PP086 Horizon Scanning In Multiple Sclerosis Decisions In Brazil

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.

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PP469 From Theory To Practice: Which Value Framework Is Applied For Onco-Hematology Therapies In Italy? A 5-year Retrospective Analysis

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462320001816 ◽

2020 ◽

Vol 36 (S1) ◽

pp. 37-37

Author(s):

Americo Cicchetti ◽

Rossella Di Bidino ◽

Entela Xoxi ◽

Irene Luccarini ◽

Alessia Brigido

Keyword(s):

Real World ◽

Ad Hoc ◽

Grey Literature ◽

National Level ◽

Pharmaceutical Products ◽

European Medicines Agency ◽

The Real ◽

R Process ◽

The One ◽

The Impact

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.

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Positive impact of cladribine on quality of life in people with relapsing multiple sclerosis

Multiple Sclerosis Journal ◽

10.1177/1352458517726380 ◽

2017 ◽

Vol 24 (11) ◽

pp. 1461-1468 ◽

Cited By ~ 6

Author(s):

Dayo Afolabi ◽

Christo Albor ◽

Lukasz Zalewski ◽

Dan R Altmann ◽

David Baker ◽

...

Keyword(s):

Quality Of Life ◽

Multiple Sclerosis ◽

Positive Impact ◽

Rank Correlation ◽

Phase Iii ◽

European Medicines Agency ◽

Total N ◽

The Impact ◽

Relapsing Multiple Sclerosis

Background: A number of elements of the pivotal ‘cladribine tablets treating multiple sclerosis orally’ (CLARITY) trial have remained unpublished. Objective: To report the impact of cladribine on health-related quality of life (QoL) in people with relapsing multiple sclerosis (pwRMS). Methods: QoL data from the phase III trial of two different doses (3.5 and 5.25 mg/kg) of oral cladribine in pwRMS were acquired from the European Medicines Agency through Freedom of Information. Spearman’s rank correlation was used to analyse the relationship between baseline QoL scores and baseline Expanded Disability Status Scale (EDSS) scores. Responses of the Euro Quality of Life 5 Dimensions (EQ-5D) and Multiple Sclerosis Quality of Life-54 (MSQOL-54) questionnaires were compared between treatment and control groups using univariate analyses of covariance. Results: In total, n = 5148 EQ-5D responses and n = 894 MSQOL-54 physical, mental health and dimension scores were extracted. Baseline EQ-5D indices correlated with EDSS scores. After 2 years, pwRMS taking 3.5 ( p = .001) and 5.25 mg/kg ( p = .022) reported significantly improved EQ-5D index scores compared with placebo. Positive, yet non-significant, differences were detected in MSQOL-54 scores between cladribine and placebo. Conclusion: Analysis of the CLARITY dataset suggests that, over and above its established clinical efficacy, cladribine leads to improved QoL over 96 weeks. ClinicalTrials.gov identifier: NCT00213135.

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Preapproval and postapproval evidence on drugs for multiple sclerosis

Neurology ◽

10.1212/wnl.0000000000005561 ◽

2018 ◽

Vol 90 (21) ◽

pp. 964-973 ◽

Cited By ~ 8

Author(s):

Chiara Gerardi ◽

Vittorio Bertele' ◽

Silvia Rossi ◽

Silvio Garattini ◽

Rita Banzi

Keyword(s):

Multiple Sclerosis ◽

Clinical Effectiveness ◽

Dimethyl Fumarate ◽

New Drugs ◽

Cochrane Library ◽

European Medicines Agency ◽

The European Union ◽

Disability Progression ◽

Disease Modifying Drugs ◽

Approved Drugs

ObjectiveTo review the evidence supporting the European Union marketing authorization of drugs for multiple sclerosis (MS) and assess how far postmarketing research addresses information gaps at the time of approval.MethodsThrough its database, we identified drugs approved by the European Medicines Agency and gathered data on pivotal trials from the European Public Assessment Reports and corresponding publications. We searched Medline, Embase, Cochrane Library, and trial registries for postmarketing randomized controlled trials testing the drugs identified in any form of the disease.ResultsSince approval of interferon and glatiramer up to 2017, the Agency has examined 10 drugs for the treatment of MS, and 8 were included in this study: alemtuzumab, daclizumab, dimethyl fumarate, fampridine, fingolimod, peginterferon-β-1a, natalizumab, and teriflunomide. We analyzed 16 pivotal trials enrolling almost 16,000 participants. Eleven compared new drugs to placebo, 5 to interferon-β-1a. Annualized relapse rate was the primary outcome in two-thirds and coprimary with disability progression in the 2 studies of alemtuzumab. Of the 52 postmarketing trials, 24 reported final results and 28 were ongoing, terminated, or completed but no results were available. None directly compared the approved drugs, thus leaving their respective therapeutic values unknown. Data on the prevention of disease progression were scarce: none of the disease-modifying drugs showed any effect on disability progression.ConclusionThe lack of comparative evidence and data on clinical effectiveness hamper the assessment of therapeutic value and place in therapy of drugs approved for MS.

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Addressing the regulatory and scientific challenges in multiple sclerosis – a statement from the EU regulators

Multiple Sclerosis Journal ◽

10.1177/1352458514546876 ◽

2014 ◽

Vol 20 (10) ◽

pp. 1282-1287 ◽

Cited By ~ 6

Author(s):

Pavel Balabanov ◽

Manuel Haas ◽

Andre Elferink ◽

Serge Bakchine ◽

Karl Broich

Keyword(s):

Multiple Sclerosis ◽

Drug Development ◽

Guideline Development ◽

New Drugs ◽

European Medicines Agency ◽

Guidance Document ◽

Flexible Approach ◽

Official Position ◽

New Drug Development ◽

The Eu

Improving and facilitating the process of making new drugs available to patients with multiple sclerosis (MS) requires cooperation among the regulators and other stakeholders. This cooperation will also positively contribute towards developing guidelines of the highest quality in medical, regulatory and scientific aspects. This would be beneficial both in areas that require further guideline development, but also in fields where existing guidance should be adapted to take into account evolution in science. Considering the input from all stakeholders, the European Medicines Agency confirmed its intention to update the relevant guideline and apply a flexible approach towards new drug development strategies in MS. This article is the first official position from the EU regulators, presenting the main changes to be expected in the guidance document.

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Chagas Disease Drug Discovery

CrossRef Listing of Deleted DOIs ◽

10.1177/1087057114550585 ◽

2014 ◽

Vol 20 (1) ◽

pp. 22-35 ◽

Cited By ~ 152

Author(s):

Eric Chatelain

Keyword(s):

Chagas Disease ◽

New Technologies ◽

Low Cost ◽

Screening Strategy ◽

New Drugs ◽

In Vivo Models ◽

The Right ◽

The Impact

American trypanosomiasis, or Chagas disease, is the result of infection by the Trypanosoma cruzi parasite. Endemic in Latin America where it is the major cause of death from cardiomyopathy, the impact of the disease is reaching global proportions through migrating populations. New drugs that are safe, efficacious, low cost, and adapted to the field are critically needed. Over the past five years, there has been increased interest in the disease and a surge in activities within various organizations. However, recent clinical trials with azoles, specifically posaconazole and the ravuconazole prodrug E1224, were disappointing, with treatment failure in Chagas patients reaching 70% to 90%, as opposed to 6% to 30% failure for benznidazole-treated patients. The lack of translation from in vitro and in vivo models to the clinic observed for the azoles raises several questions. There is a scientific requirement to review and challenge whether we are indeed using the right tools and decision-making processes to progress compounds forward for the treatment of this disease. New developments in the Chagas field, including new technologies and tools now available, will be discussed, and a redesign of the current screening strategy during the discovery process is proposed.

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The use of disease-modifying new drugs for multiple sclerosis treatment in private-sector health plans

Clinical Therapeutics ◽

10.1016/s0149-2918(04)80225-x ◽

2004 ◽

Vol 26 (8) ◽

pp. 1341-1354 ◽

Cited By ~ 15

Author(s):

Ronald J. Ozminkowski ◽

William D. Marder ◽

Kevin Hawkins ◽

Shaohung Wang ◽

Sarah C. Stallings ◽

...

Keyword(s):

Multiple Sclerosis ◽

Private Sector ◽

New Drugs ◽

Health Plans ◽

Disease Modifying ◽

Multiple Sclerosis Treatment

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Striking a balance between accuracy and timeliness in HTA: an elusive task

European Journal of Public Health ◽

10.1093/eurpub/ckz185.215 ◽

2019 ◽

Vol 29 (Supplement_4) ◽

Author(s):

E Petelos

Keyword(s):

Decision Making ◽

New Technologies ◽

Evidence Synthesis ◽

Complex Interventions ◽

Early Warning Systems ◽

High Quality ◽

Horizon Scanning ◽

Timely Access ◽

Obsolete Technology ◽

The Impact

Abstract Issue HTA is a decision-making tool. Users need high-quality and timely evidence to ensure sound resource allocation and timely access to innovation. High-quality HTA recommendations are time-consuming to generate, requiring high-quality evidence and timely evidence synthesis, as well as mechanisms to ensure timely recommendation release and feasible implementation of guidance. Description of the problem HTA submissions, issuing recommendations and implementation guidance oftentimes have no transparent timelines, with high accuracy posing unique challenges for timeliness. We can broadly examine such challenges in the context of methodologies and processes. Accuracy ensures longer-term validity of decision-making, however, HTA should not be used only for new technologies but also revision of previous decisions through timely re-assessment, ensuring obsolete technology disinvestment. Effects/changes Through the exploration of models and tools previously implemented across jurisdictions and settings, such as early-warning systems, conditional approvals and horizon scanning, it will be demonstrated that well-defined prioritisation criteria accepted by both doers and users, adequate capacity and sound preparation of stakeholders are all key elements to ensure the highest possible degree of timeliness and accuracy, and to strike a balance between the two. Particular attention will be paid to critical challenges for public health access and delivery, i.e., emerging technologies and complex interventions, highlighting how horizon scanning ought to start at the discovery, rather than development phase and transparency challenges. Technology tracking and mechanisms for standardizing methodologies and processes will also be discussed. Lessons Monitoring and evaluating the impact of HTA methodologies and processes in a transparent manner needs to become an inherent part of HTA to ensure existing and future challenges of accuracy and timeliness can be adequately addressed.

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The changing multiple sclerosis treatment landscape: impact of new drugs and treatment recommendations

European Journal of Clinical Pharmacology ◽

10.1007/s00228-018-2429-1 ◽

2018 ◽

Vol 74 (5) ◽

pp. 663-670 ◽

Cited By ~ 8

Author(s):

Irene Eriksson ◽

Joris Komen ◽

Fredrik Piehl ◽

Rickard E. Malmström ◽

Björn Wettermark ◽

...

Keyword(s):

Multiple Sclerosis ◽

Treatment Recommendations ◽

New Drugs ◽

Multiple Sclerosis Treatment

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Managing Multiple Sclerosis: Treatment Initiation, Modification, and Sequencing

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques ◽

10.1017/cjn.2018.17 ◽

2018 ◽

Vol 45 (5) ◽

pp. 489-503 ◽

Cited By ~ 17

Author(s):

Mark S. Freedman ◽

Daniel Selchen ◽

Alexandre Prat ◽

Paul S. Giacomini

Keyword(s):

Multiple Sclerosis ◽

Treatment Initiation ◽

Combination Therapies ◽

Therapeutic Approaches ◽

Optimal Outcomes ◽

Short And Long Term ◽

The Impact ◽

Multiple Sclerosis Treatment ◽

Selection Of

AbstractRecent therapeutic advances in the management of multiple sclerosis (MS) have raised questions about the selection of appropriate patient candidates for various treatments and, if the plan is to move from one treatment to another, the appropriate sequencing of these therapies. The selected approach should provide optimal disease management without limiting future therapeutic options based on safety concerns, and recognize potential future treatments and the possibility of combination therapies. Additional challenges include incorporation of patient needs and preferences into the overall therapeutic approach, in order to ensure optimal outcomes in the short and long term. The objective of this manuscript is to provide an overview of what is currently known regarding the impact of various therapies for MS on future therapeutic choices (sequencing). In this context, we reviewed the available evidence in support of various treatments and, based on the presence of disease activity, suggested a scheme for switching or escalating therapy with the main focus on sequencing of therapeutic approaches.

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