Number needed to treat based on real-world evidence for non-vitamin K antagonist oral anticoagulants versus vitamin K antagonist oral anticoagulants in stroke prevention in patients with non-valvular atrial fibrillation
2019 Journal of Medical Economicshttps://www.doi.org/10.1080/13696998.2019.1606001
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.
Effects of Droxidopa Treatment for Neurogenic Orthostatic Hypotension in Patients Concomitantly on Dopa Decarboxylase Inhibitors (P3.6-048)
Objective: To assess long-term efficacy of droxidopa for treatment of neurogenic orthostatic hypotension (nOH) in patients concomitantly on dopa decarboxylase (DDC) inhibitors (DDCIs)Background: nOH is a non-motor symptom of Parkinson disease (PD). Treatment of PD frequently includes DDCIs. Droxidopa, a treatment for symptomatic nOH, is converted to norepinephrine by DDC.Design/Methods: Patients were enrolled in a prospective cohort study comparing outcomes before initiation of droxidopa and after 1, 3, and 6 months of treatment. Patients recorded monthly number of falls using a questionnaire and dizziness/lightheadedness using Orthostatic Hypotension Symptom Assessment, Item 1 (OHSA-1). Post-hoc analysis compared outcomes in patients on DDCIs vs without DDCIs. “DDCI treatment” defined as receiving carbidopa alone or in combination with levodopa or entacapone. Influence of DDCIs was compared across time points using generalized linear mixed models adjusting for repeated measures within individuals.Results: 168 patients were included (PD: 32.1%, autonomic failure: 66.1%, other: 1.8%). 61.8% of patients on DDCIs (n=55) reported ≥1 fall at baseline vs 46.9% of patients not on DDCIs (n=113). Patients on DDCIs experienced significant reduction in fall rate (30.1%, 36.4 point reduction) and improvement in OHSA-1 scores (−1.8) from baseline to 6 months (both P<0.05). Patients not on DDCIs also experienced significant OHSA-1 scores improvement (−1.9, P<0.05), but fall rate reduction was not significant (39.8%, 6.2 point reduction, P=0.49). The differences in change from baseline to 6 months for fall risk and OHSA-1 score were not significant between groups (P=0.15, P=0.96, respectively). DDCI users (66.6%) had a greater prevalence of >1 falls vs nonusers (46.6%) at baseline.Conclusions: These post-hoc analyses suggest that DDCI users and nonusers experienced reductions in fall rate and dizziness/lightheadedness with droxidopa. Additional studies are needed to examine the impact of DDCIs on droxidopa because the current study was not powered for subgroup analyses and data were patient self-reported.Disclosure: Dr. Kymes has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Lundbeck and CVS Health. Dr. Kymes holds stock and/or stock options in Lundbeck that is greater than $10,000 in value which sponsored research in which Dr. Kymes was involved as an investigator. Dr. Francois has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Lundbeck and Creativ-Ceutical. Dr. Francois holds stock and/or stock options in Lundbeck. Dr. Sullivan has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Lundbeck. Dr. McLeod has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Xcenda, LLC. Dr. Duhig has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Xcenda, LLC. Dr. Ogbonnaya has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Xcenda LLC. Dr. Quillen has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Xcenda LLC. Dr. Cannon has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Lundbeck. Dr. Biaggioni has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Lundbeck, Theravance Biopharma. Dr. Biaggioni has received research support from Lundbeck (investigator-initiated grant) and Theravance Biopharma (Clinical Trial). Dr. Shibao has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Lundbeck. Dr. Shibao h s received research support from Lundbeck. Dr. Yue has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Xcenda LLC. Dr. Hauser has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with AbbVie Inc., Acorda Therapeutics, Academy for Continued Healthcare Learning, Acadia Pharmaceuticals, Inc., Adamas Pharmaceuticals, AstraZeneca, ApoPharma, Back Bay Life Science, Biotie Therapies, Bracket, Cerecor, Inc., ClearView Healthcare Partners, ClinicalMind Medical and Therapeutic Communications, CNS Ratings, LLC., Cowen and Company, Cynapsus Therapeutics, DDB Health LLC, Decision Resources Group (DRG), Eli Lilly & Company, eResearch Technology, Inc., Expert Connect, Extera Partners, GE Healthcare, Health Advances, HealthLogix, Health and Wellness Partners, Huron Consulting Group, Impax Laboratories, Impel Neuropharma, Intec Pharma Ltd., Jazz Pharmaceuticals, Kashiv Pharma LLC, Kyowa Kirin Pharmaceutical Development, Ltd., LCN Consulting, LifeMax, Life Sciences, Lundbeck LLC, The Lockwood Group, MEDACorp, Medscape, Medtronic, Michael J. Fox Foundation, Mitsubishi Tanabe Pharmaceuticals, Movement Disorder Society, National Institutes of Health (NIH), Neurocea LLC, Neurocrine Biosciences, Neuroderm, Neuropore Therapies, Orbes Medical Group, Outcomes Insights, Parkinson Study Group, Peerview Press, Pennside Partners, Pfizer, Inc., Pharma Two B, Ltd, Phase Five Communications, Prescott Medical Group, Prexton Therapeutics, Prilenia Development Ltd., Projects in Knowledge, Putnam Associates, Quintiles, RMEI Medical Education for Better Outcomes, SAI Med Partners LLC, Sarepta Therapeutics, Schlesinger Associates, Scion Neurostim, LLC, Seagrove Partners, LLC, Slingshot Insights, Sunovion Pharmaceuticals, Inc., Sun Pharma, Teva Pharmaceutical Industries, US WorldMeds, Vista Research, WebMD, Windrose Consulting Group. Dr. Hauser has received royalty, license fees, or contractual rights payments from the University of South Florida. Dr. Hauser has received research support from AbbVie Inc., Acorda Therapeutics, AstraZeneca, Axovant Sciences , Biogen Inc., Cavion, Enterin Inc., Impax Laboratories, LLC., Intec Pharma Ltd, Jazz Pharmaceuticals, NeuroDerm Ltd., Lundbeck, Michael J Fox Foundation for Parkinson’s Research, F. Hoffman-La Roche, Dart NeuroScience LLC, Prexton Therapeutics, Revance Therapeutics Inc., Sunovion Pharmaceuticals.
Non-profit drug research and development: the case study of Genethon
2019 J Mark Access Health Policyhttps://www.doi.org/10.1080/20016689.2018.1545514 Non-profit, ethics, orphan drugs, pharmaceutical industry, pricing, research and development,
Non-profit drug research and development (R&D) has the potential to deliver innovative treatments at affordable prices. Using the case study methodology, we discuss some ethical and economic issues, including the possible impact of non-profit companies on innovation efforts from for-profit firms. Like other non-profits, Genethon is willing to adopt an ethical attitude toward their donors by pricing their products affordably. It remains to be seen if the approach to internalize the marketing authorization, manufacturing and distribution activities prove to be efficient and sustainable. Also, the firm faces an ethical dilemma because lower prices of innovative drugs can dry the for-profit R&D in the area and prevent patient access to future innovations.
[PCV20] NUMBER NEEDED TO TREAT FOR ORAL ANTI-COAGULANTS VERSUS VKA IN STROKE PREVENTION IN ATRIAL FIBRILLATION
2018 ISPOR Europe
[PIN9] Disease burden of hepatitis C infection in Asia: an overview of systematic review
2018 ISPOR Asia
[PCP15] Why European cross-border initiatives on drug procurement may fail?
2018 ISPOR Europe
Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group
2018 Value Healthhttps://www.doi.org/10.1016/j.jval.2018.03.004 Biomedical Research/*standards, *Consensus Development Conferences as Topic, Cost-Benefit Analysis, Health Policy, Humans, *Rare Diseases/diagnosis/therapy, Technology Assessment, Biomedical/*methods, *cost-effectiveness, *health policy, *health technology assessment, *orphan designation, *rare diseases,
BACKGROUND: Successful development of new treatments for rare diseases (RDs) and their sustainable patient access require overcoming a series of challenges related to research and health technology assessment (HTA). These impediments, which may be unique to RDs or also apply to common diseases but are particularly pertinent in RDs, are diverse and interrelated. OBJECTIVE: To develop for the first time a catalog of primary impediments to RD research and HTA, and to describe the cause and effect of individual challenges. METHODS: Challenges were identified by an international 22-person expert working group and qualitative outreach to colleagues with relevant expertise. A broad range of stakeholder perspectives is represented. Draft results were presented at annual European and North American International Society for Pharmacoeconomics and Outcomes Research (ISPOR) congresses, and written comments were received by the 385-strong ISPOR Rare Disease Review Group from two rounds of review. Findings were refined and confirmed via targeted literature search. RESULTS: Research-related challenges linked to the low prevalence of RDs were categorized into those pertaining to disease recognition and diagnosis, evaluation of treatment effect, and patient recruitment for clinical research. HTA-related challenges were classified into issues relating to the lack of a tailored HTA method for RD treatments and uncertainty for HTA agencies and health care payers. CONCLUSIONS: Identifying and highlighting diverse, but interrelated, key challenges in RD research and HTA is an essential first step toward developing implementable and sustainable solutions. A collaborative multistakeholder effort is required to enable faster and less costly development of safe, efficacious, and appropriate new RD therapies that offer value for money.