The caregiver engine steers accelerated rare cancer trials
Written by Paul Kidwell, communications and patient advocacy consultant and co-host of the podcast,InSickness. Men and the Culture of Caregiving
As I write this column we are just a little over two months from the convening of ASCO (American Society of Clinical Oncology) 2025 in Chicago. The ASCO abstracts have yet to be announced but if we go by last year’snumbers,which featured over 7,000 abstracts and more than 5,000 presentations, this year’s numbers will likely match or surpass those levels.
What is also a safe bet is that the vast majority of the presentations will likely focus on those cancers for which there are the largest patient populations. The non-rare conditions like breast, lung, prostate and skin cancers. It just makes sense to concentrate on diagnosing and treating cancers where the research paths and medical need are the greatest.
Despite the imbalance between rare and non-rare cancers, there is much by which to be encouraged in with rare cancer drug development. My same eyes noticed a number of significant research funding initiatives with rare cancers as their target.
Seattle’sFred Hutchinson Cancer Centerreceived a $2.5 million FDA grant to establish an AI-driven drug accelerator. Cycle for Survival raised $34 million earmarked for cancer research and development.GSKannounced a collaboration with US biotech focused on GIST which affects around 5,000 US patients annually.
“Many of our scientists, especially our Damon Runyon-St. Jude paediatric cancer research fellows, have dedicated their careers to improving treatment outcomes for patients with rare cancers,” said Yung Lie, PhD, president and CEO, Damon Runyon Cancer Research Foundation. “Brain cancers like diffuse midline gliomas and ependymomas, though rare, are aggressive and often fatal, and patients are in desperate need of better therapeutic options. Among rare adult cancers, sarcomas in particular, have few targeted treatments and poor prognoses. This is why we fund physician-scientists like Benjamin Nacev, MD, PhD, at the University of Pittsburgh, who are motivated by their patients to make breakthroughs in the lab.”
As much as this column’s focus is on rare diseases, I have no qualms with the balance of research when I consider a couple who I interviewed for one of my caregiving podcasts last year. Lauren Huffmaster is a breast cancer survivor, and her husband, Clifton, is her dedicated and committed caregiver. The fact that Lauren has survived this horrible illness and is able to have family time with Clifton and their kids is due in large part to the treatments she has received. Well done, all.
Despite the uncertainty of the rare cancer landscape, one thing is clear. There will be no advancements unless the industry continues its adherence to the established “team sport” ethos of collaboration among industry scientists, patient foundations, regulators and caregivers. Caregivers are a somewhat new influence added to the team but of no less importance than the other participants. Particularly in their ability to accelerate clinical trials of much-needed therapeutics earmarked for patients diagnosed with a growing list of identified rare cancers.
Once considered the most underestimated industry wallflower, caregivers are now flexing their muscles among industry leaders, regulators, clinicians and advocacy groups as they increase their involvement across the entire rare cancer continuum. It’s a 24-7 involvement and the data we glean from our daily observations can improve everything about the clinical trial experience. From design and recruitment, to retention, adherence and the quality of data we pass along to clinicians.
Caregiver benefit becomes scientist benefit, becomes clinician benefit, becomes advocate benefit, becomes regulator benefit. Becomes patient medicines.
One of the more significant disease burdens of rare cancers is that it is a rare condition and not one with a rich history and legacy of disease awareness and understanding. In a somewhat true sense, rare cancer caregivers become walking and talking disease natural histories. This experience can serve as a catalyst for the development of future therapeutic advancements which may benefit a growing number of rare cancer patients by directly aiding the development of therapies for rare tumors. And as the advent of personalised medicine becomes more fully entrenched in treating narrower subsets of common tumors, the rare cancer caregiver experience gains a stronger foothold in its application for smaller cancer subtypes.
Rare cancer caregivers are becoming a vital clinical trial touch point for industry researchers as they work towards a successful trial and subsequent drug approval. There are many ways a caregiver makes an impact and as we learned with patient involvement, it is most beneficial when done early, often, and consistently.
“In TargetCancer Foundation’s decentralised rare cancer clinical trial TRACK, enrollment is purely patient driven and facilitated from home, using an online eConsent,” stated Jim Palma, CEO, Target Cancer Foundation. “However, it is very common that in the initial steps of enrollment, and throughout the process of participation in the trial, we are working directly with caregivers- spouses, sons and daughters, other friends and loved ones- in addition to the patient. While there are over 200 types of rare cancers, they share many common and very unique challenges, and these challenges often require a team effort to overcome them and ultimately identify and advocate for the most effective treatments possible.”
Early engagement comes when caregivers are invited to contribute as the trial is being designed and enrolled. Family caregivers especially serve as health proxies for loved ones and within that role are often linked to patient foundations with whom they can collaborate in identifying and recruiting patients to trials. Their willingness to offer their insight personal issues like travel (including parking), meals and impact on work can be immeasurable. Time off work for the caregiver—whether in the midst of supporting a trial or just general health proxy activities—is perhaps the one issue on which caregivers are most vocal. The care we provide is uncompensated and can impact a family financially. For someone like myself who is an independent professional, caregiving means I am unable to work and cannot charge back for my time. Salaried employees walk the fine line of corporate empathy if they have to miss time from work on a regular basis. A trial that requires clinical visits over a period of several months will always be filtered through this important lens.
Of course, the overarching goal of any clinical study is to gather patient data that will influence the life and use of a drug candidate. This is where a caregiver has the most value. From proxy assistance in determining eligibility and completing the necessary yards of paperwork, to the all-important anecdotal observations of a medicine’s side effects, caregivers are in the best position to collect this data. Between clinic visits, caregivers are no less important as they might be asked to chronicle daily activities including drug adherence, medicine side effects, physical activities like sleep, exercise or diet, or any cognitive decline. This is often done through the growing number of digital platforms and tools clinicians are employing to connect with patients.
If industry researchers aren’t doing this already, they need to give serious thought to caregiver involvement at the first inkling of a clinical study to help validate drug candidates. As I said before, early and often. And with the proper tools. Caregivers play an important role in gathering information, especially in certain rare diseases where physical and cognitive capability is limited. We are no longer an afterthought in the patient journey and have become valued collaborators in accelerating rare disease trials.
We are delighted to welcome Paul Kidwell as a regular contributor and supporter of the caregiver voice throughout 2025. For more information on Paul’s podcast check out insickness.org
