Research

Participating in research

Lauren, United States of America

“Will this (research) help?”

Sandra Bartolomeu Pires, Nurse Researcher

A few months ago, I was trying to find people interested in participating in my research project called Integrate-HD, and a member of the Huntington’s Disease community asked me “Will this help?”. Now, my research does not involve testing a new drug, it does not involve testing novel equipment, heck, I am not testing absolutely anything! Will my research help?!

You see, the problem I am trying to solve is the lack of care access and the lack of care coordination that people with Huntington’s Disease experience. I do not have to explain the reality of these problems to you, as you will know them better than me. I am not personally affected by HD, in fact, I only had my first patient with HD 10 years ago. But HD has been glued to me since then, and, for varied reasons, I always felt HD was treated like the underdog. My mum always told me I always pick the hardest path, and so here you find me, researching how to improve care with the HD community.

I started by looking at activities that had been tested that could improve care coordination in HD, but unfortunately there was none to be found. And so, I started designing those activities with the HD community, through research. First, I wanted to know how bad the situation was were I lived, in England (pretty bad), then I asked people what good care looked like through individual interviews and group discussions (results undergoing publication). And this short paragraph has taken 5 years of my life, even without any testing…

Now, for me, the testing is the next phase. I daydream that I can develop an effective intervention that can support people better and at no added cost, perhaps even cheaper to the government in the long run! If I manage to convince the people who take the decisions to reorganize care, perhaps, in time, my research will help people. You may have heard this, but research takes approximately 17 years from theory to practice. Good thing I am only 37 and I am prone to the hardest paths.

I have found people very disheartened with the system, exhausted by all the obstacles of life, too sick to take part in any research, or just wanting a break from HD related stuff. Participating in research can be very demanding. But, if you find the will, the time, the energy, join research: as a participant, as an advocate contributor, or by sharing a post you found interesting. I remain hopeful that together with the community, we can make a difference. Regardless of the time it takes us, we will keep pushing for better care and better treatment.

“The best time to start anything was yesterday. The 2nd best time to do it, is always today”.

You can find research opportunities, for testing interventions and/or for building knowledge, on many websites, for example here. You can also contact your local HD association and The International Huntington Association.

Blog written by Sandra Bartolomeu Pires, expanding on her Instagram post @integrate_hd dated 22nd October 2024. Sandra is a nurse researcher passionate in improving care for people living with Huntington’s Disease. She is writing her doctoral thesis and is not currently recruiting to any study.

Need me? Find me on socials:

Instagram @integrate_hd

LinkedIn https://uk.linkedin.com/in/sandra-bartolomeu-pires-841594190

Twitter @BartolomeuPires

Email smbp1u20@soton.ac.uk

Understanding the value of partnership in drug development

Seth Rotberg, Patient Advocacy at Prilenia

Have you ever wondered, what is the true value of a partnership among industry, patient groups, and the patient community within drug development? As someone who has been involved in each area at different timepoints of my life, I can tell you firsthand that – it’s a very big deal. Patient groups and the community must be included throughout all stages of drug development if we truly want to advance clinical research.

My own patient advocacy journey started at the age of 15 when I first learned about my mom’s diagnosis with Huntington’s Disease (HD) after being misdiagnosed for the first 5-7 years of her journey. Doctors originally diagnosed her with major depression and mood swings, while neighbors thought she was drinking during the day due to her poor balance and wobble movements. All of these signs ended up being symptoms of HD.

After taking a few years to process this new normal, I decided to get involved with the community by hosting fundraisers and raising awareness about HD. Eventually I took on different leadership roles with patient groups to help support the unmet needs of the HD community. At one point I even founded a patient organization devoted to supporting young adults with rare and chronic diseases.

Fast forward to today where I have the privilege to oversee patient advocacy and engagement efforts for a small biotech company working in HD. Here is what I have learned:

1. Patient groups and patient community members play a crucial role in drug development. They are the ones with lived experiences and understand the disease in a way not found in textbooks or in a laboratory.

2. By sharing about their lived experiences with biotechnology and pharmaceutical companies, patients can help shape the research taking place, including what diseases get attention, which endpoints are studied in trials, and what does success look like from a patient perspective. Feedback can happen through guest speaking opportunities to employees of a company, clinical trial design, educational materials, how to get the word out about clinical trial participation, among many other opportunities!

3. Patient input matters in every stage of drug development from discovery to post approval: helping researchers understand the disease, contributing to the design and recruitment strategies for clinical trials, advocating for access to therapies if approved, and sharing experiences with approved therapies.

For a partnership to be successful, it is up to the patient group and community members to get involved by asking questions or raising their hand to help push research forward. Challenge companies on what they know about diseases and the status of their work. It certainly takes a village to drive innovation, especially in rare diseases. Even more for diseases for which no treatments exists. For those of you who are already involved in some of these opportunities- thank you for continuing to advocate to help make a difference in drug development!

Seth Rotberg is a nationally recognized patient leader, healthcare industry professional, and TEDx speaker. After his mother’s 17-year battle with Huntington’s Disease (HD), Seth made it his mission to improve the patient experience in healthcare. He currently oversees patient advocacy and engagement efforts at Prilenia and the views expressed in this article are those of a Prilenia employee.

Please note this guest blog post is sponsored by Prilenia.