Paediatric Oncology: More Investment Needed

Oncology is a huge area of focus for pharmaceutical companies today, but Nicole Scobie of Childhood Cancer International warns that many of the new therapies being brought forward are not addressing the needs of paediatric patients.

“Pharmaceutical companies are very slow in developing new drugs for childhood cancers. Solutions to this issue include the European Medicines Agency (EMA)’s Paediatric Regulation, which ensures that whenever a pharmaceutical company wants to put a new drug for adults onto the market, it must also develop and test it on children.

“Without this legislation, many pharmaceutical companies would not test their drugs on children, as it is very expensive to do so. While the Paediatric Regulation has been of benefit for many childhood diseases, it has failed in cancer, with pharmaceutical companies allowed to apply for a waiver when a disease is not considered the same in children as in adults.

 

While the [EMA’s] Paediatric Regulation has been of benefit for many childhood diseases, it has failed in cancer, with pharmaceutical companies allowed to apply for a waiver when a disease is not considered the same in children as in adults

 

“For example, a waiver from having to test an Alzheimer’s drug in children makes sense. However, this is not the case in cancer. For example, a pharmaceutical company developing an ALK inhibitor – a target present in adult lung cancer but also in neuroblastoma and other cancers in children – was able to successfully apply for a waiver and not develop the drug for children, even though it has been shown to work in children by academic researchers.

“Pharmaceutical companies’ lack of engagement is a big issue, although it is understandable given the high costs of developing new drugs for cancer in children, as well as the rarity of indications like neuroblastoma.

“However, we at CCI feel that children should be a priority and have lobbied pharma companies to develop their drugs in children, even though they probably could have received a waiver from the EMA. We have also been working with the EMA so that they do not accept waivers for drugs when they can see that there is a target in children.”

Read the full interview here

 

CAR-T: Unpacking the Hype

CAR-T therapies were first approved in 2018 amid a surge of publicity. Lorna Warwick of the Lymphoma Coalition outlines the work her organisation undertook to prepare (and temper the expectations of) the lymphoma community around this ground-breaking new treatment.

“CAR-T, which was touted as a game-changing curative treatment, has been the answer for some patients, but not all. We still see a significant number of patients that are either not responsive or relapsing, usually within the two-year mark. The question is therefore how we do a better job of figuring out which treatment is best for which patient and what order we should be progressing through these treatments to take the best advantage of what is currently available. There is work to be done around how the state of a patient’s T cells impacts their response to a CAR-T therapy and whether we should be harvesting T cells earlier in the treatment paradigm in case of a relapse later. I know there are clinical trials investigating these things, but we are learning a lot from the real world.

“There are many relapsed/refractory patients wondering whether there are any treatments left for them; we need to do more research to figure out how to cure them.

 

The question is  how we do a better job of figuring out which treatment is best for which patient and what order we should be progressing through these treatments to take the best advantage of what is currently available

 

“In higher-income countries where CAR-T was a real possibility, there were more patient requests for knowledge. Back in 2018, we began to create patient materials, explaining the basic science behind CAR-T, the clinical trial experience for the Novartis, Kite, and Celgene/BMS products, as well as the differences in their reporting. This helped give patients a realistic expectation of how many trial participants got a positive response, educated them that not everyone that has their cells collected in a trial then has their manufactured cells reinfused and that CAR-T is in effect a manufacturing process within which errors sometimes occur.

“In terms of side effects, the intensity of CRS that patients with CAR-T experienced and neurotoxicity, especially when symptoms can be so subtle, was something new. Lymphoma patients have always had a heavy reliance on their caregivers, but CAR-T patients need their caregivers to be especially alert and recognise signs of neurotoxicity so they can be treated properly. If a CAR-T patient is taken to a regular hospital that has no concept of CAR-T, it may look as if they are having a stroke, the treatments for which are very different. This placed a new level of stress on caregivers.

“In countries where CAR-T was not on the agenda for the immediate future, patient groups and physician communities were reluctant to distribute information about a therapy that local patients were unable to access. However, anyone with an internet connection can access information about CAR-T; the challenge is ensuring that this information is good and credible. There has been a lot of misinformation. There have been instances where patients have been able to travel across the world and pay to obtain CAR-T therapy, whereas for many others that is far beyond their means.”

Read the full interview here

 

HTA & The Patient Perspective

Zack Pemberton-Whiteley, CEO of UK-based blood cancer charity Leukaemia Care, highlights the work still to be done to better incorporate patient perspectives into the health technology assessment (HTA) process for new medicines.

“I think the biggest question is, does the process take into account patients’ views? It is very easy to talk about involvement but sitting at the table is not equivalent to full patient involvement. At the end of the day, these organizations exist to make sure that the most effective and, particularly in the context of an organisation like NICE, the most cost-effective treatments are approved for the patients who need them.

“Organizations like ours exist for patients. It is impossible to determine cost-effectiveness and what value means without looking at what matters to patients. If quality of life measurements, for example, do not consider what matters to patients, then they are not fit for purpose. Many of the quality-of-life tools still being used today were predominantly developed in the 1980s and are now being used to assess treatments that are completely different. This is particularly relevant for cell and gene therapies.

 

One of the big limitations today is the lack of effective methodologies for using evidence of patient perspective, whether patient surveys or patient preference studies, to inform decision making

 

“We are really calling for patients to be put right at the heart of that decision making, and make sure that all methods used factor in what matters to patients. One of the big limitations today is the lack of effective methodologies for using evidence of patient perspective, whether patient surveys or patient preference studies, to inform decision making. This is not to say that the people involved in HTA are not trying to evolve, but there needs to be more speed.

“I think it would be unfair to put all of this on HTA. All stakeholders have a huge role to play in this, and if we are talking purely in the context of access, one important consideration is the evidence that is put in front of HTA bodies. The predominant stakeholder responsible for producing that information is the pharmaceutical industry, or individual pharmaceutical companies, in a single appraisal. The industry has come a long way recently in terms of trying to be more patient centric, but we are still a long way from where we want to go. It is very easy to talk about patient centricity without really walking the talk. While we are seeing lots of interesting initiatives and pilots on incorporating the patient perspective into drug development, I am yet to see any companies doing it well systematically.

“The healthcare professional perspective has been incorporated for many years; we need to think about incorporating the patient perspective in a similar manner at each stage, genuinely listening to and acting on the feedback we are receiving from individual patients and from the patient community as a whole. There is a clear tension between what regulators are asking for and what patients are asking for, and fundamentally, we are doing this for patients. Therefore, if the regulators are asking for something different from the patients, then the industry but also the regulators need to think differently about what they are asking for and what it is that truly matters to patients with that particular indication.”

Read the full interview here

 

Early Patient Engagement: Beneficial for All

Claire Saxton and Lauren Kriegel of Cancer Support Community, the largest professionally led non-profit network of cancer support worldwide, pick out the changes they would like to see in the way in which pharma companies engage with patient advocacy groups and how early patient engagement in the drug development process holds benefits for all stakeholders.

Lauren Kriegel: “Given my background as a social worker, I would love to see pharma companies opening up funds for assistance with things related to the treatment. In my experience, pharma companies have patient assistance programs that usually help solely with the cost of the medication. However, sometimes they need lodging, transportation, and help with other items.”

 

If companies bring in the patient perspective from the beginning, the insights they gain could make their clinical trial process go quicker and create a better understanding of how patients make decisions between different treatment options

 

Claire Saxton: “I have worked with some pharma companies who are ahead of the game and really involve the patient point of view from the beginning of their projects. However, there are also companies that do not think about that until they are about to go to market or launch an awareness campaign. Additionally, many start-up companies do not begin to think about patient-centricity until they are acquired by a larger firm with the infrastructure and resources to invest in it.

“If companies bring in the patient perspective from the beginning, the insights they gain could make their clinical trial process go quicker and create a better understanding of how patients make decisions between different treatment options. Having patient advocacy groups and patients and caregivers themselves represented on advisory boards can help ensure that the therapy is designed for the whole patient and not just for the cancer cell.”

Read the full interview here