Brain tumour advocacy:What is the role?

The Role of Brain Tumor Advocacy Groups

This is an excellent paper which I came across, and I find it is very pertinent. I have highlighted the most important aspects here. The full article is behind the paywall (another blog post coming up soon for this!). However, nevertheless, despite the repetitiveness, these are the essential highlights.

Brain tumor patients, caregivers, and loved ones realized that research is underfunded, which catalyzed the creation of nonprofit patient advocacy groups to bridge the gaps that the for-profit sector and the government have not addressed.

I agree here. Rare tumours are “rarely” the focus of researchers. Sadly, I have a strong feeling that bulk of current research in these tumours is dressed up to impress the charities. Because when the push comes to the shove, we hardly have moved the needle.

The challenges that brain tumor advocacy groups and organizations face and the role that they must play are reflective of the nature of their patient population. Individual efforts to combat the deadly disease are driven by a shared sense of urgency.

It is precisely my motivation to set up exclusive Telegram group and a public broadcast channel. It helps to gather all the information in one place with vetted links so that patients or their caregivers do not have to wade through the toxic Google searches.

As recently as fall of 2013, patient advocacy organizations were defined as providing “patient- and caregiver-oriented education,advocacy, and support services”

It is difficult to “define” them (advocacy groups), Definition, by its very nature, limits its scope. I would prefer to call them formal or informal support groups but more on that later.

This role may be sufficient in disease areas where there are effective treatments, but for brain tumors, where treatment options are limited and often ineffective, the community requires proactive research programs, as well as a host of advocacy and patient support services

Exactly. The issues are stark in developed economies while in most of other health care systems where research is either fledgeling or non-existent, brain tumours are likely to be axed. Breast cancer or even head and neck cancer are public health issues, and the bulk of biological research has been directed towards it. The idea is to define “targets” and spin it off to pharmaceutical companies. It does de-risk it but with it comes the high price of failure for other disease sites. Not every research leads to a breakthrough; like for example, in the case of Temozolomide. What next? Electric fields?

The goals of public policy advocacy include improving treatment options and access for brain tumor patients, supporting research funding and innovation, and optimizing conditions for drug development and approval.

As above. It is essential to raise the voice and create awareness. When this was published, using Twitter by patient advocates was not mainstream. However, in light of recent developments, it is essential that physicians also join in patient advocacy. Here’s the shocker. Very few oncologists pursue brain tumours as a career pathway. It is either because of limited treatment options (which is a fact) or restricted interest in putting efforts for what is a uniformly fatal disease. More importantly, it is likely to lead to high burnout rates. However, most of them do agree- it is intensely academically and emotionally challenging.

Owing to the nature of the disease and its treatment(s), the burden on caregivers is often very high, both emotionally and financially. Patients and their caregivers benefit from a variety of support services.

It is true.

Brain tumor treatment is expensive. Brain surgery, radiation therapy, chemotherapy, and targeted drugs all add up to a steep medical bill

Moreover, this leads to financial toxicity. It is true for most of the healthcare systems across the world.

Beyond its steep financial cost, brain tumor treatment is incredibly complicated. Coordinating care requires managing appointments, connecting with the right physicians, and finding the appropriate care services. This burden often falls on the caregiver because of the impact that the disease and treatment have on the brain tumor patient

This is what I dread the most. It is because advocacy groups are far and few in-between. Most general practitioners have a low threshold for diagnosis (which is also okay because these are rare tumours with a low index for suspicion). By the time it is understood in its context, it is usually too late. This issue is more pronounced for childhood cancers (and that breaks me entirely).

By funding research and changing the research system, advocacy groups today strive to identify a cure and transform a deadly disease into a manageable, treatable condition.

More importantly, funding the caregiver system because that is the urgent need. Basic biological research in gliomagenesis will take a long time to mature. I propose that advocacy groups should work with policymakers to push for public funding (because of a high risk of failure/difficulty in reproducing ideal lab conditions to in-vitro conditions) and at the same time, invest in support infrastructure to provide succour to affected families.

Today, the median survival for patients with GBM is approximately 15 months, with a 5-year-survival rate of less than 5% In spite of the money poured into research and although some targets and pathways have been identified, brain tumors such as these are still poorly understood in terms of causal and maintenance mechanisms. If this is a war against brain tumors, few (if any) would say that we are winning.

This is the most realistic statement. (emphasis mine)

Is this paucity of treatment options a question of a broken brain tumor drug discovery and development pipeline?
Is this a question of the research environment generally being inadequate?
What are the real barriers to advance research to treatments?
And which stakeholders are responsible for addressing these barriers? The true role of the advocacy group is to address the issues that the government and for-profit sectors cannot, or will not.

I agree, and these are the set of questions that advocacy groups should ask. (emphasis mine)

Instead, as patient groups have become more sophisticated, there has been a shift from funding research that increases scientific knowledge of the disease to targeting funding to specific points along the drug discovery and development pipeline that will provide incentives for others (such as pharmaceutical and biotechnology companies) to invest in the field.

This is one aspect. I still hold that advocacy groups should push for basic public funded research and invest in support groups on the other. It is a collaborative effort. Individual patients don’t have a voice. However, groups can collectively raise their voice- in mainstream media, social network and align with research charities.

Finally, it gets me to define the patient advocacy groups as a bridge between the policymakers to push for funding and caregivers to provide resources or access to them.

This area has not yet been fully explored by brain tumor advocacy groups, but advances in citizen science and computational platforms have opened the door to leveraging new communities of researchers. Interdisciplinary efforts in systems and computational biology have the potential to translate “big data” into meaningful analyses that further translate into clinical impact

The big data (machine learning/artificial intelligence) is still a way far off, and despite being the catchword of press releases, it is still very much in its infancy. Indeed, I get riled up because of the hype factor built in “tailored research” (which is dressing up done to squeeze in more funding). (Personalised medicine remains an elusive goal).

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