European Union centralised procedure for marketing authorisation of oncology drugs: An in-depth review of its efficiency

Netzer, Tilo

doi:10.1016/j.ejca.2005.04.045

Cited by 12 publications

(6 citation statements)

References 5 publications

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“…The EMA reviews the submission, asks questions to the company as a part of the process, and then issues a decision that is binding throughout the EU. 6 In the USA, manufacturers submit a New Drug Application (NDA) to the Food and Drug Administration (FDA) which will review the data submitted before making a decision on the request. 7 …”

Section: Introductionmentioning

confidence: 99%

Regulatory approval of pharmaceuticals without a randomised controlled study: analysis of EMA and FDA approvals 1999–2014

et al. 2016

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IntroductionThe efficacy of pharmaceuticals is most often demonstrated by randomised controlled trials (RCTs); however, in some cases, regulatory applications lack RCT evidence.ObjectiveTo investigate the number and type of these approvals over the past 15 years by the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA).MethodsDrug approval data were downloaded from the EMA website and the ‘Drugs@FDA’ database for all decisions on pharmaceuticals published from 1 January 1999 to 8 May 2014. The details of eligible applications were extracted, including the therapeutic area, type of approval and review period.ResultsOver the period of the study, 76 unique indications were granted without RCT results (44 by the EMA and 60 by the FDA), demonstrating that a substantial number of treatments reach the market without undergoing an RCT. The majority was for haematological malignancies (34), with the next most common areas being oncology (15) and metabolic conditions (15). Of the applications made to both agencies with a comparable data package, the FDA granted more approvals (43/44 vs 35/44) and took less time to review products (8.7 vs 15.5 months). Products reached the market first in the USA in 30 of 34 cases (mean 13.1 months) due to companies making FDA submission before EMA submissions and faster FDA review time.DiscussionDespite the frequency with which approvals are granted without RCT results, there is no systematic monitoring of such treatments to confirm their effectiveness or consistency regarding when this form of evidence is appropriate. We recommend a more open debate on the role of marketing authorisations granted without RCT results, and the development of guidelines on what constitutes an acceptable data package for regulators.

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Section: Introductionmentioning

confidence: 99%

Regulatory approval of pharmaceuticals without a randomised controlled study: analysis of EMA and FDA approvals 1999–2014

et al. 2016

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“…Nevertheless, these improvements in treatment come at a cost with some of these treatments ranging from tens of thousands (16) to more than a million dollars per patient per year of treatment (15). Drug costs account for around 15% of the total cost of oncologic patients (17), yet a European study evaluated that this cost has risen over time, from €4.3 to €26.3 per capita. This figure is slightly higher than the increase in the overall cost of cancer care (18).…”

Section: New Treatments New Costsmentioning

confidence: 99%

Access to Oncology Drugs in Brazil: Juggling Innovation and Sustainability in Developing Countries

Ades

2017

Medicine Access @ Point of Care

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Unfortunately, Brazil's inequalities are also seen in the public's access to health care. The Brazilian public health system (SUS-Sistema Único de Saúde) was created with the new Brazilian constitution of 1988 (4), with the purpose to deliver equal, universal, and integral health care to all Brazilian citizens. The public health system is supposed to be organized in a decentralized manner, to locally adapt to the specific health problems of each region from the tropical diseases of the Amazon forest to the pollution-related diseases of its megalopolis. However, it is underfunded, spending around US$150 per patient per year (5, 6). Table I shows the median health expenditure per capita per year in Brazilian metropolitan regions. It is not yet organized or hierarchized to promote its objective and deal with the reallife health problems of its citizens (7). Nontransmissible chronic diseases in Brazil Currently, worldwide, nontransmissible chronic diseases (NTCD) are the major cause of death, accounting for 36 million deaths per year. This group of diseases is composed of cardiovascular diseases, diabetes, lung diseases, and cancer. It has been estimated that 80% of the deaths by NTCD have occurred in middle-and low-income countries. As a convention, deaths caused by these diseases in people under the age of 60 are considered to be premature, as they may reflect the lack of proper health assistance. When comparing NTCD premature deaths, 13% occurred in developed countries, whereas 29% occurred in developing countries.

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“…In Japan, times have been decreasing: in 1998, it was 1,239 days, decreasing to 531 days in 2002 [ 15 , 16 ]. The medium time for drug approval in the EU is 418 days [ 17 ].…”

Section: The European Union Centralized Procedures For Marketing Authomentioning

confidence: 99%

“…Anticancer drugs are estimated to represent less than 15% of the total cost of oncology treatment, and around 5% of the total drug expenditure [ 17 ]. Despite this, they are a readily identified area for budget cuts, and this probably contributes most to the disparity of drug uptake across the EU [ 28 ].…”

Section: Price Negotiation and Budget Allocationmentioning

confidence: 99%

Hurdles and delays in access to anti-cancer drugs in Europe

Ades

Zardavas

Senterre

et al. 2014

ecancer

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Demographic changes in the world population will cause a significant increase in the number of new cases of cancer. To handle this challenge, societies will need to adapt how they approach cancer prevention and treatment, with changes to the development and uptake of innovative anticancer drugs playing an important role. However, there are obstacles to implementing innovative drugs in clinical practice. Prior to being incorporated into daily practice, the drug must obtain regulatory and reimbursement approval, succeed in changing the prescription habits of physicians, and ultimately gain the compliance of individual patients. Developing an anticancer drug and bringing it into clinical practice is, therefore, a lengthy and complex process involving multiple partners in several areas. To optimize patient treatment and increase the likelihood of implementing health innovation, it is essential to have an overview of the full process. This review aims to describe the process and discuss the hurdles arising at each step.

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European Union centralised procedure for marketing authorisation of oncology drugs: An in-depth review of its efficiency

Cited by 12 publications

References 5 publications

Regulatory approval of pharmaceuticals without a randomised controlled study: analysis of EMA and FDA approvals 1999–2014

Regulatory approval of pharmaceuticals without a randomised controlled study: analysis of EMA and FDA approvals 1999–2014

Access to Oncology Drugs in Brazil: Juggling Innovation and Sustainability in Developing Countries

Hurdles and delays in access to anti-cancer drugs in Europe

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