The UK participants will get support in connecting with Barcelona-Catalonia’s vast life sciences talent pool and during the soft-landing process in the region.
The Fast-Track Programme to Barcelona-Catalonia Life Science Hub is an initiative from Catalonia Trade & Investment’s office in London that works to help UK and Irish companies enter the Catalan market.
Aparito is joined by Eagle Genomics, Exploristics, GBUK Group Ltd, Kirkstall, NorthWest EHealth, Orphan Reach Ltd, Perspectum, and Talisman Therapeutics Ltd.
This is a great step for us to enhance our footprint and presence in Catalonia. We’re pleased to be part of the selected 10 companies and energised to move forward”
We’re thrilled that Aparito appear once again in the UK’s Digital Health Playbook, Beyond 100, alongside the UK’s most impressive digital health innovators as part of the Department for International Trade (DIT) campaign to showcase ground-breaking technologies that address global healthcare challenges
Digital Health is rightly seen as the answer to many of the challenges facing global healthcare. The role of digital technology in realising the dream of accessible, affordable and sustainable care has grown across the entire range of health economies in not only serving current needs but in building the basis for the healthcare of the future: personalised, digital by default and truly patient-centric.
The innovations in the Playbook can offer support to healthcare in a variety of ways from reporting data and using AI, pre-assessment, diagnostics and systems management to managing staff at scale and collaborating.
This award is great recognition from the rare disease community to acknowledge that Aparito makes a difference in the rare disease space.
Aparito has dedicated the last seven years to creating change in the clinical trial experience for patients living with a rare disease by developing standardised at-home assessments and using technology to convey what’s really important to patients.
EURORDIS is an extremely important organisation to Aparito because of the heritage of the previous winners and the esteemed company in which we find ourselves. EURORDIS’ innovative nature in pushing boundaries and acknowledging that the existing ways of working don’t work for the rare disease communities has been an instrumental enabler to change the status quo.
On behalf of everyone at Aparito we graciously accept this award and thank the judges, EURORDIS, and the patient communities that enable us to continue our mission to enable more patient-centric clinical trials.
This award is on behalf of the whole team at Aparito; all of our colleagues are key contributors to the success of our mission. But most importantly, this award is on behalf of all the patient communities that have trusted us to collaborate with them over the last seven years. Without you, our efforts would be nothing and for that, we are extremely grateful.
Dr Elin Haf Davies, CEO of Aparito
We see the award as an incentive to improve and it’s a wonderful way to reinforce our patient engagement practices. Through co-creation, we aim to develop solutions that serve the needs of the patient community and make decentralised trials a reality
Dr Elisa Ferrer Mallol, Patient Advocacy Manager at Aparito
EURORDIS congratulates Aparito for the company’s collaborative approach across rare diseases, engaging patient organisations, clinicians and sponsors.
EURORDIS awarding committee
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.
Purpose: Successful implementation of innovative Precision Medicine initiatives in the management of children with complex epilepsy is largely dependent on the caregivers’ engagement with the technology as well as its accessibility and acceptability. We investigated the feasibility of implementing these initiatives in the South African setting by gathering information on the caregivers’ experiences, perspectives, and expectations for Precision Management of Epilepsy (PME) initiatives.
Methods: We purposively recruited 12 participants from a cohort of 40 caregivers of children with complex epilepsy recruited for a PME study attending Red Cross War Memorial Children’s Hospital (RCWMCH) in Cape Town, South Africa. Face-to-face semi-structured interviews were conducted using a pragmatic qualitative approach and themes were extracted using a thematic framework approach.
Davies, E.H., Matthews, C., Merlet, A. et al. Time to See the Difference: Video Capture for Patient-Centered Clinical Trials. Patient (2022) in conjunction with Alexion, AstraZeneca Rare Disease explores the role of technology in rare disease drug development and how video recording and analysis can enable more patient-centric clinical trials, which will be a major drive to support at-home assessments, especially in rare and neurodegenerative diseases.
Developing therapeutics for the treatment of rare diseases usually requires a strong understanding of the natural history of the disease. Often, it also requires the creation of novel assessment tools and clinical trial endpoints. In diseases where mobility is impacted, the use of video to capture the impact of the disease and the assessment of specific parameters, such as gait and stride length, can help design sensitive endpoints. Video as an assessment tool also allows the use of historical videos or videos filmed by non-experts outside of clinical settings. Given the increased use of telemedicine, the use of video may be a useful addition to clinical trial assessments.
Two cases are presented: (1) the use of video in the development of asfotase alfa (Strensiq®) in hypophosphatasia is detailed as an example of the utility of this type of assessment in rare diseases; and (2) a home-setting video tool that was developed and validated (SARAhome) from a commonly used clinical scale (Scale for the Assessment and Rating of Ataxia [SARA]), allowing patients to record their own severity of ataxia. While there are certain limitations associated with video assessment, advancing technologies such as automated analysis and machine learning provide a tremendous opportunity for automated analysis of video recordings, reducing the bias associated with human assessment.
In this paper we explore key points for decision-makers, namely:
Novel assessment tools such as video technology can capture how a patient functions and also provide meaningful endpoints in rare disease development.
Video technology allows for clinical outcome assessments to be offered at home as well as in hospitals, reducing the burden of travel to sites.
Early planning to validate the approach with regulators is essential to allow for standardized data collection and alignment with real-world evidence.
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Asan O, Montague E. Using video-based observation research methods in primary care health encounters to evaluate complex interactions. Inform Prim Care. 2014;21(4):161–70. https://doi.org/10.14236/jhi.v21i4.72.
Each year, 1.4 million people attend A&E in England with a traumatic brain injury (TBI) & over 50 million people worldwide have sustained a TBI.
Advances in critical care, imaging and the reorganisation of trauma health systems mean that more people live with the damage caused by the TBI for longer.
Patient Reported Outcome Measures (PROMs)are questionnaires completed by patients that can be used to monitor the long-term effects of health conditions.
The aim of the PRiORiTy study was to develop and assess the feasibility of an electronic Patient Reported Outcome Measure (ePROM) system for inclusion within routine clinical care & TBI research, which in this instance involved getting people with a TBI to report their symptoms using questionnaires electronically utilising the Aparito Atom5TM platform.
The project is divided into three parts.
The first part was a qualitative study, which aimed to obtain the views of people with a TBI, carers and HCPs of PROMs and ePROMs.
The second part was the usability study. Using the results from the qualitative study and feedback from the PPI group, Aparito deployed Atom5TM to collect ePROM responses via a patient-facing app and a web-based clinician dashboard.
The third part is the feasibility study, which will test the electronic platform in a clinic setting.
Participants were enthusiastic about PROMs & ePROMs
PROMs help to focus consultations on what is important to patients (memory loss, anxiety, lack of concentration)
ePROMs are flexible, timesaving, facilitate evaluation of symptoms & impact on quality of life
Key features of ePROMs are conciseness and their use of lay language – they should reflect patients’ cognitive and physical ability.
Advantages of ePROMs
Less burdensome for patients & clinicians
Fewer data entry errors
Easy real-time data/remote monitoring & response
Ability to send/receive feedback easily
Participants’ positive attitudes & experience towards ePROMs in this study demonstrate the potential to capture PROs electronically in routine clinical practice and TBI research.
It is anticipated that the PRiORiTy study will increase capacity for trauma-specific knowledge and expertise in relation to PROMs, as well as inform system development in other areas of trauma research.
In this webinar recording, Aparito’s Patient Advocacy Manager, Elisa Ferrer Mallol, introduces our Patient Group Accelerator Programme and how it engages young patients and families in the development of medtech with examples from our first Accelerator programme with Duchenne UK and PCD Support UK.
Elisa’s talk took place at the Patient Engagement Open Forum 2021 hosted by the European Patients Academy on Therapeutic Innovation (EUPATI) as part of a collective effort to further children, young patients and parents’ involvement in the development of medtech and medical devices. EUPATI provides education and training for patients and patient representatives on the process of medicines research and development, as well as training on patient engagement for all stakeholders to ensure the acceleration of effective patient engagement in Europe
Watch Elisa’s ten-minute talk via the video below or on YouTube and access all of the sessions from PEOF 2021here.
Want to discover more about the Aparito Patient Group Accelerator Programme?
What we learned from our Putting Paediatrics’ Needs First in Clinical Research webinar.
We invited three luminaries from the medical field, Jenny Preston, Senior Patient and Public Involvement Lead for NIHR Alder Hey Clinical Research Facility at the University of Liverpool, Rhian Thomas-Turner, R&D Lead at Noah’s Ark Children’s Hospital for Wales, and Dr Jo Wilmshurst, Head of Paediatric Neurology at Red Cross War Memorial Children’s Hospital, University of Cape Town, to join Dr Elin Haf Davies to champion the UN International Child Day and tackle three challenging topics at our recent webinar through a series of presentations and a close-out roundtable.
Key themes discussed were
1) why it’s our duty to make the experience of clinical trials easier for children & parents
2) what remote patient monitoring has taught us about paediatric patients’ needs through the last eighteen months
3) how we can work together with all partners to break down the barriers to innovation in children’s medicine
So, what did those discussions entail?
Why it’s our duty to make the experience of clinical trials easier for children & parents
Rhian Thomas-Turner reminded us that, under the UN Convention on the Rights of the Child (CRC) it’s a legal right for children to have access to, and development of, suitable medicines and that we must recognise that the obligations to child health are legal rather than moral or ethical: a recent medical paper cited where, due to scarce resources, boys were prioritised over girls, and the paper referred to the decision as an ethical issue rather than a breach of international law.
There needs to be an interpretive community push through our collective writing and communication to ensure people understand children’s human rights to health. “Too often paediatric medicines need to be adapted in a ward setting, e.g crushing tablets where no liquid suspension is available, so we have to question whether these medicines are appropriate for paediatric needs, and without knowing fully how these medicines act in the body, whether we can suggest they do give children the right to grow and develop (as is their legal right)?”
Trials often have too many obligations on children and their families and we should seek to design trials that leverage technology to create meaningful endpoints for paediatric patients and, where possible, keep them out of a hospital setting and monitor them in their own environment.
Jenny Preston of University of Liverpool posed questions to ask ourselves when designing paediatric research and trials:
Are we asking the right research question(s)?
Are we measuring the right (or most meaningful) outcomes?
Do our research tools and instruments make sense?
How child/family-friendly is our study?
We must ask ourselves: can we design better clinical research and trial experiences that recognise children’s other rights under CRC, such as the right to play and the right to education?
What remote patient monitoring has taught us about paediatric patients’ needs through the last eighteen months
The health impact for children from the aftershock of COVID-19 is a concern, from lack of access to immunisation programmes through to delayed diagnosis that would impact interventions.
Jo Wilmhurst highlighted how COVID-19 caused huge disruption to paediatric care and low-income countries suffered disproportionately as they were less able to access telemedicine and telehealth, leading to deleterious, long-term effect on the health of its children:
“In the high-impact scenario, for every one excess COVID-19 death attributable to SARS-CoV-2 infections acquired during routine vaccination clinic visits, 84 (95% UI 14–267) deaths in children could be prevented by sustaining routine childhood immunisation in Africa.”
That raises a challenging question: under the CRC and children’s rights to medical assistance and health care, is access to technology such as remote patient monitoring and telehealth a legal imperative in instances where no alternative is available?
How we can work together with all partners to break down the barriers to innovation in children’s medicine
There is frequent discussion regarding how regulators, HCPs and drug manufacturers can work together to advance paediatric research & trials, but that omits a rather important stakeholder; the patient and their family.
As Jenny Preston posited, “If a company was developing a new soft drink, would they bring that product to market without consulting their target audience? The answer, of course, is no they would not, so why should it be any different when designing and conducting paediatric health studies?”
Jenny cited her research that found 83% of young people think they must be involved in decisions that the government makes about healthcare, and 14% believe that young people should lead the discussion on healthcare.
Children have the right to express views freely in all matters affecting the child as per article 12 of the CRC; we can, should and must work with them to ensure their voices are heard and applied through methods such as Dr Laura Lundy’s model of participation with purpose.
The University of Liverpool & NIHR created the GenerationR Alliance to set up Young People’s Advisory Groups (YPAGs) around the world. Now over 50 strong, these groups get actively involved in the design and delivery of clinical research to ensure that research is relevant to young people and their families.
“Prioritising child health needs isn’t to take away from the needs of our ageing population but if we don’t address child health now, we won’t have a healthy, prosperous society” Elin Haf Davies
Dr Jo Wilmhurst believes that preventative interventions would significantly impact on the burden of child health through trained and equipped healthcare professionals who advocate, lead, train and educate in their local areas. She cited, The African Paediatric Fellowship Programme, which brings the best doctors in Africa together, trains them, and sends them home to implement what they’ve learned. These doctors have gone to lobby for access to medications, collaborated with international associations, and completed research such as publications and PhD proposals.
Rhian suggested we improve the discourse around human rights and access to develop suitable medicines for children, saying “What we can use (human rights law) for is to have these conversations: we don’t need to move into hard legislation to change the narrative around access to medicines and development of the right medicines”.
To close, we asked our presenters “What would add the greatest value to child health?”
What did we learn?
Training and education were referenced consistently by all of the speakers, whether medical training to boost preventative interventions or discourse with the wider community to reiterate that children’s rights are legal rights and need to be respected in developing new and appropriate therapies for children.
To design better experiences in paediatric clinical research we need to use the gravitas of the law to drive participation with purpose and provide training & education so that all parties understand the benefits, not least that better patient engagement and experiences leads to better research outcomes.
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It is now 20 years since I recruited my first paediatric patient to the first randomised clinical trial that I was personally involved in.
Ironically, I remember many of those events and conversations better than I remember what I did last week, such was the impact that it has had on me: five of the children that took part in that children trial have since sadly passed away, along with many others that lived with that tragic disease, and there is still no treatment available to address the high unmet need of the neuronopathic Gaucher disease community.
Not that there hasn’t been some progress made since that failed clinical trial. We do now have other clinical trials on the horizon and some of those young people have reached adulthood and continue to inspire us. One such individual is Maddie Collins, our Project Coordinator here at Aparito, who recently became the proud mum of a baby boy.
I have been part of the EFGCP Programme Committee for a while now, and despite my passion for this topic, I have recently struggled to support the organisation of a conference where, more often than not, I felt that we were repeating the same old topics and issues since I first attended the conference back in 2007 when the Paediatric Regulation came into force here in Europe.
But there were glimmers of hope this year and I’m pleased to say I was far more enthused and energised at the end of the Better Medicines for Children conference than I have been in some time.
It was great fun to host the fireside chat with Prof Koen Norga the Chair of the Paediatric Committee (PDCO) at the European Medicine and Dr Angeliki Siapkara from the MRHA here in the UK, and a former PDCO member until Brexit enforced her departure.
As colleagues of old, we had an honest and warm conversation (despite the virtual fireside that is Zoom) and the discussion primarily focused on the value of global collaboration, a topic that was echoed with the global presence of regulators from Japan, China, and Brazil, and moderated by Marie Valentin from the WHO.
Indeed, it was the regulators’ contribution to the conference that made the show for me this year, with many of their key take-home messages resonating loudly:
It is possibly notable that two of the most impressive speakers at the conference were from the Nordic region, representing the Norwegian and The Danish Medicines Agency – Anja Schiel and Frederik Grell Nørgaard.
Over the years, I’ve heard Anja say a few times “this might not fit in our current way of thinking but…”. I have a huge amount of respect for her and she’s enabled initiatives that were otherwise facing roadblocks.
Based on a quick Google search (always a dangerous task!) it seems that the cultural traits for the Danes are for authorities, decisions and orders to be challenged! Not to make sweeping cultural generalisations but it is reported that the Danes are schooled from an early age to ask questions, challenge ideas and debate positions.
In the breakout session on Decentralised Clinical Trials (DCTs) Frederik Grell Nørgaard presented all the efforts undertaken by the Danish Agency to support the frameworks for DCTs. I loved his reflections that regulators were to be cautious and conservative but not ignorant or stubborn! The regulator’s role in DCT discussion is to ensure that the GCP regulation and rigorous requirements of clinical trials are maintained and that we don’t throw the baby out with the bathwater (ICH GCP is still the framework for DCT).
He implored the industry partners to show good examples to regulators and to include decentralised elements in paediatric trials now. This is an easy win for us all that can be evaluated iteratively, and something that we’re increasing demand for at Aparito as companies are leveraging the power of remote patient-generated data as part of hybrid studies and the ability to co-develop digital endpoints that convey what’s important to patients.
Study sponsors must justify why decentralised elements are needed in clinical trials, which are appropriate for the condition and the product and useful to answer the scientific question, but most important an advantage for the patients and for addressing the scientific question (not for saving costs for the companies!).
The advantage for the patients (and their parents) must remain central to this. One of my personal concerns that I expressed in the DCT breakout session was that we must not allow the pendulum to swing too far, where we convert children’s homes into mini laboratories, damaging the safety comfort net of home.
We must not move the burden of responsibility (or the dirty work of enforcing compliance to conduct painful tests) onto parents – they have more than enough burden to carry on their shoulders. Our duty is to make the experience easier for them, logistically and emotionally.
But progress feels painfully slow, and it seems to take an incomprehensible amount of time to make changes – I regularly get frustrated with just how long it takes for some initiatives we’re driving, or are part of, at Aparito to bear fruit.
And yet, in the development of a vaccine for Covid-19, we can only be amazed and rejuvenated by the rapid progress, including the early consideration and inclusion of children and adolescents to avoid years of off-label prescribing. The benefit of parallel working without shortcuts to quality standards led to rapid timelines that were beneficial to all.
Anja Schiel was clear – there is not too much innovation, but we risk having too many innovations at the same time. And we must be careful that we’re able to address the risk and uncertainty of each innovative approach both individually and collectively.
While the agile, ‘fail fast’ approach of the tech world should never be adopted in drug development, I’m a strong advocate that we can embrace an agile framework with pre-defined go-no go rules that keep the safety of the participants and the quality of the data coming through iteratively.
To make innovation work in drug development, we must take the regulators with us on the journey.
We must justify the approach, be transparent and plan early.
We must convey the journey, including all the bumps in the road to the regulators early and often, so that they can follow the context and understand the rationale.
After all, we all want to be part of the main storyline – not just passive passengers in someone else’s adventure.
The conference finished with concluding personal remarks by Dr Agnes Saint-Raymond as she retires after a lifelong commitment to child health, and what a highlight!
Her no-nonsense direct, to the point messaging, was a great reflection of her style which has uniquely helped her propel the Paediatric Regulation into existence, and now celebrates 350+ approved medicine with paediatric indications and 50 pharmaceutical forms, on the back of 4561 Paediatric Investigational Plans agreed.
Agnes appointed me to join the Paediatric Team at the European Medicine Agency in 2007 and I remain forever grateful for the six years as a regulator under her drive and guidance. We all need role models like Agnes in our early-mid careers.
Although advocating for an evolution rather than a revolution (which I was slightly disappointed about!) she implored for us to stop the blah blah blah talking – and move from discussion to action.
Time to stop talking and start working on definitions of unmet paediatric needs so that the development of paediatric medicine can remain in the “industry’s reality” but make faster and specific progress where it’s most needed.
She also advocated for relying even more on extrapolation and the integration of modernisation of clinical trials, including the use of AI! A life-long regulator advocating for innovation – a dream statement!
In 2010 I co-authored a paper with Agnes titled Paediatric investigation plans for pain: painfully slow! which examined the slow progress made since the regulation came into force. Our concluding sentence stated that “Now is an opportune time for clinicians, academics, learned societies and industry to collaborate for the benefit of children in pain”.
Eleven years on – can we say that it’s an opportune time NOW?
I say YES, it must be so! I refuse to be having the same conversation in another 10 years’ time! Much like Agnes, I’m not known for my patience and with the high calibre of children and young people that also contributed to the conference via pre-recorded videos or in person I am convinced by their eloquent and articulate contribution that they will not patiently wait for another 10 years either.
“Children are not the people of tomorrow, but people of today.”