(At the outset of this blog I want to make something crystal clear, this blog is not to be misinterpreted as a criticism of fellow patient advocates, we are all just trying to survive in the system that so often fails us)

Sometimes in life, you need to know when to talk up, sometimes you need to know when to shut up. Becoming a stage 4 patient advocate has been a rollercoaster lesson in knowing the difference. I don’t claim to have the authority on either scenario at present so I’m sure I will probably divide opinion on whether I’m right to talk up now. 

We have all followed the Cervical screening check story unfold in the media over the course of this year. It has to be said it’s been horrendous, as a stage 4 melanoma patient I watched in tears on many occasions. Getting any advanced diagnosis of any cancer is, without doubt, the most challenging life-changing experience anyone can have the misfortune to encounter. “Devastating” is a word often used to describe it, but even that doesn’t do it justice. 

With almost four years I have tried my best to advocate for Melanoma patients but also for improvements for all cancer patients, one of the biggest barriers we face is without doubt access to innovative treatments.  I also acknowledge the huge advances being made in cancer outcomes, but making headline-grabbing scientific advances and actually getting them in a clinic to a patient are often separated by a treatment access track that makes the grand national jumps look like pre-school sports day assault course. 

For those that don’t understand the process I will attempt to give a brief outline; To treat cancer you need to identify a target, it’s not simplistic to find these targets or weaknesses in the tumour armoury, this is one of the reasons that cancer remains so difficult to treat, but when researchers do find a “target” it starts a long process of trial and error to then find a compound that can attack that weakness and in turn kill off a tumour. This can take potentially years, firstly it has to be proven effective in repetitive lab testing, (often latched on to at this stage by the media and hyped up as major breakthrough never to be heard of again i.e effective only in mice).

Once sufficient evidence of efficacy is seen in the lab it moves on to phase 1 clinical trial, this stage will determine if it is safe to use in humans, if it is safe it will move to phase 2 trial which will look at how it reacts to cancer inside the human body this could turn out to be successful or as is often the case disappointing because killing cancer cells in a petri dish or a mouse is very different to achieving a response within a living breathing human.  At this phase, there may also be an adjustment to the dosage required for an effect. Sometimes there have been stunning results at phase 2 trials and the drug gets fast-tracked on to Phase 3. These Phase 3 trials are run in multiple locations throughout USA, Europe and globally. Ireland generally opens phase 3 trials, although we do a lot of basic lab research like I mentioned happens before Phase 1 we don’t to my knowledge run phase 1 or 2 cancer trials. 

So what happens once the big Phase 3 trial is underway, patients get recruited and get an opportunity to potentially try a treatment they wouldn’t otherwise get access to. It may turn out to be better than what’s already offered as the best standard of care, or it may turn out even at this late stage to be unsuccessful. That is the nature of research trials, now of course as these trials go on for a number of years, we can begin to get a clearer picture of the efficacy of the treatment. But what if you’re a patient who happens to get diagnosed after one of these trials has stopped recruiting in your country? what if it’s already clear that this new treatment is better than the only existing treatment available at the moment in your country? What if your life depends on having a chance to try this treatment and you try to enter the Cancer grand national race when the gates closed and everyone else is almost at the finish? 

If the trial has finished recruiting, and the interim analysis is favourable then your treatment lifeline may already have gained approval from the USA FDA (Food and Drug Administration) This approval can mean the drug is now on track to global reimbursement but hold your horses, that doesn’t mean Mary or Sean attending hospital in Cork or Dublin can have it just yet.  This is the Cancer Grand National and we are only on the first lap, tragically we have already lost some beloved companions because the track is too long and the participants are at a disadvantage from the beginning.   

What are these disadvantages you might ask, well there was the possibility to take part in the trial, but you missed the recruitment cut off potentially because it took so long for you to get referred for that MRI scan or CT your GP thought you needed months and months ago, if you go back even further you might have decided not to attend the doctors at all for that checkup because the thought of entering the HSE hospital system with all the bad news stories frighten you more than that pain your feeling inside or that little black mole that’s started to bleed. As a result of disadvantage, you will potentially end up in A & E with a much worse prognosis than if the system really supported you. Alternatively, maybe you did go early and now you find out the all clear you got was actually a mistake by an overworked under experienced locum. Maybe you did everything we are told to do but it just wasn’t reciprocated by the system. Now at least you are in the system and at the start of the Cancer race. 

What then? well, the trials out and the existing treatment is at best palliative you feel like your being dragged along the ground by your horse through all the jumps each one harder than the one before. You are being torn asunder mentally and physically. Desperately trying to cling on to that hope of pulling yourself back up into the saddle so you at least have a shot of surviving the next hurdle the next short time frame you can allow yourself to think of. The good news appears on the horizon the EMA (European Medicines Agency) has now decided to approve the treatment you want. Whoop Whoop I hear you shout it got a licence to be sold in Europe at last. It’s tantalisingly within your grasp, you can see your fellow patients from around Europe getting the treatment already, within a couple of months of the licence approval.  Some are fortunate and make it back up into the saddle, some not so lucky finish here because the treatment does not work for everyone. But you just want that chance like the patients from the Netherlands, Belgium, Sweden, UK after all we are all European, we are all the same, we all want to survive.  Don’t we all deserve a chance in this race? Arent the patient needs supposed to be at the core of our health system? 

We can see there is a process to be followed it’s been put in place to protect patients from unproven treatments that would do them harm. We have seen in the past when there was little or no regulation the damage that can happen by not following a process when it comes to the safety and regulation of medicinal treatments. If you can’t get on the Phase 3 trial maybe you can get the drug on compassionate grounds from the pharmaceutical company. It’s just that doesn’t always happen, morally you would think they should be obliged to provide it and often they do, but this is a business like it or not, and business has to answer to its investors and pay its staff. If you give your product away for free indefinitely why would anyone invest in it so you could develop new products it’s a catch 22.  

Ok so now we are really stuck; no trial, no compassionate use program, our friends in Europe are getting treated and we are left here in what can only be described as a catatonic state of despair with the only option of a treatment being offered having little chance of improving our chance of survival. Ask yourself what would you do? Sell your house, move abroad,  crowdfund, chance your life on a quack treatment, that will potentially do you even more harm. 

In Ireland patient advocates are encouraged to get involved, they want to hear our voices, where can the system be improved, how do we work together contribute? One of the ways in recent years is giving our voice to the treatment reimbursement process, letting the NCPE/HSE know why we the patients think a new drug should be reimbursed. How will it compare in terms of quality of life? sometimes its very easy to answer that one if you didn’t have it quite simply you would be dead that’s a direct improvement on quality of life right there!! 

Until today I felt we had made some progress in terms of patient engagement, there was a purpose to it until the government decided again to overturn the process we have been asked to engage in. This process of trials to gather evidence, safety assessment for licensing by the EMA, patients engaging in the established HTA reimbursement process was thrown out the window with the approval of Pembro without an EMA licence for patients with advanced Cervical cancer. Let’s be straight here before I get lynched I have no begrudgery towards these ladies receiving treatment. I wish them the very best of luck in their treatment. 

 BUT and it’s a big one, where is the fairness and transparency for all the other patients waiting sometimes up to two years in this country after the issue of an EMA licence for the reimbursement of treatment? Do we now all assemble on the streets outside Dail Eireann and demand that the NCPE (National Centre for Pharmacoeconomics) be disbanded in light of this approval, after all what need is there for such an assessment process if a TD, Advocates and media backers can get the NCPE/HSE and the Minister to overturn an entire process that all the rest of us are just expected to sit quietly and adhere to as if this isn’t relevant to our lives our survival too?

How would you feel if you were waiting for the approval of a potentially life-saving treatment, told it was too expensive, told to wait, but you don’t have time to wait,  there’s a high possibility you will die,  suddenly though money can be found to pay for a drug that doesn’t even have a market licence for cancer the HSE agreed to fund it for? Would you think there’s fairness and transparency in our reimbursement system?  Would you instead believe that Ireland continues to be not about having a fair and equal society for all its citizens, but just for the ones the government favours or the media has taken to heart?  For some, it seems the race is over before we ever even get an opportunity to participate.  

For more information on this subject pls read this link to a recent report from the Oireachtas health committee: 

https://www.irishtimes.com/news/health/high-prices-causing-delays-in-approving-breakthrough-drugs-says-hse-1.3706118

https://www.oireachtas.ie/en/debates/debate/joint_committee_on_health/2018-11-21/3/

Kay Curtin is a stage 4 patient advocate for Melanoma Support Ireland, she is PPI committee member at IACR (Irish Association Cancer Research) she is patient consultant to the Cancer Trials Ireland Melanoma Trials Group, She is a member of MPNE (Melanoma Patient Network Europe) She has been a patient adviser to the EMA scientific advisory group. She has completed the Irish Patient Education training program with IPPOSI, and made two submissions to the NCPE for the reimbursement of new Melanoma treatments. She has attended ASCO and presented at ESMO. She participates in numerous committees and remains committed to the advancement an support of scientific research to better outcomes of for cancer patients. At present, she is studying Youth and Community work at UCC  because she hasn’t lost hope in a fairer, equal society for all Irish residents.