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Does market exclusivity hinder the development of Follow-on Orphan Medicinal Products in Europe?

Does market exclusivity hinder the development of Follow-on Orphan Medicinal Products in Europe?. Brabers, AEM Moors, EHM Van Weely, S De Vrueh, RLA December 7, 2011 14 th Round Table Meeting EHC Brussel. Background – rare disorders.

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Does market exclusivity hinder the development of Follow-on Orphan Medicinal Products in Europe?

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  1. Does market exclusivity hinder the development of Follow-on Orphan Medicinal Products in Europe? Brabers, AEM Moors, EHM Van Weely, S De Vrueh, RLA December 7, 2011 14th Round Table Meeting EHC Brussel

  2. Background – rare disorders • 6,000 - 8,000 rare disorders, many of them are genetic of origin and affect children at very early age • Definition rare disorder in the EU • less than 5 / 10,000 inhabitants • life-threatening • chronically debilitating • In the EU and US together, 55 million people suffer from a rare disorder

  3. Background – legislation • Legislation introduced to stimulate development of orphan drugs • Regulations highly appreciated, however, their success has also been questioned… • Small % of orphan designations obtain approval • Certain disease classes have a high number of orphan designations and approvals • Market exclusivity most important incentive

  4. Background – market exclusivity • Roos et al. claimed that the market exclusivity incentive creates a market monopoly • Tambuyzer argued that if an approved OMP is currently the only product on the market, this is a matter of time or market size • After review of the EU register, it remains unclear whether the market exclusivity incentive serves as disincentive for follow-on OMP development or not…

  5. Objective of the study • To determine whether the market exclusivity incentive of the EU orphan drug regulation results in a market monopoly, or that absence of a follow-on OMP is a matter of time or market size

  6. Objective of the study 1.Factors elucidating follow-on OMP development 2. Effect of marketing authorization of first OMP on follow-on OMP development 3. Characterization of assumptions of significant benefit of follow-on OMPs

  7. Sample • All rare disorders with approved OMP up to 31/12/2008 • 58 OMPs obtained approval from start regulation up to 31/12/2008 • Collectively, the 58 approved OMPs have been authorized for 44 different rare disorders • For each included rare disorder the EU register was checked on follow-on OMPs. A follow-on OMP is defined as: • 1. another approved OMP • 2. another medicinal product with EU orphan designation

  8. Sample • Thereafter, rare disorders were divided into two groups: • 1. rare disorders having an approved OMP and at least one follow-on OMP(follow-on group) • 2. rare disorders having an approved OMP, but no follow-on OMP (no follow-on group) • For the 26 rare disorders in follow-on group, 120 follow-on OMPs were identified up to 30 April 2010 number of rare disorders

  9. 1. Factors elucidating follow-on OMP development • Data for each rare disorder on market-, product- and disease-related characteristics • Characteristics of rare disorders in follow-on group and no follow-on group were compared using univariate analyses

  10. 1. Factors elucidating follow-on OMP development *) Only significant characteristics are presented in the table

  11. 2. Effect of marketing authorization of first OMP on follow-on OMP development • Sponsor continued if … • 1. OD date of follow-on OMP was after date of marketing authorization first OMP • 2. continuation was verified in the public domain for follow-on OMPs having OD date prior to approval of first OMP • Overall, development is continued after marketing authorization of first OMP for same rare disorder • Out of 120 follow-on OMPs, only one could be identified for which development was discontinued (PI-88, Progen)

  12. 3. Characterization of assumptions of significant benefit of follow-on OMPs • Significant benefit is justified in the EU by demonstrating an improved efficacy, a better safety or a major contribution to patient care • Assumptions were collected in the public domain and mapped using key-words of the EMA guideline • Assumptions found for 106 out of 119 follow-on OMPs, in total 130 different assumptions

  13. 3. Characterization of assumptions of significant benefit of follow-on OMPs • Most assumptions related to improved efficacy (mainly another mechanism of action [36.2%] and sub-group patients [20.8%]) N = 130

  14. Conclusions • Objective • To determine whether the market exclusivity incentive results in a market monopoly, or that absence of a follow-on OMP is a matter of time or market size • Conclusion • This study provides evidence that absence of follow-on OMP development is a matter of time or market size, rather than that the market exclusivity incentive of the EU orphan drug regulation creates a market monopoly

  15. Conclusions • Apart from market size, turnover of first OMP, disease class, disease-specific scientific output and age of onset were identified as predictors of follow-on OMP development • Development of follow-on OMP is continued and primarily based on assumption of improved efficacy

  16. Thank you for your attention! • Contact information • Anne Brabers, MSc • a.brabers@nivel.nl • +31 (0)30 27 29 855 • Article information • Brabers AEM, EHM Moors, S van Weely, RLA de Vrueh. Does market exclusivity hinder the development of Follow-on Orphan Medicinal Products in Europe? Orphanet J Rare Dis. 2011 Sep 5;6:59

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