New treatments and new challenges: The oncology constant is change
Jack Harris at GSK discusses the UK’s effectiveness at introducing innovations to healthcare, specifically the world of oncology, and his vision for making impactful improvements in an ever-evolving field
As I took on my new oncology role with GSK in November 2021, and started to review and shape our business plans for the coming years, it struck me how much of a personal, as well as professional, interest I have in accelerating patient access to innovation. And from this I asked myself – where can I make the most positive difference day-to-day? Where can I work with others, to identify and solve some of the biggest challenges that currently result in UK patients often missing out on the widespread use of new treatments that we see elsewhere?
Not only do I work for a large UK-based global healthcare company, but I’m also a UK citizen with a UK-based family, with many of my closest friends and family here. We will all be impacted by medical advances in this country in our lifetimes. These advances rely on previous advances, and those on developments that came before, and so on. The faster we move today, the faster we will all see the benefits tomorrow. And from this, the widest possible health improvements will be felt by the widest possible number of people in the UK.
Of course, the UK is in a strong position to create fast access in the initial development and deployment phases of new treatments. The opportunities afforded by accelerated marketing authorisation initiatives, such as Project Orbis for oncology and the Innovative Licensing and Access Pathway (ILAP), are underpinned by strong commitments in the UK Government’s Life Sciences Vision. We have internationally renowned institutions, such as the MHRA, and in the NHS we have world-leading specialist centres and pioneering ambitious trials. The UK remains a key market for new approaches to the very concept of how we treat patients in the future, such as in immunology, or in potentially curative cell and gene therapies. In the early development phases of innovation, the UK is well-positioned to compete with investment against comparator economies. If the most positive difference for patients is in trialling and proving concepts here, then we can be confident that GSK and the wider industry are well-equipped to continue to play our part.
However, when bringing approved innovative medicines to NHS patients early, companies are increasingly working in a system driving towards ‘budget-neutrality’. There will always be the case for bespoke agreements in early access – be that due to unmet need or to improve clinical practice, where commercial return is minimal. The question is, will seeing this model become the norm create the biggest positive long-term difference for patients?
As a potential beneficiary of innovations in my personal life, I also look to the mechanisms that are set up to ‘bridge’ from concept to the patient, such as the Cancer Drugs Fund (CDF), the proposed Innovative Medicines Fund, and early access schemes that are in partnership with the NHS. The purpose of these is to try to make the most impactful, earliest, positive difference, in order to create a gateway from accelerated licensing routes, through managed or conditional access, and eventually into the NHS, and to provide hope for all patients; not just those in a trial or in the early access scheme itself. In this area, the biggest difference we can make to accelerated routine patient use is to do everything we can to ‘de-risk’ or share risk – risk that the treatment may not prove cost-effective, or risk that it may simply be unclear after a defined period of time (high-cost lifetimebenefit treatments, such as in cell therapy, could be cited here). Sharing risk could mean looking at new outcomes-based models, new funding vehicles, or expanding value assessment measures to encompass a wider range of data.
The CDF has been a success in terms of facilitating early access where evidence is not immediately clear, or data is incomplete. This relies on the Health Technology Appraisal (HTA) model accepting a level of uncertainty, but has perversely led to an increasing reliance on the CDF as a default access route for oncology innovation. Is the biggest positive difference we can make expanding the budget pot for CDF? I am sure that would be welcomed by many. Or could the biggest positive difference be creating the right environment to bring routine access through standard HTA processes, so that eventually we will not need a CDF?
Which leads me to think, are we actually missing the main goal? So much focus across the various strategies and system levers is on that initial early access, yet the real success measure here, and the biggest difference I feel we can make for patients, is in gearing every system lever towards the fastest and broadest eventual uptake. Whether a fast-track or more standard route to patient access is chosen, if all eligible patients are not benefitting from a technology as quickly as possible, then we have collectively failed.
I am acutely aware this is not a new insight. The 2019 Voluntary Scheme for Branded Medicines Pricing and Access makes improved patient uptake a key success measure. But three years on, we remain faced with the same challenge. We need to stress-test each and every system mechanism against how it is contributing towards rapid routine use and, if not delivering on that, then it needs to be reformed or replaced.
We must together support reforms to make the UK as good as, or better than, comparable global economies in uptake. We must learn from other markets, and not be too protective over our own approach. Indeed, the UK’s global leadership in so many areas of healthcare exists because we learn and grow from collaboration, and not because we feel our system and approach is always best. When we look at the better rates of innovation uptake in markets such as Germany or France, what are they doing differently that we can apply in the UK? In our access system, how can we improve the existing successful concept and early access models so that broad patient use is better prioritised? How can we move beyond default conditional access, whether that is fast-track appraisals, or reimbursement models that reward those that widen access and prove value in a real-world setting?
Faster use of innovation can deliver obvious population health benefits with all the knock-on positives in quality of life, and a reduced NHS burden – so there is a strong economic incentive. Faster use by patients also generates incredibly helpful data in the UK, which can subsequently be used to deliver more innovations, ahead of other markets. Real-world data will also help us see whether innovation is appropriately valued. In this, the industry has to accept that an initial assessment could be revised up or down. If trial data does not apply to the real-world setting, then that treatment is not making the difference that we believed it could.
But what else can I do? I will ensure that when relevant data is available, or when a treatment is reimbursed, our teams are out there on day one, ensuring that every possible clinician has the information they need to assess whether it is suitable for their patients. We need to ensure that our education and support materials are as clear and relevant as possible. We need to understand from colleagues in the NHS the system barriers that are preventing patients using approved treatments where they are suitable – and where possible, help to remove them by partnering on solutions.
Every day I see the amazing progress the UK makes in life sciences, and across the health system, for each and every patient. If we made the biggest difference tomorrow, then there would be another big difference to make the following day. It is not easy to meet the challenge when new obstacles will inevitably follow, but progress simply has to be made. There needs to be a willingness to disrupt our systems and processes, even if that means making hard decisions. There needs to be system investment, and a shared focus across all stakeholders that can help realise this positive difference for patients. To deliver this, we must centre all pathways towards a success measure of the highest possible patient uptake, and this is a challenge worth our time.
Jack Harrisis Vice President, Head of Oncology for GSK in the UK, responsible for GSK’s Oncology portfolio in the UK, with a particular passion for accelerating access and uptake of oncology medicines. Jack has worked in the pharmaceutical industry for almost 20 years in various marketing, sales and market access roles, both in the UK and across Europe, the Middle East & Africa.
ONCOLOGY
Embrace disruption: the changing landscape of clinical research
Georg Pirmin Meyer, Senior Vice President, International, at Blueprint Medicines, explores how precision medicine and personalised treatments can improve healthcare in Europe, and around the globe
Delivering on the promise of precision medicine in ‘one-size fits all’ health systems
Every day, new technologies are unlocking deeper insights into the molecular and cellular alterations underlying numerous diseases, and transforming our ability to diagnose and treat them. When these insights are paired with precision medicine, we can also pave the way for more precise, predictable, and powerful treatment approaches that underpin truly patientcentric care.
Researchers are now much more hopeful to treat diseases that have previously seemed untreatable, due to the lack of understanding around the underlying causes. Even some patients with certain common cancers, such as lung or breast, don’t respond to standard-of-care treatments in the same manner as other patients. Researchers and clinicians have made significant strides in the last decade to understand why changes at the genetic level may trigger an illness, and how better understanding these changes may lead to treatment options that hadn’t been there in the past. Recent progress from tools, such as genomics or big data, makes a direct contribution to care for patients living with cancer, or with rare diseases such as advanced systemic mastocytosis (advSM), which can become cancerous, or spinal muscular atrophy (SMA).
Within lung cancer, the introduction of precision therapies for patients with mutations such as Kirsten Rat Sarcoma virus (KRAS), Epidermal Growth Factor Receptor (EGFR), and Rearranged during Transfection (RET), have led to better clinical activity, supporting increased adoption of precision medicines. Historically, these patients may not have responded to standard treatments, and would have seen their disease progress with no further options. As more of these tools and technologies become available and enter into routine practice, researchers and physicians will be able to deliver the right health intervention, at the right time, and across more disease states.
Re-imagining treatment pathways
Realising the full value of these advancements requires a paradigm shift in the way medicine has been practiced for decades. Physicians have been taught that treatment approaches should be universally applied to every presenting patient. Until recently, a one-size-fits-all approach – aimed at the “average” patient, possibly with only minor, little understood variations – was the only option.
Precision medicine effectively turns this approach on its head. It recognises that complex diseases should no longer be considered as a single entity. One disease may have many different forms, or ‘subtypes’, resulting from the complex interaction of our biological make-up, and the diverse pathological and physiological processes in our bodies. This means that two patients who have the same clinical diagnosis, such as breast cancer, may really have two very different diseases, based on their genetic make-up and any mutations they have. This may impact not only the underlying disease, but also how the disease travels through their body. This also demonstrates why biomarker testing at diagnosis is essential to ensure each and every patient understands what is driving their disease, and has the potential to receive the best possible care right from the beginning of their treatment journey.
As we integrate and analyse genomics and other data, we can find common factors and causes of variation. This means we are constantly discovering new pathways and presentation of disease, and with those discoveries, changing how diseases are thought of and treated. It enables us to recognise that the same underlying change in our DNA or genome can lead to problems in very different parts of the body, which would not have been previously identified with a more traditional care approach.
An example of this is AdvSM, which, in 95% of cases, is driven by a D816V mutation to the KIT gene. The mutation leads to uncontrolled mast cell production across multiple organ systems, and is associated with poor overall survival. Symptoms are varied across the body and may include skin lesions, chronic anaphylaxis, hypotension, migraine, and bone and muscle pain. Individually, these symptoms would be treated by different physician specialties, and the connection may not be made to the underlying cause, which impacts time to diagnosis for the patient.
The emerging precision medicine ecosystem
While precision medicine represents incredible potential for physicians and their patients, we must also recognise the co-ordination needed to operate in a myriad stakeholder ecosystem. Joining the dots between patients, clinicians, laboratories, clinical information systems, and government or other industry research sponsors, is an incredibly complex and delicate balance of information systems, personal data, and best supportive care. As we continue to grow in our ability to execute on precision medicine, we will need further collaboration among the developers and regulators of precision medicine, professional societies who will train the next generation of researchers, providers, and the regional and national health technology bodies who provide recommendations on medicines, and the other health technologies that can be financed or reimbursed by the healthcare system.
Translating the science into value
As healthcare systems aim to reset and accommodate precision medicine into daily practice, they have also needed to rethink access and reimbursement processes. Innovation uptake rarely coincides at pace, and bringing precision medicines into clinical practice in Europe has been gradual, given barriers to adoption among Health Technology Assessment systems in many European countries.
What might not be immediately recognisable is the value precision medicines can bring to the overall health system. When the right treatment is identified and deployed at the earliest possible stage of disease, it will naturally decrease the use of ineffective or inappropriate treatments, reduce hospitalisations and other costs associated with chronic conditions, and more efficiently deploy the use of healthcare resources. However, there are also a number of environmental and organisational challenges that currently prevent the effective uptake of personalised medicines, and potentially hinder their development.
Collaboration by a range of stakeholders including leading payers, policymakers, and healthcare professionals, is needed to drive patient access and reimbursement for new therapies, as well as to maximise their positive impact on health systems in Europe.
Embracing the disruption
Advances in our understanding of the genome and disease pathogenesis, combined with collaboration of families and carers of patients living with cancer, are changing the landscape for clinical research and drug development. However, to fully realise the disruptive potential of precision medicine will require a multipronged scientific, clinical, and policy agenda.
The speed at which breakthroughs in precision therapies translate into advances in European healthcare, and improve patient outcomes for debilitating diseases, will ultimately depend on how stakeholders collaborate to tackle some uniquely European challenges. Only a continuously evolving and connected healthcare system will be able to accelerate the advancement of precision medicine technologies.
Georg P Meyer, MD, SVP & General Manager International at Blueprint Medicines, brings nearly 20 years of industry and clinical experience in a broad variety of diseases from inline and launch products (cardiovascular, bone, cystic fibrosis), to rare diseases in early pipeline and peri-launch settings (inflammation, oncology). From 2018 to 2019, he served as Blueprint Medicines’ Vice-President & General Manager, taking strategic and tactical ownership of the region, including access strategies to prepare Blueprint Medicines’ long-term success in DACH region, with accountability for six indication launches and two launch products.
Prior to joining Blueprint Medicines, Georg served as General Manager Germany, Vertex Pharmaceuticals 2018 to 2019, with a business focus on cystic fibrosis stakeholders, including payer HTA and price negotiations. Georg was also tasked to drive strategic innovation for Vertex International, with a focus on digital and multi-channel customer engagements, as well as driving value-based healthcare projects.
Prior to that, he held roles of increasing responsibility related to clinical development in bone, inflammation, cardiovascular, oncology, diabetes and urology within Amgen, Sanofi-Aventis and Sanofi- Synthélabo. Georg holds an MD from Albert- Ludwigs-Universität Freiburg.