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Table 1 Patients’ open-text comments on the ‘value of a medicine’ (excerpts)

From: Quantifying benefit-risk preferences for new medicines in rare disease patients and caregivers

On hope
 ‘To have no hope is to have no life or anything to live for – any chance […] of not just a cure but hope that life will be longer or will not deteriorate makes a difference beyond anything.’
 ‘Any new medicine that can give some hope to those suffering with such diseases is invaluable.’
 ‘I would prefer research into new medicines [addressing my main symptoms], rather than research into a ‘cure’ for my condition.’
 ‘We live in hope.’
 ‘Any port in the storm.’
On risk
 ‘With motor neurone disease any improvement outweighs any side effects. Better to die than live with this cruel disease.’
 ‘We have nothing to lose but everything to gain.’
 ‘As I am symptom-free at present, I am against health risks. However, once my condition deteriorates, my attitude to health risks will change.’
 ‘[Minor] side effects should not be underestimated or played down. [They] can be extremely wearing and challenging when they occur every day.’
 ‘I have discovered I am better off with no medicine, the side effects made me feel worse.’
On interactions with healthcare professionals
 ‘Regular contact with health care professionals […] is invaluable.’
 ‘Doctors need to give us the information so we can decide for ourselves.’
 ‘Health professionals take a very ‘paternalistic’ stance and give minimum information – “they know best”.’
 ‘Each new medicine should have the side effects explained in percentages like this survey.’
 ‘Anything that engages the sufferer in discussions determining how to handle treatment/medication can only be beneficial to the patient.’
On the impact of long-term use of steroids
 ‘[The medication] I object to most are steroids because of the physical changes they have made to me. This side effect may not be as relevant/important to other patients. This is an area the medical profession need to be more sympathetic towards.’
 ‘Doctors prescribe [steroids] and do not explain the side effects and long term effects and risks without discussing it with you or giving you the options. They got me into remission after two years but I am still suffering with other diseases caused by [steroids]. I would have opted for other options if I was given a choice.’
Patients’ introspection
 ‘Really interesting survey, I learnt a lot about my attitude to risk.’
 ‘I found the survey quite thought provoking and made me think about my current treatment plan and other options facing me.’
 ‘Answering the questions about value made me realise I regard the chance of a beneficial effect as outweighing any possible risk or discomfort.’
  1. Note: More patient quotations can be found in Additional file 8: Appendix H