Approvals of drugs with uncertain benefit–risk profiles in Europe

Banzi, Rita; Gerardi, Chiara; Bertelè, Vittorio; Garattini, Silvio

doi:10.1016/j.ejim.2015.08.008

Cited by 84 publications

(93 citation statements)

References 32 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…Concerning expedited‐approval programmes in the EU, it has been reported that there were delays or discrepancies in the fulfilment of the post‐market obligations in more than one third of the conditional approvals (Banzi et al , 2015). Moreover, as in the example of Midodrine in the USA (Dhruva and Redberg, 2010), it has sometimes become difficult for authorities to withdraw products of questionable efficacy from the market because of appeals made by professional organizations, health care professionals and patients.…”

Section: Discussionmentioning

confidence: 99%

“…A large number of obligations to collect and report post‐marketing data on approved drugs have been bypassed by sponsors (Moore and Furberg, 2014). In the EU also, there have been delays or discrepancies in fulfilling post‐marketing obligations in more than one third of granted conditional approvals (Banzi et al , 2015). Objections by the US congress or patient advocacy groups have reversed withdrawal decisions previously made by the legislative authorities (Dhruva and Redberg, 2010).…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Comparison of the new Japanese legislation for expedited approval of regenerative medicine products with the existing systems in the USA and European Union

Jokura

Yano

2017

J Tissue Eng Regen Med

View full text Add to dashboard Cite

Legislation for expedited‐approval pathways and programmes for drugs, biologics or medical devices has been enacted for rapid commercialization of innovative products in the United States of America (USA) and the European Union (EU). However, less innovative products are increasingly benefitting from these expedited‐approval pathways, and obligations to collect and report post‐marketing data on approved products are being bypassed frequently. The Japanese government recently enacted legislation for a new conditional and time‐limited approval pathway dedicated to regenerative medicine products. The current study examines this new legislation and compares it with existing US and EU regulatory frameworks, with a particular focus on how it addresses the limitations of existing systems. Regulations, guidance documents and approval information were gathered from the websites of the respective authorities in the USA, the EU and Japan, and the systems were categorized through qualitative analysis. The pathways and programmes from each region were categorized into four groups, based on the requirement of pre‐ or post‐marketing clinical data. Expedited‐approval pathways in the USA and the EU provide similar qualification criteria, such as severity of target disease; however, such criteria are not specified for the new pathway in Japan. Only the Japanese pathway stipulates a time limitation on exceptional approval, requiring post‐marketing study for conditional and time‐limited products. Continuous improvement is necessary to solve previously addressed issues within the expedited‐approval pathways and programmes and to ensure that innovative medical products are rigourously screened, but also readily available to patients in need. The time limitation of conditional approval could be a potential solution to some of these problems. Copyright © 2017 The Authors. Tissue Engineering Regenerative Medicine published by John Wiley & Sons, Ltd.

show abstract

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Comparison of the new Japanese legislation for expedited approval of regenerative medicine products with the existing systems in the USA and European Union

Jokura

Yano

2017

J Tissue Eng Regen Med

View full text Add to dashboard Cite

show abstract

“…13 Conditional authorisation is used most often for drugs used in oncology and infectious diseases and for orphan-designated medicines. 13–15 Examples include ▾bedaquiline for the management of multidrug-resistant tuberculosis and pazopanib hydrochloride (Votrient) for renal cell carcinoma. 15,16 …”

Section: Regulatory Tools For Faster Approval or Pre-approval Accessmentioning

confidence: 99%

“…13–15 Examples include ▾bedaquiline for the management of multidrug-resistant tuberculosis and pazopanib hydrochloride (Votrient) for renal cell carcinoma. 15,16 …”

Section: Regulatory Tools For Faster Approval or Pre-approval Accessmentioning

confidence: 99%

Pathways to faster access to medicines

2018

DTB

View full text Add to dashboard Cite

Before a medicine can be marketed in the UK, marketing authorisation approval is needed from the European Medicines Agency (EMA) or the Medicines and Healthcare products Regulatory Agency (MHRA). However, the time it takes to appraise a medicine is considered by some to delay access to new treatments for people with serious or life-threatening conditions who have no other treatment options. Also, the standard regulatory process may be less suitable for medicines for rare conditions in which it is difficult to gather a large amount of clinical trial data. Here we look at a range of new regulatory and access pathways that have been developed to respond to these challenges and consider some of their potential pitfalls. In a future article we will review the impact that the UK's departure from the European Union (EU) will have on licensing processes.

show abstract

“…Drei KHSG-Maßnahmen adressieren dieses Problem: Seltenheit (vgl. HAI et al 2015;Banzi, R. et al 2015;Fain, K. et al 2013). Damit steigt das Risiko von Fehlentscheidungen für Ärzte, Patienten, Versicherte und das Gesundheitssystem dauerhaft (Zentner, A., Haas, A.…”

Section: Abbildung 10: Belegungstage Und Durchschnittliches Alter (19unclassified

Neuerungen im Krankenhaus- und Arzneimittelbereich zwischen Bedarf und Finanzierung

Wille¹

2017

View full text Add to dashboard Cite

Approvals of drugs with uncertain benefit–risk profiles in Europe

Cited by 84 publications

References 32 publications

Comparison of the new Japanese legislation for expedited approval of regenerative medicine products with the existing systems in the USA and European Union

Comparison of the new Japanese legislation for expedited approval of regenerative medicine products with the existing systems in the USA and European Union

Pathways to faster access to medicines

Neuerungen im Krankenhaus- und Arzneimittelbereich zwischen Bedarf und Finanzierung

Contact Info

Product

Resources

About