Dept of Clinical and Molecular Biomedicine, University of Catania, Catania, Italy.
Eur Respir J. 2013 Feb;41(2):262-9. doi: 10.1183/09031936.00115112. Epub 2012 Aug 16.
Idiopathic pulmonary fibrosis (IPF) is a progressive fibroproliferative disease that results in increasing morbidity. To date there is only one licensed therapy for this condition and other agents are needed for this attritional disease. Efforts to study other agents have been obstructed by an increasing division of opinion about the most clinically meaningful end-point of phase III clinical trials to demonstrate efficacy. Many clinicians believe that an agent that impedes progression of the disease is more than acceptable and will encourage the pharmaceutical industry to further develop their IPF programmes. We have been impressed by the behavioural and biological similarities of cancer and IPF, and wondered if lessons could be learned about clinical trial design from lung cancer studies. Here, we set out our arguments that the similarities with cancer justify comparing the magnitude of therapeutic effects in clinical trials in nonsmall cell lung cancer with those in successful trials in IPF. We demonstrate that efficacy is of a similar magnitude in the two chronic lung diseases. We recommend that the demonstration of similar magnitudes of progression-free disease effect in IPF, using appropriate indices, should be considered as clinically meaningful benefit in future phase III clinical trials of novel therapies.
特发性肺纤维化（IPF）是一种进行性纤维增生性疾病，会导致发病率不断上升。迄今为止，这种疾病只有一种获得许可的治疗方法，因此需要其他药物。由于对 III 期临床试验中最具临床意义的疗效终点的意见分歧越来越大，研究其他药物的努力受到了阻碍。许多临床医生认为，一种能够阻止疾病进展的药物是完全可以接受的，并将鼓励制药行业进一步开发他们的 IPF 项目。我们对癌症和 IPF 的行为和生物学相似性印象深刻，并想知道是否可以从肺癌研究中吸取关于临床试验设计的经验教训。在这里，我们提出了我们的观点，即与癌症的相似性证明了比较非小细胞肺癌临床试验中的治疗效果幅度与 IPF 中成功试验的治疗效果幅度是合理的。我们证明，这两种慢性肺部疾病的疗效幅度相似。我们建议，在未来的新型疗法 III 期临床试验中，使用适当的指标，在 IPF 中证明无进展疾病效应的相似幅度应被视为具有临床意义的益处。
