Article Text

other Versions

PDF
Study the past to divine the future. Confucius' wisdom doesn't work for idiopathic pulmonary fibrosis
  1. Martin Kolb1,
  2. Gisli Jenkins2,
  3. Luca Richeldi3
  1. 1Department of Medicine, McMaster University and Firestone Institute for Respiratory Health, St. Joseph's HealthCare Hamilton, Hamilton, Ontario, Canada
  2. 2Nottingham Respiratory Research Unit, University of Nottingham, Nottingham, UK
  3. 3NIHR Southampton Respiratory Biomedical Research Unit, University Hospital Southampton, Southampton, UK
  1. Correspondence to Professor Luca Richeldi, National Institute for Health Research, Southampton Respiratory Biomedical Research Unit, Mailpoint 813, E Level, South Academic Block, University Hospital Southampton NHS Foundation Trust, Tremona Road, Southampton, SO16 6YD, UK; l.richeldi{at}soton.ac.uk

Statistics from Altmetric.com

Predicting the future is one of the greatest challenges and for many people one of the greatest hopes of humanity. This applies to any aspect of human life and medicine included. In respiratory medicine, predicting the future is particularly difficult for chronic remodelling disorders, such as pulmonary hypertension or fibrosis.1 The course and recovery from an acute illness are usually easier to foresee than the progression and rate of decline for chronic diseases. In particular, one of the major current challenges is actually predicting the effect of the available pharmacological treatments on the course of idiopathic pulmonary fibrosis (IPF), which is of paramount importance but is still rarely possible. Nonetheless, the more we enter the era of the so-called personalised medicine, anticipating the response to a specific drug is becoming part of realistic expectations.2

Safety and efficacy of drugs are assessed in the context of placebo-controlled randomised clinical trials (RCTs). Although a well-established and worldwide accepted methodology, RCTs still have limitations: one of these is the fact that necessarily trials last for a definite period of time, for IPF typically 12 months, during which time all participants are blinded to the active treatment or to a placebo. This limitation is intrinsic and unavoidable, given the need to balance between harm and benefit when new drugs with unknown effects are tested in patients. However, once approved, all new drugs undergo a mandatory postapproval surveillance of several years. While this type of postmarketing surveillance provides valid information about long-term safety of new drugs, there is no formal way of assessing long-term efficacy, and even if these studies report on efficacy, they are never controlled and therefore the evidence base is less rigorous than for prospective trials. For this reason, other forms of clinical research may be used to inform clinical …

View Full Text

Request permissions

If you wish to reuse any or all of this article please use the link below which will take you to the Copyright Clearance Center’s RightsLink service. You will be able to get a quick price and instant permission to reuse the content in many different ways.

Linked Articles