Patients with rare diseases and risk of sharing too much
Nicole Gusset wears two hats. She has a young child with spinal muscular atrophy (SMA) and is also the head of the patient advocate group—SMA Switzerland.
She believes patients participating in clinical trials should know how their data is used and analysed. As a private person and mother, as a patient advocate and from a rare diseases and clinical trials perspective, she explores the three main angles from which she looks at the issue of privacy.
The personal perspective
"I have very strong feelings when it comes to my personal privacy. However, living with a family member who requires assistance for simple, everyday activity means that I have to balance my opinion.
Today, people with physical limitations have the opportunity to shape their lives to a certain extent by employing help or assistants. At least this is the case in Switzerland, and we are very fortunate. This is important for the development of a young adult’s personality. But it also means that there are ‘outsiders’ in their lives. There is no privacy when it comes to bank details, salaries, or when it comes to a friend or partnership. Neither is there any privacy when it comes to personal ups and downs and in the sensitive area of body hygiene.
As a patient advocate
Privacy is closely linked to data. As a patient advocate, I think the systematic collection and analysis of data and information leads to knowledge that can improve health and well-being. But what is the price to be paid by an individual? How can patients have their say in the collection, analysis and use of this data? How can we as patient advocates support innovation but also defend the claims of patients?
Personal health data is a valuable asset. Health information is sensitive and intimate and can lead to discrimination. This may take the form of higher life insurance premiums or the denial of insurance coverage. Records can contain details of prescriptions, sexually transmitted infections, abortions or fertility treatments, genetic testing, hospital admissions and even addiction treatment. With the rise of digital health solutions in our daily personal and business lives, this becomes very important.
However, the inconsistency between our privacy concerns versus our online behaviour is pronounced. Today, a lot of medical and health information is controlled by internet companies. They have both the ability and the interest, to combine this data with a wide range of other personal information such as data from daily activities, transactions, movements and demographics. Individuals have to be made aware that health data is not only collected in the monitored environment of a clinical trial setting or registries, but is also out of our control in social media or by using some health apps or wearables.
This is extremely valuable information and I, as a patient advocate, am eager to know in what way this data is beneficial to patients in the cases in which it is used. The enormous amount of data we generate holds exciting potential for researchers who can use it to improve care or find new treatments and insights into a disease. How do we balance the competing interests of privacy and data sharing?
Privacy, rare diseases and clinical trials
In rare diseases, data and privacy are hot topics. But there is a dilemma. By definition, there are only few patients who can be identified quite easily without having names but only by other markers such as geographic distribution. To protect their privacy, rigorous measures are required. On the other hand, there is usually a high unmet need for treatments for serious rare diseases. Sample sizes in clinical trials are usually small, and having detailed natural history data or reusable data that was collected for other purposes as comparators can help avoid some burden for patients (e.g. avoid placebo-controlled trials). To be of best use, this data usually must also include highly sensitive genetic information.
How can patients have their say in the collection, analysis and use of this data? How can we as patient advocates support innovation but also defend the claims of patients? ”
In rare diseases for which there are only a few, and in some cases no effective treatments, people usually volunteer gladly to participate in a clinical trial. In some instances this is their only chance to keep their quality of life or even to potentially survive. Thus, these patients are at risk of sharing whatever they have to and signing what is needed, whatever the consequences. An ethical committee including patient representatives is needed here.
Even if as a patient one would prefer not to share certain data in a clinical trial setting, they might not have a real option to deny it as this is usually mandatory. Therefore, I think patients with a serious rare disease are in a very weak position when it comes to negotiating what data they want to share. You either take it or leave it. This is why early involvement of patient advocates is important."