covid sbq picture of man in mask

The Symptom Burden Questionnaire™ for Long COVID

The University of Birmingham’s Centre for Patient-Reported Outcomes Research (CPROR) have designed the Symptom Burden Questionnaire™ for use in research and clinical care to enable patients to report symptoms and help develop new treatments for Long COVID leveraging Aparito’s Atom5™ Clinical Trial Platform.

The approach used to develop the Symptom Burden Questionnaire™ tool appears in the BMJ titled “Development and validation of the symptom burden questionnaire for long covid (SBQ-LC): Rasch analysis”

Hughes S E, Haroon S, Subramanian A, McMullan C, Aiyegbusi O L, Turner G M et al. Development and validation of the symptom burden questionnaire for long covid (SBQ-LC): Rasch analysis BMJ 2022; 377 :e070230 doi:10.1136/bmj-2022-070230

The University of Birmingham’s release notes the tool “was developed with extensive patient input following regulatory guidance, meaning its scores may be used to support regulatory decisions around the approval of new therapies for Long COVID and by policymakers.”

The study was carried out in partnership with Aparito and funded by the National Institute for Health Research (NIHR) and UK Research and Innovation (UKRI).

Abstract: Development and validation of the symptom burden questionnaire for long covid (SBQ-LC): Rasch analysis

Objective To describe the development and validation of a novel patient reported outcome measure for symptom burden from long covid, the symptom burden questionnaire for long covid (SBQ-LC).

Design Multiphase, prospective mixed methods study.

Setting Remote data collection and social media channels in the United Kingdom, 14 April to 1 August 2021.

Participants 13 adults (aged ≥18 years) with self-reported long covid and 10 clinicians evaluated content validity. 274 adults with long covid field tested the draft questionnaire.

Main outcome measures Published systematic reviews informed development of SBQ-LC’s conceptual framework and initial item pool. Thematic analysis of transcripts from cognitive debriefing interviews and online clinician surveys established content validity. Consensus discussions with the patient and public involvement group of the Therapies for Long COVID in non-hospitalised individuals: From symptoms, patient reported outcomes and immunology to targeted therapies (TLC Study) confirmed face validity. Rasch analysis of field test data guided item and scale refinement and provided initial evidence of the SBQ-LC’s measurement properties.

Results SBQ-LC (version 1.0) is a modular instrument measuring patient reported outcomes and is composed of 17 independent scales with promising psychometric properties. Respondents rate their symptom burden during the past seven days using a dichotomous response or 4 point rating scale. Each scale provides coverage of a different symptom domain and returns a summed raw score that can be transformed to a linear (0-100) score. Higher scores represent higher symptom burden. After rating scale refinement and item reduction, all scales satisfied the Rasch model requirements for unidimensionality (principal component analysis of residuals: first residual contrast values <2.00 eigenvalue units) and item fit (outfit mean square values within 0.5 -1.5 logits). Rating scale categories were ordered with acceptable category fit statistics (outfit mean square values <2.0 logits). 14 item pairs had evidence of local dependency (residual correlation values >0.4). Across the 17 scales, person reliability ranged from 0.34 to 0.87, person separation ranged from 0.71 to 2.56, item separation ranged from 1.34 to 13.86, and internal consistency reliability (Cronbach’s alpha) ranged from 0.56 to 0.91.

Conclusions SBQ-LC (version 1.0) is a comprehensive patient reported outcome instrument developed using modern psychometric methods. It measures symptoms of long covid important to people with lived experience of the condition and may be used to evaluate the impact of interventions and inform best practice in clinical management.


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On-Demand Webinar: Video Capture for Patient-Centered Clinical Trials

Aparito Webinar | Video Capture for Patient-Centered Clinical Trials 

Aparito On-demand-webinar-video-capture
To view the webinar on-demand, please enter your details via Zoom to gain instant access.

The Topics

In this webinar, our panel explored the topics from our paper, Time to See the Difference: Video Capture for Patient-Centered Clinical Trials, and examined:

  1. a case study in the development of a home-setting video tool for ataxia using the Aparito Atom5™ platform
  2. where technology can support more patient-centric clinical trials, which will be a major drive to support recruitment and retention
  3. how new endpoints can not only support modern solutions to existing rare diseases but might also be essential to design and develop new scales that are tailored to rare and neurodegenerative diseases

The speakers

  • Dr Elin Haf Davies – CEO of Aparito
  • Dr Clare Matthews – Data Scientist at Aparito
  • Adeline Merlet – Director, Global Regulatory Affairs, Development Strategy at Alexion Pharmaceuticals, Inc.
  • Dr Marcus Grobe-Einsler (MD) – Deutsches Zentrum für Neurodegenerative Erkrankungen e.V. (DZNE, the German Centre for Neurodegenerative Diseases)

Watch the webinar now

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Dr Rashmi Narayana Chief Scientific Officer at Aparito

Aparito appoints Chief Scientific Officer

Aparito is pleased to announce the appointment of Dr Rashmi Narayana as Chief Scientific Officer (CSO).

Dr Rashmi Narayana Chief Scientific Officer at Aparito
Dr Rashmi Narayana

Rashmi qualified as a psychiatrist and made her way to healthtech via leadership roles at the American Red Cross, academia, and research management. In health tech, she has held roles as Clinical Director at uMotif, as Head of Clinical Development and Head of Clinical and Regulatory Affairs at Huma and, as Director of Operations at Our Mobile Health.

She has numerous peer-reviewed publications to her name. She holds a Masters in Public Health from the London School of Hygiene and Tropical Medicine and an MBA from Imperial College Business School, London. Over the pandemic, she trained and certified as a leadership coach.  

With a wealth of experience in navigating complex healthcare environments and partnering with cross-functional teams in business, academia, start-ups and the NHS, Rashmi joins Aparito at a time of rapid growth and will support the senior team in the key role of CSO. 

She will focus her operational knowledge and practical expertise to help Aparito facilitate decentralised and hybrid clinical trials with our biopharma clients.

I’ve known Elin and Chris for years. Elin embodies a mix of expertise, experience, and energy which is essential in healthtech. Her passion and commitment to delivering high quality, patient-centred solutions are infectious. The fact that she’s a female tech founder was an added incentive to join Aparito.
Chris has an uncanny ability to translate the complex tech and regulatory needs of biotech and pharma clients into requirements that make sense for tech teams who build solutions. This is no small feat in the world of decentralised and hybrid clinical trials.

I’m excited at the opportunity to support them and the wider team to drive Aparito’s growth while retaining and attracting people who connect with Aparito’s mission.

Dr Rashmi Narayana, Chief Scientific Officer at Aparito

We’re very pleased to have appointed Rashmi to our senior management at this critical time.

Her wealth of experience and insight will be a great asset to drive Aparito’s growth forward!

Dr Elin Haf Davies, CEO of Aparito 

Meet the rest of the Aparito team

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Aparito collecting the Innovation and Technology Award at the 2021 Daily Post Business Awards

Aparito Win Innovation and Technology Award

Aparito won the Innovation and Technology Award at the 2021 Daily Post Business Awards for their Atom5TM clinical trial software platform!

Aparito at the Daily Post Business awards at Banger University, Wales. Picture Jason Roberts
Daily Post Business awards at Banger University, Wales. Picture Jason Roberts

The award recognises the transformational work of Aparito’s software developers, data scientists and support teams who help bring clinical trials to patients and unlock real-world data through the Atom5TM platform, video assessments & wearable devices, making it simpler for more diverse patient groups to engage in clinical trials by reducing the time and cost required to participate. 

We’re thrilled to win this award and have the work of a Wrexham company that competes with global businesses recognised by the judges.

North Wales is a vibrant and supportive location for tech companies and has helped us build a world-class team who in turn have built a world-class platform. 

Diolch yn fawr iawn i’r Daily Post a Prifysgol Bangor.    

Dr Elin Haf Davies, Founder & CEO of Aparito

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Celebrating International Women’s Day

Acknowledging the contributions of women to the fields of Science, Technology, Engineering and Mathematics (STEM) is a crucial part of the Aparito story.

Aparito was founded by Dr Elin Haf Davies, a visionary nurse turned PhD doctor who wanted to change the patient experience of clinical trials and was frustrated by existing solutions.

Women make up just 4% of UK tech startup founders

11 out of 251 of the fastest growing tech start-ups in the UK were founded by women

Elin went on to build a team that champions inclusivity and diversity. Innovation is one of our guiding values and we put it into practice with our recruitment policies.

The Aparito team
The Aparito team

40% of the Aparito team are women

That’s more than twice the industry average for tech businesses!

We also introduced two new outstanding executives in the first quarter of 2022: Graciëlle Schutjens as our Scientific Business Director and Dr Rashmi Narayana as our Chief Scientific Officer.

100% of the Aparito Data Science team are women

Our Data Science team, started by Dr Clare Matthews and Sandra Komarzynski, continues to grow with two additional hires this year to date.

There is always more to be done but we are proud of our commitment to furthering equal representation of women in STEM, and not just because greater female representation at senior levels within businesses makes those businesses more profitable and more socially responsible, but because it’s the right thing to do to create a more equitable society.

Five women who paved the way in STEM

Aparito stands on the shoulders of giants when it comes to innovating in STEM and so we celebrate five women whose contributions to the world made our work possible.

1. Elizabeth Garrett Anderson

The first English female doctor; Elizabeth Garrett Anderson (1836 – 1917) was an astounding and resilient woman whose plight and determination enabled other women to also achieve greatness.

Having been inspired by successful women such as Elizabeth Blackwell, the first female doctor in the US, Elizabeth Garrett Anderson opted to contradict the submissive life she was expected to lead and become a doctor. Denied entry to any medical school, she was forced to study nursing alongside male peers whose objections led to her dismissal.

After Elizabeth qualified as a doctor through the Society of Apothecaries, they immediately implemented a ban on female entrants. The sexism and adversity Elizabeth faced only fuelled her strength and resolve.

Having taught herself French in order to study at the University of Paris, Elizabeth finally earned her medical degree. However, this was still not enough to allow her onto the British Medical Register, so she established the New Hospital for Women, which was to become the London School of Medicine for Women.

Her vocal campaign efforts eventually paid off and in 1876 female entry into the profession of medicine was legalised.

Even once she had retired from medicine, Dr Anderson was still grinding down the patriarchy, becoming the first female mayor in England. She was influential in the suffragette movement and inspired her daughter, alongside many other intrepid women, to follow in her esteemed footsteps and strive toward gender equality.

2. Betsi Cadwaladr

Betsi Cadwaladr (1789 – 1860) was one of 16 children from Bala in North Wales. When Betsi grew up she became a traveller of note. She travelled around the world extensively in an age when most working-class men hardly ever moved outside the area they were born. Betsi was not officially a nurse but her various jobs involved her in nursing duties and she was convinced of the need for cleanliness as an aid to recovery from disease and illness.

In 1854, the Crimean War broke out and a total lack of care for the wounded and dying soldiers became evident. Betsi applied to join Florence Nightingale’s group of nurses to go out to help, when she arrived she demanded to be sent to the front, Nightingale thought that Betsi was argumentative and wanted nothing more to do with her.
Betsi lived and worked just behind the front line where she cared for wounded and injured soldiers. She nursed for up to 20 hours a day and usually slept on the floor with seven other nurses by which time she was over 70 years of age.

When Nightingale visited the battlefront and saw what amazing work Betsi had done, she changed her mind about a woman whom she had only previously considered as being irritating. She begged Betsi to stay on, but Betsi knew she had reached her limit by then and returned home.

Her heroic efforts in the field of healthcare were not acknowledged during her lifetime, however, she did gain some degree of recognition in 2009 when the Betsi Cadwaladr Health Board was created to include the six local health boards along the North Wales coast.

3. Ada Lovelace

Ada Lovelace (1815-1852) was unquestionably one of the most important women in science history. She was born in 1815, the daughter to Lord Byron and Lady Byron. Lady Byron believed that if she could tame Ada’s imagination, this would prevent Ada from going down the line of imaginative self-indulgence that Byron himself had.

She set out to use mathematics as the method of taming Ada’s imagination, figuring that if she could arrange for Ada to be educated in mathematics. When Ada was only 17 years old, and on the evening of 5 June 1833, she met a man who would become arguably her most important friend.

His name was Charles Babbage. He was 24 years older than her, and she quickly became fascinated, after talking to him, with his plans for building a calculating machine called a ‘Difference Engine’. The purpose of this, although Babbage never managed to complete it, was to calculate mathematical tables automatically without error.

Babbage eventually abandoned the Difference Engine in favour of the Analytical Engine: the world’s first digital computer, with a store, a processor, a memory, a sub-routing function and all the other essential features of a modern digital computer.

It is Lovelace’s work on the Analytical Engine that has led to her being known as the first computer programmer.

4. Katherine Johnson

Katherine Johnson (1918 – 2020) was a mathematician who worked on NASA’s early space missions and was portrayed by Taraji P Henson in the film ‘Hidden Figures‘.

She was one of the “computers” who solved equations by hand during NASA’s early years and those of its precursor organisation, the National Advisory Committee for Aeronautics. Johnson and other black women initially worked in a racially segregated computing unit in Hampton, Virginia, that was not officially dissolved until NACA became NASA in 1958. Signs had dictated which toilets the women could use.

She focused on planes and other research at first. But her work at NASA’s Langley Research Centre eventually shifted to Project Mercury, the nation’s first human space programme.

She and her co-workers had been relatively unsung heroes of America’s ‘Space Race But in 2015, President Barack Obama awarded Johnson (then 97 years old) the Presidential Medal of Freedom, the nation’s highest civilian honour.

5. Barbara McClintock

We live in an age where we have mapped the human genome and developed tools such as CRISPR, but all of this was possible thanks to the dedication and lifelong study into genetics by Barbara McClintock (1902 –1992) She spent her entire career analysing maize, and in the 1930s developed a staining technique that allowed her to identify, examine and describe its individual chromosomes.

Armed with her research, she was able to determine the existence of jumping genes, which are sequences of DNA that move between the genome. Jumping genes were considered junk DNA by much of the scientific community at the time. Still, McClintock pressed on and suggested they might in fact determine which of the genes in cells are switched on – vital in creating differences between cell types.

It was not until 1983, when she was awarded The Nobel Prize in Physiology or Medicine, that the scientific community began to recognise not only just how important these jumping genes are, but how much of the genome they make up – some estimates suggest they make up 40 per cent of the human genome.

McClintock also was the first to suggest the idea of epigenetics, where genes alter their activity in response to external factors, some 40 years before it was formally studied.

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Impact of assessment frequency of patient-reported outcomes: an observational study using an eHealth platform in cancer patients

Abstract

Background and aim: The evaluation of patient-reported outcomes (PRO) in cancer has proven relevant positive clinical impact on patients’ communication with healthcare professionals, decision-making for management, well-being, and overall survival. However, the optimal frequency of PRO assessment has yet to be defined. Based on the assumption that more frequent sampling would enhance accuracy, we aimed at identifying the optimal sampling frequency that does not miss clinically relevant insight.

Methods: We used pilot data from 31 advanced cancer patients who completed once daily the 19-item MD Anderson Symptom Inventory at home. The resulting dataset allowed us to compare different PRO assessment frequencies to daily sampling, i.e., alternate days (q2d), every third day (q3d), or once a week (q1w). We evaluated the sampling frequencies for two main outcomes: average symptom intensity and identification of severe symptoms.

Authors: Pasquale F Innominato 1 2 3Sandra Komarzynski 4Robert Dallmann 5Nicholas I Wreglesworth 6 7Mohamed Bouchahda 8 9 10 11Abdoulaye Karaboué 8 12Ayhan Ulusakarya 8 11Christian P Subbe 7 13David Spiegel 14 15Francis A Lévi 5 8 11 16

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Graciëlle Schutjens joins Aparito

Aparito is pleased to announce the appointment of Graciëlle Schutjens as Scientific Business Advisor and Director of Aparito B.V, Aparito’s Dutch entity.

Graciëlle joins Aparito following a distinguished career starting as a Clinical Research Project Manager with Quintiles (now IQVIA) before her passion for education and training saw her promoted to Training and Quality Control Manager for Europe at Parexel.

Graciëlle’s love for developing young CRAs led to her forming her own CRO in 2000 which she sold to Venn Life Sciences in 2010 where she stayed on as Chief Operating Officer to oversee an AIM listing and growth through M&A with companies throughout the UK and Europe before leaving in 2018 to work as a freelance consultant.

Graciëlle will apply her operational knowledge and expertise as Aparito grows its global customer base and maintain excellence as Aparito wins more new business facilitating decentralised and hybrid clinical trials with clients.

I need to feel a ‘click’ before I take on a challenge like this – I need to feel the energy – and I recognised myself in Elin Haf (Davies, CEO of Aparito).

The pandemic accelerated the importance of companies like Aparito and we are ahead of the companies that saw this opportunity during the pandemic; Aparito were there in time to lead this change towards more decentralised medicine and personalised healthcare.

Graciëlle Schutjens, Scientific Business Advisor and Aparito B.V. Director
Graciëlle Schutjens

I am delighted that Graciëlle has joined us. Her extensive knowledge and deep understanding of this space is second to none, and as such she brings a wealth of insight to help us grow.
Personally, I’m thrilled to be supported by a business leader in life science who’s been there already. The fact she’s a woman is a major advantage for me, too.

Dr Elin Haf Davies, CEO of Aparito

About Aparito

Aparito is a global health tech company that brings clinical trials to patients and unlocks real-world data through mobile apps, video assessments & wearable devices.

We provide innovative patient-centric clinical trials solutions that integrate specialist medical & regulatory expertise to capture patient data and develop digital endpoints for hybrid and decentralised clinical trials. 

We do all this through Atom5™, our iOS & Android-compatible web and mobile app platform. Atom5™ captures multiple, high-frequency data points from video, voice, wearables, and electronic Patient Reported Outcomes, then analyses the results in conjunction with our team of data scientists to provide rich, real-time insights to clinical teams. 

Atom5™ is regulator-friendly by design and features eConsent via a partnership with DocuSign, computer system validation and complete data privacy. The global platform operates under ISO14385 QMS and ISO/IEC 27001 ISMS accreditations and is FDA CFR21 Part 11 compliant.

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Media relations

Alexander Guest – Head of Marketing, Aparito

E-Mail: alexander.guest@aparito.com Tel: +44 1978 896 191

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DT4RD logos

Announcing the Digital Tools for Rare Disease (DT4RD) Project

Aparito are proud to announce the launch of the three year 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 to develop non-invasive tools for measuring mobility in rare disease patients. 

The DT4RD project brings together a multinational consortium consisting of Aparito, Paris Institut de Myologie (Institute of Myology), The John Walton Muscular Dystrophy Research Centre at Newcastle University, Yumen Bionics, Metabolic Support UK, along with biotechnology and pharmaceutical industry partners, CSL Behring and Chiesi Pharmaceuticals who will both serve as the sponsors of the project. 

In order to meet the objective set by the call the project consortium will develop a digital platform capable of assessing upper limb function remotely and continuously, capturing the point between being ambulant and non-ambulant, a pivotal juncture in the progression of rare diseases which is often not accounted for with the use of conventional clinical methodology and tools. 

Over the course of three years, Aparito’s Atom5TM software platform will be integrated with state-of-the-art wearable sensors developed by Yumen Bionics to create an integrated platform which will amass data covering physiological and psychosocial parameters and will be tested across two clinical centres in Paris and Newcastle, UK, respectively. 

Improving the lives of patients with rare diseases is very important to us here at Aparito so it is a privilege to collaborate with a very dedicated and talented team who also share this passion. We are very excited about what we can achieve together and can’t wait to see the positive impact on the lives of patients.

Dr Elin Haf Davies, Aparito CEO

The DT4RD project is one of the 3 projects selected for funding within the RDR Challenges call led by Fondation Maladies Rares and EJP RD. We are very excited about this innovative project fostering public-private partnerships and combining EC funding and co-funding from industry partners to the benefit of patients.

Dr Christine Fetro, Foundation for Rare Diseases

Projects like DT4RD are a prime example of the type of collaboration that is needed between industry, academia, and patient organizations to bring about positive changes in the lives of patients living with a rare disease. That is why the EJP RD decided to initiate, support and is proud to facilitate RDR Challenges.

Dr. Daria Julkowska, European Joint Programme on Rare Diseases

We are very excited and honoured to take on the challenge to innovate future clinical trials. Together with a complementary team of clinical, tech and experience experts we’ll explore the possibilities of remote monitoring to change the experience of rare disease patients. We can’t wait to get started.

Paulien Klap, Lead Product Engineer – Yumen Bionics

DT4RD represents a great opportunity to move towards a new evaluation paradigm. Recent events have unfortunately shown that patients sometimes suffer a loss of clinical follow-up. New digital solutions should avoid such situations. The Institute of Myology is enthusiastic to participate to the development and validation of such novel tools.

Dr Jean-Yves Hogrel, Director of the Neuromuscular Physiology and Evaluation Lab, Institut de Myologie (Paris)

This is a very timely and exciting project, as the COVID-19 pandemic has illustrated how important it is to assess patients remotely. Exploring novel ways to monitor motor function remotely using smart technologies will ultimately make lives of patients with motor impairments easier.

Professor Volker Straub, Director of The John Walton Muscular Dystrophy Research Centre and Deputy Dean, Translational and Clinical Research Institute, Newcastle University and Newcastle upon Tyne Hospitals NHS Foundation Trust

We are very much looking forward to this cross-cutting collaboration where experts in technology, remote monitoring, clinicians and individuals living with motor impairments can deliver meaningful gains suitable for wider populations.

Dr Anna Mayhew, Consultant Research Physiotherapist at Newcastle University

This innovative project, bringing together a group of leading international experts, is a step forward for patients with rare disease. Our patient communities need access to non-invasive tools that allow them to contribute to research but do not increase their everyday burden. We are also delighted that, by involving a patient organisation from the outset, the DT4RD project has committed to embedding the lived experience of rare disease patients in the project.

Metabolic Support UK

As a patient focused organization, CSL is always looking for innovative ways to enhance the experience of rare disease patients in our clinical trials. We are hopeful that this project will have a broad impact for our industry by creating a novel approach to measuring mobility. We are excited for the opportunity to partner on the promising DTR4D project with world class researchers, Chiesi, as well as EJP RD, EURORDIS and French Rare Disease society.

Deirdre BeVard, Senior Vice President of R&D Strategic Operations, CSL Behring

We are proud to join together with EJP RD, EURORDIS, FFRD as well as CSL Behring in efforts to address areas of high unmet need and support innovations that may ultimately help us provide optimal support to patients and families affected by rare diseases. Being part of DT4RD project is very exciting. We look forward to work with the Team to improve the quality of life of rare disease patients.

Chiesi Group

For more information on the respective members of the project team please visit the links attached below: 

About the European Joint Programme on Rare Diseases – https://www.ejprarediseases.org/fundings-and-calls/rare-diseases-challenges/

About Aparito

Aparito is a health tech company that transforms clinical trials by unlocking real-world patient data through mobile apps, video-based assessments and wearable devices. Aparito provides innovative patient-centric remote monitoring solutions that integrate specialist clinical & regulatory expertise to capture patient data and develop digital endpoints for both hybrid and decentralised clinical trials through the flagship product Atom5™.

Atom5™ is an iOS & Android-compatible web and mobile app platform. Atom5™ captures multiple, high-frequency data points from video, voice, wearables, and electronic Patient Reported Outcomes, then analyses the results in conjunction with the team of in-house data scientists to provide rich, real-time insights to clinical teams.

Atom5™ is regulator-friendly by design and features eConsent via a partnership with DocuSign, computer system validation and complete data privacy. The global platform operates under ISO14385 QMS and ISO/IEC 27001 ISMS accreditations and is FDA CFR21 Part 11 compliant.

For further information please visit: https://www.aparito.com/ 

About Fondation Maladies Rares (Foundation For Rare Diseases (FFRD))

FFRD is a unique cooperative framework coordinating research resources and expertise in the Rare Disease (RD) field. Flagship of the 2nd French National Rare Diseases Plan, FFRD acts as a federative hub to fund research and accelerate scientific, clinical and social innovation by stimulating cross-sector cooperation towards effective healthcare to the benefit of RD patients. Its active financial support spans from biomedical sciences to Social Sciences and Humanities. Since its creation in 2012, FFRD has funded 417 projects (out of 1270 submitted) for a cumulated amount of 12,3M€. With headquarters located in Paris, FFRD also relies on a team of regional field coordinators in direct contact with academic and clinical teams all over the national territory to ensure that priorities are driven by grounded needs. FFRD main objectives include: accelerating the translation of research into clinical development; detecting business development opportunities; enhancing access to innovative technologies; facilitating cross-sector partnerships. With its close contacts with academic researchers and clinicians in the field, FFRD is contributing to early identification of proofs of therapeutic concept and making links with other partners in the research value chain, namely Small and Middle-sized Enterprises (SMEs), Pharmas, and Technology Transfer Offices (TTOs). For further information please visit: https://fondation-maladiesrares.org/

About Yumen Bionics 

Yumen Bionics is a Duchenne focused medtech SME, developing technology for people living with neuromuscular diseases. Yumen Bionics was founded by the Duchenne Parent Project NL resulting in a profound collaboration with the community. For further information please visit: https://yumenbionics.com/ 

About Paris Institut de Myologie 

The Institute of Myology was created in 1996 by AFM-Telethon to diagnose, treat patients and study muscle diseases, in partnership with five public bodies (AP-HP, CEA, Inserm, Sorbonne Université and CNRS). This centre of expertise, unique in the world, focused on the patient, promotes the existence and recognition of Myology by bringing together in one-place activities of evaluation, diagnosis and care, fundamental, applied and clinical research and teaching. With 250 muscle experts (technicians, doctors, researchers, etc…) it has innovative investigation and measurement tools at its disposal for diagnosis, management and monitoring of neuromuscular diseases: a Myology Research Centre composed of 10 teams; a Neuromuscular Exploration and Evaluation Centre composed of three laboratories and a Biological Resources Centre, Myobank-AFM ; two clinical research platforms and a Registries and Databases Centre; a Neuro-Myology clinical service for neuromuscular patients; an international training centre ensuring the dissemination of knowledge on Myology. For further information please visit: https://www.institut-myologie.org/en/ 

About The John Walton Muscular Dystrophy Research Centre, Newcastle University: 

The John Walton Muscular Dystrophy Research Centre works across Newcastle University and the Newcastle Hospitals NHS Foundation Trust and is located within the University’s Institute of Translational and Clinical Research, which is part of the Faculty of Medical Sciences. The Centre has a strong interest in the development and application of genomic and translational medicine for neuromuscular diseases with a clear emphasis on improving health outcomes for this patient group. The John Walton Muscular Dystrophy Research Centre sits in crucial strategic areas of increasing importance to the national and international research agendas – translational medicine, genomic and personalised medicine and rare diseases. The team has an extensive track record of leadership in the international community in projects on translational neuromuscular research, policy direction in rare diseases, patient registries, biobanking, muscle imaging and clinical trials. The Centre is part of the International Centre for Genomic Medicine in Neuromuscular Diseases (ICGNMD) and works closely with the World Muscle Society, EURO-NMD and the TREAT-NMD Alliance. For further information: John Walton Muscular Dystrophy Research Centre – John Walton Muscular Dystrophy Research Centre (newcastle-muscle.org)

About Metabolic Support UK

Metabolic Support UK are the leading patient organisation for Inherited Metabolic Disorders supporting thousands of patients worldwide. For more information please visit: Home – Metabolic Support UK 

About CSL Behring

CSL Behring is a global biotherapeutics leader driven by our promise to save lives. Focused on serving patients’ needs by using the latest technologies, we discover, develop and deliver innovative therapies for people living with conditions in the immunology, hematology, cardiovascular and metabolic, respiratory, and transplant therapeutic areas. We use three strategic scientific platforms of plasma fractionation, recombinant protein technology, and cell and gene therapy to support continued innovation and continually refine ways in which products can address unmet medical needs and help patients lead full lives.

CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. The parent company, CSL Limited (ASX:CSL;USOTC:CSLLY), headquartered in Melbourne, Australia, employs more than 25,000 people worldwide, and delivers its life-saving therapies to people in more than 100 countries. For inspiring stories about the promise of biotechnology, visit Vita CSLBehring.com/vita and follow us on Twitter.com/CSLBehring.

About Chiesi Group:

Based in Parma, Italy, Chiesi is an international research-focused pharmaceuticals and healthcare group with over 85 years’ experience, operating in 30 countries with more than 6,000 employees (Chiesi Group). To achieve its mission of improving people’s quality of life by acting responsibly towards society and the environment, the Group researches, develops and markets innovative therapeutic solutions in its three focus areas: AIR (products and services that promote respiration, from new-born to adult populations), RARE (treatment for patients with rare and ultra-rare diseases) and CARE (products and services that support specialty care and consumer-facing self-care). The Group’s Research and Development centre is based in Parma and works alongside 6 other important research and development hubs in France, the U.S., Canada, China, the UK, and Sweden to pursue its pre-clinical, clinical, and regulatory programmes. Chiesi, since 2019, is the world’s largest B Corp certified pharmaceutical group. The global B Corp movement promotes business as a force for good. Moreover, Chiesi Farmaceutici S.p.A. has changed in 2018 its legal status to a Benefit Corporation, by incorporating a double purpose for the creation of shared value, and to generate value for its business, for society and the environment. As a Benefit Corporation, Chiesi Farmaceutici S.p.A. is required by law to include objectives of common benefit in its bylaws and to report annually in a transparent way. The Group is committed to becoming carbon neutral by the end of 2035. For further information: www.chiesi.com 

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Media relations

Alexander Guest – Head of Marketing, Aparito

E-Mail: alexander.guest@aparito.com Tel: +44 1978 896 191

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Designing and refining mHealth solutions for epilepsy patients

Why epilepsy? 

Epilepsy is a debilitating disease that poses a significant challenge in all settings, especially in those that are resource-limited. Difficulties in its management within such environments can be attributed to:

  • Early diagnosis and access to care 
  • Medication access and/or non-adherence to prescription
  • Limited visibility of seizure frequency and general well-being: seizure incidence and general well-being can be difficult for clinicians to monitor after patients leave a clinic so clinically relevant events may be missed

Tackling unmet clinical needs

Acknowledging these challenges, Aparito and Red Cross War Memorial Children’s Hospital (RCWMCH) were motivated to take action, launching the Precision Management of Epilepsy study where mHealth technologies were combined with genetic and pharmacogenomic analysis to improve the treatment outcomes of children with refractory epilepsy in Cape Town, South Africa. Qualitative research was also carried out in parallel to understand how epilepsy patients and their caregivers understood precision medicine, their experience of mHealth, and how the technology could be further developed to meet their needs. 

Design and deployment of mHealth technology 

The mHealth technology was powered by Aparito’s software platform Atom5™ and consisted of a mobile app paired with a wearable device. 39 patients aged between 4 and 16 made use of the software platform with the following data sets being collected on a 24/7 basis:

  • Physiological profiles (e.g. seizure incidence/type and sleeping patterns)
  • Psychological wellbeing and general quality of life via validated patient-reported outcome measures
  • Medication adherence

The continuous nature of data collection allowed the clinicians to have a thorough understanding of the patient experience.  

Evaluating feasibility of mHealth for epilepsy patients

Generally, caregivers were fond of the prompts provided via the app as they felt that it helped medication adherence, with some liking the fact that they could record seizures in real time with a great deal of accuracy. Caregivers also made mention of their ability to contact healthcare providers remotely which they valued. Engagement rates for mobile patient-reported outcome measures (mPROs) and the wearable device were 57.1% and 30.8% respectively. The majority of participants were given smartphones so they could use the app and it was acknowledged by some caregivers that the new technology was challenging to understand and they were not sure if they could use it properly. Caregivers also acknowledged that owning new technologies placed them at risk of being victims of crime, and that the wearables were inconvenient for their children (due to their physical or intellectual disabilities). Difficulties posed by high mobile data costs and internet access were also mentioned.

The results of the feasibility study were published in a paper, Demonstrating the feasibility of digital health to support pediatric patients in South Africa.

Conclusion 

The work carried out to date highlights the clinical utility of mHealth for epilepsy patients. Informative data sets were collected that facilitated routine care and helped caregivers (and their children) to better understand and manage the disease. However, patient-centric healthcare technology designs are needed as emphasised by the caregivers that were impacted by socioeconomic dilemmas which include: clinical literacy, digital health literacy/digital literacy, crime and internet access.

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