agreement.Conclusions: MEAs should only be used when HTA identifies issues or concerns about key outcomes and/or costs and/or organizational/budget impacts that are material to a reimbursement decision. They provide patient access and can be useful to manage technology diffusion and optimize use. However, they are administratively complex and may be difficult to negotiate and their effectiveness has yet to be evaluated.

scholarly journals Price and reimbursement of advanced therapeutic medicinal products in Europe: are assessment and appraisal diverging from expert recommendations?

2021 ◽  
Vol 14 (1) ◽  
Author(s):  
Virginia Ronco ◽  
Myriam Dilecce ◽  
Elena Lanati ◽  
Pier Luigi Canonico ◽  
Claudio Jommi
Keyword(s):  
Selection Process ◽  
Grey Literature ◽  
Primary Objective ◽  
Medicinal Products ◽  
Health Authorities ◽  
Advanced Therapy ◽  
Additional Funding ◽  
Managed Entry Agreements ◽  
R Process ◽  
Expert Recommendations

Abstract Background Advanced therapy medicinal products (ATMPs) represent an important cornerstone for innovation in healthcare. However, uncertainty on the value, the high average cost per patient and their one-shot nature has raised a debate on their assessment and appraisal process for pricing and reimbursement (P&R) purposes. This debate led experts providing for recommendations on this topic. Our primary objective is to investigate the ATMPs P&R process in the main five European countries and to understand if this process is consistent with published P&R expert recommendations. We also investigated the current ATMP pipelines to understand if future ATMPs will create challenges for their P&R process. Methods P&R framework for ATMPs in the European Major five (EU5) countries was investigated through a literature search on PubMed, institutional websites of National Health Authorities and grey literature. The ATMPs pipeline database was populated from a clinical trial database (clinicaltrials.gov), relying on inclusion and exclusion criteria retrieved from the literature. Results Reimbursement status of ATMPs is different across the EU5 countries, with the exception of CAR-Ts which are reimbursed in all countries. Standard P&R process in place for other medicinal products is extended to ATMPs, with the exception of some cases in Germany. List prices, where available, are high and, tend to be aligned across countries. Outcome-based Managed Entry Agreements (MEAs) have been extensively used for ATMPs. Extra-funds for hospitals managing ATMPs were provided only in Germany and, as additional fund per episode, in France. The accreditation process of hospitals for ATMPs management was in most countries managed by the national authorities. As far as ATMPs pipeline is concerned, ATMPs in development are mostly targeting non-rare diseases. Conclusions Expert recommendations for ATMPs P&R were partially applied: the role of outcome-based MEAs has increased and the selection process of the centres authorized to use these treatments has been enhanced; additional funding for ATMPs management to accredited centres has not been completely considered and annuity payment and broader perspective in cost considerations are far from being put in place. These recommendations should be considered for future P&R negotiations to pursue rational resource allocation and deal with budget constraints.

scholarly journals The latest orphan drug designations and the Commission Communication on Regulation (EC) 141/2000

2005 ◽  
Vol 11 (3) ◽  
Author(s):  
Rashmi R Shah
Keyword(s):  
European Union ◽  
Rare Diseases ◽  
European Medicines Agency ◽  
Biological Plausibility ◽  
The European Union ◽  
Medicinal Products ◽  
Market Exclusivity ◽  
Community Regulation ◽  
Orphan Medicinal Products ◽  
Patient Groups

The implementation of Community Regulation on orphan medicinal products in the European Union in April 2000 has resulted in a deluge of applications for designation of medicinal products as orphan for rare diseases. By April 2004, the Committee for Orphan Medicinal Products had already given positive opinion on 63 per cent of the 316 applications considered by them. A significant number of these positive designations have already matured into full marketing authorisations. Three major reasons – failure to meet prevalence or significant benefit criteria or provide evidence of biological plausibility – have equally contributed to either the negative opinion on or the applicants withdrawing the remaining applications. In July 2004, the European Commission issued a communication setting out its position on certain matters relating to the implementation of the designation and market exclusivity provisions. The Commission, the European Medicines Agency (EMEA) and the Committee for Orphan Medicinal Products (COMP) continue to be proactive and provide as much guidance and incentives as practical, engaging themselves with sponsors, patient groups and academia. As experience builds up and issues are clarified, there are expectations that the Community Regulation on orphan medicines will prove to be a spectacular success.

scholarly journals Discovering Key Topics From Short, Real-World Medical Inquiries via Natural Language Processing

2021 ◽  
Vol 3 ◽  
Author(s):  
A. Ziletti ◽  
C. Berns ◽  
O. Treichel ◽  
T. Weber ◽  
J. Liang ◽  
...  
Keyword(s):  
Language Processing ◽  
Real World ◽  
Medical Information ◽  
Pharmaceutical Companies ◽  
Medicinal Products ◽  
Medical Treatments ◽  
Key Topics ◽  
Linear Dimensionality Reduction ◽  
Improving Patient Care ◽  
Insight Into

Millions of unsolicited medical inquiries are received by pharmaceutical companies every year. It has been hypothesized that these inquiries represent a treasure trove of information, potentially giving insight into matters regarding medicinal products and the associated medical treatments. However, due to the large volume and specialized nature of the inquiries, it is difficult to perform timely, recurrent, and comprehensive analyses. Here, we combine biomedical word embeddings, non-linear dimensionality reduction, and hierarchical clustering to automatically discover key topics in real-world medical inquiries from customers. This approach does not require ontologies nor annotations. The discovered topics are meaningful and medically relevant, as judged by medical information specialists, thus demonstrating that unsolicited medical inquiries are a source of valuable customer insights. Our work paves the way for the machine-learning-driven analysis of medical inquiries in the pharmaceutical industry, which ultimately aims at improving patient care.

scholarly journals Implementasi Value For Money, Input Output Outcome dan Best Value Sebagai Alat Pengukuran Kinerja Sektor Publik

2018 ◽  
Vol 11 (1) ◽  
pp. 72-82
Author(s):  
Kariyoto Kariyoto
Keyword(s):  
Input Output ◽  
Value For Money ◽  
Best Value

Pengukuran organisasi sektor publik menjadi penting untuk mengetahui tingkat pencapain pelayanan kepada masyarakat. Kinerja organisasi sektor publik dapat diukur menggunakan alat dan indikator yang sesuai. Tujuan dari penelitian ini adalah untuk mengetahui konsep nilai waktu uang, masukan keluaran hasil, nilai terbaik sebagai indikator pengukuran kinerja sektor publik. Penelitian ini berlokasi di Perpustakaan Universitas Brawijaya Malang. Jenis penelitian melalui studi perpustakaan. Pengumpulan data dilakukan dengan observasi dan dokumentasi buku yang berhubungan dengan kinerja organisasi sektor publik. Analisis data digunakan reduksi data, penyajian data, dan penarikan kesimpulan atau verifikasi data. Hasil penelitian menunjukkan bahwa nilai waktu uang, masukan keluaran hasil dan nilai terbaik dapat dipakai sebagai alat pengukuran kinerja organisasi sektor publik.

scholarly journals Sistem Informasi Pendataan Siswa/Siswi di RA Sami’na Waathanaa Berbasis Web

KREA-TIF ◽  
2017 ◽  
Vol 5 (2) ◽  
pp. 58
Author(s):  
Dahlia Widhyaestoeti ◽  
Guntara Guntara
Keyword(s):  
Data Collection ◽  
Query Language ◽  
Problem Analysis ◽  
Collection System ◽  
Web Based ◽  
Data Collection System ◽  
Student Data ◽  
Structured Query Language ◽  
Student Data Collection ◽  
Microsoft Office

<h1 align="center">Abstrak</h1><p>Sistem informasi pendataan siswa Di RA Sami’na Waathanaa, proses pendataan siswa/siswi baik ketika siswa/siswi yang baru masuk, atau yang sudah lulus di data oleh operator sekolah tersebut masih menggunakan Microsoft Office. Hal tersebut menghambat pekerjaan operator serta data-data siswa mudah hilang atau rusak. Pengembangan sistem berupa sistem informasi pendataan siswa berbasis web, sehingga membantu kecepatan dan kualitas dalam penyampaian informasi. Tujuan dari penelitian ini adalah untuk menyediakan Sistem Pendataan Siswa terkomputerisasi dalam database. Metode pengembangan sistem informasi yang digunakan model waterfall, dari mulai analisis masalah, perancangan, hingga implementasi. Menu pada sistem pendataan siswa ini dapat diakses oleh user tertentu yaitu siswa, guru dan admin. Sistem informasi pendataan siswa ini berbasis web dengan pemanfaatan bahasa pemprogram Personal Hypertext Prepocessor (PHP) dan Structured Query Language (SQL). Pengunaan sistem informasi pendataan siswa ini dapat menghemat waktu dan menghasilkan informasi yang dibutuhkan.</p><h1 align="center"><em>Abstract</em></h1><p><em>Student data collection system At RA Sami’na Waathanaa, the student data collection process is good when students who have just entered, or who have graduated in data by the school operator are still using Microsoft Office. This hinders the work of operators and student data is easily lost or damaged. The development of the system in the form of a web-based student data collection system, so that it helps speed and quality in delivering information. The purpose of this study is to provide a computerized Student Data Collection System in a database. The information system development method used is the waterfall model, from problem analysis, design, to implementation. Menus in the student data collection system can be accessed by certain users, namely students, teachers and admins. This student data collection system is web based with the use of Personal Hypertext Prepocessor (PHP) and Structured Query Language (SQL) programming languages. Using this student data collection system can save time and produce the information needed.</em></p><p align="left"> </p>

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