Back in March 2018, Datatrial were successfully included in two of the three Advanced Therapies Treatment Centres (ATTC) being established in the UK and funded by Innovate UK as part of the Industrial Strategies Challenge Fund. Our role is to provide an informatics backbone to facilitate information sharing. Delivery of these innovative therapies to patients involves a complex supply chain coupled with extensive data collection expectations and Nucleus is being used as the central hub for information from multiple sources.
What is an advanced therapy?
The description of an advanced therapy from the European Medicines Agency (EMA) website is as follows:
“Advanced therapy medicinal products (ATMPs) are medicines for human use that are based on genes, tissues or cells. They offer ground breaking new opportunities for the treatment of disease and injury.”
ATMPs can be classified into three main types:
- Gene therapy medicines: these contain genes that lead to a therapeutic, prophylactic or diagnostic effect. They work by inserting ‘recombinant’ genes into the body, usually to treat a variety of diseases, including genetic disorders, cancer or long-term diseases. A recombinant gene is a stretch of DNA that is created in the laboratory, bringing together DNA from different sources;
- Somatic-cell therapy medicines: these contain cells or tissues that have been manipulated to change their biological characteristics or cells or tissues not intended to be used for the same essential functions in the body. They can be used to cure, diagnose or prevent diseases;
- Tissue-engineered medicines: these contain cells or tissues that have been modified so they can be used to repair, regenerate or replace human tissue;
In addition, some ATMPs may contain one or more medical devices as an integral part of the medicine, which are referred to as combined ATMPs. An example of this is cells embedded in a biodegradable matrix or scaffold.”
Since 2010, over £500m has been invested into this area and there is an expectation that the global market will be worth £14bn by 2025. For the UK, a focus on this area offers significant growth opportunities; both jobs in ATMP developers and supply chain companies and infrastructure within our health care system.
But as with all cutting-edge medicines, these therapies come with their own set of challenges – some of which are still unclear at this stage. With the recent licensing of Kymriah (Novartis) and Yescarta (Kite/Gilead) as CAR-T treatments, the NHS is quickly having to develop process and infrastructure to enable and improve access to these treatments by patients.
Accreditation (JACIE) and manufacturer oversight have become expectations that have not present for more traditional medicines. For example, Newcastle Hospitals has been granted the ability to deliver Kymriah to children but has had to demonstrate to the medicine manufacturer that they have the clinical expertise and infrastructure to ensure safe delivery to patients. For example, the Intensive Care Unit must be proximal to the area used to administer the therapy because of the risk of life threatening side effects.
Any patient given these therapies must be followed up for 15 years or more – one reason for this is the ongoing risk of infection as a patients’ B Cells are eliminated. Emily Whitehead, the first child to be treated for Acute Lymphoblastic Leukaemia (ALL) with CAR-T at the University of Pennsylvania has bi-monthly immunoglobulin transfusions to support her immune system. A small proportion of the engineered T cells also remain in the patient’s system – it is uncertain whether the patient needs these cells and again the long-term follow up will provide more insight.
The objectives of the UK ATTC network
As this is a new paradigm in therapy delivery, the most prominent objective is to increase patient access to ATMPs both regionally and nationally by growing a cost-effective clinical delivery pathway which meets the needs of the providers of advanced therapy products. The networks are populated with companies, NHS and universities with expertise in ATMP development and manufacture, specialist supply chain, pharmacy, National Blood Transfusion centres, clinicians in oncology and regenerative medicine. It is an astonishing collection of stakeholders all with the same overall objective; to better facilitate access to these new therapies.
Covering 3 large geographic regions in the UK (Manchester – IMATCH; The Northern Alliance – NAATTC including Scotland, Newcastle and Leeds; Midland Wales ATTC including Birmingham, Wales and Nottingham), this offers an opportunity to define best practice and develop process and systems that fit into the clinical pathways within the NHS.
Scaling up delivery of these therapies is already being highlighted as a risk (https://medcitynews.com/2018/10/car-t-therapies-face-logistical-bottleneck-as-market-set-to-see-major-growth/), one that the UK centres have an unprecedented chance of addressing.
Traditional drug development and delivery has been siloed with data from each process sitting separately; manufacturing and logistics almost irrelevant to clinical outcomes and long term follow up. Advanced Therapies are forcing industry to link these systems; the data flowing from the patient, through manufacture and its delivery and administration to the patient is critical in its aggregation. We need to be able to scrutinize this information in combination for better clinical decision making as well as a broader view on outcomes for health economics.
Where do we fit in?
Datatrial has an incredible opportunity via the ATTCs. As a software company, rarely do you get direct access to all stakeholders when developing requirements. We are working with supply chain (TrakCel, Asymptote, World Courier), clinicians and most recently patients to design a system that can provide real time information end to end as well as keeping patients informed and engaged.
The vision is that all relevant data generated through the process can be brought into the Nucleus hub, can then be aggregated and digested via API (application programming interface)by analytics tools. For the patient, we are anticipating providing some access to information as well as a way for patients themselves to stay in touch with each other, their clinicians and the treatment process they are going through. Patient centric trials are a great aspiration but with these therapies, it is a necessity, particularly as the follow-up requirements are so lengthy. Nucleus should be able to handle data, which also include the long-term follow-up, both patient reported and clinically collected.
We are in month 9 of the project and our requirements capture is almost complete. Watch this space…