NIVEL, Netherlands Institute for Health Services Research, PO Box 1568, 3500 BN Utrecht, The Netherlands.
Orphanet J Rare Dis. 2011 Sep 5;6:59. doi: 10.1186/1750-1172-6-59.
We determined whether the market exclusivity incentive of the European Orphan Drug Regulation results in a market monopoly or that absence of another Orphan Medicinal Product (OMP) for the same rare disorder, a so-called follow-on OMP, is a matter of time or market size. In the interest of rare disorder patients better understanding of the effect of the market exclusivity incentive on follow-on OMP development is warranted.
First, the impact of various market-, product- and disease-related characteristics on follow-on OMP development in the EU was determined by comparing rare disorders with an approved OMP and at least one follow-on OMP (N = 26), with rare disorders with an approved OMP and no follow-on OMP (N = 18). Next, we determined whether manufacturers continued development of a follow-on OMP upon approval of the first OMP for the intended rare disorder. Since in the EU significant benefit of an OMP has to be established, we determined for each follow-on OMP for which development was continued on what grounds significant benefit was assumed by the sponsor. Data were collected from the public domain only.
The likelihood of a rare disorder with an approved OMP to obtain at least one follow-on OMP development was strongly associated with disease prevalence, turnover of the first OMP, disease class, disease-specific scientific output and age of onset. Out of a total of 120 follow-on OMPs only one follow-on OMP could be identified for which development was discontinued upon approval of the first OMP for the same rare disorder. Only a substantial level of discontinuation of follow-on OMP development would have indicated the existence of a market monopoly. Moreover, sponsors that continued development of a follow-on OMP predominantly assumed that their product had an improved efficacy compared to the first approved OMP.
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 European Orphan Drug Regulation creates a market monopoly.
我们确定欧洲罕见病药物法规的市场独占激励是否会导致市场垄断，或者是否缺乏针对同一罕见疾病的另一种孤儿药物（OMP），即所谓的后续 OMP，这是时间问题还是市场规模问题。为了让罕见病患者更好地了解市场独占激励对后续 OMP 开发的影响，有必要进行研究。
首先，通过比较已批准 OMP 和至少一种后续 OMP 的罕见疾病（N=26）与仅批准 OMP 而无后续 OMP 的罕见疾病（N=18），确定各种市场、产品和疾病相关特征对欧盟后续 OMP 开发的影响。其次，我们确定制造商是否在批准针对预期罕见疾病的第一个 OMP 后继续开发后续 OMP。由于在欧盟，必须证明 OMP 具有显著益处，因此我们确定了每个继续开发后续 OMP 的制造商，其依据的是赞助商假设的显著益处的理由。数据仅从公共领域收集。
已批准 OMP 的罕见疾病获得至少一种后续 OMP 开发的可能性与疾病流行率、第一个 OMP 的周转率、疾病类别、疾病特异性科学产出和发病年龄密切相关。在总共 120 种后续 OMP 中，只有一种后续 OMP 可以被确定为在同一罕见疾病的第一个 OMP 获得批准后，其开发被停止。只有大量的后续 OMP 开发停止才表明存在市场垄断。此外，继续开发后续 OMP 的赞助商主要假设他们的产品与第一个批准的 OMP 相比具有更高的疗效。
本研究提供了证据表明，缺乏后续 OMP 开发是时间问题或市场规模问题，而不是欧洲罕见病药物法规的市场独占激励创造了市场垄断。
