By Blair VanBrunt, Director of Community Development - firstname.lastname@example.org
As a mom of a daughter with a rare disease, Shwachman Diamond Syndrome, I wear or have worn many hats in this rare disease space. I am a mom, guardian, advocate, past president of the non-profit SDS Foundation with all that entailed, and now a member of a for-profit company, TREND Community, that helps individuals and caregivers with many different rare diseases bridge the gap between advocacy and industry.
It really struck me while at the past Global Genes Patient Advocacy Summit just how many hats I and so many others wear in the rare disease space and how so many of us are bridging the gap between non-profit and for-profit; advocacy for their child and advocacy for the disease at large.
We met people who currently have or lost a child with rare disease who are now working for the pharmaceutical industry who are trying to make a difference within the drug development process. We met people who started a foundation because of their loved one with a rare disease or people who have close friends who have a child with a rare disease and want to participate by joining and/or fundraising for an organization.
You wouldn’t expect to meet so many people who radiate positivity, joy and generosity at a rare disease conference where so many families need treatments and support. But go to the Global Genes Patient Advocacy Summit and you will meet many who exude all of these qualities who are buoyed by hope, who wear many hats and strive to bridge the gap.
How Doctors’ Language in Medical Records Impacts Patient Care….. and Rare Disease Drug Development
By Blair Van Brunt
Patient advocate, Casey Quinlan, posted an article a couple of weeks ago on LinkedIn and I can’t stop thinking about it. The author of the article, Emily Bradley, wrote a blog piece about the clinical language that is used by doctors in recording patient visits that can negatively affect the direction of a patient’s care and medical community in general. The article is poignant and insightful with a dose of solution.
Emily Bradley noted a number of incidents in which her disease’s impact on her life, as well as on her financial situation and her job, forced her to miss doctor’s appointments or forego expensive treatment options. Despite her best efforts to communicate this to her doctors, when she looked at her medical records, the words she read made her feel unseen and unheard.
Can we change this practice?
“Perhaps instead of “patient failed X treatment” we can begin with “X treatment failed for patient” or incorporate more phrases such as “patient elected to…” and “patient shared X regarding medication XYZ.”
I relate to this from a rare disease mom point of view, from my job point of view - at TREND Community, trying to help patients and caregivers with their daily struggles while providing crucial patient experience data to the companies that can get them the treatments they need -and, of course, from the point of view as a patient myself.
In fact, could this language barrier also be preventing patients from gaining access into clinical trials? Is this kind of language not only hindering individual patient care, but perhaps the drug development process at large? And in the rare disease world, with so many of the diseases classified as life threatening, this is a very serious problem.
This is a heads up to all of us to:
By Sarah Inoue
Growing up, we never celebrated Mother's Day because my mother's birthday was on or near the day and because, as a family, we felt an extra day was unnecessary. I have always had mixed feelings about the holiday. On the one hand, taking time to thank someone and express one's love is always a good idea, and some people do need a reminder to do so. On the other hand, the holiday can be so difficult for non-traditional families, people without mothers, people with abusive mothers, the list of problems can be long. Once, a college friend of mine and I went out for brunch on Father's Day and the waitress asked why we didn't bring our dads - well, because her father had died ten years ago. Holidays can often seem as if they are designed to remind us that our lives are imperfect, not normal, and lacking.
I don't mean to diminish the experience of people who have wonderful Mother's Day celebrations full of wonderful presents and crafts from the kids and beautiful expressions of love. I'm so happy for you, truly. Enjoy the moment and the day fully and without guilt.
I am also, as a feminist mother, aware all the time of the amount of work mothers in the USA have to do - the Mental Load - without either strong infrastructure and society support and sometimes without spousal support. I'm constantly trying to un-gender parenting. The person who started Mother's Day, Anna Jarvis, according to Wikipedia, "envisioned a day venerating the daily services and sacrifices of mothers within the homeervices and sacrifices of mothers within the home" (https://en.wikipedia.org/wiki/Mother%27s_Day_(United_States)).
In my work with the rare community, I notice that mothers often bear the brunt of the work taking care of the child with a disease and this can overtake their other work and change the course of their lives and their careers. In some ways, this can be a beautiful undertaking, where mothers help the whole community and our society deal with these illnesses, but in other ways, it is may be just another way that our society depends on unpaid work to solve problems no one wants to look at.
In 2017, on Mother's Day, Rare Disease Report recognized the work of some of these mothers. I hope these stories make all of us feel better about ourselves and our lives and what we can accomplish. I applaud these women and everyone dealing with rare disease.
As part of the implementation of PDUFA VI and 21st Century Cures, the FDA is continuing to develop guidance to enable more widespread usage of patient experience data to inform regulatory decision-making and is trying to best address the questions of defining it and what methods to collect it that will be most acceptable for benefit-risk assessments.
What is Patient Experience Data?
Data that are collected by any persons and are intended to provide information about patients’ experiences with a disease or condition. It includes the experiences, perspectives, needs and priorities of patients related to (but not limited to):
– Symptoms of their condition and its natural history
– Impact of the conditions on their functioning and quality of life
– Experience with treatments
– Input on which outcomes are important to them
– Patient preferences for outcomes and treatments
– Relative importance of any issue as defined by patients
(Source: Title III, Section 3002(c) of the 21st Century Cures Act)
Who can collect this data?
Any persons including patients, family members and caregivers of patients, patient advocacy organizations, disease research foundations, researchers, and drug manufacturers
How to Collect This data?
Gathering patient experience data is about understanding a human perspective, and therefore uses data collection techniques from the social sciences, not the bio-chemical and medical techniques that pharmaceutical researchers have been using. Thus, the emerging guidance discusses ways to do good research in the social sciences. The main methods discussed are surveys/questionnaires, interviews and focus groups. These are good methods for eliciting people’s opinions and experiences, although there are others such as observation or analysis of conversations on social media.
TREND Community uses natural language processing and the machine learning techniques that accompany it on social media conversations to effectively and efficiently gather patient perspective and experience data to listen to what patients and caregivers are saying in their everyday lives. This novel technique has captured the interest of the FDA. We have had and are continuing to have conversations with the FDA regularly to hopefully clear the way for social media analysis techniques to be acceptable to help support better data collection and get the true patient voice heard.
Slides from FDA Workshop on Draft Guidance for Patient Experience data
Statement from FDA Commissioner 3/30/18
Patient-Focused Drug Development: Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data; Public Workshop; Request for Comments
A call to the rare disease community: Don't stop sharing your experiences! Reconsider WHERE you share them.
Maria Picone, Founder/CEO
In light of recent reports concerning privacy violations of millions of users’ Facebook data, patients and caregivers are telling us that they are nervous to discuss private matters on social media. As the mother of a child with a rare disease, I fully understand your concerns. However, we implore you: Do not stop! Reconsider where you are posting and find private spaces for private matters.
Why? When patients and caregivers share their experiences about their disease, its progression, possible treatments and under-recognized symptoms amongst themselves they are creating rich data that companies like TREND Community can analyze to accelerate the discovery of promising treatments and bring them to market faster.
How does TREND protect my privacy?
How does TREND Community secure my data?
TREND Community secures your data with state-of-the-art, private cloud services. Our security practices comply with all current HIPAA guidelines. Our internet security measures are continually updated and monitored in order to keep private information protected and safe. We use a world-class cloud-computing provider which has signed a HIPAA compliance agreement and earned SAS Type II certification. We take several technical and procedural measures to secure electronic personal health information (ePHI) transacted through the TREND Community platform.
Why should my community move its discussions to TREND?
How can my conversations help research?
Using computer algorithms, TREND performs an analysis of patient and caregiver discussions on social media to gain valuable insights into community perspectives on living with rare disease and a better understanding of the natural history of the disease. Our technology can quickly analyze vast amounts of data from years of discussions and identify important trends. These data can be used to generate hypotheses, sparking research interest in previously under-served patient communities.
Does TREND Community sell my data?
Yes, selling de-identified community experience data is part of our business model. We foster collaborations between our rare disease communities and the companies/organizations whose products and services are focused on helping them. As part of these efforts, we may sell summary data about your community to our partners to help them better understand your disease, perspectives and priorities (for example, risks and benefits) so that they may develop or repurpose products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.
Are there any risks to sharing my data?
We ask that our participants use discretion when conversing with others online. When sharing information about your diagnosis or health, there is always a risk that someone could use this information against you. Despite the risks, we hope that you will choose to participate in these important conversations because we believe that the more health information you share, the more we can advance science and improve your life and the lives of patients and caregivers living with your disease.
Founded by parents of a child with a rare disease, the mission of TREND Community is:
To accelerate the search for treatments, interventions and cures while improving the quality of life for individuals living with rare disease.
Follow us on social media @trendcommunity!
Note: The following viewpoint was written in collaboration with The Chion Foundation (chionfoundation.org).
Traditionally, drugs that are approved by the FDA must go through many layers of clinical trials in order to be approved. Typically, these trials must take place through an FDA-approved protocol in the United States or in another country. The gold standard trial is the phase III, randomized, double-blind study which is used to gather data on the safety and efficacy of medical products. Such phase III trials are very expensive and time consuming. They often occur in highly specific patient populations and in very controlled clinical settings. Unfortunately, in the rare disease world, such clinical trials are rare because patients are few and far between and may be difficult to recruit.
The United States government and the FDA have recognized this difficulty and are making efforts to remedy this problem. The 21st Century Cures Act was signed into law in December 2016. The Cures Act specifies that patient experience data may be submitted to the FDA for review. This data may include data on patients’ experience with a disease as well as patients’ preferences with respect to treatment.
The Cures Act also requires the FDA to develop a plan to collect and use patient experience data in drug development. The intention is for the FDA to use patient experience data in its benefit-risk assessment framework so that it can inform decision-making. The Cures Act also re-emphasizes the need to encourage treatments for rare pediatric diseases. Additionally, the Orphan Drug Act has been expanded to include not just clinical and preclinical studies in rare diseases, but “prospectively planned and designed observational studies and other natural history analysis.”
Many clinical researchers would like to be able to successfully integrate clinical research into real-world settings by accessing data from electronic health records, electronic devices, and social media. The Cures Act shepherds in a new emphasis on this real-world data, with the understanding that such data have the potential to complement data obtained from traditional clinical trials. Experts note that such real-world data have the potential advantages of helping researchers answer questions about drug treatment effects and outcomes in an efficient and cost-effective manner. There are challenges, of course, to using data that may be collected in less than scientifically rigorous settings and that is the reason why we believe that we must be as diligent as possible as we collect our patient experience data. The FDA is currently evaluating whether they can use real-world evidence to help support the approval of new indications for an approved drug.
The New England Journal of Medicine recently published an article written by leaders at the FDA that discussed some ideas on how this real-world evidence can be incorporated into drug approval. The authors wrote that, “We believe that real-world evidence can be used across a wide spectrum of research, ranging from observational studies to studies that incorporate planned interventions, whether with or without randomization at the point of care.” The article described the challenges and opportunities of real-world data. In particular, it emphasized the importance of having the data address defined populations using defined methodologies. TREND Community aims to systematically document patient experience data to inform regulatory decision making.