Author Archives: Alex

August 09 2023, 09:00 CEST

Since announcing the launch of the Digital Tools for Rare Disease project (DT4RD), a product of the Rare Disease Research call placed by the European Joint Programme on Rare Diseases in partnership with Fondation Maladies Rares sponsored by Chiesi and CSL Behring, extensive progress has been made!

Non-invasive tools for measuring rare disease patient mobility in daily living and distinguishing between voluntary and involuntary movements were put in place using state-of-the-art wearable sensors integrated with Aparito’s Atom5^TM clinical trial platform to capture physiological and psychosocial parameters. 

The main objective of this project has been focused on developing and validating a way to acquire, transfer, store and process heterogeneous data related to movement measured at home and anchored to assessments performed at the hospital, as Clinical Outcome Measures (eCOA).

This European study involves great collaboration with renowned clinical experts located at the Institute of Myology (Paris, France), and at the John Walton Muscular Dystrophy Research Centre (Newcastle, UK).

After the French Ethics Committee’s approval, the Institute of Myology is proud to announce their First Patient recruited into the study on the 3rd of July 2023!

The enrolment went very well although a bit longer than expected to explain in detail the protocol and train the patient with the digitised tools including the Atom5^TM platform with video tasks and ePRO to complete, the Garmin Vivosmart5 wearable device and the Yumen Bionics wearable sensors.

Dr Jean-Yves Hogrel, Director of the Laboratory of Neuromuscular Physiology and Evaluation at the Institute of Myology

The patient expressed her motivation for taking part in this study related to the mobility of patients affected by rare neuromuscular diseases. Afterwards, she declared: “I felt comfortable, not too tired and confident to carry the tasks at home independently”.

The Institute of Myology Team added: “We are in the process of recruiting very soon our next patient on the 1st of September 2023 and keep on enrolling a total of 20 patients in the next few months.”

This is a great milestone which reflects a wonderful collaboration to date. We are always grateful when patients enrol on our studies and appreciate their time and dedication to develop innovation that will hopefully be used in patient-centric clinical trials to convey what’s important to patients.

Dr Elin Haf Davies, CEO of Aparito

By digitising critical procedures within the clinical trial process, Aparito leads this project with European Clinical partners as well as renowned technology and industry partners to provide real-time visibility of data collection and integrity, reducing patient and site burden, enhancing the patient experience, and improving patient retention.

This EJP-DT4RD study is supported by the Atom5^TM platform’s innovative technology in the patient’s homes using disease-specific smartphone apps, video assessments, and wearable technology to deliver meaningful data through eCOAs and the development of digital biomarkers.

About Aparito
Aparito’s mission is to digitize clinical trials and accelerate drug development for patients with life-limiting diseases by supporting patient-centric clinical trials with innovative treatments. Clinical trials can be conducted within patients’ homes by leveraging our Atom5™ platform incorporating disease-specific smartphone apps, video assessments and wearable technology to deliver digital clinical trials using novel eCOA to generate continuous real-world data. Aparito’s patient-generated data platform is disease-agnostic and scalable, ready for rapid deployment in global rare disease studies.

About the Institute of Myology

The Institute of Myology, located in Paris, coordinates medical management, research, and education related to muscle diseases and injuries. It is an international reference centre that participates in numerous trials and clinical studies, with a focus on neuromuscular diseases, high-performance sports, and ageing. The Institute of Myology Association facilitates the coordination of site activities in partnership with five public guardianship organisations, and the institute includes a complete Center of Research in Myology and several scientific poles as well as clinical activities.

Find them on https://www.institut-myologie.org/en/

About the EJP-DT4RD project

A unique project involving Academics (Institute of Myology, John Walton Muscular Dystrophy Research Centre), SMEs (Aparito, Yumen Bionics), large pharmaceutical companies (Chiesi, CSL Behring), and a patient organisation (MSUK) working towards the same objective for and with patient involvement. It widens the horizon of tremendous possibilities for the future of research in rare diseases and warrants hope for future clinical trials using Real World data.

Find out more at https://www.ejprarediseases.org/rare-diseases-research-rdr-challenge-1-digital-tools-for-rare-disease-dt4rd/

July 31 2023, 09:00 EST

Aparito and TruLab, two companies focused on improving rare disease studies, have formed a partnership to improve the efficiency and experience of clinical trials.

TruLab’s technology has gained traction in the rare disease space due to the difficulty of obtaining samples, especially from children, and the criticality of the sample for the patient’s eligibility in the study. Both Aparito’s and TruLab’s mobile platforms are designed to maintain data integrity and minimize the potential for data loss.

By leveraging Aparito’s Atom5™ platform, clinical trials can be supported in the patients’ homes using disease-specific smartphone apps, video assessments, and wearable technology to deliver meaningful data through eCOAs and digital biomarkers.

Collectively the companies intend to reduce the burden on parents and caregivers seeking treatment for their children. The application of innovative technologies in this setting ensures that patient samples and data points are collected according to study procedures and are not lost, ultimately increasing the chances of success in the clinical trial.

By digitizing critical procedures within the clinical trial process, TruLab and Aparito can provide real-time visibility to data collection and integrity, reduce patient and site burden, enhance the patient experience, and improve patient retention.

“We are excited to partner with TruLab to improve the collection and management of samples in rare disease studies,” said Dr Elin Haf Davies, CEO of Aparito. “Our Atom5™ platform will help ensure that the patient-generated data collected is of the highest quality, ultimately leading to a better clinical trial experience for people living with rare diseases.”

“We are delighted to partner with Aparito to provide a seamless solution for rare disease studies,” said Jim Wahl, Vice President of Client Services at TruLab. “Our combined technologies will enable us to provide high-quality data to support the development of treatments for rare diseases.”

The partnership between Aparito and TruLab is a significant step forward in rare disease research. With the two companies working together, researchers will have access to platforms that provide real-time visibility and improve the quality of data collected and ultimately lead to better treatments for patients with rare diseases.

About Aparito
Aparito’s mission is to digitize clinical trials and accelerate drug development for patients with life-limiting diseases by supporting patient-centric clinical trials with innovative treatments.

Clinical trials can be conducted within patients’ homes by leveraging our Atom5™ platform incorporating disease-specific smartphone apps, video assessments and wearable technology to digitise clinical trials using novel eCOA and generate continuous real-world data. 

Aparito’s patient-generated data platform is disease-agnostic and scalable, ready for rapid deployment in global rare disease studies.

Discover more at aparito.com.

About TruLab
TruLab, Inc. is a sample management software company that provides advanced point-of-collection workflow solutions for the clinical research industry.

TruLab’s technology is designed to help clinicians, labs and pharmaceutical sponsors manage their clinical samples more effectively, while ensuring consistent compliance with study protocols and achieving target levels of data quantity and quality.

TruLab also enables real-time visibility of clinical procedures and sample status for pharmaceutical and lab customers, especially in a decentralized trial environment.

Learn more at trulab.com.

Introduction

The advent of a new disease in 1981 would spark a revolution in science and society, and lead to the birth of modern patient advocacy. When AIDS (Acquired Immune Deficiency Syndrome) was identified as a global health concern in the early 1980s, few would have realised the impact it would have on society and especially on healthcare.

Those early days were grim, the reporting of HIV was often horrific, and there were many myths and fears surrounding the disease. And all the while, people were dying.

The medical community, patients, and regulatory bodies had to navigate the challenges of a new disease without many of the tools and processes we take for granted today.

We spoke to Neil Bertelsen, an advocate for the HIV community, to explore the role of HIV patients in the birth of modern patient advocacy and how this changed the regulatory landscape for the better.

Legacy Perceptions of HIV and COVID-19

Neil notes how legacy perceptions of HIV and COVID-19 can influence how we react to new diseases.

“In the 1980s, when HIV first appeared, there were many myths and fears surrounding the disease that were perpetuated by the media, and led directly to discrimination of those with the disease and the communities it affected,” he says. Similarly, in the early days of COVID-19, we witnessed the same fear response, leading to discrimination against healthcare workers treating the first wave of COVID patients. Neil suggests that society needs to “become more adult in its response to infectious diseases and that we need to work together to address these challenges, including a better understanding of risks and relative risks, and more stringent guidance for the media on reporting this”.

The Birth of Modern Patient Advocacy

The 1980s saw the birth of coordinated and sophisticated activism by the HIV-affected communities and through that decade the birth of modern patient advocacy, which was a response to the challenges faced by people with HIV. Those affected and medical professionals came together to learn, at the same time and at the same speed, about the disease. One of the key challenges was a mismatch between the needs of the community and the regulatory rules in place for approving new medicines. To overcome this, activism and then advocacy led to the formation of patient advocacy groups and councils, which advised regulatory bodies on patient perspectives and helped shape regulatory policy.

This marked a significant shift towards patient involvement in regulatory decision-making, which has become a hallmark of modern healthcare.

Neil points out, “Now we have so many advocacy groups that are laser-focused on shaping policy and regulations, some operate at the global level, others cover a region, and of course, we have the national patient groups that are connected to policymakers and understand how their healthcare system works, how decisions are made. These groups are building their own evidence to support patients within the healthcare systems and policy landscape.”.

The FDA’s Role in the Fight Against HIV

During the early days of HIV, the FDA became a lightning rod for dissatisfaction. “The regulators were taking too long to approve medicines. Placebo-controlled trials were the norm, and from the view of the HIV-positive community the regulatory system was just not geared for the fast turnaround needed for a deadly infectious disease,”,” explains Neil. “From the perspective of the HIV community, promising experimental medicines were spending years under review while many thousands were dying.”.

People with HIV and advocates were angry with the slow pace of drug approvals and the regulatory hurdles that stood in the way of life-saving treatments. In essence, similar to today, this was all about access to medicines. The difference is that today it is the payers that control access, back then it was the regulators.

The collective activism led to a change in the summer of 1989 when Neil recalls, “The FDA said, ‘Okay, we’re going to come up with a new system only for life-saving medicines, and this parallel track will allow patients to access a medicine while we’re still going through the review process’. And it’s from that moment that a lot of things that we take for granted in the regulatory world actually came from and marked a significant shift towards patient-centred regulatory policy.”.

Further reading from The Atlantic. “Before Occupy: How AIDS Activists Seized Control of the FDA in 1988“

Patient Advocacy and Regulatory Bodies

The growth of patient involvement in regulatory bodies has been a significant development in the healthcare industry. Patient advocates now sit on committees and councils that advise regulatory bodies on patient perspectives and help shape policy.

This is a direct result of the advocacy efforts of patients and medical professionals during the HIV epidemic. As Neil says, “What happened in those last years of the 1980s was something that we hadn’t seen before: a genuine coming together of both the medical community and the patient community, where we became hand-in-glove, basically learning at the same speed. At the same time, we had a community of people affected by a disease that self-taught themselves in how regulatory processes worked.”.

This shift in the way the regulators engaged with patients was driven by “the community of people with HIV started to make contact and sit down with regulators to say, ’Hey, how do we work together on this?’” Neil recollects. “That led to the FDA forming what is now their patient engagement policy, and it started with its HIV-specific patient council that advised the FDA and was later expanded to include more diseases”.

However, there is still work to be done to ensure that patient perspectives are fully integrated into regulatory decision-making. One of the emerging trends is the use of ‘patient experience data’ – evidence beyond the clinical that details how patients experience a condition, its treatment and the effects it has on their lives. “We need to make sure that patients, advocates and the affected community are co-creators of this evidence to ensure it is providing decision-makers with evidence on what is important to people with the condition. This means we need to democratise evidence generation so that all sections of society have a hand in shaping the evidence used for decisions.”.

Eye-to-Eye Partnership and Fragmented Networks

Neil notes that one of the missing ingredients in modern patient advocacy is the eye-to-eye partnership that existed between patients and medical professionals during the HIV epidemic. “This partnership enabled a true collaboration, where patients and medical professionals learned at the same time and grew together”. Additionally, Neil argues that the healthcare industry is becoming increasingly fragmented, with many different networks and initiatives that are poorly connected.

He suggests that organisations need to step back and find ways to create a “seamless, interconnected network”. In those early days of HIV, there was no internet or email. Fax machines provided the technology and personal contacts were the network dynamics. People attending seminars in San Francisco would fax the details over to contacts in London, Paris and around the world, and vice versa. “It seems clunky by today’s standards, but it worked,” says Neil. “Today, we have so many different networks we are meant to tap into and the information overload is immense. I do worry about the duplication of efforts that I see all around me these days.”.

Conclusion

The birth of modern patient advocacy can be traced back to the HIV epidemic of the 1980s. Patients and medical professionals came together to navigate the challenges of a new disease and the affected community built a framework for patient involvement in regulatory decision-making as well as patient involvement in medicine development. The lessons learned have led to significant improvements in the role of people with conditions, new regulatory approaches to speed up access to life-saving medicines, and a realisation that patient involvement is not a ‘nice-to-have’, it is essential for our healthcare systems.

This framework has led to significant improvements in healthcare, but there is still work to be done to ensure that patient perspectives are fully integrated into regulatory, health policy and access decisions.

Healthcare organisations need to find ways to connect more efficiently and work together to avoid duplication. We need to find ways to engender parity between the clinician and patient communities. The AIDS/HIV epidemic was devastating on so many levels “Who knows what our society would be like today if we hadn’t lost that generation of people,” reflects Neil. Yet, something grew from that devastation that enriches us all today.

As we continue to face the unique challenges that rare diseases present, we can look back on the experiences of patients with HIV and draw strength from their activism and advocacy efforts.

Sign up for our newsletter to receive our latest papers, posters and platform updates direct to your inbox!

We are thrilled to announce that Aparito CEO Dr Elin Haf Davies has won the Made WITH Patients Rising Star Award for her work in developing our Patient Group Accelerator Programme. To quote Elin, “The recognition is not just for us but for every individual who has contributed to our mission of digitising what’s important to patients in a patient-centric way”.

A Collaborative Approach to Patient-Centric Solutions

At Aparito, we firmly believe in the power of co-creation. Elin developed the Patient Group Accelerator Programme to provide a unique platform where patients, caregivers, and advocacy groups can collaborate with us to create solutions that genuinely work for patients.

With Dr Elisa Ferrer and Navdeep Sahota now running the Accelerator, we work closely with patient organisations to understand and fulfill their needs by finding new endpoints relevant to their specific conditions.

By developing technological solutions, novel endpoints, and digital biomarkers designed for and with the patients, our objective is to collect sufficient exploratory data as a starting point for future validation and use in clinical trials.

Transforming the Patient Experience of Clinical Trials

Our ultimate goal is to bring equity to clinical trials, ensuring that every individual, regardless of their circumstances, has equal access to life-changing treatments.

Our Patient Group Accelerator Programme has laid the foundation for future validation and utilisation in clinical trials. These groundbreaking tools have not only allowed us to collect insightful data but have also transformed the patient experience of clinical trials and we firmly believe that this innovative approach will revolutionise the way clinical trials are conducted, making healthcare accessible to all.

Full Partnership with Patients

The novelty of our Patient Group Accelerator Programme lies in our full partnership with patients.

Working with the patients from the beginning until the end of the process, we ensure that we understand their needs and develop solutions that work for them.

The programme started in 2020 with two patient groups that help patients living with Duchenne Muscular Dystrophy (DMD) and Primary Ciliary Dyskinesia (PCD) and has since added PHA UK (Pulmonary Arterial Hypertension) and ALS Liga (amyotrophic lateral sclerosis, ALS). By collaborating with patients and advocacy groups, we can develop solutions that are tailored to meet their needs, breaking barriers, and transforming lives.

Our Patient Group Accelerator Programme is a testament to our commitment to co-creation and patient-centric solutions and we thank the patient communities who have trusted us and collaborated with us on this transformative journey.

This recognition serves as a reminder of the tremendous impact we can achieve when we come together with a shared purpose.

Let us continue to work hand in hand, breaking barriers, and transforming lives, as we strive for a future where healthcare is accessible to all.

Dr Elin Haf Davies, Aparito CEO

Sign up for our newsletter to receive our latest papers, posters and platform updates direct to your inbox!