UK PATIENTS AND FAMILIES SUPPORT GROUP

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  Lenwood | published 6 months ago | Originally written in English UK PATIENTS AND FAMILIES SUPPORT GROUP

  Hello

  Thanks Nicholas for directing me to this community. Already as with the Foundation for children with aHUS site I have found much information to absorb.

  It must say something, either about the global nature of aHUS ,or the slowness of the UK to catch on, that earlier postings about the conference in Newcastle in June should be posted on a european site by someone from the USA!!.

  Anyway a new UK organisation may help to remedy that in future.

  Out of the conference in June came two UK organisations

  - aHUS Action which is a campaign group set up to get political support for the national funding and commissioning of treatment for HUS (including eculizumab) in England;

  - aHUSUK a patient and family support group set up to bring together patients with aHUS and to do on a UK level what other countries have done for their patients.

  aHUS patients are a small group and even now 5 months on from the Newcastle conference aHUSUK is only aware of less than 10% of the aHUS patient cohort in the UK!

  A simple illustration of how remote aHUS patients are, is that tonight my daughter spoke to another patient who became ill with aHUS at the same time as her and has had an almost parallel treatment experience with her at the same hospital for the past 10years and they only found out about each other a week or so ago.

  As I say the hope is that aHUSUK will also remedy that.

  There is more news about what is happening in the UK but I will leave it at that for now!!
  15 replies to the topic — see all replies

  • 
    LindaBurke published 6 months ago | Originally written in English

    Hi Len,

    Thanks for joining this community - feel free to add links to your UK aHUS event posted on www.atypicalhus.org to assure the widest outreach to aHUS patients and their families. As you mentioned with your daughter's experience, connecting with others is valuable to sharing experiences and vital to progress on various aHUS fronts!
  • 
    Lenwood published 6 months ago | Originally written in English

    To continue the story of what is happening in the UK.

    Funding of treatments for rare diseases by our National Health Service (NHS) is haphazard resulting in what is known as a “postcode (zip code) lottery” with some Primary Care Trusts prepared to fund treatments like eculizumab ,whereas other Trusts might not. This is further compounded by the NHS being separately organised in England, Scotland, Wales and Northern Ireland.

    Currently the only application for the treatment of aHUS with eculizumab has been made to the NHS England. The application is for it to be nationally commissioned and funded. For the application to be successful it has to be reviewed by an Advisory Group for NHS Special Services (AGNSS). Having reviewed the cases for all the illnesses applying for funding from 2012 onwards, AGNSS will make a recommendation to the Government’s Health Minster (Earl Howe who sits in the House of Lords) for a decision on whether to accept it.

    This is why we have an organisation called aHUS Action, which is coordinating the lobbying of politicians and mustering a public awareness and support for the application to be accepted.

    aHUS patients have been supporting this campaign by getting signatures for an e petition, calling for the government to provide eculizumab. They have been writing to their MPs with their personal story of what aHUS has done to them and seeking their support to influence the Heath Minister.

    They have also supplied their personal stories so that aHUS Action can produce a brochure about aHUS and its consequences. This is to be launched at a Parliamentary Seminar in Westminster on 17 January 2012 with MPs, Peers, patients/families and others attending.

    Meanwhile AGNSS have begun their evaluation and Alexion are already in the process of supplying them with the evidence they need. The other UK organisation, aHUSUK, the patient and families support group, will also be contributing to AGNSS’s evaluation because they want to hear what patients have to say and understand what it is like to be a aHUS patient.

    A Patient’s Voice Group has been formed with a cross section of patients to supply the evidence to AGNSS on behalf of aHUSUK. AGNSS are due to complete their evaluation by 14 June 2012.
    
    If a positive recommendation is accepted by the Minister, eculizumab could become the universal treatment being delivered to aHUS patients in England from October next year.

    What do other countries have to do to get eculizumab for aHUS?
  • 
    cherylbiermann published 6 months ago | Originally written in English

    Hi Len,

    To my knowledge, FDA approval is normally sought by the drug companies. The drug company may, contact patients and advocates before approval to hear their stories and gather information about how to help them, if and when approval is met. However, they are NOT able to push the drug in any sense.

    In the case of aHUS in the United States, the Foundation for Children with Atypical HUS had already met with the NIH, (a government funding agency), various universities and researchers globally, so perhaps, the disease was on the radar for the FDA. The FDA has an avenue to take when a drug has previously been through trials for another disease and been found to be safe and highly effective called the "Fast Track". Whether or not that is the official name, I don't know, lol!

    The great reputation and track record of Alexion and their attention to get every detail right, certainly didn't hurt when the FDA was considering if they should put the drug on the "Fast Track' and the stellar results from the clinical trials, of course, clinched the approval process.

    Hope this very simple explanation benefits your efforts in some way!
  • 
    Lenwood published 6 months ago | Originally written in English

    Hi again Cheryl,

    Thanks it does help and will make aHUSUK more authoritative on the subject.

    I guess the FDA is the equivalent of the European Medicine Agency (EMA) .

    What part does your funding agency, NIH, play in the process? And with both NIH and FEDA on board, is the treatment then commissioned universally in the USA by whoever is going to apply it.

    If the EMA follow up the postitive statement that has already been given,by approving the market license for Soliris/Eculizumab in Europe, then it is stll left to member states to decide whether and how to deploy it.

    I have described above how that is being done in the UK; but it would be interesting to see where other countries in Europe are going with it.

    I hope someone can enlighten us.
  • 
    cherylbiermann published 6 months ago | Originally written in English

    The FDA, Federal Drug Administration is our "safety net". They gather the information from the clinical trials and base the decision to approve or disapprove based on efficacy and safety.

    The NIH, National Institutes of Health, is a taxpayer funded organization which decides what research gets funded. The medical side of it is strict. For example, when we approached them way back around 2001 or 2002, we were told the aHUS group was just too small to spend the taxpayer dollars. Another attempt was made grouping regular HUS with aHUS and it was never funded.

    In addition, we have private organizations, like the NKF, National Kidney Foundation, which raises money and provides it for a wide range of services and research, even the Transplant Olympics. We have never received direct funding or indirect from any of these organizations. However, the organizations were all aware of our little group, so we did have name recognition.

    Dr. Remuzzi was the first person to respond to our inquiries and willing to work with any small dollar amount we might raise. He also helped Bill tremendously in defining the disease and current research at the time. Bill also went to a nephrology convention and set up a booth, there he met Dr. Goodship, who introduced him to a US researcher whom Bill had attempted to reach several times because he is literally in our own backyard.

    In this country, once you go into renal failure,after 30 months, your private insurance becomes secondary and Medicare, (taxpayer funded), becomes primary. Medicare offers many choices, hospitalization, medicine, ect. Because we kept our private insurance and continued paying for it, they picked up the tab for the Soliris, even before FDA approval, using the Compassionate Use Clause, which is implimented when there is sufficient data and/or it is a life-saving or life-changing option, our insurance approved the drug. Medicare paid for the transplant and related costs for the donor.

    To confuse matters even more, there is another government funded option federally, patientsmay receive Medicaid if they are unable to obtain isurance. Then each state may or may not also have the option of insuring children, we live in Missouri, so this is called Missouri MC + for Kids.

    The complicated thing for American's to understand is that if an when your government approves the drug, it will then be automatically covered, but not over the whole UK, but each state must also then approve?

    Now all of this is coming from my own perseptions and experience it is on a very simple explanation of a very complicated system! Lol!

    Hope this furthers your efforts!
  • 
    cherylbiermann published 6 months ago | Originally written in English

    Len,

    Sorry, I see I didn't cover one part of your initial question, and that is it is universally covered. In theory, that would be the case, but private insurance sometimes has caps, or a certain amount spent yearly or even a lifetime cap. (I believe currently, the lifetime cap is legally gone now with new legislation.) I am no expert here, but this a very genral overview of how things currently stand.
  • 
    Lenwood published 6 months ago | Originally written in English

    Thanks Cheryl for that clarification of what happens in the USA, I am familiar with some of the organisations names but did not understand their roles.

    Am I right in thinking therefore that if an insurer accepts, under the terms and conditions of their insurance, that eculizumab is the treatment to be used for aHUS ,then should a time limit or cost limit be exceeded, as a result the safety net organisation Medicare are obligated to continue with eculizumab.

    Is there a similar obligation on Medicaid for those who are under or not insured to use the best treatment approved by the FDA?

    In the UK private health insurers would have little to do with renal care except perhaps for kidney cancer treatment. The key benefits of the private sector are quicker treatment in better surroundings.

    The NHS is the main provider of renal care. The NHS is funded from some of the National Insurance contributions made by those in employment and their employers.

    The funds go to 150 or so NHS Primary Care Trusts which pay for the medical services for the patients in their geographical area. Although technically PCTs could pay for eculizumab to be used as a renal treatment, it is unlikely that they would do so because of insufficient funds in their budgets.

    This is why 60 or so illnesses are funded nationally and the treatment is also commissioned nationally and deployed through one or two centres of excellence. In the current application for national funding for aHUS treatment, Prof. Tim Goodship’s hospital in Newcastle upon Tyne is nominated as the UK centre of excellence.

    The fragmentation of the NHS between England Scotland Wales and Northern Ireland is a recent development because of devolution of political powers to these regions. They can choose to use their NHS funds how they want. For example Wales provide free medicines to all its patients; whereas in England a small prescription charge is levied.

    If NHS England decided to use eculizumab however, it is likely that Wales will do so too. It is unlikely that Scotland would follow England’s lead and I am not sure about NI.

    I am not sure whether the rest of Europe follows the US model or UK model for health care provision. Or maybe they have a third way. Perhaps someone could enlighten us on that.

    I am not sure if you are aware of another forum that has been discussing this but I am going to start a discussion on the economic benefits to society of using eculizumab for the treatment of aHUS compared with existing conventional therapies.

    I am confident that members of this community could provide a comprehensive of societal costs that could be avoided if renal failure and its complications could be prevented.

    Len
  • 
    cherylbiermann published 6 months ago | Originally written in English

    Hi Len,

    Excellent questions and observations. Traditionally, your assumptions regarding "caps" or exceeding limits would be correct; however, health care is a huge political ball of fire right now. Currently, the caps are not allowed anymore. The way in which we came to this legislation however, has many people wanting to change it again. Some want to revamp the entire systems, some seem to want a one-payer system and still others want to see minor tweaks. What this will all end up as, no one can predict.

    Adding to the confusion is that because of the costs of Eculizumab, I'm not sure if it will become the first treatment of course with diagnosis. I know that currently people are still using some form of plasma either exchanges or plasma transfusions. The people requiring plasma exchange, or plasma pheresis, the terms are interchangeable, have the best shot because these costs are also astronomical; add that to the fact that it means there would be the constant risk of flare-ups and I think the governmental programs will see the wisdom in covering it. I'm not sure what they will wind up doing in the case of aHUS being managed with FFP or steroids, for example. Much cheaper, but even with constant flare-ups, cheaper than Soliris, at least for now. In other words, I think what may happen is that insurance/governmental programs will cover if the patient fights for it, but I'm not sure it will be the first drug of choice, unfortunately.

    That's why all efforts are so important, EDUCATION of patients as well as the medical professionals is vital.

    Umm, I see your comment about dialysis, ESRD or End Stage Renal Disease, (I think you all call it ESRF (Failure?), is covered by Medicare...after 30 months, and your private insurance becomes secondary.

    Does this make sense? There are still a lot of unaswered questions here too...we need to keep educating everyone.
  • 
    Lenwood published 6 months ago | Originally written in English

    Hi Cheryl

    In the end, health treatment comes down to a matter of economic choices. The clinical and political case for aHUS patients to receive eculizumab, before patients progress to end stage renal failure or worse is more or less won. The economic argument, however, because of the high price of eculizumab is more or less conceded. Which means, as you say, patients have a fight on their hands.

    There are two ways in which the economic cost of relative treatments can be arrived at:

    -Willingness to pay
    - Societal cost of illness outcomes;

    Willingness to pay is based on free market principles, and is the price people are prepared to pay to avoid the chance of something bad happening to them. If Private Health insurance is going to provide eculizumab as the universal treatment of choice for aHUS, insurers would have to reflect it in the insurance premiums and hope that people would be willing to pay them.

    The societal cost of illness outcomes is the way Medicare and our NHS would perceive the cost of treating a HUS which would go beyond just the direct cost of treatment options and take into account the consequential costs of the relative effectiveness of treatments. You mention the wisdom needed for Medicare to see the cost of future treatment costs. That is part of that evaluation.

    To arrive at the true cost of aHUS all the direct costs of treating it in the acute stages need to found which means costing out every medical procedure and add these to the direct costs of the chronic stage if there is a progressive deterioration of renal function including and the range of complications that arise from long term dialysis, including transplants and the effects of psychological trauma.

    In addition to direct medical costs, the indirect costs incurred by patients ,their families and others which arise because aHUS results in time and cost spent on hospital visits care costs, loss of employment which in turn leads to loss of skilled employees to employers.

    Add the intangible costs of pain,suffering and grief and the cost gap between eculizumab and the alternatives begins to close rapidly.

    Many of the academic studies of the true cost of illness have come out of the USA, a leading researcher being the economist Jean Busby. Busby’s studies into the societal cost of foodborne illnesses, including e coli and the costs of typical HUS, influenced your government’s “apparent” costly intervention programmes/legislation on hygiene practices in the catering industry.
    
    In the UK AGNSS are going to evaluate the application for eculizumab against 12 criteria, one of which is the cost to society if eculizumab is not chosen as the treatment. Until aHUSUK’s Patient’s Voice Group got underway, the chances of AGNSS arriving at a positive view of the economic case looked unlikely but now we are not so sure.

    This aHUS community could help aHUSUK in arriving at a true cost of aHUS or at least provide a comprehensive list of what cost elements should be included.There is no better group to do it!

    Len
  • 
    cherylbiermann published 6 months ago | Originally written in English

    I agree with your assumptions in full, however, it isn't even clear if private insurance will jump right to Eculizumab or need proof of on-going complications, which as you know, some strains are so aggressive kidney function is lost within a few months.

    Our docs actually went back over ten years of treatments, including every single visit, hospitalization, time in the ICU, every procedure, every consultation every treatment and all the medications and still fell short. Nathan actually sees three specialists, neurology, nephrology and the sleep disorder docs. (Not sure what you call them!) Believe you me, Nathan was in the hospital more often than he was at home, but he was receiving FFP transfusions and not pheresis. He was not one of these kids who have lapses between relapses. Before losing his kidneys at age 6, he never went any longer than 3 weeks, (and that was a one-time event), without relapsing. I couldn't even begin to imagine what it costs us in gas, and the wear and tear on the other kids. We were very fortunate to both have very large families and a wonderful group of friends at our church who did many tasks we were unable to do. However, I wonder sometimes if a couple of my kids could have done better in school had they had an intack family. We are pretty lucky because the are all doing well in school, but you wonder, could they have done better? We have managed to keep Nathan at grade level, but he had a great homebound program and I taught him where the homebound left off. How do you measure things like that?

    It sounds as if you have a good chance compiling all this information with your group, if information from USA would help, I'm pretty sure I could get our numbers for you, but if it didn't help us, it probably won't do you any good.
  • 
    Lenwood published 6 months ago | Originally written in English

    Cheryl you made an excellent start here.

    Don’t worry about putting costs on items that can be done by someone later. Even if such costs cannot be attributed in the end and it becomes an “intangible” we can still use it. Those sitting on the AGNSS group can put their own value to it, whether that is 1£/$/€ or 1 million£/$/€, it depends on how lucky they have been to not experience anything like it!

    From your posting I have got:

    Direct medical costs :

    In patient stays, outpatient visits, ICU stays:
    Procedures, Consultations, Treatments, Medication:
    FFP

    Each of these has a frequency and I would imagine there are not many who have the number experienced by Nathan for the pre ESRF stage of care.

    Also I have an idea what the above might mean for kidney treatment but what is the range of treatments etc. for neurological effects and sleep disorders.

    Indirect costs:

    Gas for visits
    Stress on siblings
    Child care of siblings whilst parents are focussed on child in hospital
    Marginal loss of educational attainment
    Additional tutoring costs at home

    From the global experience of aHUS this list can be both extended for other costs areas and supported as people confirm similar effects on them. No health organisation would have anything like it!

    Others in the USA could help build up the list I am sure, particularly with side effects and especially the “indirects”.

    Remember do not worry about estimating cost or the frequency of treatments at this stage. But make a note of any you know.

    Would it help if I produced an initial list from input I have got so far? People might then see the gaps in my awareness (like four of the indirect costs you mention, for the 5th in the UK we use petrol not gas!!!!).

    Len
  • 
    cherylbiermann published 6 months ago | Originally written in English

    Hi Len,

    So you are saying it is beneficial to have a list of ways families are impacted by the direct and indirect costs of caring for a loved one with aHUS? Would you like to post the pertinent parts of our conversation on the .org site? Would you like me to do so? We could easily ask everyone to share anything they have encountered which we have missed, and if necessary, ask for more precise dollar amounts later.

    You could probably get a good idea of the common hardships by perusing the blogs, but it would be much quicker and more accurate to state we are making a list of hardships, both tangible and intangible, for the purpose of approval in other countries. (I see this being useful for many people.) You are most welcome to address the whole aHUS community as I know of people who have different co-morbidities than my son.
  • 
    Lenwood published 6 months ago | Originally written in English

    Hi Cheryl

    Yes I appreciate that it may seem a bit hard hearted; but if we are going to confront people who need to demonstrate that they have made a rational decision; it would help them, certainly in the current economic climate, that it makes economic as well clinical sense to recommend our application..

    I would be grateful if you could lead on it within the USA community I can send you the list as it stands with your items added.

    I agree that some hardships can be found on the blogs. Jaen-Francois account on this site introduces "blue finger" and " loss of fingers and toes", which I was not aware of. Indeed aHUS ultimately caused his divorce.But it would be better if people make a contribution themselves.

    Tomorrow I am off to London for a meeting with the trustees of aHUSUK and aHUS Action.

    We are also going to Parliament to watch proceeedings, although the MP who we thought might be raising the issue of aHUS in the House of Commons may sadly not now be called on to speak this time!

    It will be Wednesday before I can send the revised list to you. Can I send it direct through the Foundation mail service?

    Len
  • 
    cherylbiermann published 6 months ago | Originally written in English

    Hi Len,

    That is fine to send it via the other site, I will look for it. Hopefully the our Thanksgiving celebration Thursday will be an easy clean up, I have my twins home to help. I wouldn't expect to see anything before Friday. It shouldn't be too hard to come up with a title that will draw attention and a call to action, so I look forward to your list.
  • 
    Lenwood published 3 months ago | Originally written in English

    Events over took this but Alexion were able to produce a societal cost submission to AGNSS as part of their case ;although the details of it have not yet been made public.

    There was however a public statement at the meeting in Portcullis House in Westminster, to raise awareness of aHUS, that clinicians in the UK have demonstrated that eculizumab has cost advantages over other forms of treatment

    The campaign continues in the UK.

    aHUSUK are to make a submission to AGNSS and a Patient Voice Group will be active in supplying the evidence needed.

    I have no concept from reading information on this site on how other european countries are progressing on making eculizumab available to aHUS patients in their respective countries .

    I will set up a new forum about the issue.