Our letter (written by Trevor Grant and Lou Williams) to Pharmaceutical Benefits Advisory Committee and also the response received. It is thanks to everyone in unity sharing this petition that we are so close to fast tracking Keytruda on to the PBS for Mesothelioma and other rare and less known cancers. Dear Professor Wilson Ref: Keytruda (Pembrolizumab) fast tracked PBS for Mesothelioma We are writing to ask for your help in getting the immunotherapy drug, Keytruda, fast-tracked to the Pharmaceutical Benefits Scheme (PBS) for the lethal asbestos cancer, Mesothelioma. We are two people among the thousands in Australia suffering from Mesothelioma, a disease inflicted upon us by corporate greed and government neglect. As the body responsible for assessing drugs for the PBS, the Pharmaceutical Benefits Advisory Committee (PBAC) is a key player in prolonging the lives of so many of us. In recent correspondence with the Federal Health Minister, Ms Sussan Ley, which we enclose, she indicated the first step was for Keytruda developer, Merck Sharp Dohme (MSD), to submit an application to the Therapeutic Goods Administration (TGA), which hadn’t been done so far. MSD, though, in a paper dated September, 2015, calls for “a new approach to listing promising therapies for Rare and Less Common (RLC) cancers”, such as mesothelioma. It points out that more than 42,000 Australians are diagnosed with an RLC cancer in a typical year, and half will die. This represents half the cancer deaths in Australia per annum, yet less than 15% of the PBS spend, and less than 20% of cancer research spending, is allocated to RLC cancers. As the paper states: “This lack of resource and funding for RLC cancers results in an inequity for 42,000 Australians each year who are unlucky enough to be diagnosed with a cancer that falls into this category rather than the more common cancers like breast and prostate cancers.” The MSD paper says that the current approval process is “not fit for purpose for RLC cancers and long delays are evident in recent PBAC evaluations for RLC cancer treatment.” It says that regulatory agencies unfairly apply similar standards to RLC cancers as common cancers. It means “there is limited opportunity to collect and present the data that would be required to address the uncertainty within the current PBAC HTA framework. It goes on to say “the limited evidence base for RLC cancers means that submissions are often rejected or held up in protracted negotiations.” One given example is sunitinib for pancreatic neuroendocrine cancer, which took “over three years and four PBAC submissions to get approved for public funding despite PBAC acknowledging the high clinical need for this medication and lack of alternatives at the start of the process….” Yet, as the paper points out, the TGA and PBAC processes have, on occasions, recently shown “increasing flexibility.” This is evident in the recent PBS listing of Keytruda for melanoma cancer, which “demonstrated that there is capacity within the registration and reimbursement systems for accelerated access where a treatment may have potential.” Keytruda, it says, received regulatory approval in nine months, based on early data, despite there being no formal TGA fast-track process. The PBAC “considered a managed access scheme for Keytruda in melanoma which addresses the uncertainty associated with the immaturity of the data submitted, allowing a positive recommendation on the first submission.” The obvious question is why can’t this be done for other cancers, such as mesothelioma? We also note with interest a recent story (13/5/2016) in the Melbourne Herald Sun which states that there are 250 Keytruda trials going on around the world. How many more are needed before the acceptance of the obvious evidence that Keytruda is a very effective treatment for Mesothelioma and other cancers? The same article quoted the researcher responsible for developing MSD’s PD-L1 bio-marker test, which is behind the success of Keytruda. He said MSD was developing this in 30 different tumour types – presumably one is Mesothelioma - and urged the TGA to change its way of approving cancer drugs so they can be fast-tracked. Would you support this? Both of us – Lou and Trevor – have been lucky enough to be able to afford access to Keytruda, at between $5000 and $7000 every three weeks. It has transformed us from pain-wracked victims waiting in line to die to people with a bright new outlook on life. We also endure the immense frustration of often sitting next to melanoma patients in oncology wards who pay as little as $6 for exactly the same treatment. Is that fair? Lou’s case is well-documented. She was diagnosed in 2003 with peritoneal Mesothelioma and pleural Mesothelioma in 2009. She went from death’s door after years of fighting the disease to someone who saw the disappearance of the crippling pain that is part of Mesothelioma. Soon after starting Keytruda treatment in April 2015, she was doing things that were simply impossible for her, including long walks without her usual oxygen aid, gardening, interstate travel and, generally, an excellent quality of life with no pain. Lou is back involved in global asbestos advocacy, education and support for thousands of people living with Mesothelioma including supporting daily those who contact her asking about Keytruda, wanting information for them or family members and desperate for Keytruda to be put on the PBS so that everyone can have this option rather than the current protocol treatments that have little effect on Mesothelioma. Trevor, too, has experienced a remarkable transformation. Last year the disease had transformed him from a fit healthy 63-year-old who ran 5km three times a week to someone unable to walk more than 100 metres without severe pain. Now, his tumours have shrunk considerably, he’s off all pain medication, walks the dog for an hour each day, and has even been able to return occasionally to his favorite sport of golf. All because of Keytruda. We know of many similar stories. One of the most important benefits of Keytruda is that it allows you to live a decent life while being treated, unlike chemotherapy, which has severe side effects in most people and condemns you to a miserable life of mostly home confinement in dressing gown and pyjamas. Of course, as outlined here, the cost is prohibitive for so many people. We sit next to these people in oncology waiting rooms, and watch them deteriorate because they can’t afford the $85,000 -- $120,000 per year it costs for mesothelioma patients to access the lifeline drug Keytruda. All we ask is for an old-fashioned Australian “fair go” and we believe PBAC is vital to helping deliver it. We look forward to your response. Yours faithfully Lou Williams Email: eradicateasbestos@gmail.com Social Media Voice, ADFA (Asbestos Diseases Foundation Australia) Australian National Director (GBAN) Global Ban Asbestos Network Our response received from PBAC. Lou Thanks for your email. I am very conscious of the matters you raise here. I do not agree with the statement quoted from the MSD paper which infers that Rare and Less Common Cancers are specifically disadvantaged in the PBAC assessment process. This is not the case, indeed we recognise that for rare diseases that we need to be flexible in our expectations of the trial evidence. As you correctly identify, companies can and do make applications to both TGA and PBAC based on early results when these are promising and based on need we have recommended special arrangements with companies. Indeed this was the basis of the listing for pembrolizamab for advanced melanoma. I have personally encouraged both of the companies marketing PD-L1 inhibitors in Australia to bring forward as soon as possible an application for mesothelioma in recognition of the need. It is worth noting that pharmaceutical companies make decisions about their priorities for when and where they trial new cancer treatments based on a range of factors not just the feasibility of achieving the requisite number of patients to conduct a statistically valid trial. As you are very aware, because of the history of asbestos exposure in Australia we have more than 600 cases a year. Looking at the the US clinical trials registry trials being conducted for mesothelioma for both pembrolizumab and one of its competitors nivolumab, the size of these studies could be achieved in Australia particularly given the well organised Australian community of concern around asbestos related disease. I hope we can work together to encourage them to do so in future. I would be happy to discuss further with you. Aw Professor Andrew Wilson Chair Pharmaceutical Benefits Advisory Committee Department of Health