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

2014 ◽  
Vol 20 (10) ◽  
pp. 1282-1287 ◽  
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.

PP086 Horizon Scanning In Multiple Sclerosis Decisions In Brazil

2017 ◽  
Vol 33 (S1) ◽  
pp. 111-111
Author(s):  
Andrea Brígida de Souza ◽  
Avila Vidal ◽  
Pollyanna Gomes ◽  
Vania Canuto ◽  
Clarice Petramale
Keyword(s):  
Multiple Sclerosis ◽  
New Technologies ◽  
Grey Literature ◽  
New Drugs ◽  
European Medicines Agency ◽  
National Committee ◽  
Administration Route ◽  
Horizon Scanning ◽  
The Impact ◽  
Multiple Sclerosis Treatment

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.

Preapproval and postapproval evidence on drugs for multiple sclerosis

Neurology ◽  
2018 ◽  
Vol 90 (21) ◽  
pp. 964-973 ◽  
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.

Paternalism and Public Health

2017 ◽  
Author(s):  
Jessica Flanigan
Keyword(s):  
Public Health ◽  
Public Policy ◽  
Drug Development ◽  
New Drugs ◽  
Health Policies ◽  
Clinical Context ◽  
Public Health Policies ◽  
Patients Rights ◽  
Medical Paternalism ◽  
New Drug Development

Since medical paternalism is wrong in the clinical context, it should be rejected in public policy as well. But even if paternalistic public health policies were permissible, it is not clear that prohibitive pharmaceutical regulations are necessary to promote public health. Prohibitions could undermine health in some cases, for example, if prescription requirements make patients more deferential to physicians and tolerant of medical risks. Premarket testing requirements cause people to suffer and die waiting for new drugs to get approved, and they discourage new drug development. This is not to say that regulation serves no purpose. Pharmaceutical regulators provide a valuable pubic good by overseeing testing for new drugs and by certifying drugs that they deem generally safe and effective. But the benefits of regulation do not require that the regulations be prohibitive, and prohibitive regulations not only violate patients’ rights, they may also cost lives.

The Effect of Price on Pharmaceutical R&D

2009 ◽  
Vol 9 (1) ◽  
Author(s):  
Abdulkadir Civan ◽  
Michael T. Maloney
Keyword(s):  
United States ◽  
Drug Development ◽  
Price Elasticity ◽  
The United States ◽  
Mortality Rates ◽  
New Drugs ◽  
New Drug ◽  
Development Pipeline ◽  
New Drug Development

Abstract This work extends prior research that finds drug development is driven by demand factors such as mortality rates of the diseases new drugs are aimed at. Here we find that the number of drugs in the development pipeline is strongly positively related to the price of existing drugs treating those diseases. This gives us a direct price elasticity measure from which we can draw some inference about the effect on new drug development that might occur if the pricing regime in the United States were to change.

scholarly journals Strategies and Techniques of Drug Discovery from Natural Products

2017 ◽  
Vol II (I) ◽  
pp. 34-43
Author(s):  
Parniya Akbar Ali ◽  
Farah Hanif ◽  
Hosna Nettour ◽  
Mubashar Rehman
Keyword(s):  
Natural Products ◽  
Drug Discovery ◽  
Drug Development ◽  
Positive Impact ◽  
New Drugs ◽  
Active Materials ◽  
Natural Sources ◽  
Innovative Strategies ◽  
Therapeutic Properties ◽  
New Drug Development

New drugs are mostly obtained from Natural sources. The traditional and ethic medicines have provided evidence on the therapeutic properties and resulted in some distinguished drug discovery of natural products. The microorganisms and the endogenous active materials from human or animal have also become a significant approach to the discovery of a drug. Bioinformatics and artificial intelligence have facilitated the study and development of products. For discovery of natural products different software have been used. Different computational software needed in the future for the predicting features in new drug development, for instance pharmacokinetic and pharmacodynamics, in drug development lead positive impact. This review focus on natural product drug discovery and uses innovative strategies and techniques as a part of discovery of drugs from natural products.

scholarly journals Antimicrobial Resistance Surveillance and New Drug Development

2019 ◽  
Vol 6 (Supplement_1) ◽  
pp. S5-S13 ◽  
Author(s):  
Helio S Sader ◽  
Paul R Rhomberg ◽  
Andrew S Fuhrmeister ◽  
Rodrigo E Mendes ◽  
Robert K Flamm ◽  
...  
Keyword(s):  
Public Health ◽  
Health Care ◽  
Antimicrobial Resistance ◽  
Drug Development ◽  
Antimicrobial Agents ◽  
Regulatory Agencies ◽  
European Medicines Agency ◽  
New Drug ◽  
Antimicrobial Resistance Surveillance ◽  
New Drug Development

Abstract Surveillance represents an important informational tool for planning actions to monitor emerging antimicrobial resistance. Antimicrobial resistance surveillance (ARS) programs may have many different designs and can be grouped in 2 major categories based on their main objectives: (1) public health ARS programs and (2) industry-sponsored/product-oriented ARS programs. In general, public health ARS programs predominantly focus on health care and infection control, whereas industry ARS programs focus on an investigational or recently approved molecule(s). We reviewed the main characteristics of industry ARS programs and how these programs contribute to new drug development. Industry ARS programs are generally performed to comply with requirements from regulatory agencies responsible for commercial approval of antimicrobial agents, such as the US Food and Drug Administration, European Medicines Agency, and others. In contrast to public health ARS programs, which typically collect health care and diverse clinical data, industry ARS programs frequently collect the pathogens and perform the testing in a central laboratory setting. Global ARS programs with centralized testing play an important role in new antibacterial and antifungal drug development by providing information on the emergence and dissemination of resistant organisms, clones, and resistance determinants. Organisms collected by large ARS programs are extremely valuable to evaluate the potential of new agents and to calibrate susceptibility tests once a drug is approved for clinical use. These programs also can provide early evaluations of spectrum of activity and postmarketing trends required by regulatory agencies, and the programs may help drug companies to select appropriate dosing regimens and the appropriate geographic regions in which to perform clinical trials. Furthermore, these surveillance programs provide useful information on the potency and spectrum of new antimicrobial agents against indications and organisms in which clinicians have little or no experience. In summary, large ARS programs, such as the SENTRY Antimicrobial Surveillance Program, contribute key data for new drug development.

scholarly journals Flexible and Expedited Regulatory Review Processes for Innovative Medicines and Regenerative Medical Products in the US, the EU, and Japan

2019 ◽  
Vol 20 (15) ◽  
pp. 3801 ◽  
Author(s):  
Sumimasa Nagai
Keyword(s):  
New Drugs ◽  
European Medicines Agency ◽  
Regulatory Review ◽  
Medical Products ◽  
Gene Therapies ◽  
Advanced Therapy ◽  
The Us ◽  
Innovative Drugs ◽  
Original Feature ◽  
The Eu

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.

scholarly journals Scorpion Peptides: Potential Use for New Drug Development

2013 ◽  
Vol 2013 ◽  
pp. 1-15 ◽  
Author(s):  
BenNasr Hmed ◽  
Hammami Turky Serria ◽  
Zeghal Khaled Mounir
Keyword(s):  
Drug Development ◽  
Functional Diversity ◽  
Biological Activities ◽  
New Drugs ◽  
Biological Mechanisms ◽  
Neoplastic Diseases ◽  
New Drug ◽  
New Drug Development ◽  
Potential Use

Several peptides contained in scorpion fluids showed diverse array of biological activities with high specificities to their targeted sites. Many investigations outlined their potent effects against microbes and showed their potential to modulate various biological mechanisms that are involved in immune, nervous, cardiovascular, and neoplastic diseases. Because of their important structural and functional diversity, it is projected that scorpion-derived peptides could be used to develop new specific drugs. This review summarizes relevant findings improving their use as valuable tools for new drugs development.

Novel GABAA Agonist Entities: Pharmacological Investigation and Molecular Modeling Study of Thiazolo- and Thiadiazolo-[3,2-a][1,3] diazepine Analogs

2020 ◽  
Vol 21 ◽  
Author(s):  
Hussein I. El-Subbagh
Keyword(s):  
Molecular Modeling ◽  
Drug Development ◽  
Biological Evaluation ◽  
Neuromuscular Blocking ◽  
Present Review ◽  
Pharmacological Investigation ◽  
Important Class ◽  
Pharmacological Activities ◽  
Short Acting ◽  
New Drug Development

Abstract:: Thiazolo- and thiadiazolo-[3,2-a][1,3]diazepines and their patented derivatives, tested with diverse CNS pharmacological activities, constitute an important class of compounds for new drug development. Therefore, research efforts were continued to design, synthesize, and evaluate compounds for their ultra-short, short-acting hypnotic, anticonvulsant, and neuromuscular blocking activities. The present review provides a summary of the work accomplished by these heterocycles and their biological evaluation.

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