Medicine characteristics affecting the time to guidance publication by National Institute for Health and Care Excellence in the single technology appraisal process

Objective In England, the time gap between marketing authorization (MA) and guidance publication by National Institute for Health and Care Excellence (NICE) can limit patients’ access to new medicines. In this study, our aim was to identify medicine characteristics associated with the long time gap between MA and guidance publication and explore the influencing factors. Methods We identified 116 single technology appraisals from 2016 to 2020 using publicly available data, and extracted information on the year of appraisal completion, application type, experiences of similar appraisals, orphan medicinal products (OMPs), cancer medicines, and accelerated assessment. Multiple regression analyses were performed to analyze the associations between the medicine characteristics and key time periods related to health technology assessment and MA processes. Results OMPs were associated with a long period between MA and guidance publication. Specifically, OMPs and cancer medicines were associated with slow guidance publication after the final scope (FS) development. However, there was no association between OMPs and the period between validation of MA application and FS development. Non-double-blinded randomized clinical trials and the use of comparators not specified in the FS were associated with slow guidance publication after the FS development. Conclusions Our results demonstrate that OMPs are associated with a longer period between MA and guidance publication by the NICE than non-OMPs; this may be attributed to the slow guidance publication after the FS development. These findings indicate the necessity to shorten the appraisal process for OMPs.

PP106 Twenty Years Of Orphan Medicines Regulation: Have Treatments Reached Patients In Need Across Europe And Canada?

IntroductionThe European Union regulation for orphan medicinal products (OMPs) was introduced to improve the quality of treatments for patients with rare conditions. To mark 20 years of European Union OMP regulation, this study compared access to OMPs and the length of their reimbursement process in a set of European countries and Canadian provinces. Access refers to their full or partial reimbursement by the public health service.MethodsData were collated on European Medicines Agency orphan designation and marketing authorizations, health technology assessment (HTA) decisions and reimbursement decisions, and the respective dates of these events for all the OMPs centrally authorized in 14 European countries (Belgium, England, France, Germany, Hungary, Italy, the Netherlands, Norway, Poland, Scotland, Slovakia, Spain, Sweden, and Switzerland) and four Canadian provinces (Alberta, British Columbia, Ontario, and Quebec).ResultsSince the implementation of the OMPs Regulation in 2000, 215 OMPs obtained marketing authorization. We found that Germany had the highest level of coverage, with 91 percent of OMPs being reimbursed. The three countries with the lowest reimbursement rates were Poland, Hungary, and Norway (below 30%). We observed that Germany had the quickest time to reimbursement following marketing authorization, followed by Switzerland and Scotland. We observed that Poland, Hungary, and Slovakia consistently had the longest time to reimbursement.ConclusionsWe observed substantial variation in the levels and speed of national reimbursement of OMPs, particularly when comparing countries in Eastern and Western Europe, which suggests that an equity gap between the regions may be present. The data also indicated a trend toward faster times to reimbursement over the past 10 years.

Health Technology Assessment Challenges for Orphan Medicinal Products in Low- and Middle-Income Countries in Europe

The purpose of this research is to provide a short overview of the differences in access to and health technology assessment of OMPs in selected low- and middle-income countries in Europe and discuss these in terms of some challenges in the face of HTA comparing with high-income countries. This is a retrospective, desktop study of already published official inquiries of national or international institutions, scientific publications, and reports from national governing bodies who regulate and monitor HTA in low- and middleincome economies in Europe, with further regards to HTA of orphan medicinal products. Among all selected low- and middle-income countries in Europe - Bulgaria, Serbia, North Macedonia, Moldova, Ukraine, and Georgia, have available and up-to-date information regarding their health care legislation. During 2000-2019 EMA approves 164 orphan medicines and out of them from 23 to 133 were found in the selected countries. We found that Bulgaria has a special HTA body. Since 2015 in Ukraine the health care system is experienced dynamic reform. In particular, a legislative framework has been adopted that regulates pharmaceutical pricing and reimbursement policies, the introduction of health technology assessment (HTA), etc. Despite the fact that Serbia is not part of the European Union there is an HTA commission established by the Ministry of Health where members of the HTA committee have 5- year mandates. According to our research, Georgia and North Macedonia do not use HTA. Meanwhile, United Kingdom is well known to show a strong interest in evidence health care. system. On the other hand, ?he Statutory health insurance system (SHI reform), since 2000, established a federally funded HTA program with a database of relevant HTA results. Even though that the high-income countries have more developed HTA guidelines and stronger and more secure health systems, all the countries mentioned in this work are facing similar challenges. All the countries have limite

Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit–The EU Regulator's Perspective

Since the implementation of the EU Orphan Regulation in 2000, the Committee for Orphan Medicinal Products at the European Medicines Agency has been evaluating the benefits of proposed orphan medicines vs. satisfactory treatment methods. This type of evaluation is foreseen in the Orphan Regulation as the orphan designation criterion called the “significant benefit.” In this article, based on 20 years of experience, we provide a commentary explaining what is considered a satisfactory method of treatment in the context of the EU Orphan Regulation and for the purpose of the assessment of significant benefit. We discuss the challenges posed by continuously changing clinical practise, which is associated with the increasing number of treatment options, evolving nature of medicinal therapeutic indications and our understanding of them.

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

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.

Pharmacotherapeutic Patterns and Patients’ Access to Pharmacotherapy for Some Rare Diseases in Bulgaria – A Pilot Comparative Study

Provision of the latest innovative and advanced therapies for rare diseases (RDs) patients, following the international therapeutic recommendations, is crucial and necessary for both practitioners and patients. The goal is to assess the access of Bulgarian patients with the most cost-consuming RDs to medicines and to compare the pharmacotherapeutic patterns in Bulgaria and the relevant European professional associations. Pharmaco-therapeutic guidelines for treating the most cost-consuming RDs in Bulgaria were analyzed to assess their compliance with the European ones. Market entrance was evaluated through analysis of the availability of medicines in the Positive Drug List (PDL) and their date of inclusion since marketing authorization. Guidelines’ compliance index was calculated and patient access was analyzed through evaluation of the National Health Insurance Fund (NHIF) standards, which provide additional criteria for treatment initiation. The analyzed guidelines follow the adopted recommendations by the relevant European professional associations. NHIF have exclusion and inclusion criteria for initiating treatment with medicines for rare diseases and for continuation. The average time-lag between centralized procedure approval and inclusion in the Bulgarian PDL for orphan medicinal products (MPs) is 6.75 years (SD = 4.96) with the longest time observed for eptacog alfa (20 years) and the shortest for rurioctocog alfa pegol, octocog alfa and simoctocog alfa (1 year). Bulgarian patients with cystic fibrosis with pulmonary manifestation had a wait time of only 1.6 years to get access to innovative, centrally authorized medicines, whereas the period for access to acromegaly treatment was 8.2 years. The main factors influencing market entrance and patient access are the time to inclusion in the PDL and the NHIF criteria.

High Price Medicines and Health Budgets: The Role Patients’ and Consumers’ Organisations Can Play

Healthcare systems face challenges due to budget constraints, complex therapies, and new treatments for rare diseases. One of the most successful patient advocacy campaigns of all times was initiated by people living with HIV and AIDS in African countries. Facing industry giants, they won court cases allowing governments to ignore intellectual property rights when the price of a medicine was abnormally high. This led to the approval of the international ‘Agreement on Trade Related aspects of Intellectual Property Rights’, which contributed to improving the availability of AIDS treatments for millions of people. Beyond this successful patient advocacy campaign, patient organisations have an important role to play in national discussions on prices of treatments and pharmaceuticals, especially for rare diseases. This article first discusses the specificity of the pricing of orphan medicinal products, and then provides an overview of some of the important actions that can be carried out by patients’ organisations.