Accelerating development, registration and access to medicines for rare diseases in the European Union through adaptive approaches: features and perspectives

Uguen, David; Lönngren, Thomas; Cam, Yann Le; Garner, Sarah; Voisin, Emmanuelle; Incerti, Carlo; Dunoyer, Marc; Slaoui, Moncef

doi:10.1186/1750-1172-9-20

Cited by 9 publications

(7 citation statements)

References 3 publications

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“…In particular, the rare conditions with the highest number of failures were in the oncology area and included: glioma (12), acute myelogenous leukaemia (11), pancreatic cancer (10) and chronic lymphocytic leukaemia (7). In the respiratory group, six failures were for cystic fibrosis.…”

Section: Data Analysis By Risk Factorsmentioning

confidence: 99%

“…Other relevant causes were linked to sponsors, that is, (a) lack of data, (b) inactive company, (c) company strategy blocking the developmental process. For MA failures, the identified reasons were: efficacy/ safety issues (26), insufficient data (12), issues related with the quality of the IMP (eg, manufacturing issues) (7) and regulatory issues on trials (4), such as trials without a control arm or not compliant with Good Clinical Practice and/or no commercial protection (ie, market exclusivity granted for other products already authorised for the same condition). In just one case, a commercial reason was declared by the sponsor (1).…”

Section: Reasons For Failuresmentioning

confidence: 99%

“…Few analyses on the developmental status of OMPs and regulatory pathway have been published. [6][7][8][9] In conclusion, the real extent and reasons for the failures in the developmental process of OMPs in European Union (EU) are currently mainly unknown.…”

Section: Introductionmentioning

confidence: 99%

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Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs

Giannuzzi

Landi

Bosone³

et al. 2017

BMJ Open

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ObjectivesThe research and development process in the field of rare diseases is characterised by many well-known difficulties, and a large percentage of orphan medicinal products do not reach the marketing approval.This work aims at identifying orphan medicinal products that failed the developmental process and investigating reasons for and possible factors influencing failures.DesignDrugs designated in Europe under Regulation (European Commission) 141/2000 in the period 2000–2012 were investigated in terms of the following failures: (1) marketing authorisation failures (refused or withdrawn) and (2) drugs abandoned by sponsors during development.Possible risk factors for failure were analysed using statistically validated methods.ResultsThis study points out that 437 out of 788 designations are still under development, while 219 failed the developmental process. Among the latter, 34 failed the marketing authorisation process and 185 were abandoned during the developmental process. In the first group of drugs (marketing authorisation failures), 50% reached phase II, 47% reached phase III and 3% reached phase I, while in the second group (abandoned drugs), the majority of orphan medicinal products apparently never started the development process, since no data on 48.1% of them were published and the 3.2% did not progress beyond the non-clinical stage.The reasons for failures of marketing authorisation were: efficacy/safety issues (26), insufficient data (12), quality issues (7), regulatory issues on trials (4) and commercial reasons (1). The main causes for abandoned drugs were efficacy/safety issues (reported in 54 cases), inactive companies (25.4%), change of company strategy (8.1%) and drug competition (10.8%). No information concerning reasons for failure was available for 23.2% of the analysed products.ConclusionsThis analysis shows that failures occurred in 27.8% of all designations granted in Europe, the main reasons being safety and efficacy issues. Moreover, the stage of development reached by drugs represents a specific risk factor for failures.

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Section: Data Analysis By Risk Factorsmentioning

confidence: 99%

Section: Reasons For Failuresmentioning

confidence: 99%

See 1 more Smart Citation

Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs

Giannuzzi

Landi

Bosone³

et al. 2017

BMJ Open

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“…Furthermore, consideration of the condition and population to be treated, as well as the expected duration of treatment, has a direct effect on the preclinical and early clinical investigations required to bring a drug into clinical trials for a given indication. 3 Hence, it is advisable to involve patient representatives and disease experts early in the therapeutic development process.…”

mentioning

confidence: 99%

Considerations for development of therapies for cutaneous neurofibroma

et al. 2018

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The number, size, and distribution of cNF is highly variable among patients with NF1 and it is possible that different phenotypes will require different drug development paths. The nonfatal nature of the disease and relatively limited patient numbers suggest that for any product to have a higher likelihood of acceptance, it will have to (1) demonstrate an effect that is clinically meaningful, (2) have a safety profile conducive to long-term dosing, and (3) have a low manufacturing cost.

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“…In recent years, though there is relatively small number of people with hemophilia, for meeting the challenge of the ageing hemophilia population, the management of hemophilia becomes more complex [9]. We will have come a long way to supply assistance treatment information for discovering new characteristic rules and seeking new treatment strategies [24] [25] [26]. One of the important challenging issues in the near future is registration available information for all affected individuals worldwide.…”

Section: Discussionmentioning

confidence: 99%

A Preliminary Study of Age and Sex of People with Hemophilia

Yang¹,

Miao²,

Liu³

et al. 2017

Health

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Aim: This paper aims at providing more information of people with hemophilia for more targeted treatment. Methods: 1149 people with hemophilia are surveyed in 45 units, and then established by EpiData software. The number of people with hemophilia in 9 age intervals for the male and female is analyzed by Population Pyramid and Pareto Analysis. Results: It is found that the people with hemophilia are appearing in every age interval. Relative to the number of male patients, the number of female patients is extremely rare. Of 1134 male patients, relative frequencies for male ranked the first (23.0%), second (17.4%), third (17.2%) are 7 -12, 2 -3, 13 -18 years old, respectively, while of 15 female patients, relative frequencies for female in these age intervals are only 13.3%, 13.3%, 6.7%, respectively. However, the most relative frequency of female patients is between 26 -45 years old. Conclusion: There is a challenge of the aging hemophilia population. Most of male patients are between 7 -12 and 2 -3 years old. Most of female patients are between 26 -45 years old.

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Accelerating development, registration and access to medicines for rare diseases in the European Union through adaptive approaches: features and perspectives

Cited by 9 publications

References 3 publications

Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs

Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs

Considerations for development of therapies for cutaneous neurofibroma

A Preliminary Study of Age and Sex of People with Hemophilia

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