IDA Ireland, the agency responsible for the attraction and retention of inward foreign direct investment (FDI) into Ireland, has recently published an article entitled ‘Why Ireland is a proving ground for medicinal breakthroughs like cell and gene therapy’. Hitech Health is delighted to be featured as part of Ireland’s exciting growth in the cell and gene therapy space. Hitech was the first commercial company to have a licence to manufacture these advanced medicines in Ireland. It works with some large global companies, but Managing Director Brian Harrison says most of the companies in the cell and gene therapy space are smaller companies including university spin outs.
In many cases, these advanced medicine companies have products in development, and need expertise to manufacture pre-clinical materials as is required for toxicology studies. Hitech Health can also manufacture clinical trial materials for these advanced medicine companies once the potential treatment has been approved by regulators to be used in patients . In some cases, large pharma companies outsource these advanced medicine products to contractors and they continue to use Hitech for support.
Temperature is a critical parameter in the transportation and storage of medicinal products. The temperature must be controlled and monitored from the manufacturing site through to the distributor and site of medicine use. When temperatures go outside the specified range, there is a risk that the medicine may alter and degrade with the potential for adverse outcomes for the patient. In addition to this, there may be significant financial losses for the medicinal product owner.
The manufacturing of medicinal products is highly regulated with strict GMP quality control standards to ensure that a high-quality medicine is provided to patients. When it comes to transportation, it is just as important to maintain the same level of quality standards and ensure medicinal products are not compromised during the many touchpoints that can occur between the sending and receiving sites. Good Distribution Practice (GDP) is a set of standards, guidelines, and principles for the storage and transportation of pharmaceutical products. Developers must implement robust processes to guarantee the quality and safety of their products during distribution. GDP compliance can help avoid major quality impacts and potential product recalls.
Medicinal product shipments can generally be categorised as:
Ambient (controlled room temperature, 15°C to 25°C)
Refrigerated (2°C to 8°C)
Frozen (Below 0°C)
Cryogenic (Below -150°C)
As transportation may involve many modes of transport including road, air and sea, then continuously controlling and monitoring temperature can be challenging. Furthermore, cross border customs checks can cause unwanted delays. There is a risk that custom checks can interfere with outer packaging and cause exposure of the medicinal products to outside temperatures. The sensitivity of new advanced therapies such as cell and gene therapies increase the demand on both the shipping system and on the integrity of the entire temperature-controlled supply chain.
The FDA’s 21 CFR Part 11 guidance addresses the traceability challenges of electronic temperature records. Records must be reliable and accurate to ensure the quality and safety of the medicinal product. Pharmaceutical companies are strongly advised to use calibrated temperature monitoring dataloggers that can generate accurate and reliable reports. In addition to this, electronic records of all alarms and events must be kept.
With so many variables in transportation, successful shipments depend on reliable transportation and the correct choice of shipping system for the type of medicinal product. Using shipping systems that are validated and appropriate for the medicinal product is crucial. Performing shipping qualification studies is advised to ensure that a system is in place for seamless transportation and quality assurance.
To help navigate the challenges with temperature control, it is key to find partners experienced in handling complex medicinal products. HiTech Health have a team Responsible Personnel (RPs) who are specialists in GDP and can advise and support you with:
Minimising supply chain risks and ensuring compliance with applicable regulations
Shipping qualification studies
Due diligence and auditing of transportation providers
We can work with your team to ensure that effective processes are established to ensure that high quality medicinal products are available for patients on time.
Schedule a call with our team today to learn more about our services and how we can support your organisation.
Many Cell and Gene Therapy (CGT) companies outsource product development and manufacturing activities to a Contract Development and Manufacturing Organisation (CDMO). It is critical to ensure that an informed decision is made when selecting a CDMO to prevent future disruption to the intricate supply chains of cell and gene therapies and ultimately patients. HiTech Health explores 5 key considerations for a cross-functional team to examine when partaking in the CDMO selection process.
Does your CDMO have an experienced CGT team?
The complex and time-sensitive nature of CGT manufacturing processes makes it imperative to select a CDMO with a team who have deep knowledge of cell and gene therapies. The technical and regulatory landscapes in the CGT industry are changing frequently and ensuring your CDMO has in-depth expertise and flexibility is crucial to success. Assessing the CGT experience of the proposed CDMO team could reduce the risk of knowledge gaps later. A CDMO may have a strong track record of developing and manufacturing small molecule products and biologics but may have limited experience of CGTs. Are sufficient resources in place now and into the future?
Is there an integrated project management approach defined by the CDMO?
If you have shortlisted potential CDMO partners, have you enquired about their project management approach? How do the proposed approaches differ among the potential partners? Having an integrated project management approach that ensures seamless alignment between production, supply chain, QC testing personnel and many other functions is paramount given the stringent timeframe for supplying CGTs to patients. Understanding how frequently you will meet with the assigned CDMO project manager and how they will communicate with you should be clearly defined from the beginning.
Does the CDMO practice effective Quality by Design (QbD)?
Considering if a CDMO practices Quality by Design (QbD) is an important part of the due diligence process. The CDMO team may work closely with you to measure, analyse and report on the quality attributes of the CGT product. The team members who develop your upstream regulatory strategy and initial CMC plan need to be able to work closely with a trusted CDMO partner who can ensure quality and compliance goals are met. Have you considered an on-site audit of the CDMO before the final selection?
How flexible is the potential CDMO partner?
During the due diligence assessment, look at the CDMO’s internal systems and workflows for a true indication of their fit as a potential partner. The CDMO should be experienced and agile to help you navigate unforeseen challenges, open and transparent to help you mitigate risks and control your costs as well as being fully committed to the ultimate goal of providing CGTs to the patients who vitally need them. These are the characteristics of a resilient and long-lasting partnership, and the indicators of future success.
From the start of a project, the right CDMO will encourage an ongoing dialogue about the quality, pace and cost considerations of your project. They will focus on developing a robust and scalable approach that will work for the entire lifecycle of your project.
Is there a Business Continuity Plan (BCP) that has been implemented and managed effectively by the CDMO?
The CDMO’s ability to maintain essential functions during and after an unforeseen event has occurred is very important and should be assessed prior to signing a contract. Business continuity planning, in this case, relates to establishing risk management processes and procedures that aim to prevent interruptions to manufacturing and supplying CGTs. If an unforeseen event occurs, such as critical components not being available, how will the CDMO mitigate or manage the issues to ensure the effects on operations are minimised. Have you requested a copy of your shortlisted CDMO’s business continuity plan?
Learn more about our cell and gene therapy services by clicking here
HiTech Health today announced that the company has been granted a Manufacturer’s/Importation Authorisation (MIA) for Investigational Medicinal Products (IMP) by the Medicines and Healthcare Products Regulatory Agency (MHRA). The company is now authorised to perform batch certification and import oversight for UK clinical trials. This achievement underscores commitment to meeting the highest standards of quality and safety in pharmaceutical products and advanced therapies, as set forth by the MHRA.
HiTech Health’s Qualified Personnel (QPs) can perform batch importation and certification for EU countries and the UK, thereby streamlining clinical supply chains for the sponsors. HiTech Health is dedicated to working closely with pharmaceutical and advanced therapy developers to bring new products to patients.
At the Advanced Therapies Congress 2024 in London, Amina Al-Mossawi, a Qualified Person (QP) at Hitech Health, delivered vital insights on the crucial considerations for biotech firms when selecting a Contract Development and Manufacturing Organisation (CDMO). With extensive experience in both clinical and pharmaceutical settings, Amina’s guidance is invaluable for companies aiming to navigate the complex landscape of cell therapy manufacturing.
During her presentation, Amina emphasised the importance of aligning with a CDMO that meets the specific needs of a therapy from development through to commercialisation. She also explained that this alignment proves crucial to the success rate of new therapies in the highly competitive and rapidly evolving biotech industry.
Understanding the Full Scope of CDMO Services
Amina also pointed out that a competent CDMO must fully grasp the intended purpose of the therapy and the demographics of the patients it targets. This deep understanding proves critical for developing, scaling up, and managing the commercial supply chain of new therapies. She further stressed the importance of technical proficiency, particularly in adhering to Good Manufacturing Practice (GMP) and Good Distribution Practice (GDP).
The Financial and Strategic Stakes of CDMO Selection
“Selecting the right CDMO involves more than evaluating technical capabilities,” Amina stated. As a result, financial considerations and strategic planning play pivotal roles. Thus, biotech firms must prepare to manage unexpected costs and complexities, such as those arising from Brexit and other regulatory changes.
Risk Management in CDMO Partnerships
In particular, A significant portion of Amina’s speech addressed the risks associated with miscommunication and the dangers of partnering with CDMOs that do not tailor their services to meet client needs. “Clear, realistic communication and expectations form the foundation of any successful partnership,” Amina advised.
A Call for Tailored CDMO Services
Hitech Health is advocating for a shift away from the ‘one-size-fits-all’ approaches prevalent in the CDMO industry, urging companies to seek partners that offer flexibility and customisation. This tailored approach ensures that the unique needs of each therapy and patient group are met, thereby enhancing the potential for clinical and commercial success.
If you’d like to arrange a meeting with Amina to learn more about our services then please email: aal-mossawi@hitech-health.com
Hitech Health is delighted to announce its latest partnership with CCRM Australia, a not-for-profit organisation supporting regenerative medicine commercialisation in the Australian market. CCRM Australia works to accelerate the commercialisation of regenerative medicine (RM) therapies and related technologies by providing specialist expertise, funding opportunities, and fostering connections between industry, clinicians, researchers, and key stakeholders throughout the country
HiTech Health helps advanced therapy providers through their development, manufacture and supply of products including cell and gene therapies for clinical trials and also for commercial supply to patients around the world. HiTech Health can assist Australian partners by providing strategic input for entering the EU and UK markets, QP certification, auditing and quality assurance support, supply chain and project management. HiTech Health is a CDMO for advanced medicines including cell and gene therapies and can provide analytical and process development as well as GMP manufacturing from new facilities in Galway, Ireland.
CCRM Australia’s vision is to enable unique translational platforms that address the bottlenecks in RM commercialisation, integrate Australia’s strength in stem cell and biomaterials sciences with dynamic business leadership, engage industry partners and link to global nexus of RM commercialisation, and internationalise Australian activities leading to RM Powerhouses.
