Orphan Drugs in Oncology
2019 Recent Results Cancer Reshttps://www.doi.org/10.1007/978-3-030-01207-6_8 Humans, Neoplasms/*drug therapy, Orphan Drug Production/*economics/*legislation & jurisprudence, Rare Diseases/*drug therapy, Equity, Health policy, Health technology assessment, Oncology, Orphan drugs, Patient access, Rare diseases,
Rare diseases represent a group of conditions affecting a very limited number of patients. Low profitability resulting from the small size of target population coupled with difficulties in conducting the research causes the lack of interest from the pharmaceutical industry. In order to promote research and development of medicines for rare diseases, a special ‘orphan’ legislation was introduced in a number of regions. These measures led to a significant increase in the number of approved orphan molecules. The high per patient cost of orphan drugs, as well the rapid growth of orphan drug sector, raised concerns regarding the sustainable funding of therapies for rare diseases. Rare cancers represent the majority of the current orphan drug market and are often associated with very high revenues. This chapter provides a review of orphan legislations and health technology assessment framework, analyses the position of oncology drugs on the orphan drug market and discusses future perspectives.
Adherence and persistence to hyperlipidemia medications in patients with atherosclerotic cardiovascular disease and those with diabetes mellitus based on administrative claims data in Japan
2019 Atherosclerosishttps://www.doi.org/10.1016/j.atherosclerosis.2018.12.026 Adherence, Cardiovascular disease, Diabetes mellitus, Hyperlipidemia, Persistence,
BACKGROUND AND AIMS: Real-world data on treatment patterns in Japanese hyperlipidemia patients with diabetes mellitus (DM) or prior atherosclerotic cardiovascular diseases (ASCVD) are lacking. METHODS: This is a retrospective, longitudinal cohort analysis of administrative claims data (Japan Medical Data Center [JMDC] and Medical Data Vision [MDV] databases) for patients prescribed a new hyperlipidemia medication between 2014 and 2015. Patients were followed for >/=12 months. Outcomes included prescribing patterns, persistence (discontinuations), and adherence (proportion of days covered). RESULTS: Data were analyzed for 11,718 and 27,746 DM, and 4101 and 14,356 ASCVD patients from the JMDC and MDV databases, respectively. Among previously-untreated patients, index prescriptions were primarily for moderate statins in the DM (JMDC: 74.7%, MDV: 77.5%) and ASCVD (JMDC: 75.4%, MDV: 78.5%) sub-cohorts. Combinations were rarely prescribed (=2.5%). Previously-treated patients were most frequently prescribed combinations in the DM (JMDC: 46.7%, MDV: 53.6%) and ASCVD (JMDC: 49.3%, MDV: 53.3%) sub-cohorts. Intensive statins were rarely used by previously-untreated (=1%) or previously-treated (=8%) patients in either sub-cohort. Approximately half of previously-untreated patients discontinued hyperlipidemia therapy within 12 months. Adherence was >/=80% across most drug classes. CONCLUSIONS: Many Japanese hyperlipidemia patients with DM or ASCVD are prescribed single-agent lipid-lowering therapy. Use of intensive therapy is lower than expected, and is suggestive of under-treatment. The low persistence rates are concerning, and warrant further study.
Health-care resource use and current treatment of adult atopic dermatitis patients in Japan: A retrospective claims database analysis
2019 J Dermatolhttps://www.doi.org/10.1111/1346-8138.14947 Adult, Ambulatory Care/statistics & numerical data, *Cost of Illness, Cyclosporine/therapeutic use, Databases, Factual/statistics & numerical data, Dermatitis, Atopic/economics/*therapy, Dermatologic Agents/*therapeutic use, Drug Prescriptions/statistics & numerical data, Female, Glucocorticoids/therapeutic use, Hospitalization/statistics & numerical data, Humans, Japan, Male, Middle Aged, Patient Acceptance of Health Care/*statistics & numerical data, Phototherapy/*statistics & numerical data, Retrospective Studies, Young Adult, atopic dermatitis, claim database, health-care resource use, treatment patterns, study. A. I. received consultancy fees from AbbVie Inc., Eli Lilly Japan K.K.,, Novartis Pharma K.K., Maruho Co. Ltd, Torii Pharmaceutical Co. Ltd., Japan Tobacco, Inc. and Sanofi K.K., including fees for this study. H. F., K. A., T. I. and Y. T., are employees of Sanofi K.K. J. D. and A. F. are employees of Creativ‐Ceutical. The, authors were responsible for all content and editorial decisions, and received no, honoraria related to the development of this publication.,
The real-world evidence on the profiles of patients suffering from atopic dermatitis (AD) in Japan is sparse. A retrospective claim database analysis was conducted to estimate the health-care resource use (HCRU) and current AD treatment. Data from October 2013 to September 2016 were extracted from the JMDC (Tokyo, Japan) claims database. HCRU was assessed by a comparison of AD patients and matched non-AD controls. A multivariate analysis was performed to estimate HCRU attributable to AD. AD patients (n = 39 893) have more claims of certain diagnoses such as rhinitis, viral and fungal infections, sleep disorders and conjunctivitis as well as higher HCRU (outpatient visits, prescriptions of AD-related and non-AD-related medications, phototherapy, laboratory tests) than matched non-AD controls (n = 39 893). Treatment pattern analysis included treatment-naive patients (n = 8478) and previously treated AD patients (n = 30 109). Approximately 20% of previously treated patients were on the continuous systemic treatment during 18-month follow up. Systemic corticosteroids were the most frequently used systemic treatments. Oral cyclosporin was less frequently used in both groups, but for the longest duration. Almost half of previously treated patients with oral cyclosporin continued treatment for more than 3 months. In conclusion, HCRU was higher in AD patients than non-AD controls, indicating a high burden of the disease imposed on AD patients. Continuous administration of systemic treatment, such as oral cyclosporin, systemic corticosteroids and phototherapy, observed in AD patients sheds light on the difficulties of managing AD in Japanese clinical practise.
New Horizons of Treatment Technologies: The Emerging Gene Therapies
2019 2nd HTA Academy
Meta-analysis of real-world evidence comparing non-vitamin K antagonist oral anticoagulants with vitamin K antagonists for the treatment of patients with non-valvular atrial fibrillation
2019 J Mark Access Health Policyhttps://www.doi.org/10.1080/20016689.2019.1574541
ABSTRACTIntroduction: Numerous real-world studies have compared non-vitamin K antagonist oral anticoagulants (NOACs) with vitamin K antagonists (VKAs) in patients with non-valvular atrial fibrillation (NVAF). A meta-analysis was performed to synthesize the available evidence.Methods: Systematic searches were performed through 12/2016 to identify non-randomized NVAF studies comparing NOACs with VKAs, and reporting effectiveness, safety, or persistence.Results: Of 562 citations identified, 49, 79, and 18 compared rivaroxaban, dabigatran, and apixaban, respectively, with VKAs and were included. Compared with VKAs, rivaroxaban was associated with a reduced risk of ischemic stroke (IS) (hazard ratio [HR] = 0.83, 95% confidence interval [CI] = 0.75?0.93), intracranial haemorrhage (ICH) (HR = 0.69, 95% CI = 0.52?0.90), and non-persistence (HR = 0.62, 95% CI = 0.60?0.65). Dabigatran was associated with a significantly lower risk of IS (HR = 0.80, 95% CI = 0.65?0.98) and ICH (HR = 0.45, 95% CI = 0.36?0.58), but not for non-persistence (HR = 0.91, 95% CI = 0.53?1.55), compared with VKAs. Apixaban was associated with a lower risk of ICH than VKAs (HR = 0.41, 95% CI = 0.28?0.60), but was not different to VKAs in terms of IS (HR = 1.01, 95% CI = 0.87?1.17) or non-persistence (HR = 1.08, 95% CI = 0.81?1.45).Conclusion: NOACs appear to be at least as effective and safe as VKAs for stroke prevention in patients with NVAF.
Patient-Reported Outcomes in Oncology, Beyond Randomized Controlled Trials
2019 Recent Results Cancer Reshttps://www.doi.org/10.1007/978-3-030-01207-6_5 Humans, *Neoplasms, *Patient Reported Outcome Measures, Quality of Life, Randomized Controlled Trials as Topic, Clinical trials, Health-related quality of life (HRQoL), Oncology, Patient preference, Patient-reported outcomes (PROs), Payers, Symptom assessment,
The goal of the treatment of a disease has moved from treating organs and diseases through symptoms, biological parameters and imaging towards treating a human being as a whole. The treatments should deliver benefits that patients can personally perceive. However, the patient’s perspective does not always match the one of those surrounding them. Illustratively, patients’ symptom assessments are more predictable for daily health status, whereas clinicians’ symptom measurements are more related to clinical outcomes. The term, patient-reported outcomes (PROs), includes any data that are reported directly by the patient without an intermediary, such as a family member or a healthcare professional. The use of PROs in oncology trials is increasing and the U.S. Food and Drug Administration has published guidelines on the review and evaluation of PROs. However, while PROs are increasingly used in clinical trials, they are rarely used in daily clinical practice. Further, healthcare payers are concerned with issues related to relevance, quality, and interpretability of these outcomes.
PIN37 COST-EFFECTIVENESS ANALYSIS OF FIDAXOMICIN FOR CLOSTRIDIUM DIFFICILE INFECTION IN JAPAN
2019 Value in Health
Treatment patterns, persistence and adherence rates in patients with type 2 diabetes mellitus in Japan: a claims-based cohort study
2019 BMJ Openhttps://www.doi.org/10.1136/bmjopen-2018-025806 adherence, administrative claims-based study, antidiabetic drug therapy, dipeptidyl peptidase-4 inhibitors, persistence, type 2 diabetes, Nippon Boehringer Ingelheim, Eli Lilly Japan K.K., Kissei Pharmaceutical,, Medtronic Japan, MSD, Novartis Pharma KK, Novo Nordisk Pharma, Sanofi KK and, Takeda Pharmaceutical and contract research fees for collaborative research with, the Japan Diabetes Foundation. HK, KK, AO and YS are employees of Takeda, Pharmaceutical. SH was an employee of Takeda Pharmaceutical at the time the study, was conducted. FG and YO are employees of Creativ-Ceutical K.K.,
OBJECTIVE: To determine real-world trends in antidiabetic drug use, and persistence and adherence, in Japanese patients with type 2 diabetes mellitus (T2DM). DESIGN: Retrospective evaluation of administrative claims data (2011-2015) using the Japan Medical Data Center (JMDC) and Medical Data Vision (MDV) databases. SETTING: Analysis of two administrative claims databases for Japanese patients with T2DM. PARTICIPANTS: Adults (aged >/=18 years) with an International Classification of Diseases, 10th Revision code of T2DM and at least one antidiabetic drug prescription. MAIN OUTCOME MEASURES: Treatment patterns in untreated (UT) or previously treated (PT) patients receiving antidiabetic therapy; persistence with treatment at 12 months; adherence to treatment at 12 months. RESULTS: 40 908 and 90 421 patients were included from the JMDC and MDV databases, respectively. The most frequently prescribed therapy at the index (first prescription) date was dipeptidyl peptidase-4 inhibitor (DPP-4i) in UT patients (JMDC: 44.0%, MDV: 54.8%) and combination therapy in PT patients (74.6%, 81.1%). Most common combinations were DPP-4i plus: biguanide (BG; 11.4%, 10.9%), sulfonylurea (SU; 8.4%, 11.0%) or BG+SU (7.8%, 9.1%). In UT or PT patients from either database whose index prescription was for any antidiabetic drug class(es) other than DPP-4i, the most frequent add-on or switch was to DPP-4i. 12-month persistence with index monotherapy was highest with DPP-4i and BG. Adherence was high (>/=80%) for all monotherapy schedules, except insulin and glucagon-like peptide-1 agonist, and for the five most frequent two-drug and three-drug combinations. Persistence was greater in elderly UT patients and in those receiving =5 medications, but comparatively worse in UT patients with >/=3 index antidiabetic drug classes. CONCLUSIONS: The findings indicate that DPP-4i is the most commonly used antidiabetic drug class in Japanese patients with T2DM, and persistence and adherence to this antidiabetic drug class are high.
Health Technology Assessment of Gene Therapies in Europe and the USA: Analysis and Future Considerations
2019 Cell and Gene Therapy Insightshttps://www.doi.org/10.18609/cgti.2019.112
Prevalence of hepatitis C infection among the general population and high-risk groups in the EU/EEA: a systematic review update
2019 BMC Infect Dishttps://www.doi.org/10.1186/s12879-019-4284-9 Adult, Blood Donors/statistics & numerical data, Europe/epidemiology, European Union, Female, Hepatitis C/*epidemiology, Hepatitis C Antibodies/blood, Homosexuality, Male, Humans, Male, Pregnancy, Pregnancy Complications, Infectious/epidemiology/virology, Pregnant Women, Prevalence, Prisoners/statistics & numerical data, Sexual and Gender Minorities/statistics & numerical data, Europe, Hepatitis C, High-risk groups, Men who have sex with men, People who inject drugs, Prisoners, Systematic review,
BACKGROUND: Although significant improvement in efficacy measured by a sustained virological response, the high acquisition costs of direct-acting antivirals limit the access for patients and influence the costs of healthcare resource utilisation in hepatitis C. It is important to have the latest estimates of prevalence, especially in high-risk groups, for cost of illness, cost-effectiveness and budget impact studies. METHODS: Original studies on the estimates of the prevalence among general and high-risk groups in the European Union/European Economic Area (EU/EEA) were retrieved from Medline and Embase for the period from 2015 to 2018. All included studies were evaluated for risk of selection bias and summarised together in a narrative form. Results from previous reviews and updated searches were compared per country among different populations, respectively. RESULTS: Among the 3871 studies identified, 46 studies were included: 20 studies were used for the estimate of the general population; 3 for men who have sex with men (MSM); 6 for prisoners; and 17 for people who inject drugs (PWID). Compared with the results reported in previous systematic reviews, the updated estimates were lower than previously in most available countries. Anti-HCV general population prevalence estimates ranged from 0.54 to 1.50% by country. The highest prevalence of anti-HCV was found among PWID (range of 7.90-82.00%), followed by prisoners (7.00-41.00%), HIV-positive MSM (1.80-7.10%), HIV-negative MSM (0.20-1.80%), pregnant women (0.10-1.32%) and first-time blood donors (0.03-0.09%). CONCLUSIONS: Our study highlights the heterogeneity in anti-HCV prevalence across different population groups in EU/EEA. The prevalence also varies widely between European countries. There are many countries that are not represented in our results, highlighting the need for the development of robust epidemiological studies.