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 How Risky Is That Risk Sharing Agreement? Mean-Variance Tradeoffs and Unintended Consequences of Six Common Risk Sharing Agreements

2021 ◽  
Vol 6 (1) ◽  
pp. 238146832199040
Author(s):  
Gregory S. Zaric
Keyword(s):  
Risk Sharing ◽  
Financial Risk ◽  
Unintended Consequences ◽  
Drug Costs ◽  
New Drugs ◽  
Total Drug ◽  
Number Of Patients ◽  
Clinical Benefits ◽  
The Impact ◽  
Mean Variance

Background. Pharmaceutical risk sharing agreements (RSAs) are commonly used to manage uncertainties in costs and/or clinical benefits when new drugs are added to a formulary. However, existing mathematical models of RSAs ignore the impact of RSAs on clinical and financial risk. Methods. We develop a model in which the number of patients, total drug consumption per patient, and incremental health benefits per patient are uncertain at the time of the introduction of a new drug. We use the model to evaluate the impact of six common RSAs on total drug costs and total net monetary benefit (NMB). Results. We show that, relative to not having an RSA in place, each RSA reduces expected total drug costs and increases expected total NMB. Each RSA also improves two measures of risk by reducing the probability that total drug costs exceed any threshold and reducing the probability of obtaining negative NMB. However, the effects on variance in both NMB and total drug costs are mixed. In some cases, relative to not having an RSA in place, implementing an RSA can increase variability in total drug costs or total NMB. We also show that, for some RSAs, when their parameters are adjusted so that they have the same impact on expected total drug cost, they can be rank-ordered in terms of their impact on variance in drug costs. Conclusions. Although all RSAs reduce expected total drug costs and increase expected total NMB, some RSAs may actually have the undesirable effect of increasing risk. Payers and formulary managers should be aware of these mean-variance tradeoffs and the potentially unintended results of RSAs when designing and negotiating RSAs.

scholarly journals Health Canada’s use of expedited review pathways and therapeutic innovation, 1995–2016: cross-sectional analysis

BMJ Open ◽  
2018 ◽  
Vol 8 (8) ◽  
pp. e023605 ◽  
Author(s):  
Joel Lexchin
Keyword(s):  
Anatomical Therapeutic Chemical ◽  
Cross Sectional Study ◽  
New Drugs ◽  
Cross Sectional ◽  
Time Period ◽  
Therapeutic Value ◽  
Kappa Value ◽  
Innovative Drugs ◽  
Sectional Analysis ◽  
Health Canada

ObjectivesThis study examines the use of expedited approval pathways by Health Canada over the period 1995 to 2016 inclusive and the relationship between the use of these pathways and the therapeutic gain offered by new products.DesignCross-sectional study.Data sourcesTherapeutic Products Directorate, Biologics and Genetic Therapies Directorate, Notice of Compliance database, Notice of Compliance with conditions web site, Patented Medicine Prices Review Board, La revue Prescrire, WHO Anatomical Therapeutic Chemical classification system.Primary and secondary outcomesPercent of new drugs evaluated by Health Canada that went through an expedited pathway between 1995 and 2016 inclusive. Kappa values comparing the review status with assessments of therapeutic value for individual drugs.ResultsOf 623 drugs approved by Health Canada between 1995 and 2016, 438 (70.3%) drugs went through the standard pathway and 185 (29.7%) an expedited pathway. Therapeutic evaluations were available for 509 drugs. Health Canada used an expedited approval pathway for 159 of the 509 drugs, whereas only 55 were judged to be therapeutically innovative. Forty-two of the 55 therapeutically innovative drugs received an expedited review and 13 received a standard review. The Kappa value for the entire period for all 509 drugs was 0.276 (95% CI 0.194 to 0.359) indicating ‘fair’ agreement between Health Canada’s use of expedited pathways and independent evaluations of therapeutic innovation.ConclusionHealth Canada’s use of expedited approvals was stable over the entire time period. It was unable to reliably predict which drugs will offer major therapeutic gains. The findings in this study should provoke a discussion about whether Health Canada should continue to use these pathways and if so how their use can be improved.

Treatment of Advanced Colorectal Cancer (CRC) in Daily Practice: Results of a Survey in two Italian Regions, Piemonte and Valle D'aosta

1998 ◽  
Vol 84 (5) ◽  
pp. 562-566 ◽  
Author(s):  
Alessandro Comandone ◽  
Roberto Berardo ◽  
Roberto Faggiuolo ◽  
Antonella Boglione ◽  
Paola Bergnolo ◽  
...  
Keyword(s):  
Colorectal Cancer ◽  
Performance Status ◽  
Daily Practice ◽  
New Drugs ◽  
Therapeutic Choice ◽  
Italian Regions ◽  
Important Health ◽  
Definition Of ◽  
No Gold Standard

Aims and background Colorectal cancer (CRC) is one of the most important health problems in Western countries: it is the fourth cancer in terms of incidence and the second cause of cancer death. Surgery is the main therapeutic choice and there is broad consensus on the role of adjuvant chemotherapy (CT) after resection. Unfortunately, 50% of the patients will relapse and die of the disease. Palliative CT based on 5-fluorouracil (5FU) may induce a 9-48% response rate with a median survival of 11.5 months. At present there is no gold standard for CT in advanced CRC and the situation has become more complicated since the advent of new drugs (Raltitrexed, Irinotecan, Oxaliplatin). The aim of this study was the identification of the different approaches to treatment of advanced CRC among the clinicians (oncologists, radiologists, internal medicine specialists, surgeons) who practice CT. Methods and study design Forty-six clinicians from two Italian Regions (Piemonte and Valle d'Aosta) were interviewed by telephone. Results 5FU modulated with Lederfolin according to the classic Machover scheme is the main option in daily practice. More sophisticated therapies are reserved to patients with a good performance status (PS) and are prescribed only in the larger centers. The planned therapies usually consist of six courses. Restaging may be performed after three or six courses. A marked difference has been recorded in the evaluation of a situation of no change (NC): 25.5% of the clinicians evaluate stable disease as a positive result. In the event of disease progression or relapse, 35% of the clinicians do not prescribe second-line CT. In case of further treatment, the options are totally subjective. Conclusions A national survey on this issue is necessary under the auspices of AIOM (Associazione Italiana Oncologia Medica) and involving oncologists, epidemiologists and statisticians, in order to define the reasons for variations in therapy in advanced CRC and determine the differences between clinicians of different age, specialization and location. This survey could lead to a definition of guidelines for the treatment of advanced CRC.

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.

scholarly journals How can we improve patients’ access to new drugs under uncertainties? : South Korea’s experience with risk sharing arrangements

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Boram Lee ◽  
Eun-Young Bae ◽  
SeungJin Bae ◽  
Hyun-Jin Choi ◽  
Kyung-Bok Son ◽  
...  
Keyword(s):  
South Korea ◽  
Risk Sharing ◽  
Pharmaceutical Expenditure ◽  
Developed Countries ◽  
Orphan Drugs ◽  
Drug Coverage ◽  
New Drugs ◽  
Current Status ◽  
Time Gap ◽  
The Uk

Abstract Background New drugs including cancer drugs and orphan drugs are becoming increasingly more expensive. Risk sharing arrangements (RSAs) could manage the risk based on both financial impact and the health outcome of new drugs if reimbursed. To improve patients’ access to new drugs under uncertainties, many developed countries have adopted RSAs. In this study, we aimed to understand the effects of RSAs in South Korea on patients’ access. Methods We reviewed current status of RSA drugs in South Korea. The number of appraisals and time gap between market approval and reimbursement per RSA drug were considered to quantify improvement of patients’ access as they showed how rapidly decisions on reimbursement of RSA drugs were derived. Then, we applied a comparative analysis to determine whether the RSA drugs in South Korea were reimbursed in the UK, Italy, and Australia. Most data for this study were obtained from websites of the governmental department/agencies responsible for appraisal of drug reimbursement in each country. And literatures related to RSAs were investigated as well. Results The eligibility for Korean RSAs had two key components - drugs for cancer and rare diseases and not having other alternative treatments. As of the first half of 2019, there were 39 RSA drugs reimbursed in South Korea, the majority of which were financial-based schemes. Refund and expenditure cap were the representative types (89.7%). After introduction of RSAs, the time gap and number of appraisals were decreased. Based on the indications of RSA drugs, the level of drug coverage in South Korea was found lower than Italy, similar to the UK, and higher than Australia. Conclusions RSAs in South Korea significantly enhanced patients’ access to new drugs and led to the alleviation of patients’ out-of-pocket expenses. The drug coverage of South Korea had a level comparable to that of other countries. This study provides implications for countries that have a dual mission of containing pharmaceutical expenditure and improving access to new drugs.

Outcomes Research: Issues of Evidence, Timing and Application

1995 ◽  
Vol 8 (4) ◽  
pp. 178-184
Author(s):  
Joseph D. Jackson
Keyword(s):  
Outcomes Research ◽  
New Drugs ◽  
Value For Money ◽  
Basic Premise ◽  
Research Issues ◽  
Mortality And Morbidity ◽  
Populations At Risk ◽  
Related Quality ◽  
Health Related

We have entered the era of value for money, and it will likely last into the next century. Increasingly, the use of pharmaceuticals will be a function of the value for money they bring to specific populations at risk. As an integral part of this era, the outcomes research movement is pursuing the quest for value; however, the movement is in its infancy. There is a great appetite for systematic outcomes research that would assess unmet clinical need, the novelty of the clinically meaningful outcomes, and the potential for monetary benefit. The basic premise of outcomes research is that choices between alternatives must be made to promote efficiency without compromising quality of care. Although choices must be made, the foundation for these decisions is properly good research evidence. The process of evidence generation is time-intensive, especially in diseases with long episodes of illness. Therefore, it will take time to produce clinically meaningful data on factors such as mortality and morbidity gains, health-related quality of life, productivity enhancements, patient and provider satisfaction and compliance, especially for new drugs. The needs are acute and the promise is great, but the movement is probably more evolutionary than revolutionary. Copyright © 1995 by W.B. Saunders Company

scholarly journals The AIFA time-to-reimbursement: a comparison between the last two committees from 2015 to 2020

2020 ◽  
Vol 7 (1) ◽  
pp. 109-114
Author(s):  
Paola Raimondo ◽  
Giorgio Casilli ◽  
Martina Isernia ◽  
Dario Lidonnici ◽  
Roberto Ravasio ◽  
...  
Keyword(s):  
Time Delay ◽  
Average Duration ◽  
Economic Assessment ◽  
New Drugs ◽  
Economic Conditions ◽  
European Medicines Agency ◽  
Official Journal ◽  
Managed Entry Agreements ◽  
R Process ◽  
Innovative Drugs

Objective. To compare the time-to-reimbursement of the last two committees of the Italian Medicines Agency (AIFA), respectively appointed in 2015 and in 2018. Methods. The analysis was run through a specific internal database created by MA-Provider. The database was populated with information regarding European Medicines Agency (EMA) approved new drugs, including each step of the Italian Price and Reimbursement (P&R) process reported in the monthly outcomes of Technical Scientific Committee (CTS) and Price and Reimbursement Committee (CPR) meetings from September 2015 to April 2020. Results. The 2015 and the 2018 committees have reimbursed respectively 39 and 28 drugs by comparing their initial 19 months of activity. Significant differences have been observed in negotiated economic conditions, in particular an increase in the number of drugs with confidential discount (2018-committee: 96.4% vs 2015-committee: 64.1%; p = 0.003) and a reduction in the application of Managed Entry Agreements (MEAs) (2018-committee: 10.7% vs 2015-committee: 33.3%; p = 0.036). The average duration of the P&R procedure managed by the 2018-committee has increased by 45 days compared to the 2015-committee (287 days vs 242 days; p = 0.071) and this trend of delay is associated to the active scientific/economic assessment phase by CTS and CPR (particularly by the latter) and not to administrative phases (e.g. Official Journal publications). Conclusions. The observed differences between committees may be explained by the higher number of oncological and/or innovative drugs assessed by the 2018-committee (regarding the time delay, probably linked to greater difficulties in finding a win-win agreement able to satisfy both AIFA and Pharmaceutical Company).

scholarly journals How innovative are new drugs launched in the UK? A retrospective study of new drugs listed in the British National Formulary (BNF) 2001–2012

BMJ Open ◽  
2014 ◽  
Vol 4 (10) ◽  
pp. e006235 ◽  
Author(s):  
Derek J Ward ◽  
Angharad Slade ◽  
Tracey Genus ◽  
Orsolina I Martino ◽  
Andrew J Stevens
Keyword(s):  
Unintended Consequences ◽  
New Drugs ◽  
British National Formulary ◽  
Secondary Outcome ◽  
National Formulary ◽  
Biological Drugs ◽  
New Drug ◽  
New Chemical Entities ◽  
The Uk ◽  
Innovative Drugs

ObjectivesInnovative new drugs offer potential benefits to patients, healthcare systems, governments and the pharmaceutical industry. Recent data suggest annual numbers of new drugs launched in the UK have increased in recent years, and we sought to understand whether this represents increasing numbers of highly innovative drugs being made available or the introduction of increasing numbers of drugs with limited additional therapeutic value.Design and settingRetrospective observational study of new drug entries in the British National Formulary (BNF).Primary and secondary outcome measuresNumber of new drugs launched in the UK each year (based on first appearance in the BNF) from 2001 to 2012, including new chemical entities and new biological drugs, categorised by degree of innovativeness according to published criteria that incorporate both clinical usefulness and the nature of the innovation.ResultsHighly innovative, moderately innovative and slightly innovative drugs made up 26%, 18% and 56% of all newly launched drugs, respectively, for the study period (n=290). There was an upward trend in annual numbers of slightly innovative drugs from 2004 onwards (R2=0.44), which aligned closely with the recovery in total numbers of new drugs launched each year since that time. There were no discernible time trends in the highly or moderately innovative categories. New drugs for malignancy and skin disease were most likely to be characterised as highly innovative (44% and 57%, respectively).ConclusionsHighly innovative new drugs comprise only around a quarter of all new drug launches in the UK. In contrast, drugs categorised as only slightly innovative comprised well over half of all new drugs and annual numbers in this category are increasing. Current policy initiatives that seek to increase the supply of innovative new drugs have long-lead times to impact, and will need careful assessment to ensure they deliver their aims without unintended consequences.

Anticancer prescription appropriateness: The APA score—A new method to assess anticancer prescriptions in the clinical setting.

2011 ◽  
Vol 29 (27_suppl) ◽  
pp. 261-261
Author(s):  
V. Launay-Vacher ◽  
O. Aujoulat ◽  
M. Brandely ◽  
R. Chevrier ◽  
L. Dumas ◽  
...  
Keyword(s):  
Anticancer Drugs ◽  
Real Life ◽  
New Drugs ◽  
University Hospitals ◽  
Level Of Evidence ◽  
The People ◽  
Common Process ◽  
Set Up ◽  
Innovative Drugs ◽  
Standardized Screening

261 Background: Innovation is crucial in cancer care and access to innovation cannot be—only—economically ruled. We need new drugs, more effective and/or better tolerated to improve the care of our patients. In this context, we need new tools to evaluate the appropriateness of anticancer drugs prescriptions. In fact, the better the appropriateness, the better is the patient care, and the overall balance, including economically. Methods: A group of 9 pharmacists from university hospitals, general hospitals, and cancer centres joined under the umbrella of the European Fellowship for Pharmacists (EFP) association. The aim was to set up a common process in evaluating the appropriateness of anticancer drugs prescriptions (ADP), focusing on innovative drugs, but not only. Results: The backbone of our work was to put back the patient at the centre and not to focus on the costs as themselves. We defined a composite criterium called “value of an ADP” which included the costs of course in addition to several factors such as the existence of alternatives for a particular patient, the level of evidence from literature data, the existence of other indications, and several other items. First, a decision tree was elaborated, which allows systematic and standardized screening of ADP leading to early identification of ADP which do not necessitate thorough evaluation: prescription within the terms of either a market authorisation or a Temporary Therapeutic Protocol, or a Temporary Utilization Authorization (ATU), and not on a clinical trial. For the other prescriptions, an evaluation grid coupled to a score was set up. This APA Score (Anticancer Prescription Appropriateness) will allow to decide whether or not a ADP, in a particular clinical context and patient, should be judged as appropriate or not. Conclusions: At the time of this abstract: the evaluation grid has been discussed, modified, and validated with the participation of two clinical oncologists, specialized in Breast Cancer; the APA scoring is currently under testing by a team of methodologists and bio-mathematicians; and the grid and the APA Score will be tested in “real-life” within the departments of the people involved in the group by the end of 2011.

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