INSIGHTS INTO CONTRACT SERVICES
PREDICTING THE NEXT DISEASE WITH ANALYTICS
COLD CHAIN LESSONS IN THE PANDEMIC
March/April 2022
REMOTE WORKING
HOW THE CLINICAL TRIALS INDUSTRY MOVED AWAY FROM TRADITIONAL WORKING METHODS DURING THE PANDEMIC TO GO REMOTE.
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Contents
March/April 2022 | Volume 22 Issue 2
REGULARS 5: EDITOR’S DESK Looking back.
6: A SMALL DOSE
A brief round-up of some of the latest developments in the industry.
10: OPINION
The latest research of the microbiome and why it could be essential for cancer care.
13: IN THE NEWS
A short selection of stories from the world of science.
14: COVER STORY
How the clinical trials industry moved away from traditional working methods during the pandemic to go remote.
26: TALKING POINTS
Stories to consider and what to look out for in EPM over the coming weeks.
FEATURES 8: PERSPECTIVE ON PHARMA
How analytics can be used to predict the spread of future disease.
16: LYOPHILISATION
The lessons learned during Covid-19 for the cold chain sector.
18: CONTRACT SERVICES
An in-depth look at the latest challenges and developments within contract services.
4
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5
REFLECTIONS
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Undoubtedly the government will stand behind the success of its vaccine rollout to ensure us it is staying on top of the pandemic.
F
or a long time now, European Pharmaceutical Manufacturer’s content has been influenced by the Covid-19 pandemic. That I’ve worked on this title at such a time has been remarkable. Seeing the response of the life sciences industry was nothing short of inspiring and despite the challenges, the tragic death toll and restrictions on living, the industry’s
actions compounded the fact that this is a sector which ultimately wants to do good. The pharma industry had to adapt to the challenges of Covid-19 much like the rest of us and this has been reflected in developments across the sector’s entire supply chain. We’ve seen remote working start to become a core component in clinical trials, logistics suppliers work out the best ways to transport crucial
EDITOR’S DESK therapies and vaccines, and regulators find ways to expedite approvals so people would have some form of protection against the virus. If this editor’s letter sounds reflective, well that’s because it is. This will be the last edition I helm for European Pharmaceutical Manufacturer and as such I can’t help but consider the journey this title has been on as we’ve navigated the complexity and
developments of the industry. And for all that Covid-19 has dominated in recent years, there have been many other fascinating topics that we’ve covered. Rising environmental concerns within the industry, the use of artificial intelligence (AI) in cancer care, and the provision of mental health treatments using psychedelic compounds - these are just some of the topics that stick in my mind when I think of European Pharmaceutical Manufacturer. So too do the fascinating and — admittedly — far more intelligent people than I, that have kindly given me their time and attention. But still Covid-19 lingers. Here in the UK cases are rising again and the government’s decision to remove restrictions in England means that people are vulnerable. It seems strange that, when at the start of the pandemic people were more cautious of the virus and its spread, restrictions should now be casually thrown away to play up to some vague notion of nation-wide freedom. Now, with testing limited, masks optional and isolating no longer required, the choice has been placed firmly on the shoulders of the public to control the spread of Covid-19. Undoubtedly the government will stand behind the success of its vaccine rollout to ensure us it is staying on top of the pandemic. However, people should remember that it was the hard work of the brilliant scientists, academics and all those working in life sciences that helped us get through the worst of the pandemic. Truly, it’s been a pleasure.
6
A small dose
Almac Pharma Services expands services for ATMPs
A
lmac Pharma Service has expanded its ultra-low temperature commercial packaging, labelling and distribution solutions to support the launch of Advanced Therapy Medicinal Products (ATMPs) from its European Centre of Excellence. The company’s bespoke solutions of Just-in-Time labelling,
packaging and serialisation at ultra-low temperatures (-20°C to -80°C) has been enhanced to offer clients a tailored experience to ensure every step of the process is adjusted to meet the specific requirements of both the client and the valuable product. The dedicated Centre of Excellence is located in Europe and offers an
expansive storage facility with specialist suites and freezer capacity to enable inventory stock of crucial product supply ready for rapid distribution when required. Robert Smith, fellow of the Royal Pharmaceutical Society of Great Britain and Consultant QP to Almac Pharma Services said: “As a result of the nature of these
DEMAND FOR LAB SPACE DRIVES NORTH EAST LIFE SCIENCES
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uge demand for commercial lab and office space at Newcastle’s Biosphere means private sector investment is now being sought to take the lead on expanding the the North East’s £1.7bn life science eco-system. Located at Newcastle Helix, The Biosphere launched in 2019 as a publicly funded project, led by Newcastle City Council; it represented a significant investment for the local economy and showed a clear commitment to life sciences growth. The Biosphere is now home to more than 25 life science businesses and features a specialist lab facility tailored to commercialisation of life sciences, providing
biology and chemistry laboratories and office space. Councillor Nick Forbes, leader of Newcastle City Council said: “Almost three years on from its opening, the Biosphere is a widely recognised success story and quite simply, there is currently more demand for lab space at the Biosphere than can be accommodated. It will be encouraging for any potential investor to know that a strong pipeline made up of existing businesses wanting to grow, alongside inward investors looking for city centre commercial lab and office space, has already been identified. “The health and life science cluster is
The Biosphere is now home to more than 25 life science businesses and features a specialist lab facility tailored to commercialisation of life sciences.
It’s imperative, therefore, to partner with an experienced CDMO. therapies, QP release of ATMPs requires an advanced level of knowledge, training and certification to ensure they understand the biological processes and the variability this brings. It is imperative,
therefore, to partner with an experienced CDMO – like Almac - to facilitate and expedite this crucial stage and have confidence that product will be delivered to the patient as quickly as possible.”
a big employer in the city and a key part of the future economy of Newcastle city centre; we’re ambitious, and looking for a private investor to share the ambition to be a leading international location for innovation in the sector.» Now, the North East Local Enterprise Partnership (NELEP) is providing funding for Newcastle City Council, to develop a business case that will go to market later this year. With the support of Newcastle Gateshead Initiative and Invest Newcastle the aim is to secure a private sector investor to lead the project and create further city centre commercial lab and office space for spinouts, start-ups, growing and established companies to complement the provision already in place. The North East of
England’s regional life science eco-system employs around 7,000 professionals in almost 200 companies, who operate globally from the region. Jen Hartley, director at Invest Newcastle said: “There is already huge interest in the North East as a leading UK location in which to invest, develop or locate, and our time at MIPIM will be spent talking with influential investment and real estate delegates from across the world about opportunities that exist in Newcastle, and at the same continuing to promote the wider region and its strengths. “The Biosphere has created jobs, supported graduate retention and further enhanced our global reputation as a hotspot for life science businesses. Securing private investment will be critical to the growth of the sector,” she said.
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8
PERSPECTIVE ON PHARMA How powerful AI is turning intervention into prevention
W
e know disease exists, where it comes from and how it evolves but we don’t yet know ahead of time when something changes to mark the start of a new disease, a new variant or just an increase in transmission. Often reports and reflection on major events will talk about key points in time where ‘intervention’ or specifically ‘earlier intervention’ could have happened to change outcomes. In respect of Covid-19, there are many, some debatable, points in time where as a collective, nations, governments, health professionals, scientists and data experts could have made earlier interventions to reduce the spread of the virus and ultimately save lives. But that’s the challenge in itself. Without knowing when a disease is going to spread among the population, we are already on the back foot and playing catch-up to get ahead of what is often an incredibly complex and rapidly changing situation. This is where the world’s health professionals and data scientists want to move from intervention to prevention - and why so many clinicians are now relying increasingly on digital health analytics and AI. SIMON TILLEY - global lead for Healthcare and Life Science, SAS Looking at how analytics can predict future spread of disease.
Covid is of course going to be the great example for how we spot and act to prevent future pandemics, epidemics and reduce the spread of disease in our population. We can learn so much from it. Researchers at University of
Covid is of course going to be the great example for how we spot and act to prevent future pandemics, epidemics and reduce the spread of disease in our population.
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California San Diego School of Medicine, and at the University of Arizona and Illumina, estimate that the SARS-CoV-2 virus was likely circulating undetected for at most two months before the first human cases of Covid-19 were described in Wuhan, China in late-December 2019. Chinese authorities cordoned off the region and implemented mitigation measures nationwide. By April 2020, local transmission of the virus was under control - but by then more than 100 countries were reporting cases. That’s five months it took to get the virus under control within one area - but it was already too late to contain. This
is the exact type of scenario where data is already being used to try and prevent similar delays from happening again. Using data to monitor the behaviour of viruses is nothing new. During a Cholera outbreak in London in 1850, physician John Snow discovered, through data analysis, that the areas that were being served by a particular water pump were more affected than others. Shutting down that pump helped to control the pandemic. Also before the days of computers, epidemiologists used marbles to model the spread of an infectious disease. A white marble represented a susceptible individual, a red marble an infected individual
and a black one a recovered immune survivor. What the epidemiologists did was place a red marble randomly into a group of white marbles. Where the red touched the whites, those marbles became infected, and so on, while adding recovering marbles over time. Although very basic, this is actually a good way to visualise what machine learning models do. The difference now is that there are powerful AI and analytical tools available to healthcare professionals, governments and scientists to not only report but predict the future spread of disease. AI uses algorithms to analyse huge datasets - both structured and unstructured - to make accurate predictions in a matter of seconds or minutes. During the pandemic we have seen the UK government consistently use data to reflect on how the virus is spreading and then scientists have used that data to understand the geographical spread and peak of the spread. And of course contact tracing has been used to reduce and slow transmission. But this is using data on the go to track the behaviour of the virus retrospectively, rather than being able to predict events ahead of time. Of course without a modern day pandemic to compare against, it was incredibly difficult to predict how the virus would spread and mutate. Now, organisations like WHO (World Health Organisation) and Center For Disease Control have masses of public data that, alongside government and private medical businesses data, and even mobile phone network operators’ data, can all be powered by machine learning to give us real insights into how to prevent and limit future outbreaks of similar viruses. SAS supports clinical
9 organisations all around the world through its powerful AI and Analytics to help track and understand how infectious diseases spread, so the world can respond more effectively. As one example, we have partnered with PERSOWN. It is developing a novel point-of-care diagnostic platform that plans to provide instant test results using ultra low-cost disposable test strips and a handheld reader, fully integrated with a robust suite of electronic health records applications. This platform will present a cost-effective option for testing in parts of the globe where access to sophisticated medical diagnostics is scarce, potentially creating billions of integrated data points across various disease states and among previously undiagnosed populations. Through the partnership and the use of visual analytic and data management software we hope that PERSOWN will be able to effectively mine diagnostic data to uncover infection trends and visualise disease hot spots to better monitor and predict outbreaks. These early insights will help governments and health organisations implement practices that reduce disease spread, mitigate impact and create early interventions. Through AI and machine learning, data sources are connected mathematically to predict how viruses will behave. A model is the representation of these mathematical connections and more and more clinicians around the world will be using these machine learning models to effectively predict and prepare to prevent the spread of infectious disease. Data scientists and health professionals are working tirelessly to finally answer the question, when does something change that marks the spread of a virus?
10
Opinion
Greater understanding of the human microbiome, particularly that of the gut, is helping drug developers to identify mechanisms of resistance and response to certain therapies.
CANCER IMMUNOTHERAPY: THE BEST IS YET TO COME Author: JUSTIN WILSON - partner and patent attorney at Withers & Rogers.
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The promising innovations in the microbiome and the role of IP in supporting the discovery of new drugs and treatments.
arnessing the therapeutic powers of the human microbiome is an exciting area of scientific research which is hoping to unlock the potential of the immune system and improve the efficacy of cancer treatments. But which novel therapeutics and combination therapies will deliver the best patient outcomes? The successful use of viral vector technology in the development of OxfordAstraZeneca’s two-dose Covid-19 vaccine has raised public awareness of the role of immunotherapy in the treatment of diseases. Building on this, research scientists from the University of Oxford and the Ludwig Institute for Cancer Research are developing a new cancer vaccine that makes use of similar technology. The vaccine flags cancer-specific antigens to the immune system which causes an increase in the response of tumour-infiltrating T-cells. In a study of mouse tumour models, the vaccine was shown to increase the levels of anti-tumour T cells infiltrating the tumours, and as such, improved the efficacy of cancer immunotherapy. The cancer vaccine is now being assessed in a Phase 1/2a clinical trial featuring 80 patients with non-small cell lung cancer.
12
OPINION
As Adrian Hill, Lakshmi Mittal and Family Professorship of Vaccinology and Director of the Jenner Institute, University of Oxford puts it: “This new vaccine platform has the potential to revolutionise cancer treatment.” Over the past decade, improved understanding of the immune system has inspired the pharmaceutical industry to explore its use in the treatment of cancer. Improved knowledge in these areas is helping drug companies to identify subsets of cancer patients who would benefit from specific cancer vaccines or other therapies. Improved detection of target biomarkers can also facilitate smaller, precision trials and shorten drug development timelines. In particular, greater understanding of the human microbiome, particularly that of the gut, is helping drug developers to identify mechanisms of resistance and response to certain therapies. Using this knowledge, treatments can be directed to individuals who are pre-disposed to achieve a better outcome. It is also hoped that better understanding of a patient’s microbiome could help to decrease the risk of toxic side effects and improve the efficacy of specific treatments. Much innovation activity to date has centred on tumour-infiltrating lymphocytes (TILs) and CAR T-cell therapies - such as those targeting the antigens CD19, and more recently, BCMA. Some treatments focus on activating TILs, whilst others work by isolating and proliferating TILs from solid tumours to boost the quantity of tumour-reactive T-cells. CAR T-cell therapy is a type of immunotherapy involving the adoptive transfer of T-cells that have been genetically modified with a chimeric antigen receptor to target a tumour. CAR T-cell therapies have been found to be most effective in the treatment of blood cancers; however, considerable research activity is directed towards their utilisation and optimisation for solid tumour cancers. Another important area of R&D is focused on immune checkpoint inhibitors (ICIs), which have shown great promise in improving the survival rates of some cancer patients. These treatments have been found to be particularly effective in the treatment of melanoma and non-small cell lung cancers. ICIs, such as anti-PD1, essentially work by releasing the brakes on T-cells to target and kill the cancer cells. However, the overall response rate of these therapies remains unsatisfactory and high-grade toxicity is a significant problem. For example, over 50% of those receiving antiCTLA4 and anti-PD1 blockade may require hospitalisation. To improve outcomes for cancer patients, some innovators are looking for ways to alter a patient’s gut microbiome in combination with immunotherapy. The gut’s microbiome comprises around 100 trillion micro-organisms, encoding over three million genes and producing thousands of metabolites. Convincing evidence is now emerging, suggesting that the gut microbiome could impact the development of some types of cancer – mainly those relating to the gastrointestinal and hepatobiliary systems. It could also impact responses to other cancer therapeutics such as ICIs. Research focuses on various ways of leveraging the microbiome such as by microbial consortia or genetically modified microbial strains. Live biotherapeutics for use in the treatment of cancer may also prove particularly beneficial in improving outcomes when used as an adjuvant for treatments such as ICIs. For example, UK-based 4D Pharma PLC have an expanding portfolio of over 1,000 patents in the microbiome sector and their current pipeline focuses on the efficacy of lead products and candidates as monotherapies and combination treatments for cancer. With drug companies learning more about how the gut microbiome can impact the outcome of patients receiving immunotherapy for the treatment of cancer, this strand of research and development is expected to continue to develop. With commercial protection in place, those responsible for discovering new ways to improve the efficacy of immuno-oncology treatments have an opportunity to leverage their innovations commercially, while improving outcomes for cancer patients the world over.
13
IN THE NEWS
Social media could be key to vaccinating older generation, study shows S
ocial media advertising could be a cost-effective of way of encouraging older people in deprived communities to get vaccinated, new research shows. Research from the International Longevity Centre-UK (ILC) found that targeting older users directly on social media can be a more effective way of encouraging them to get vaccinated compared to using younger relatives to convince them. ILC conducted a largescale target social media campaign to test whether or not older people were less likely to use social media and how they would engage with a vaccination campaign. ILC’s campaign aimed to find out whether social media could be used to engage older people via their younger relatives using a large-scale targeted social media advertising campaign. ILCs campaign reached over a million people and generated over 5 million impressions. Through these campaigns, ILC was able to costeffectively encourage marginalised older adults to get their flu jab. For the pneumococcal vaccine, the campaigns had an even greater impact and cost less than a third of the price per booking link click than the flu adverts – most likely because many
people had already received the flu vaccine, while the pneumococcal vaccine was relatively unknown. ILC are now calling for the government and NHS to invest more in targeted social media campaign to improve vaccination uptake. ILC is also arguing for the NHS to make it easier for people to access information on vaccination and the vaccines themselves through the use of community pharmacies and a single online hub where people can book routine vaccination appointments. Sophia Dimitriadis, senior economist at ILC and lead researcher on the project, said: “Social media often gets a bad rep, especially surrounding the disinformation and fake news it can be used to spread. But we’ve shown that social media has huge potential when it comes to immunisation – including for people living in deprived communities, who are typically hard to reach through the traditional healthcare system. And yet, there is so much untapped potential still left to unlock. Our campaigns generated more engagement and at a cheaper rate than the average healthcare industry advert. Social media should become part of our arsenal for promoting and protecting public health.”
Did you know?
More than half (59.1%) said they were aware of “fake news” surrounding Covid-19.
The top concern for respondents was the risk of friends and family catching Covid-19.
In a World Health Organization (WHO) study of 23,500 people, 43.9% of respondents said they would likely share “scientific” content on social media.
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14
D
uring the pandemic, we all got used to doing things remotely, whether in our professional or personal lives. The same has also been true of clinical trials. As patients and clinicians alike were forced to stay home whenever possible, reducing the necessity to conduct trial activity on-site became a key focus of the drug development industry, driving interest in the concept of decentralised clinical trials (DCTs).
COVER STORY
How the clinical trials industry moved away from traditional working methods during the pandemic to go remote.
WHAT EXACTLY IS A “DECENTRALISED CLINICAL TRIAL”? It refers to shifting trial activities out of the traditional clinical site setting and making them remote or virtual. That doesn’t necessarily mean the entire trial is conducted remotely. It could, for example, mean that patients are enrolled remotely via a tool like electronic informed consent (eConsent), but then have in-person checkups moving forward. Or we might reduce the number of face-to-face clinician meetings by using remote consultation or electronic Clinical Outcome Assessment (eCOA). It’s a spectrum and it’s about the ability to be flexible and patient-centric - the remote elements can be increased or decreased depending on the specific clinical trial or even the patient’s preference. This can help decrease burden on both patients and sites. HOW DO YOU DECIDE ON THE RIGHT LEVEL OF DECENTRALISATION? This often comes down to the therapeutic area, the nature of the patient population and the study design. In an oncology study, where patients are likely to have a higher burden of illness, they will often be coming on-site anyway to see their healthcare providers so it might make sense to continue conducting most of the trial
REMOTE WORKING
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Author: PAUL O’DONOHOE - senior director of eCOA Product and Science at Medidata, a Dassault Systèmes company
activities on-site. However, the patient – or the trial itself – could still benefit from aspects of decentralisation. It’s still quite easy for patients to complete questionnaires or patient diaries at home, or have their activity levels monitored between site visits using wearable devices or sensors. At the other end of the spectrum, if you are conducting a trial in rare diseases, where patients are scarce and often need to travel long distances, it is likely to be of significant benefit to the patient to be able to complete more elements of the trial remotely. The “right” level of decentralisation will vary a lot from study to study, even within the same therapeutic area. It’s important that contract research organisations (CROs) and pharma/biotech companies, or sponsors, think about this early on in the process to ensure the optimal approaches are included in the study protocol. WHAT ARE THE BENEFITS FOR PATIENTS? One of the key drivers behind DCTs has been the desire to improve the patient experience. We consistently hear from patients that trial participation is too burdensome and that visits to trial sites in particular are difficult to navigate. People have to take time off work, find childcare and/or travel to trial sites which can be far away. Being able to reduce or remove the need for such visits makes a massive difference in reducing the burden on patients. There are other, more intangible, benefits. Regular digital communication with clinicians or interactions with apps and software helps patients feel more connected to a trial. Rather than only being involved when they are on-site every two months, they feel like they are being listened to throughout the trial journey. WHAT ARE THE BENEFITS FOR CLINICIANS? If patients feel more engaged in a trial, they are more likely to be compliant, more likely to deliver high-quality data, more likely to provide it faster, and will be less likely to drop out of the trial. This all has the knock-on effect of improving the quality and rigour of the trial, making it easier to understand the safety and effectiveness of treatments.
The “right” level of decentralisation will vary a lot from study to study, even within the same therapeutic area.
15
DCT solutions can also provide clinicians a near real-time insight into how a trial is progressing. This means any issues with the study or a specific trial site can be identified and addressed as soon as possible. TO WHAT EXTENT WERE CROS AND SPONSORS USING THE FULL RANGE OF DCT TECHNOLOGY BEFORE THE PANDEMIC? WHAT HAS CHANGED? Electronic data capture (EDC) and eCOA tools have been in use for decades and there has been great interest in the use of sensors and wearables in trials in recent years. However, the pandemic made the uptake of DCTs a necessity. In response to sites closing due to lockdowns, regulators showed a lot of flexibility and open-mindedness towards using technology as a way of mitigating the impact and ensuring patient safety. This has led to a psychological change in the industry which is now feeling more comfortable using these technologies. There has also been a trend towards making different tools and solutions more ‘joined-up’. With data coming in from various sources, it’s important to have a way to consolidate it and properly be able to store, review and analyse it. WHAT ROLE DID CROS PLAY IN FACILITATING THIS CHANGE TO DCTS? Global CROs have a lot of exposure to the latest technologies and need to be flexible and able to work with all tools and solutions on the market. As such, they tend to be at the forefront of driving change and adopting improved approaches. They’ve always been strong proponents of DCTs. At the start of the pandemic, while the need for rapid change posed many challenges, CROs did an excellent job of working closely with service providers, sponsors and regulators to onboard tools and solutions effectively in order to support the continuation of clinical research. WHAT NEXT FOR DCTS? We are already starting to see many sponsors build in contingency planning in case we experience further lockdowns or even another pandemic. However, we are also moving away from thinking of using DCT technologies only for emergencies and starting to see CROs and sponsors using the full range of these technologies – from EDC to eCOA to wearables – more systematically, with decentralisation planned from the earliest stages of trial design. The rise of DCTs may have come about due to necessity, but it will stay because it can offer a more efficient and effective approach to drug development.
16
LYOPHILISATION
FROM QUALITY CONTROL TO COLD CHAIN
– WHAT THE PANDEMIC TAUGHT US The impact of Covid-19 on the pharma supply chain and the lessons learned in the race to a vaccine.
Author: JEFF SMYTHE - commercial development manager, Datwyler
I
n the wake of the Covid-19 pandemic, the pharmaceutical industry and its suppliers had to mobilise overnight in order to implement a healthcare solution as quickly as possible. Although the world had seen pandemics as recent as the spread of H1N1, the severity and impact of Covid-19 presented a global challenge due to how easily transmittable the disease is and the number of deaths worldwide. Capacity reservation and emergency protocols are not a new concept but following H1N1, the industry seemingly moved away from a preventative approach to emergency response planning. The current pandemic is a testament to this shift, with the industry taking a reactive approach by drawing on past experiences with a certain level of preparedness and planning. As the pandemic continues to unfold and unprecedented demands for drug delivery components and packaging continue to arise, it is imperative that we prepare for future occurrences and move closer to a proactive approach. TRANSPARENCY IS KEY Drug manufacturers faced exponential volume increases as mass vaccination plans
progressed in 2021. These demands rippled out to their suppliers of packaging components like vials and stoppers. What became clear from the outset is that communication channels needed to be as clear and transparent as possible to deliver what was realistic in a short timeframe. Prior to the pandemic, pharmaceutical
companies were less inclined to share challenges with their production, especially since competing manufacturers can use that information to their advantage. However, the pandemic opened communication channels beyond what we would generally see across the board. The pandemic forced the
industry from a norm of closely guarded planning to a more open and communicative industry as all players involved had to work closely to resolve major supply chain challenges, anticipate potential personnel and raw material shortages, and navigate planning amid a global crisis. It forged the most united front this industry has seen in decades.
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THE CORE TO AN EFFECTIVE RESPONSE: RISK MANAGEMENT AND QUALITY Just as supply chains were impacted, so was the ability to mitigate packaging supply chain risk with the lack of necessary materials and machinery to meet demands. Across the industry, the impact varied as countries reacted based on the severity of the situation that unfolded domestically. Moreover, in addition to supply chain challenges as well as impacted labor and difficulty sourcing equipment such as PPE, expanding capacity in terms of machinery and materials was also difficult. As the industry moves forward, it will benefit from
being better prepared for any interruption in supply of materials, lockdowns, both locally and nationally, as well as a hugely reduced labor force. In the short-term, it is critical to build significant safety stocks to mitigate the impact of disruption. In terms of cold chains, formulators and drug development experts continue to work on 2-8 Celsius options or even vaccines that can be stored at room temperature. During the pandemic, initial storage conditions were reexamined to see if less severe storage conditions could be used. As supported by stability data, the next generation of vaccines and boosters will likely have
During the pandemic, initial storage conditions were reexamined to see if less severe storage conditions could be use. storage conditions optimised for the best possible global distribution. That said, the industry continues to work to simplify the cold chain by increasing the temperatures at which drugs need to be stored. QUALITY CONTROL IS PARAMOUNT Quality is codified by global authorities. It is not optional, nor is it flexible – and rightly so. The pharma industry is incredibly stringent and holds itself to the highest standards and with the goal to disseminate vaccines to every person on the globe, this is more important than ever before. It is critical that even
when volume is increased, quality standards are still met. There are certain standards to which manufacturers must adhere to in this industry. All involved in the response to Covid-19 have been acutely aware of this fact from the outset. Even prior to the pandemic, the industry has operated with the understanding that cutting corners could have potentially life-threatening consequences. Sacrificing quality and safety is not an option. With so much pressure to meet unprecedented demand, flexibility became paramount for drug manufacturers. If minor changes were required,
they were allowed some latitude to get things done more quickly. Datwyler for example, proactively streamlined the pathway to qualification, and qualified components in the greatest volume in the shortest time. The authorities were a bit more flexible in their approach to the production of pharmaceuticals, but only up to a point. For the packaging and distribution elements there was no change. RISK REDUCTION IS KEY Whether it’s a Covid-19 vaccine or any other critical drug, risk reduction is a vital element throughout the entire drug delivery process. As such, packaging suppliers are evolving to take already stringent manufacturing practices to another level. FirstLine manufacturing, for example, is based on ultramodern cleanroom technology, automated production cells, automated camera inspection, and a unique validated washing process. Designed and operated under a zero-defect philosophy, FirstLine manufacturing facilities keeps particulate extremely low to avoid contamination once the vaccine is packaged and ensures that what reaches the end user is of the highest quality. These strategies combined with a fluoropolymer spray coating, can significantly reduce risk of contamination. As a result, particulates or unanticipated interactions between the vaccine or drug and the closure are greatly reduced or eliminated, lowering overall risk in the production of the vaccine. Further automation capabilities like those used in FirstLine facilities allow for increased production with the exact same world-class quality levels without having to add labor. Before the pandemic compromised employees’
17 safety and, in most cases, prevented people from going to work, interruptions in labor were not fully considered. The industry had already been moving towards greater levels of automation to ensure quality, but reduced labor only reinforced the need for solutions that address capacity limits and prevent disruptions. The pandemic highlighted the fact that in addition to reducing human error and increasing efficiency, automation tackles unexpected interruptions to labor. In the event of a global pandemic, demand can still be met when automation tools are in place. MANY LESSONS HAVE BEEN LEARNED Just as any pandemic before it, Covid-19 brought core lessons to be learned that helped push the industry forward and improve global health. For one, transparency is vital and embracing greater communication helps the industry better plan and tackle unanticipated occurrences. An industry-wide proactive approach towards preparing for something like another global healthcare crisis will be the rule going forward. It is imperative to have a formal pandemic plan in place with protocols that identify the types of closures, the overall packaging and better ensure immediate capacities along with scale up plans. The pharma industry is one of the most impactful in the world and it has risen to the challenge of the Covid-19 pandemic in ways few would have thought possible. With a continued focus on collaboration, cooperation, and transparency, that impact can be harnessed even further to tackle challenges in the future and contribute to better and healthier communities all around the world.
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CONTRACT MANUFACTURING
Cutting it Lonza on APIs Steps to manufacturing highly potent APIs under shorter timelines
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ver the last few years, highly potent active pharmaceutical ingredients (HPAPIs) have become more prevalent in the drug development pipeline driven by research in enhancing the precision and bioavailability of these therapies. Currently, more than 30% of the small molecules in production are HPAPIs, and its increased use has led to a pipeline of more effective treatment options, with potentially
lower dose requirements and/or fewer side effects. Oncology research drives the majority of HPAPI production as it has been associated with inhibiting tumour cell growth and demonstrated usefulness in cancer treatments. Outside of oncology, HPAPI molecules have also shown effectiveness in treating conditions such as autoimmune diseases and diabetes. With more research at
EPM speaks to Swiss contract development manufacturing organisation (CDMO) Lonza to learn about the advances taking place across the industry to manufacture high potency active pharmaceutical ingredients (HPAPIs).
An active market
Lonza on APIs
EPM: What are some of the emerging trends we’re seeing right now in the HPAPI market? RL: We see a direct relationship between the continued validation of precision medicine approaches and the growth of high potency APIs (HPAPIs) in the development pipeline and in regulatory approvals. Medicinal chemists have become extremely efficient at innovating exquisitely targeted protein modulators with impressive binding affinities. Precise delivery mechanisms and bioavailability enhancing technologies are enabling HPAPIs to create a pipeline of more effective medicines, with
potentially lower dose requirements and fewer side effects. Interest in highly potent drugs is largely driven by oncology research and more targeted therapies across a number of indications. Currently, over 1,000 highly potent small molecules are in development, with approximately 30% targeting oncology, 20% for antidiabetics, 20% for autoimmune diseases and the remainder for other indications. Due to their wide range of potential uses and benefits for patients, the growth in the HPAPI market is outpacing the overall API market by almost two-to-one. The segment
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hand, regulators understand that HPAPI-based therapies fill an unmet need of targeted therapies with fewer adverse side effects, thus making it easier to inform approvals. This combination of factors has led to HPAPIs making up over 30% of the drug development pipeline, outpacing the growth of the overall API market by almost two-to-one. This upward trend presents significant manufacturing challenges for innovators and their external partners. This is especially relevant for smaller biotech and start-ups who continue to bring innovative HPAPI molecules and products to the table. Particularly, these companies are submitting more HPAPI drug products for approval with orphan designations and/or to treat serious medical conditions with no or limited treatment options available, which
Managing shortened timelines for HPAPI drug products will continue to be a top priority for pharma and biotech companies. usually means operating under accelerated timelines to the market. As they navigate these shorter timelines, smaller companies may benefit from a strategic external partner with access to in-house expertise and manufacturing assets. In these cases, contract development and manufacturing organisation (CDMO) partners can help by providing their expertise, engaging in technology transfer activities, and exchanging information, ideas, and best practices across the drug development cycle. On top of that, the right partner can help emerging companies create
robust manufacturing processes for two critical deliverables in shortened timelines: drug substance and drug product. SQUARE ONE: DEVELOPING HPAPI DRUG SUBSTANCE As emerging companies enter the development pipeline, their first challenge is ensuring enough of the drug substance is produced to cover current and future clinical phases. However, manufacturing processes in the pre-clinical phase are different from those in later stages as priorities change. With phase-appropriate objectives in mind, there are
is growing at about 10% compound annual growth rate, compared to 6% for the overall small molecule market. The market value from existing and new HPAPI product launches is expected to double between 2018 and 2025, from around $18 billion to $35 billion.
Authors: RYAN LITTICH, director, Strategy & Planning, Small Molecules
CONRAD ROTEN, group leader R&D Clinical Phase and HPAPI Projects at Lonza.
EPM: How have CDMOs adapted to be able to manufacture HPAPI products? RL: In response to the growing need for sophisticated HPAPI manufacturing, some CDMOs are building flexible, integrated capabilities dedicated to HPAPI development from preclinical to commercial production. These programmes place an emphasis on safe procedures from equipment start-up to handling, cleaning and decontamination, and they come as outsourcing of HPAPI production is increasing at a rapid pace. Demand for highly potent small molecule API manufacturing and development services is
key factors to consider when developing HPAPI drug substance in accelerated timelines: Manufacturing scale: Pharma companies should have phaseappropriate manufacturing scales proposed early in the development process that reflect current needs and future growth. Manufacturing processes: Robust processes mean that manufacturing inefficiencies are trimmed immediately when they are identified. This ensures these inefficiencies aren’t carried out throughout the development and confirms a close eye on process performance and quality. Containment: Companies must remain compliant with local containment regulations to ensure the safety of their employees. This allows for efficient technology transfer and manufacturing timelines. As early-stage companies navigate drug substance production for clinical trials, it is critical to always have a
keeping pace with the overall HPAPI market value, and is expected to exceed $7.5 billion by 2023, more than doubling the $3.5 billion market size in 2015. For biopharma innovators, working with a single partner across the development pipeline can shorten timelines and reduce risk. CDMO partners can access in-house experts across a range of drug substance and drug product challenges, engage in technology transfer activities and exchange information, ideas and best practices across the drug development cycle. A study from Tufts Center for the Study of Drug Development has found that a single-source outsourcing model can shorten the drug development cycle by 14 weeks and lead to financial gains of up to $45 million. For HPAPI compounds, drug product capabilities will include parenteral formulations / sterile fill-finish services and oral delivery options such as liquid-filled hard capsule
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rounded view of manufacturing and its different stages. This will help them create efficient drug substance procedures as well as prepare for the next stages of development. GETTING THE DOSAGE FORM RIGHT: DRUG PRODUCT DEVELOPMENT • After producing the HPAPI drug substance, pharma companies will need to develop drug product intermediates (DPI) and drug products (DPs). Finding the right dosage form in accelerated timelines requires expertise in the following topics: • Formulation technology: Understanding of the formulation problem statement as well as the best approach to meet the target drug product profile. Similarly, any bioavailability-related issues and the right enhancing technology to resolve it. • Enabling technology: Identifying the appropriate enabling technology and/or specialised
processing required is critical while putting in place a phase-appropriate process for non-cGMP feasibility and cGMP clinical manufacture. • Devices: Understanding of any special devices required for drug administration and their impact on the formulation and/ or dosage form. • Drug product supply: How to navigate market demand fluctuations regarding regulation and manufacturing to support product supply. These factors are critical in both creating the drug product and establishing robust processes that save time in the long run. With a qualified partner, emerging companies can make more appropriate DPI and DP decisions. For example, they can conduct feasibility studies in the early stages to tell whether a candidate’s insufficient clinical effect is due to the candidate itself or the formulation. This allows avoiding abandoning candidates that could otherwise
technology. Contained particle engineering can also be required for safe and effective jet milling or spray dry processing. Outsourcing development to a qualified CDMO can help meet accelerated production timelines and help deliver life-saving therapies more quickly to patients who need them. EPM: What are some of the best practices in terms of containment and handling when it comes to HPAPIs? CR: This trend towards greater use of HPAPI molecules presents significant potential benefits for patients, but it also comes with handling, containment and manufacturing challenges for innovators and their development partners, since these molecules can be dangerous if mishandled. Safe HPAPI handling can be ensured by focusing on facility design, protection strategy & procedures and personal protective equipment.
have been optimised by a different formulation approach. A SINGLE INTEGRATED CDMO PARTNER Conventionally, pharma companies work with several CDMOs in different stages of the development process: working on the drug substance with one and then the drug product with another. However, each partner in this value chain will again sample, test, and release the product before moving forward – creating redundant activities that consume more time and resources. Many issues cross between different stages of the process, including packaging requirements, quality control and quality assurance, liability questions, optimising schedules and timelines to avoid full retesting. This is especially true for manufacturing HPAPI payloads for antibody-drug conjugates (ADCs), which requires both small and large molecules manufacturing expertise. Having
Sophisticated facility design elements include several tools to ensure safe handling of potent materials. Units are available for primary and secondary containment of the entire process including solid charging containment, sampling and unloading. For product sampling, liner ports can help lock in and lock out glass sampling bottles. To aid in the product unloading process, manufacturers can use endless liner systems and customised flex isolators (in some cases more rigid, hard-walled isolators may be preferred). Well-designed protection strategies include nuanced and detailed processes for how workers can use and clean HPAPI manufacturing assets. For example, cleaning procedures should include clear acceptance criteria for opening equipment after precleaning. And the safest equipment start-up sequences are embedded into the risk assessment process, with leak test and rinse prior to production and defined criteria
Authors: JAKOB BONDE, director, CMC Regulatory Affairs, Small Molecules RYAN LITTICH, PhD, Director, R&D Small Molecules at Lonza
The CDMO industry will have to listen to its customers’ needs and ideally be involved from the very early stages of the product life cycle in a close and collaborative manner. allowing for production release. Personal protective equipment (PPE) is of course extremely important to keep workers safe. Reliable equipment including coveralls, speciality hoods and isolators, gloves, chemical suits, supplied air (if appropriate) and other implements should be available in ample supply for anyone working with highly potent materials. However, the use of PPE should be the last resort and not the main method of protection for routine operations. Adequate organisational measures and
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BIOPHARMA & MANUFACTURING
multiple partners to produce your ADC’s biologics and chemical components may unnecessarily elongate timelines as they will be in separate locations. The best way to solve these issues is from a holistic perspective, rather than homing in on one value chain point. This means that working with fewer partners who will have a bird’s eye view of the processes will save complexity, effort, time, and costs. One solution is having an integrated partner. A study from Tufts Center for the Study of Drug Development found that a single-source outsourcing model shortens the drug development cycle by up to 14 weeks while increasing financial gains by up to $45 million. This data suggests that outsourcing to a qualified CDMO where all activities are managed by one system overseeing all early and late-stage activities may give
emerging companies a critical leg up that would put them ahead of competitors. SELECTING THE RIGHT PARTNER Managing shortened timelines for HPAPI drug products will continue to be a top priority for pharma and biotech companies. Emerging companies will need to be tactical in choosing their manufacturing partner to scale up their products and compete with larger companies. To ensure that they find the right fit, leaders should evaluate technical expertise while visiting facilities and conducting interviews with site management. This approach will allow them to gather the right insights into whether that specific CDMO candidate has the resources and expertise specific to their programme to help them overcome accelerated timelines for their HPAPIs.
procedures can often accommodate PPE-free operations, keeping the PPE only for non-routine operations. EPM: What’s next for HPAPI drug products in terms of the future? CR: As HPAPI molecules continue to play a growing role in new pharmaceutical products, biopharma companies will need access to flexible, sophisticated manufacturing assets to bring their innovative products to market and to patients. Given the high potency of these molecules, safety and containment are key components of effective HPAPI manufacturing. Working with a qualified CDMO can help biopharma companies advance HPAPI compounds and products at high speed and low risk. When evaluating external partners, biopharma innovators should base their choice on the CDMO’s experience in the HPAPI space and the containment, handling and manufacturing assets the partner brings to bear.
EPM: With the rise of specialised medicines is there enough manufacturing expertise to match demand from the biotech sector? RL: Close communication and alignment between customers and the CDMO industry are required to ensure that the appropriate manufacturing expertise is built and put into place when it is needed. The CDMO industry will have to listen to its customers’ needs and ideally be involved from the very early stages of the product life cycle in a close and collaborative manner. In terms of nurturing talent and expertise in CDMOs, at Lonza our people are a core part of our strategy. We focus on keeping our expertise in-house and have defined action plans that further develop critical skillsets. We commit ourselves to improving the customer experience through an engaged workforce and continue to be strategic in attracting strong talent.
35 T
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EDITION
35TH
31 May - 1 June 2022 Messe Frankfurt, Germany
The place to be for the fine & speciality chemicals industry Find bespoke solutions & innovative substances for your enterprise and take your networking to the next level: • custom synthesis • pharmaceuticals • fine chemicals • chemical intermediates • agrochemicals • adhesives & sealants • paints & coatings
• colourants & dyestuffs • flavours & fragrances • green chemicals • household & industrial cleaning • biocatalysts • bio-based chemicals
• plastics additives • cosmetics • polymers • surfactants • petrochemicals • electronic chemicals • and much more
Top conferences and panel discussions offer valuable industry insights! Agrochemical Lecture Theatre Chemspec Careers Clinic Pharma Lecture Theatre Regulatory Services Lecture Theatre RSC Lecture Theatre Innovative Start-ups
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MANUFACTURING & PROCESS SOLUTIONS
Castle Creek Biosciences discuss their lessons learned in establishing an in-house cGMP manufacturing facility with the capability to produce clinical and potentially commercial supply of cell and gene therapies.
Learning along the
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ith the rise of precision medicine over the last decade, including many cell and gene therapies, companies need to manufacture potential treatments in a reliable and reproducible way while ensuring safety and efficacy for patients. There are several unique challenges in the development of cell and gene therapies and a range of factors to consider, such as administration methods, vector type, therapeutic approach (e.g., in vivo or ex vivo), target indication and size of eligible patient populations. The nuances of cell and gene therapy production require specific expertise and skills; for many companies this means outsourcing manufacturing capabilities to third parties, including contract development and manufacturing organisations (CDMOs). Nevertheless, there are a select few cell and gene therapy companies that have learned to overcome the inherent challenges and have developed in-house current good manufacturing practice (cGMP) manufacturing capabilities. As a result, they have realised the benefits of having an in-house facility. For example, companies can manage many aspects of manufacturing, quality control, logistics, and supply chain and have oversight of processes and timelines at
different stages of production. There is less risk of wait times for technology transfer or product delays compared to using a CDMO or outside vendor. If an in-house facility is established early enough in the drug development process, companies can also control manufacturing and delivery of products for clinical trials as well as, ultimately, commercial supply, pending market approval. Another advantage of in-house manufacturing is that it employs a highly skilled and dedicated staff who have a vested interest in the patient communities they serve. Also, these employees take ownership of their roles and responsibilities to consistently perform the many precisionbased techniques required for producing cell and gene therapies.
At Castle Creek, our cGMP facility benefits from the validated systems and processes previously implemented at the site for manufacture of a prior FDAapproved autologous cell therapy. But that does not mean the path to building an in-house facility for manufacturing our proprietary autologous cell and gene therapies wasn’t challenging, particularly because there was no precedent-setting approach to create the operation. Following are five key strategies that we learned along the way that other cell and gene therapy companies might consider. 1. BE FORWARD-LOOKING AND THOROUGH, WITH NO SHORTCUTS When building an in-house manufacturing operation,
ideally, companies should design the facility to allow for future expansion of its pipeline and add the technology, processes, and systems to support it. Manufacturing processes for most cell and gene therapies are lengthy, complex, and may be difficult to expand as production needs increase. In just one example, the manufacture of genetically-modified fibroblast cells for an autologous gene therapy requires a process that must be replicated in individualised batches for each patient. To meet quality control (QC) and demand at every stage, production may require customised technologies and innovations in manufacturing techniques. Companies should always be thorough in assessment of needs, not take any shortcuts, and
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way
invest in advanced technologies that are critical to developing clinical and commercial-ready products. Accepting sub-optimal technologies or any underdeveloped processes that are not productive for scale up at the commercial level would not be a prudent investment in time or resources. 2. COMPLIANCE IS ESSENTIAL cGMP compliance is a critical requirement in developing personalised therapies. Cell and gene therapy companies must focus on implementation of quality management systems to ensure cGMP-compliant processes and methods, and advanced laboratory equipment and technologies play a central role in these efforts. It is especially important to focus on compliance as production transitions from research to clinical to commercial stages. The
transition from a development and pre-clinical environment to an organisation that fully bears the compliance mindset requires strong leadership, discipline, and a keen eye for detail. Compliance is not just a bolt-on set of procedures – it is a behaviour and a way of life. 3. BE INSPECTION-READY As companies are focused on manufacturing safe and effective cell and gene therapies, whether for clinical trials or commercial supply, keep in mind that maintaining a safe and compliant operation is imperative at all times. Processes and systems should be used and managed daily, including consistent and timely preparation and assembly of key documents and records, making them readily accessible, keeping all files current, and having risk monitoring systems in place. In addition, maintenance of a facility’s physical space and equipment should always be a priority to ensure a safe, functional, and compliant working environment. Constant attention is essential to keep the people, plant, and processes in an inspection-ready mode. There should be no need to scramble to prepare for an audit or inspection – once you attain a high level of readiness, it’s all about staying there. 4. BE COLLABORATIVE, AND ACCOUNTABLE Another key factor in successfully establishing an in-house manufacturing facility is fostering a positive and collaborative corporate culture, which starts with strong company management who lead by example. Management must also be able to recognise their team’s skill sets and put the right people in positions to succeed as well as support them along the way. Team members, from executive leaders to managers to employees at
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all levels, are encouraged to collaborate and hold each other accountable, including when addressing any issues or concerns. 5. RECOGNISE KEY INFLECTION POINTS During the product lifecycle, companies will experience key inflection points when important decisions must be made that may determine their success. For example, companies may need to decide when the time is right to move from manual to electronic systems in terms of documentation or logistics. Assessing, prioritising, and investing in upgrades to operations is an ongoing process. When appropriate, companies may consider striking a balance between working with an outside vendor and conducting work internally.
Authors: MATTHEW GANTZ & MICHAEL MAURER - Castle Creek Biosciences
The major challenges in building an in-house manufacturing facility for cell and gene therapies are determining the appropriate timing to do so and acquiring funding to make it happen. Companies must prepare for every stage of drug development and continually assess processes, equipment and logistics and determine if and when upgrades are necessary. In these efforts, the overall and long-term goal continues to be to bring safe and effective therapies to patients in need.
Management must also be able to recognise their team’s skill sets and put the right people in positions to succeed as well as support them along the way.
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England launches rare disease action plan
ngland has launched a Rare Diseases Action Plan in a widespread effort to support faster diagnosis and understanding of rare diseases. The plan includes 16 commitments to improve care for people living with or who have an undiagnosed rare disease. Health and Social Care Secretary Sajid Javid said: “This action plan will speed up diagnoses and care and allow our fantastic workforce to better support patients, by drawing upon the UK’s world-leading science and technology. “I am committed to levelling up our health system so that everyone regardless of their condition can receive treatment that is tailored to their needs.” The plan includes commitments on research, including an announcement of £40 million of new funding to the National Institute for Health Research (NIHR) BioResource, to further its work in characterising and understanding rare diseases. Another part of the plan also aims to increase the ability to spot genetic conditions during the screening of newborn babies so diagnoses can be made earlier. This includes a new research pilot using whole genome sequencing to screen for rare genetic conditions in healthy newborns, and improvements to the way the UK National Screening Committee makes decisions on rare diseases.
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UK INVESTS IN LIFE SCIENCES
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p to £260 million of government funding will be made available to expand life sciences manufacturing in the UK. The majority of the funding (£200 million) will be used to invest in digital capabilities in clinical trial services and to help researchers better access NHS data through Trusted Research Environments. The government hopes the funding will make crucial data more secure and quicker to access for research. It’s also intended to ensure that the NHS will be able to deliver new treatments to patients faster and support more diverse and inclusive clinical research to tackle health inequalities and improve patient care. A further £60 million will be available to support commercial-scale manufacturing investments by companies developing a range of technologies including medical
devices and diagnostics, and cell and gene therapies. The funding for manufacturing investments will be distributed through the new Life Sciences Innovative Manufacturing Fund (LSIMF), which has been launched to grow the UK’s life sciences sector. Business Secretary Kwasi Kwarteng said: “The past two years have shown just how important our fantastic medical and life sciences industry is for the UK not only in dealing with the pandemic, but also for the vital economic opportunities they create throughout the country. By providing £260 million to boost UK medical manufacturing and medical research, we are ensuring the industry has the support it needs to improve patient outcomes and generate high-skilled jobs while building up Britain’s manufacturing base in the process.”
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s the leading event for medical device and supply chain intelligence, Med-Tech Innovations News explores the top 10 benefits of exhibiting at Med-Tech Innovation Expo. Make sure to read the full article on www.med-technews.com
BE SURE TO LISTEN TO The latest episode of The MedTalk Podcast features Mike Rea, CEO and founder of IDEA Pharma and Protodigm, who discusses drug development and the challenges that are prevalent throughout the industry.
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