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

2021 ◽  
Vol 12 ◽  
Author(s):  
Maria Kamusheva ◽  
Maria Dimitrova ◽  
Konstantin Tachkov ◽  
Guenka Petrova ◽  
Zornitsa Mitkova
Keyword(s):  
Rare Diseases ◽  
Time Lag ◽  
Wait Time ◽  
Professional Associations ◽  
Insurance Fund ◽  
Patient Access ◽  
Orphan Medicinal Products ◽  
Advanced Therapies ◽  
Therapeutic Guidelines ◽  
Therapeutic Recommendations

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.

scholarly journals Do Guidelines for the Diagnosis and Monitoring of Diabetes Mellitus Fulfill the Criteria of Evidence-Based Guideline Development?

2008 ◽  
Vol 54 (11) ◽  
pp. 1872-1882 ◽  
Author(s):  
Eva Nagy ◽  
Joseph Watine ◽  
Peter S Bunting ◽  
Rita Onody ◽  
Wytze P Oosterhuis ◽  
...  
Keyword(s):  
Diabetes Mellitus ◽  
Methodological Quality ◽  
Guideline Development ◽  
Evidence Based ◽  
Test Results ◽  
Time Period ◽  
Therapeutic Guidelines ◽  
Therapeutic Recommendations ◽  
Evidence Based Guideline

Abstract Background: Although the methodological quality of therapeutic guidelines (GLs) has been criticized, little is known regarding the quality of GLs that make diagnostic recommendations. Therefore, we assessed the methodological quality of GLs providing diagnostic recommendations for managing diabetes mellitus (DM) and explored several reasons for differences in quality across these GLs. Methods: After systematic searches of published and electronic resources dated between 1999 and 2007, 26 DM GLs, published in English, were selected and scored for methodological quality using the AGREE Instrument. Subgroup analyses were performed based on the source, scope, length, origin, and date and type of publication of GLs. Using a checklist, we collected laboratory-specific items within GLs thought to be important for interpretation of test results. Results: The 26 diagnostic GLs had significant shortcomings in methodological quality according to the AGREE criteria. GLs from agencies that had clear procedures for GL development, were longer than 50 pages, or were published in electronic databases were of higher quality. Diagnostic GLs contained more preanalytical or analytical information than combined (i.e., diagnostic and therapeutic) recommendations, but the overall quality was not significantly different. The quality of GLs did not show much improvement over the time period investigated. Conclusions: The methodological shortcomings of diagnostic GLs in DM raise questions regarding the validity of recommendations in these documents that may affect their implementation in practice. Our results suggest the need for standardization of GL terminology and for higher-quality, systematically developed recommendations based on explicit guideline development and reporting standards in laboratory medicine.

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 Clinical diagnostic and therapeutic guidelines of stroke neurorestoration (2020 China version)

2020 ◽  
Vol 8 (4) ◽  
pp. 241-251
Author(s):  
Xiaoling Guo ◽  
Qun Xue ◽  
Jianhua Zhao ◽  
Yi Yang ◽  
Yang Yu ◽  
...  
Keyword(s):  
Therapeutic Strategy ◽  
International Association ◽  
Stroke Patients ◽  
Clinical Diagnostic ◽  
Therapeutic Guidelines ◽  
Effective Therapeutic Strategy ◽  
Chinese Association ◽  
Therapeutic Recommendations ◽  
Made In

Stroke is the main cause of death and disability among Chinese, and neurorestoration is an effective therapeutic strategy for patients with stroke. In recent years, many achievements have been made in stroke neurorestoration, but viewpoints for managing stroke vary per discipline. In order to promote standardization of diagnosis and treatment for stroke neurorestoration, the Chinese Association of Neurorestoratology (CANR; Preparatory) and China Committee of International Association of Neurorestoratology (IANR-China Committee) organized professional experts in the field to integrate fragmented neurorestorative methods and establish clinical diagnostic and therapeutic guidelines for stroke neurorestoration. This guideline includes the diagnosis and staging of stroke and therapeutic recommendations for neurorestoration at different stages of stroke in order to improve survival and quality of life of stroke patients.

PP452 Impact Of Patient Access Schemes On Health Technology Assessment Agency Guidance For Rare Diseases In England and Scotland

2021 ◽  
Vol 37 (S1) ◽  
pp. 38-38
Author(s):  
Karen Mark ◽  
Prachi Manchanda ◽  
Judith Rubinstein ◽  
Riza Veronica Inumerable
Keyword(s):  
Cost Effectiveness ◽  
Health Technology Assessment ◽  
Incremental Cost ◽  
Rare Diseases ◽  
Technology Assessment ◽  
Health Technology ◽  
Incremental Cost Effectiveness Ratio ◽  
Base Case ◽  
Patient Access ◽  
Cost Effectiveness Ratio

IntroductionPatient access schemes (PAS) are agreements that may enable patients to access drugs or other treatments that may not be cost effective under normal circumstances. The aim of this study was to determine whether the use of PAS by the National Institute for Health and Care Excellence (NICE) and Scottish Medicines Consortium (SMC) for recommended drugs can lead to greater access to medications for rare diseases.MethodsReimbursement data for rare diseases between 2004 and 2021 from health technology assessment (HTA) agencies, namely the SMC (Scotland) and NICE (England), were included. The reviews with positive HTA decisions were considered, while those with negative decisions were excluded. Several observations were made from these data and reported.ResultsAmong the total positive reviews (n = 81), 43 included PAS. The inclusion of PAS in manufacturer submissions was more frequent for NICE than for the SMC (79% and 40% percent, respectively). Most of the drugs with PAS were included in the HTA guidance from both agencies. The positive NICE reviews contingent on PAS consisted of 20 drugs. For the same set of drugs, the SMC recommended 14 with PAS and one without PAS; five drugs were not assessed. Adalimumab was recommended by NICE with a PAS (base-case incremental cost-effectiveness ratio of GBP12,336 [EUR14,256]; GBP13,676 [EUR15,804]) and by the SMC without a PAS (base-case incremental cost-effectiveness ratio of GBP22,519 [EUR26,023]). Hence, without a PAS, the drug was costlier per quality-adjusted life-year for the National Health Service (NHS) Scotland.ConclusionsPAS submissions for rare diseases are more frequent for NICE than for the SMC. With the PAS discounts, the overall cost of the drugs is reduced, resulting in cost effectiveness. The SMC approved some drugs for which NICE required a PAS to improve the economic argument. Hence, the use of PAS for these drugs could lead to potential cost-savings to the NHS Scotland.

scholarly journals Legal assessment of current situation on orphan patients in Lithuania

Medicina ◽  
2008 ◽  
Vol 44 (8) ◽  
pp. 571 ◽  
Author(s):  
Indrė Špokienė
Keyword(s):  
Health Care ◽  
European Union ◽  
Rare Diseases ◽  
Comparative Method ◽  
National Level ◽  
Practical Implementation ◽  
The European Union ◽  
Medicinal Products ◽  
Member States ◽  
Orphan Medicinal Products

After Lithuania joined the European Union, the Regulation (EC) No. 141/2000 on orphan medicinal products and Commission Regulation (EC) No. 847/2000 came into force as part of national legislation. Member States must adopt specific measures to increase knowledge on rare diseases and to improve their detection, diagnosis, and treatment. The aim of this article was to present and to assess the current legal situation on orphan patients and their treatment in Lithuania, to identify legislation gaps, and to propose some ideas how to facilitate the solution of the existing problems in this field. For this purpose, European Union and Lithuanian legal documents on rare medicinal products are examined using a comparative method. With reference to inventory of Member States’ incentives for rare diseases in national level, the most important issues, which orphan patients face to in Lithuania, are singled out. In Lithuania, the situation of orphan patients in terms of protection of patient rights is insufficiently determined. The access to effective health care services or approved therapies in some cases is restricted. Working relationships between genetic services and various clinical specialists as well as with those in primary care are not legally determined; the number of clinical trials aimed at orphan medicinal products is low. These results suggest a need for awareness raising among Lithuanian Government, health care specialists, patient organizations about the importance to improve practical implementation of European Union legislation and progressive experience of some European countries in this field.

Comparative analysis of the scope of European Union paediatric investigation plans with corresponding orphan designations

2017 ◽  
Vol 103 (5) ◽  
pp. 427-430 ◽  
Author(s):  
Andrea Ecker ◽  
Segundo Mariz ◽  
Frauke Naumann-Winter ◽  
Koenraad Norga ◽  
Ingeborg Barisic ◽  
...  
Keyword(s):  
European Union ◽  
Comparative Analysis ◽  
Rare Diseases ◽  
Market Forces ◽  
The European Union ◽  
Medicinal Products ◽  
Paediatric Investigation Plan ◽  
Pharmaceutical Development ◽  
Orphan Medicinal Products ◽  
Small Patient

BackgroundMarket forces may not be sufficient to stimulate research and development of medicines for small patient populations, such as children and patients with rare diseases. Both the European Union Orphan and Paediatric Regulations were introduced to address the unmet public health needs of these smaller patient populations through the use of incentives, rewards and obligations. Developers for new medicines for rare diseases must agree a paediatric investigation plan (PIP) or waiver with the European Medicines Agency’s (EMA) Paediatric Committee (PDCO), and can also apply for an orphan designation (OD) from the EMA’s Committee of Orphan Medicinal Products (COMP). The scope of both the OD and the PIP (or waiver) is defined by the agreed condition.ObjectivesThe aim of this study was to analyse the approach of PDCO and COMP in defining the appropriate condition for a PIP or OD, respectively, in order to investigate potential challenges in the paediatric development of orphan medicines which have to meet the requirements of both legislations.MethodsA comparative analysis of PIP conditions and OD conditions was performed for medicines that have been reviewed by both Committees.ResultsWe found that in the substantial majority of cases there is no divergence between the conclusions of COMP and PDCO with regard to the condition for which a medicine is to be developed.ConclusionThese findings demonstrate that a collaborative approach allows both Regulations to work synergistically to foster pharmaceutical development for rare diseases in childhood.

scholarly journals Atypical clinical variants, predisposing factors and new therapeutic guidelines in bullous pemphigoid

2021 ◽  
Vol 97 (4) ◽  
pp. 183-191
Author(s):  
Ágnes Kinyó ◽  
Keyword(s):  
Elderly People ◽  
Bullous Pemphigoid ◽  
Skin Lesions ◽  
Predisposing Factors ◽  
European Guidelines ◽  
Therapeutic Guidelines ◽  
Blistering Disease ◽  
Clinical Variants ◽  
Therapeutic Recommendations

Bullous pemphigoid is the most common autoimmune subepidermal blistering disease in elderly people. The disease typically manifests on the skin and the mucosa with tense bullae or erosions with surrounding erythema, but in up to 20% of afected patients, bullae may be completely absent. The localized or generalized skin lesions usually present with itch. The author presents the rare, atypical forms of the disease, the most common triggering and predisposing factors, and the new therapeutic recommendations according to the European guidelines.

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.

Assessing the economic challenges posed by orphan drugs

2007 ◽  
Vol 23 (1) ◽  
pp. 36-42 ◽  
Author(s):  
Michael F. Drummond ◽  
David A. Wilson ◽  
Panos Kanavos ◽  
Peter Ubel ◽  
Joan Rovira
Keyword(s):  
Rare Diseases ◽  
Research Agenda ◽  
Cost Effective ◽  
Health Technology ◽  
Orphan Drugs ◽  
Patient Access ◽  
Standard Methods ◽  
Societal Value ◽  
Small Market ◽  
Societal Preferences

Historically, patients with rare diseases have been underserved by commercial drug development. In several jurisdictions, specific legislation has been enacted to encourage the development of drugs for rare diseases (orphan drugs), which would otherwise not be commercially viable. However, because of the small market, these drugs are often very expensive. Under the standard methods of health technology assessment (HTA) incorporating economic evaluation, orphan drugs do not usually prove to be cost-effective and this, coupled with their high cost, means that funding and patient access may be limited. However, these restrictions may not be in line with societal preferences. Therefore, this study discusses whether the standard methods of HTA are adequate for assisting decisions on patient access to and funding of orphan drugs and outlines a research agenda to help understand the societal value of orphan drugs and issues surrounding their development, funding, and use.

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