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

Author(s):  
Paola Raimondo ◽  
Giorgio Casilli ◽  
Martina Isernia ◽  
Dario Lidonnici ◽  
Roberto Ravasio ◽  
...  

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).

ABOUTOPEN ◽  
2020 ◽  
Vol 7 (1) ◽  
pp. 89-94
Author(s):  
Mariangela Prada ◽  
Letizia Rossi ◽  
Matteo Mantovani

Introduction: The main purpose of this study was comparing median time (TTR, time to reimbursement) between the first Agenzia Italiana del Farmaco (AIFA) pricing and reimbursement (P&R) dossier’s evaluation and patient access in Italy and to observe the key P&R negotiation results for all new active substances approved by the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use between January 2014 and December 2019. We analysed the different factors influencing TTR. Methods: A panel of medicines for human use approved by the EMA in the period 2014-2019 was considered. All information about authorisation and reimbursement in Italy, including timelines and results from the negotiation, were gathered through EMA and Italian Official Journal databases. Results: Of 213 new active substances approved from January 2014 to December 2019, 137 obtained reimbursement in Italy, with a median TTR of 7.6 months (228 days). Even if orphan designation, oncology indication, application of Managed Entry Agreements (MEAs; both outcome and financial based) or a discount did not show an impact on TTR, recognition of full innovativeness (n = 27; 20%) was associated with a reduction of 1 month in median TTR. Interestingly, drugs reimbursed with a lower price/daily defined dose showed a reduced TTR (−22%). Conclusions: Even if the lack of impact of some negotiation conditions was predictable (e.g. oncology indication or orphan status) or the application of a MEA helped to manage possible uncertainties, it did not lead to a quicker completion of the negotiation procedure. Likewise, full innovative drugs showed a shorter TTR underlying the AIFA commitment in recognising, promoting and rewarding innovation.


2019 ◽  
Vol 20 (15) ◽  
pp. 3801 ◽  
Author(s):  
Sumimasa Nagai

Several expedited regulatory review projects for innovative drugs and regenerative medical products have been developed in the US, the EU, and Japan. Each regulatory agency has elaborated an original regulatory framework and adopted regulatory projects developed by the other regulatory agencies. For example, the Food and Drug Administration (FDA) first developed the breakthrough therapy designation, and then the Pharmaceuticals and Medical Devices Agency (PMDA) and European Medicines Agency (EMA) introduced the Sakigake designation and the priority medicines (PRIME) designation, respectively. In addition, the necessity of the product being first development in Japan is the original feature of the Sakigake designation, while actively supporting the development of advanced-therapy medicinal products (ATMPs) by academia or small/medium-sized sponsors is the original feature of the PRIME; these particular features are different from the breakthrough therapy designation in the US. In this review article, flexible and expedited review processes for new drugs, and cell and gene therapies in the US, the EU, and Japan are described. Moreover, all the drugs and regenerative medical products that were granted conditional approval or Sakigake designation in Japan are listed and analyzed herein.


2020 ◽  
Vol 36 (S1) ◽  
pp. 37-37
Author(s):  
Americo Cicchetti ◽  
Rossella Di Bidino ◽  
Entela Xoxi ◽  
Irene Luccarini ◽  
Alessia Brigido

IntroductionDifferent value frameworks (VFs) have been proposed in order to translate available evidence on risk-benefit profiles of new treatments into Pricing & Reimbursement (P&R) decisions. However limited evidence is available on the impact of their implementation. It's relevant to distinguish among VFs proposed by scientific societies and providers, which usually are applicable to all treatments, and VFs elaborated by regulatory agencies and health technology assessment (HTA), which focused on specific therapeutic areas. Such heterogeneity in VFs has significant implications in terms of value dimension considered and criteria adopted to define or support a price decision.MethodsA literature research was conducted to identify already proposed or adopted VF for onco-hematology treatments. Both scientific and grey literature were investigated. Then, an ad hoc data collection was conducted for multiple myeloma; breast, prostate and urothelial cancer; and Non Small Cell Lung Cancer (NSCLC) therapies. Pharmaceutical products authorized by European Medicines Agency from January 2014 till December 2019 were identified. Primary sources of data were European Public Assessment Reports and P&R decision taken by the Italian Medicines Agency (AIFA) till September 2019.ResultsThe analysis allowed to define a taxonomy to distinguish categories of VF relevant to onco-hematological treatments. We identified the “real-world” VF that emerged given past P&R decisions taken at the Italian level. Data was collected both for clinical and economical outcomes/indicators, as well as decisions taken on innovativeness of therapies. Relevant differences emerge between the real world value framework and the one that should be applied given the normative framework of the Italian Health System.ConclusionsThe value framework that emerged from the analysis addressed issues of specific aspects of onco-hematological treatments which emerged during an ad hoc analysis conducted on treatment authorized in the last 5 years. The perspective adopted to elaborate the VF was the one of an HTA agency responsible for P&R decisions at a national level. Furthermore, comparing a real-world value framework with the one based on the general criteria defined by the national legislation, our analysis allowed identification of the most critical point of the current national P&R process in terms ofsustainability of current and future therapies as advance therapies and agnostic-tumor therapies.


Author(s):  
Virginia Ronco ◽  
Myriam Dilecce ◽  
Elena Lanati ◽  
Pier Luigi Canonico ◽  
Claudio Jommi

Abstract Background Advanced therapy medicinal products (ATMPs) represent an important cornerstone for innovation in healthcare. However, uncertainty on the value, the high average cost per patient and their one-shot nature has raised a debate on their assessment and appraisal process for pricing and reimbursement (P&R) purposes. This debate led experts providing for recommendations on this topic. Our primary objective is to investigate the ATMPs P&R process in the main five European countries and to understand if this process is consistent with published P&R expert recommendations. We also investigated the current ATMP pipelines to understand if future ATMPs will create challenges for their P&R process. Methods P&R framework for ATMPs in the European Major five (EU5) countries was investigated through a literature search on PubMed, institutional websites of National Health Authorities and grey literature. The ATMPs pipeline database was populated from a clinical trial database (clinicaltrials.gov), relying on inclusion and exclusion criteria retrieved from the literature. Results Reimbursement status of ATMPs is different across the EU5 countries, with the exception of CAR-Ts which are reimbursed in all countries. Standard P&R process in place for other medicinal products is extended to ATMPs, with the exception of some cases in Germany. List prices, where available, are high and, tend to be aligned across countries. Outcome-based Managed Entry Agreements (MEAs) have been extensively used for ATMPs. Extra-funds for hospitals managing ATMPs were provided only in Germany and, as additional fund per episode, in France. The accreditation process of hospitals for ATMPs management was in most countries managed by the national authorities. As far as ATMPs pipeline is concerned, ATMPs in development are mostly targeting non-rare diseases. Conclusions Expert recommendations for ATMPs P&R were partially applied: the role of outcome-based MEAs has increased and the selection process of the centres authorized to use these treatments has been enhanced; additional funding for ATMPs management to accredited centres has not been completely considered and annuity payment and broader perspective in cost considerations are far from being put in place. These recommendations should be considered for future P&R negotiations to pursue rational resource allocation and deal with budget constraints.


2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 1425.1-1425
Author(s):  
P. Herrera-Sandate ◽  
G. Figueroa-Parra ◽  
D. Vega-Morales ◽  
J. A. Esquivel Valerio ◽  
B. R. Vázquez Fuentes ◽  
...  

Background:Early referral of patients with suspicion of progression to rheumatoid arthritis (RA) is of paramount importance in disease prognosis. We had previously described a time delay of 28 months between symptom onset and evaluation by a rheumatologist, and a mean wait time of 9.5 weeks for referral to a secondary-level public hospital (1). The availability of specialized interdisciplinary evaluation of patients in a third-level of care raises the possibility of shortening this time gap, as well as describing patient and physician decisions amidst the referral to a Rheumatology center.Objectives:Describe the diagnosis profile of patients with hand arthralgia and time of referral to Rheumatology in a Family Medicine clinic.Methods:A cohort study was conducted in 110 patients from October 2018 to December 2020 in a Family Medicine clinic within the tertiary-care University Hospital “Dr. Jose Eleuterio Gonzalez” in Monterrey, Mexico. Patients with hand arthralgia as their chief complaint were recruited. An observational, descriptive compilation of patient history was retrieved prospectively through medical records. Variables included time of inclusion, number of medical visits until referral and definitive diagnosis. Descriptive statistics, Kaplan-Meier curves and log-rank tests were used to test the association between time of diagnosis and clinical variables of interest.Results:Assessed variables are shown in Table 1. Out of 110 patients with hand arthralgia, a quarter received a final diagnosis within 3 medical visits. Less than half of patients were referred, and only a third attended the referral indication. It takes 39.3 days from the first medical visit to be referred, and 69 days and 2.89 consultations to receive a definitive diagnosis. Around half of patients will have a definitive diagnosis, osteoarthritis being the most common. The log-rank test for categoric variables including a positive squeeze test or ≥4 criteria of clinically suspect arthralgia did not show a significant association for time of referral and definitive diagnosis (data not shown).Table 1.Diagnostic and referral characteristics of patients with hand arthralgia attending a Family medicine clinicPatients recruited in a Family Medicine clinicn = 110Female, n (%)90 (81.8)Age in years, mean ± SD49.69 ± 14.90RF, ACPA, or hand radiography request, n (%)100 (90.9)Diagnosis in Family MedicineDiagnosed patients after 1 medical visit, cumulative n (%)5 (4.6)Diagnosed patients after 2 medical visits, cumulative n (%)22 (20.0)Diagnosed patients after 3 medical visits, cumulative n (%)26 (23.6)Referral to Rheumatology for diagnostic doubt or clinical follow-upPatients referred to a Rheumatology clinic, n (%)49 (44.5)Patients attending Rheumatology referral, n (%)34 (30.9)Time for referral, days ± SD39.37 ± 38.64Global definitive diagnosisPatients with a definitive diagnosis, n (%)51 (46.4)Osteoarthritis diagnosis, n (%)23 (20.9)Rheumatoid arthritis diagnosis, n (%)13 (11.8)Overlap syndrome diagnosis, n (%)5 (4.5)Time for definitive diagnosis, days ± SD68.96 ± 106.57Number of consultations for definitive diagnosis, mean ± SD2.86 ± 1.05RF, rheumatoid factor; ACPA, anticitrullinated protein antibodies; SD, standard deviation.Conclusion:Patients with hand arthralgia evaluated in a tertiary-care Rheumatology center receive a timely referral in one month and a definitive diagnosis after 3 medical visits in around two months.References:[1]Vega-Morales, D., Covarrubias-Castañeda, Y., Arana-Guajardo, A. C., & Esquivel-Valerio, J. A. (2016). Time Delay to Rheumatology Consultation: Rheumatoid Arthritis Diagnostic Concordance Between Primary Care Physician and Rheumatologist. American journal of medical quality: the official journal of the American College of Medical Quality, 31(6), 603.Graphs:Disclosure of Interests:None declared


2012 ◽  
Vol 7 (1) ◽  
pp. 35-38
Author(s):  
Mohammad Salman ◽  
Syed Allahsan ◽  
Manzoor Mahmood ◽  
Md Khairul Anam ◽  
Shahed Mohammad Anwar ◽  
...  

Acute heart failure is a major health problem responsible for several million hospitalizations worldwide each year. Standard therapy has not changed for long time and includes diuretics and variable use of vasodilators or inotropes. Recently Nesiritide and Levosimendan are two drugs for the treatment of acute heart failure which have been approved by the Food and Drug Administration (FDA) and European Medicines Agency (EMEA), respectively. There was little concern that Nesiritide can worsen the renal failure but recent trials had abolished this concern. DOI: http://dx.doi.org/10.3329/uhj.v7i1.10208 UHJ 2011; 7(1): 35-38


BMJ Open ◽  
2018 ◽  
Vol 8 (8) ◽  
pp. e023605 ◽  
Author(s):  
Joel Lexchin

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.


2019 ◽  
Vol 22 ◽  
pp. S716
Author(s):  
A. Enrici ◽  
D. Lidonnici ◽  
E. Lanati
Keyword(s):  

2017 ◽  
Vol 33 (S1) ◽  
pp. 111-111
Author(s):  
Andrea Brígida de Souza ◽  
Avila Vidal ◽  
Pollyanna Gomes ◽  
Vania Canuto ◽  
Clarice Petramale

INTRODUCTION:In Brazil, the pharmaceutical sector has requested an individual incorporation in the Brazilian public health system (SUS) for each new drug for multiple sclerosis that receives sanitary authorization for marketing. Horizon Scanning within Brazilian Ministry of Health has played a key role in the recommendations made by the National Committee for Health Technology Incorporation (CONITEC). Horizon Scanning seeks to predict which technologies have potential to impact health care in SUS, before their formal request. This study aims to present the impact of horizon scanning in two assessments made by CONITEC on drugs to treat Multiple Sclerosis.METHODS:Grey literature was searched to find new and emerging drugs for multiple sclerosis treatment. Regulatory agencies were also searched: European Medicines Agency (EMA), Food and Drug Administration (FDA) and Brazilian Regulation and Health Surveillance Agency (Anvisa). A pre-defined standardized form was used. Information extracted about each drug was identified as: drugs name, mechanism of action, indication, administration route, finished phases of clinical trial and registration in other countries.RESULTS:In 2014, horizon scanning identified seven drugs while CONITEC was assessing Fingolimod for multiple sclerosis. In this case, the drug's administration route was a differential, as only three new drugs identified were also orally administrated. Thus, Fingolimod received a positive recommendation for incorporation. In 2016, horizon scanning identified fourteen drugs while Teriflunomide was under assessment. At this moment, the orally administrated Fingolimod was already available and it was identified other eight new drugs with the same route. Therefore, the initial recommendation was against its incorporation.CONCLUSIONS:Horizon scanning has proved to be of major importance for assisting recommendation-making process of the committee. In the two cases presented, horizon scanning information could predict which technologies were being developed and could be registered in Brazil. These new technologies had influenced the recommendations made by CONITEC's members. As a result, a horizon scanning section in all CONITEC's reports became mandatory.


BMJ Open ◽  
2019 ◽  
Vol 9 (5) ◽  
pp. e027625 ◽  
Author(s):  
Derek J Ward ◽  
Lucy Doos ◽  
Andrew Stevens

ObjectivesTo investigate the trend in the launch price of new drugs for five common health conditions.DesignCross-sectional study using data on new drugs launched in the UK between 1981 and 2015 for hypertension, asthma, rheumatoid arthritis, schizophrenia and colorectal cancer.Data and sourcesAll drugs marketed in the UK between 1981 and 2015 (inclusive), and licensed specifically for the treatment of one of the five chosen conditions were included in the study. Newly launched medicines and their launch prices were identified by hand-searching all editions of the British National Formulary in addition to searching the websites of relevant regulatory agencies (European Medicines Agency and Medicines and Healthcare products Regulatory Agency). The launch price in UK pounds for a 28-day supply of each medicine at a typical or usual maintenance dose was adjusted for the effects of general inflation using the gross domestic product deflator series.Results104 drugs were included in our study with a mean inflation-adjusted 28-day launch price of £288 (SD £678). The launch price of new drugs varied significantly across the five conditions, with drugs for hypertension having the lowest mean price (£27) and drugs for colorectal cancer having the highest mean price (£1590) (p<0.001). There were large increases in launch prices across the study period, but the magnitude and pattern was markedly different between therapeutic areas. Biological drugs represented 13.5% of all included drugs and had a significantly higher launch price than non- biological drugs (£1233 vs £141, p<0.001). 22.1% of included drugs were first-of-kind and had a significantly higher launch price than follow-on drugs (£768 vs £151) (p<0.0001).ConclusionDrugs prices continue to increase across different therapeutic areas. This has some association with novelty, but, it is not clear if this increase in price is associated with medical benefits.


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