What if?

[DonnaBoll]

I mentioned to you that one of the main premises of the Summit was to think about "what ifs" that we could change about how PBC is perceived and/or treated.

One of these suggestions was "what if" we could totally eliminate the use of placebos in all clinical trials. Some of you may have participated in a clinical trial to evaluate a new potential drug usually treating fatigue, disease progression or itch associated with PBC. The nature of any trial is to have one group taking the drug itself and the second group taking a sugar pill of sorts or a 'placebo'. This group is not actually receiving the drug at all. Trials can last last months to sometimes 8-10 years. Just think about the patient who is receiving a placebo for that length of time. So much time wasted for him or her. When they are in a trial because they are desperate for something to help them or they are advancing in their disease they don't often have that kind of time to waste in every sense of the word.

Eliminating the use of placebos could change the whole dynamics of the trial. Every patient would be taking the drug... perhaps in different doses. No time is wasted taking nothing besides their Urso or Ocaliva. Every patient deserves equal chance of benefiting from a new drug potentially.

Any thoughts on this? The lobbying of the pharmacuetical companies is crucial for this to happen. The PBC Foundation is committed to make this happen!

[DebatDG9]

Radical!

So instead of comparing effect of drug vs no drug the comparison would be of each patient’s experience pre drug and with drug. Makes absolute sense. Licensing authority and NICE would have to be on board

[DonnaBoll Administrator in reply to DebatDG9]

Thanks for sharing how you feel about this proposal. At this point there is much work to be done getting organizations on board. This is truly just in 'what if' stages of planning a proposal. The PBC Foundation has felt strongly about this for several years. The Summit was a likely place to propose this to a group of pharmas, clinicians, patients and support group from literally around the world.

[Candy12]

I may end up being in the minority here and that’s perfectly okay . I find it hard to comprehend what I’ve just read, I totally, disagree with doing away with the placebo arm of any clinical trial . It’s there to gather the information needed on efficacy and safety. Giving everyone a drug to see if it works does not make it safe to do so. It sounds more like something the pharmaceutical companies would want to push for, to get drugs to the market quicker. Than something the PPC foundation would endorse.. Astonishing.

[DonnaBoll Administrator in reply to Candy12]

I really appreciate your sharing your point of view. This proposal did not come from big pharma. This is something that The PBC Foundation has felt strongly about for some time, knowing that someone who is in need of a different drug to retard their disease progression does not have the years (in some cases) to possibly be on a placebo. Each of the drug companies that spoke at The Summit go thru rigorous testing mandated from the FDA to make sure that their drug is safe, often leading to years of 'stages' of the trials and usually spending upward of $1 billion dollars on this research that could likely end up useless. I'm really glad you wrote how you felt. This is what is needed for sure... discussion by any party that could be potentially affected by this measure. Please feel free to reach out to The PBC Foundation with any of your concerns. pbcfoundation.org.uk

[Candy12]

Thank you for your reply, I’ve no need to reach out, I fully understood it was the PBC Foundation pushing for it . I just don’t agree with it for my own personal reasons, and not everyone can have the same opinions which is good because it drives conversations. I could go into my reasons why, but it’s beyond the scope of this platform.

I would like to say thank you for feeding back to us what you learn though, whether it’s something we agree with or not, it’s all important information.

[DonnaBoll Administrator in reply to Candy12]

I couldn't agree more. Discussions from many points of view make many differences that change lives. In the coming days I will continue to share information with the group from my experience in Edinburgh.

[Ancolie]

I was on a trial for a itch medication and I know that I was on the placebo for most of the trial since I had bruises all over from my scratching. Unfortunately I got the real medication for the last 4 weeks only and most of my bruises went away. Took away the itch for about 75 to 80 %. Sad I didin't get the real medication in the first place. But I wonder how they get to know if a medication is realy working if they dont give a placebo to a portion of the patients on trial ?

[DonnaBoll Administrator in reply to Ancolie]

Great question! There are 2 (if I remember correctly) officially used 'itching scores' that pharma would evaluate those participating in the trials. In some cases there are different doses of the medication being trialed. The results from that part of the trials are evaluated as well. The bottom line is that the patient is the one who really tells pharma if the itch is improving. There are no hard numbers like LFT's to go on in most cases. What a game changer this could be for so many. I will share info on more drugs in the coming days.