scholarly journals Pricing Zolgensma – the world’s most expensive drug

2021 ◽  
Vol 10 (1) ◽  
Author(s):  
Mark Nuijten
Keyword(s):  
Expensive Drug

scholarly journals Reduction of Vascular Inflammation, LDL-C, or Both for the Protection from Cardiovascular Events?

2018 ◽  
Vol 12 (1) ◽  
pp. 29-40 ◽  
Author(s):  
Andromachi Reklou ◽  
Michael Doumas ◽  
Konstantinos Imprialos ◽  
Konstantinos Stavropoulos ◽  
Dimitris Patoulias ◽  
...  
Keyword(s):  
Vascular Inflammation ◽  
Low Density Lipoprotein ◽  
Density Lipoprotein ◽  
Myocardial Damage ◽  
Lipid Lowering ◽  
Human Antibody ◽  
Low Grade ◽  
Cvd Risk ◽  
Expensive Drug ◽  
Arterial Inflammation

Background: Low density lipoprotein cholesterol (LDL-C) and low grade arterial inflammation are key pathogenic factors for atherosclerosis and its manifestation, cardiovascular disease (CVD). Objective: In this narrative review we assessed if decreasing LDL-C levels or inflammation or both is more effective in reducing CVD events. Results: In the Scandinavian Simvastatin Survival Study (4S), all statin trials of the 90s’ and the Further Cardiovascular Outcomes Research with PCSK9 Inhibition in Subjects with Elevated Risk (FOURIER) the benefit came from the LDL-C reduction. In the GREak and Atorvastatin Coronary heart disease Evaluation (GREACE), the Treating to New Targets (TNT), and the Justification for the Use of Statins in Prevention: an Intervention Trial Evaluating Rosuvastatin (JUPITER) trials both mechanisms in combination produced significant benefits. In the Atorvastatin for Reduction of MYocardial Damage during Angioplasty (ARMYDA) trials and the Canakinumab Antiinflammatory Thrombosis Outcome Study (CANTOS) with a human antibody targeting IL-1β with no lipid lowering effect, the reduction in arterial inflammation played the only beneficial role because there was no change in lipids levels. Conclusion: Both LDL-C and inflammation reduction are beneficial to the reduction of CVD risk. However, canakinumab is a very expensive drug that only induced a 15% reduction in CVD events, thus drastically reducing the possibility for it to be used in clinical practice. Besides, canakinumab is associated with increased infections, some fatal. A potent statin with anti-inflammatory effects is probably the best choice for the majority of those needing hypolipidaemic drug therapy.

scholarly journals Ensuring access to medicines at a fair price? Innovative contracting experiences in France

2020 ◽  
Vol 30 (Supplement_5) ◽  
Author(s):  
I Alaoui ◽  
C Izambert ◽  
A Toullier
Keyword(s):  
Public Investment ◽  
Access To Medicines ◽  
Added Value ◽  
Fair Price ◽  
Real World Data ◽  
Expensive Drug ◽  
Patient Organizations ◽  
Performance Contracts ◽  
Performance Based Contracts ◽  
Innovative Contracting

Abstract Issue Innovative contracting models are developed to ease price-setting negotiations in case an extremely expensive drug has not proven sufficient efficiency in clinical trials. As disruptive HIV treatments are expected in the near future, French patient organizations evaluated the ability of these innovative contracts to ensure accessible medicines at a fair price. Description Performance-based schemes condition prices paid by the State to the efficiency of the medicine observed through real-world data. In France, thirteen performance-based contracts have been concluded between 2008 and 2015. They are presented as a triple solution: innovative treatments are available to patients, manufacturers access markets, and states ensure healthcare within limited budgets. Establishing the added value of these models implies determining if they allow rapid access to treatments with substantial savings for payers, while ensuring rigorous price and cost transparency. Results Performance-based contracts indeed ensure patient access to treatments, but other mechanisms (such as temporary use authorizations) already serve this purpose. Regarding expenditure reduction however, these schemes have not proven their worth. The Court of Auditors' evaluation showed they do not generate substantial savings, as final prices correspond to those that would have applied with the European price guarantee. Lastly, as contracts are protected by business secrecy, the public cannot access neither to actual prices negotiated by payers, nor the amount of public investment that have been used for the research and development of the drug. Lessons The derogatory nature of performance contracts invites us to consider them on a case-by-case basis if ensuring access to a specific innovation is necessary. These contracts are certainly innovative, but they cannot be presented as technologies providing access at a fair price. Finally, their contractual and derogatory nature raises serious transparency issues. Key messages Performance-based contracts should be considered as alternatives to existing administrative channels provided that they lead to substantial savings and are drawn up in full transparency. Patient organizations need to assess innovative schemes such as performance-based contracting to ensure access to treatments without undermining historical struggles for fair and transparent pricing.

What price for a year of life? A survey of U.S. and Canadian oncologists

2009 ◽  
Vol 27 (15_suppl) ◽  
pp. 6565-6565
Author(s):  
S. R. Berry ◽  
P. J. Neumann ◽  
C. Bell ◽  
E. Nadler ◽  
W. C. Evans ◽  
...  
Keyword(s):  
Contextual Information ◽  
Metastatic Cancer ◽  
Therapeutic Benefit ◽  
New Drugs ◽  
Expensive Drug ◽  
Hypothetical Treatment ◽  
New Treatment ◽  
The Stability ◽  
To Receive ◽  
The U.S

6565 Background: New cancer drugs are increasingly expensive and raise difficult questions about the magnitude of therapeutic benefit needed to justify their incremental cost. In this context, it is unclear whether oncologists endorse standard thresholds of $50,000 to $100,000 per year of life. Methods: We surveyed 1,379 U.S. and 356 Canadian (Cdn) oncologists and asked how much longer a patient would need to survive metastatic cancer to justify the expense of a new treatment. To determine the stability of attitudes towards cost-effectiveness (CE) we randomized oncologists to receive two different versions of the scenario in which the price of the new treatment was varied (higher versus lower drug cost). In the U.S. survey, oncologists were also randomized to receive surveys in which we varied the provision of contextual information about the CE of several familiar interventions. Both U.S. and Cdn oncologists were asked to indicate what they “thought was ‘good value for money’ expressed as cost per life-year gained (LYG).” Results: Response rate was 57% in the U.S. and 48% in Canada. CE ratios implied by oncologists’ responses differed significantly between the groups randomized to the higher versus lower price of the hypothetical treatment (p < 0.001 U.S., p < 0.0001 Canada), but were independent of randomization to varying contextual information (p > 0.1). The median willingness to pay for a quality-adjusted year of life ranged from $150,000 (for oncologists considering the lower priced drug) to $250,000 (for those considering the more expensive drug) in both countries. Among those who considered the more expensive drug, 25% of respondents implicitly endorsed a CE ratio greater than $600,000 (U.S.) and $500,000 (Canada). In contrast, when asked directly to indicate CE ratios that were good value for the money outside of the clinical scenario, 70% (U.S.) and 64% (Canada) of respondents indicated values of less than $100,000 per LYG. Conclusions: Oncologists responding to our survey provided inconsistent views on how much benefit expensive new drugs should provide to be worthwhile. This suggests that means of eliciting input from physicians that reflect more stable attitudes need to be developed to appropriately inform decision-makers. No significant financial relationships to disclose.

High Risk Pregnancies: Teenagers, Poverty, and Drug Abuse

1997 ◽  
Vol 27 (3) ◽  
pp. 541-562 ◽  
Author(s):  
Judith Waters ◽  
Albert R. Roberts ◽  
Keith Morgen
Keyword(s):  
Drug Abuse ◽  
Behavioral Problems ◽  
Teenage Pregnancy ◽  
Family System ◽  
Single Mother ◽  
Sexual Relationships ◽  
Readiness For Change ◽  
Real Problem ◽  
Expensive Drug ◽  
Effective Prevention

The real problem is not teenage pregnancy alone, but the destructive combination of single motherhood, poverty, and drug abuse. The consequences of being a poor, inner-city, single mother with an expensive drug habit may include prostitution, drug dealing, dependency upon welfare and/or extra support from an already overwhelmed family system, and neglected children. Almost inevitably, the children exhibit cognitive deficits and behavioral problems, may turn to drugs at a young age, and may have an early involvement with the criminal justice system. To design effective prevention programs it is essential to understand the myriad complex risk factors that affect the lives of these vulnerable young women. As the first stage in a larger study of motivation and readiness for change and the development of self-protective health practices, interview data were collected from 30 participants. Sixteen were residents and staff who had graduated from a drug treatment program and 14 were undergraduate and graduate students, all of whom had been pregnant as teenagers. The results yielded a surprising commonality of experiences for both groups that included childhood physical and sexual abuse, coercive adolescent sexual relationships, early first sexual contact, early first pregnancies (often as a function of rape or incest), abortions, and chemical dependency, especially during pregnancy.

scholarly journals Il governo dell’innovazione farmaceutica in Italia: quali strumenti di governance regionale proporre?

2016 ◽  
Vol 3 (Suppl. 1) ◽  
pp. S7-S12
Author(s):  
Sabina Nuti
Keyword(s):  
Risk Sharing ◽  
Decisive Role ◽  
Regional Governance ◽  
New Drugs ◽  
Adoption Process ◽  
Value For Money ◽  
Expensive Drug ◽  
Italian Regions ◽  
Governance Tools ◽  
Innovative Drugs

Managing pharmaceutical innovation in Italy: which regional governance tools can be adopted? Within the Italian federalist framework, national and regional governance tools for pharmaceutical care have been developed in recent years. From a financial perspective, the pharmaceutical outpatient expenditure has already been put under control and this has markedly contributed to reducing the overall costs. The hospital pharmaceutical expenses instead have grown. On this last element bears how innovative and expensive drugs are introduced and managed; among these drugs, cancer drugs have a decisive role. In the last years the AIFA (Agenzia Italiana del Farmaco – Italian Drugs Agency) policies regarding the adoption process of new drugs have stressed the concept of value for money: any innovative and expensive drug is linked to a webbased control register in order to monitor outcomes. The aim is to relate the drug price to the obtained results (payment by result and risk-sharing) or at least to institute simple financial agreements (cost-sharing) that can be defined as managed entry agreements (MEA). The critical point that can cause equity problems is the way these national governance tools are applied in different regional contexts. There are in fact marked differences among Italian regions. Most regions are aware that the only way to rule the system, and in particular the use of innovative drugs, is to have stronger evidence-based management tools able to connect different systems of oncological prescriptions. The aim is to follow patients in different care settings in order to measure the pathway phases in terms of consumption, costs and quality outcomes and therefore evaluate in actual practice the value for money of innovative drugs.

scholarly journals Fluconazole in Hypercalciuric Patients with Increased 1,25(OH)2D Levels: The Prospective, Randomized, Placebo-Controlled, Double Blind FLUCOLITH Trial.

2022 ◽  
Author(s):  
Aurélia Bertholet-Thomas ◽  
Aurélie Portefaix ◽  
Sacha Flammier ◽  
Carole Dhelens ◽  
Fabien Subtil ◽  
...  
Keyword(s):  
Vitamin D ◽  
Case Reports ◽  
Renal Diseases ◽  
Dietary Advice ◽  
Expensive Drug ◽  
Double Blind ◽  
Efficient Treatment ◽  
Tolerability Data ◽  
Stage Renal Disease ◽  
End Stage

Abstract BackgroundHypercalciuria is one of the most frequent metabolic disorders associated with nephrolithiasis and/or nephrocalcinosis possibly leading to chronic kidney disease (CKD) and bone complications in adults. Orphan diseases with different underlying primary pathophysiology share inappropriately increased 1,25(OH)2D levels and hypercalciuria, e.g., hypersensitivity to vitamin D and renal phosphate wasting. Their management is challenging, typically based on hyperhydration and dietary advice. The antifungal azoles are known to inhibit the 1α-hydroxylase and therefore decrease 1,25(OH)2D levels; they are commonly used, with well described pharmacokinetic and tolerability data. Fluconazole has been successfully reported to reduce calciuria in patients with CYP24A1 or SLC34A3 mutations, with no safety warnings. Thus, based on these case reports, we hypothesize that fluconazole is effective to decrease and normalize calciuria in patients with hypercalciuria and increased 1,25(OH)2D levels.MethodsThe FLUCOLITH trial is a prospective, interventional, randomized in parallel groups (1:1), placebo-controlled, double blind trial. A total of 60 patients (10-60years) with nephrolithiasis and/or nephrocalcinosis history, hypercalciuria (> 0.1 mmol/kg/d), increased 1,25(OH)2D levels (> 150 pmol/L) and 25-OH-D levels >20 nmol/L, will be included. Inclusions will be performed only from mid-September to the beginning of February to avoid bias due to sunlight-induced vitamin D synthesis. The primary endpoint will be the proportion of patients with normalization of 24-hour calciuria between baseline and 16 weeks, or with a relative decrease of at least 30% of 24-hour calciuria in patients who still display at W16 a 24-hour hypercalciuria. DiscussionThe current challenge is to propose an efficient treatment to patients with hypercalciuria and increased 1,25(OH)2D levels in order to prevent later complications and notably CKD that can ultimately lead to end-stage renal disease. Based on improvement of knowledge in phosphate/calcium metabolism, pathophysiology and genetics, the “off-label” use of fluconazole was recently reported to be useful in hypercalciuric patients with increased 1,25(OH)2D levels. Thus, the FLUCOLITH study is a unique opportunity to develop a new indication of a well-known and not expensive drug in orphan renal diseases, the ultimate objective being the secondary prevention of CKD worsening in these patients. Trial RegistrationClinicalTrial.gov, ID: identifier: NCT04495608. Registered on July 23rd, 2020

scholarly journals A Prospective Drug Utilisation Study and Pharmacoeconomic Analysis in Paediatric Dentistry at a Tertiary Hospital

2020 ◽  
Vol 19 (2) ◽  
pp. 60-64
Author(s):  
Binaya Shrestha ◽  
Megha Pradhan
Keyword(s):  
Drug Utilisation ◽  
Pharmacoeconomic Analysis ◽  
Drug Prescription ◽  
Tertiary Care ◽  
Tertiary Hospital ◽  
Drug Expenditure ◽  
Paediatric Dentistry ◽  
Expensive Drug ◽  
Cross Sectional ◽  
Utilisation Data

Introduction: Paediatric dentistry is the branch of dentistry dealing with children from birth to adolescence. Children tend to suffer from various dental conditions which can be primarily infective in nature and hence requires drugs that can be expensive. Drug utilisation data are needed for developing and monitoring policies because data on drug expenditure are not sufficient. Methods: This was a prospective cross-sectional study conducted in the Department of Pedodontics (paediatric dentistry) of a tertiary care teaching institute in Kathmandu, Nepal from July 2019 to December 2019. During the study period a total of 200 prescriptions were collected. Analysis was conducted using MS excel 2016 and further required analysis was done using Statistical Package for the Social Sciences v22.0. Results: A total of 200 prescriptions were analysed during the study period, of which 126 were males whereas 74 were females. The most common drug prescribed was paracetamol whereas amoxicillin was the most commonly prescribed antibiotics. Most common condition was periapical abscess but space infection was the most expensive condition.There was no statistically significance between the cost of drugs prescribed for the male and female patients. Conclusions: The drug prescription pattern was found to be rational. Every institution must have their own Drugs and Therapeutics Committee as has been suggested by WHO, so that patients will be benefited through decreasing economical burden, and adverse drug reactions and that by this, therapeutic effect will be increased.

Therapeutic vaccination against HIV: current progress and future possibilities

2005 ◽  
Vol 110 (1) ◽  
pp. 59-71 ◽  
Author(s):  
Rebekah L. Puls ◽  
Sean Emery
Keyword(s):  
Antiretroviral Therapy ◽  
Viral Vectors ◽  
Therapeutic Vaccine ◽  
Viral Disease ◽  
Clear Understanding ◽  
Therapeutic Vaccination ◽  
Expensive Drug ◽  
Drug Regimens ◽  
Anti Hiv ◽  
Hiv 1

Although effective in reducing mortality, current antiretroviral therapy for HIV infection involves complex and expensive drug regimens that are toxic and difficult to take. Eradication of HIV reservoirs is not possible with existing therapies. The concept of therapeutic vaccination has been investigated to increase the potency and breadth of anti-HIV immune responses in order to delay or reduce antiretroviral therapy use. A variety of approaches targeted to both cell- and antibody-mediated immunity have been developed, including whole inactivated HIV-1, protein subunits and synthetic peptides, DNA vaccines and a number of viral vectors expressing HIV-1. These investigations have occurred in the absence of a clear understanding of disease pathogenesis or the correlates of protective immunity. At this time, there is no licensed therapeutic vaccine for any viral disease, including HIV; however, this review will consider recent progress in the field and summarize the challenges faced in the development of a therapeutic HIV vaccine.

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