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  • Jo Waters — United Kingdom

Amazing advances in medicines hold huge promise for a better world

The past century has been one of enormous progress in the understanding of human pathology and treatment of disease. We are now looking at a future in which technology and state-of-the-art treatments offer previously unimaginable potential for health care that is specifically targeted to individual patient requirements.

The pace of scientific research holds the promise of dramatically improving the lives of people living with non-communicable diseases (NCDs). Finding ways of extending the benefits of the most innovative treatments to all who need them will be the biggest challenge facing global health policymakers.

Re:solve Global Health asked five global health experts what the future of medicines for non-communicable disease (NCDs) looks like and what innovations we can expect to see.

The research voice

“What’s happening now is amazing—it’s almost science fiction in some ways”

Mike Fraser, general manager at Novartis Gene Therapies for Europe, Middle East and Africa

Q: How will gene therapies transform NCD medicines?

A: We all know that NCDs are the cause of 70% of all deaths and even with the current chronic [illness] treatment options there is still an urgent need to develop breakthrough technologies as we do need to treat them more effectively, not only to save resources spent on them by healthcare providers but to im- prove healthcare for society.

What’s interesting now is that we have discovered that some people with NCDs have a genetic make-up that predisposes them to these conditions; we’re seeing this with diabetes for instance. There is a lot of work going on in this space now in industry. I think gene therapies (or rather a collection of different gene therapies including gene editing, gene addition and some types of cell therapy) can target the underlying causes of diseases and this is going to change health care and society.

Q: What diseases are being investigated now for treatment with gene therapies?

A: Cancer and diabetes are two that come to the fore. If we can understand the genes that are predisposing people to these diseases it will accelerate the development of products. We have already come a long way in treating blood cancers such as leukaemia and lymphoma with CAR-T drugs [drugs genetically engineered to produce an artificial T-cell receptor for use in immunotherapy] and that knowledge will now be applied to solid organ cancer tumours.

I can envisage a time when people won’t have to take daily pills over a long period as we will be able to treat the root cause of the problem. I also believe it is very important to identify those who are predisposed to certain conditions as early as possible—as we are doing with newborn screening to look for babies with the genes for Spinal Muscular Atrophy (SMA), a muscle wasting condition. Once identified we can then correct the gene defect as we are doing currently in SMA babies with a one-off treatment that targets the underlying gene dysfunction by introducing a fully functioning gene to replace it. This is done by using a virus vector that has been ”de-diseased”, so it is not harmful to humans.

Q: Can you foresee a time when people with type-1 diabetes will no longer need insulin supplied?

A: Yes, with type 1 diabetes at least, I could imagine a point where we will be able to get the pancreas functioning again so people would not need insulin shots. But I’m talking hypothetically at the moment. What we have done with our product for SMA and another gene therapy for retinal dystrophy, is showing that you can address the underlying root cause and treat it. What we are seeing today with these treatments and what we hope will be their long-lasting effects is amazing—it’s almost science fiction in some ways. I spoke to a parent of a SMA child recently and she said to me: “Imagine a time when you won’t be able to tell which child in the playground has SMA because they’ve had gene therapy and they’re completely normal.” When you think about all diseases, including rare ones, but also NCDs such as cancer and diabetes, can you imagine the time when they are just gone? That’s what drives me and my team to work every single day.

The person with lived experience

“Genomic testing of cancer tumours will be a game changer”

Pancreatic cancer survivor Ali Stunt is CEO and founder of Pancreatic Cancer Action in the UK and a past president of Pancreatic Cancer Europe

Q: What can you see on the horizon in terms of new treatments for cancer patients?

A: Things haven’t advanced much in pancreatic cancer treatments in the past 50 years, but we’re beginning to see some chinks of light, mainly along the lines of more personalised medicine for all cancer patients—and a lot of this is being patient-driven. Biomarker tests, genomic testing and targeted treatments are nudging up survival rates. But there is huge geographical variation. Australia has a five-year survival rate of 15%, compared to only 6.9% in the UK. The UK had only a 24% one-year survival rate but the figure is 50% in Austria and Belgium.

The Know Your Tumour Registry study of 1000 cancer patients with biopsy-confirmed metastatic pancreatic cancer, carried out by the US Pancreatic Cancer Network, found that 26% had genomic alterations that were actionable with currently available drugs. This could be potentially game-changing if health systems routinely tested the genome of each tumour. The same study found that those given genetically targeted treatments rather than standard care had a year’s improvement in survival. When you consider at the moment that average life expectancy is four to six months with metastatic pancreatic cancer, an extra year of life is a phenomenal gain. Genetically targeted treatment is definitely the way we want to go in treating pancreatic cancer; immunotherapy doesn’t appear to be working as there is a stroma around it which prevents drugs getting to the tumour.

Q: Why is genomic testing of tumours so key?

A: To take an example of how genome testing can expand treatment options, around 4% to 7% of pancreatic cancer patients have the BRCA1 and BRCA2 gene mutations found in breast, ovarian and other cancers. We know from research done into other BRCA cancers that these patients respond better to platinum chemotherapy as a first line treatment. Last year, the POLO trial in the US showed that a PARP inhibitor drug, which helps to repair damage inside cells, doubled progression free survival in patients with metastatic cancer who had been first treated with platinum chemotherapy.

Understandably, there is a lot of buzz about genomic testing of cancer tumours—I’d like to see it offered routinely by health care systems as this will determine if patients could be treated with targeted treatments that could extend their survival time. Information is key. We have patients walking around today with cancer gene mutations which could benefit from therapy, but they don’t know about it and will probably die without knowing.

Q: Any new breakthroughs in identifying biomarker tests?

A: Yes, there have been some exciting results in April [2021] from a study that used a blood test together with a biomarker for pancreatic cancer. The study was designed to detect early-stage cancer in patients in high-risk groups with symptoms that are concerning but not specific. The test detected cancer with 92% specificity and 81% sensitivity.

This could mean that we have a non-invasive test that we could use to screen relatives of patients who have had pancreatic cancer. Non-invasive biomarker tests for cancer have been the Holy Grail, and it looks like we could have this potentially by the middle of 2021.

The lawmaker

“If we don’t find a new financing model for NCD medicines in the next decade we will be creating a two-tier system—one for the rich and one for the poor”

Ricardo Baptista Leite, physician, Portuguese member of parliament and head of public health at the Catholic University of Portugal in Lisbon

Q: What are the trends in NCD medicine development?

A: Medical technology is going after retail medicine, developing products for conditions that affect millions of people (such as diabetes) for the mass market. What we are seeing in the pharma industry is a trend to invest in fields of medicine where there are currently no solutions (conditions such as pancreatic cancer).

What I can foresee—especially when you add in all that is currently happening in the genomic and precision medicine fields—is that in the next decade, if we do not find a new financing model, we’ll basically be creating a two-tier system, one for the rich and one for the poor. Those that have the resources will get access [to the newest medicines] and those without, will just have access to whatever is currently available. This is exactly the opposite of what universal health coverage is aiming to achieve.

Q: What are the cost implications of some of the new NCD medicines such as genomic drugs for cancer and ”cure” drugs?

A: The issue from the pharmaceutical company’s point of view is the discussion around value and what the cost of that value should be based on—including not only what they have invested, but also the drugs that have failed during that process and other pipelines they are building. Many of the technologies are life changing and some of them are real cures.

The issue from policymaker perspectives and for those having to manage the finances, either private insurers or those controlling public spending, is that it’s not an issue about cost but affordability.

Nobody can dispute a technology that saves lives or cures disease. The issue is about how you get that value embedded within your system in a sustainable manner that doesn’t undermine the other efforts of a health delivery system—and that’s where I think we need to discuss how payers and pharmaceutical companies can work together to find these solutions.

Q: Do you think we need new payment models for financing more expensive types of NCD treatments?

A: Following the introduction of new drugs for hepatitis C, a new kind of payment model has come into being, the so-called “Netflixing” of medicines, with the idea that you pay a rent or fee for medicines and use as much as you need. This type of approach is aimed at curative drugs for diseases affecting a big population, where there is need and you can see the value and true benefit that technology brings to the health system. Wider use of this model could keep costs within budget and create a true incentive to treat as many people as possible. If you think about hepatitis C, your incentive is to cure all people as soon as possible. The more effective you are in diagnosing and treating those people, the more you free yourself from the expense moving forward.

I think we need to incorporate different ideas and find solutions that work. I also think that pharmaceutical companies need to rethink the way they brand and commercially present themselves to the payers— be they states, ministries or insurance companies. The “one-size-fits-all” approach many pharmaceutical companies use when addressing different countries doesn’t look at the specifics of that health system. They need to provide solutions to overcoming barriers that exist in that health system to make sure the technology is made accessible. At the end of the day, instead of selling a pill, companies will be selling a solution that adds value that can be measured at the end.

There is also this idea that countries, governments, and insurers can pay after the benefits have been proven in the real world with real-world data. These are all different models which I think could be considered to make sure everyone gets a fair deal.

Q: Should countries be working together on innovation and procurement of new technologies?

A: For rare diseases and some cancers, it may be that we need a joint procurement approach between countries. This would create a critical mass to allow us to negotiate in a way to make a win-win solution for those investing and for those buying.

On the other hand, it may be necessary for countries to allocate specific funds for innovation. At the moment, it’s becoming impossible for health systems to manage their budgets as innovation comes in and disrupts everything. To have central funds dedicated to innovation would enable access and ensure that innovation doesn’t disrupt the normal functioning of the health system. Those that own the patents, who are also investing in R&D, would also know the limits of the system, so it becomes more transparent; this would make negotiations more realistic so everybody knows what there is to gain and how far each party can go.

The industry view

“Medical innovation is meaningless unless it reaches those in need”

Vanessa Peberdy, Associate Director, NCD Policy & Advocacy & WHO Engagement, International Federation of Pharmaceutical Manufacturers and Associations

Q: What in your view are some of the most exciting breakthroughs in the pharmaceutical pipeline for NCDs?

A: Some of the most exciting developments in treating NCDs have been in relation to treating cancer and cardiovascular disease (CVD). Innovative regimens based on progenitor cells [powerful cells with the ability to form new blood vessels] are also emerging as promising approaches for the treatment of a variety of CVDs as this technique could play a key role in repairing damaged heart tissues.

Investments in R&D are providing tools and techniques to exponentially decrease the costs of genetic sequencing and allow clinicians to increasingly treat each cancer patient with a personalised combination of drugs.

The field of immunotherapy holds much promise, as evidence has shown that increasing the strength of the patient’s immune system to attack tumour cells can lead to a cancer-free diagnosis. Another immunotherapy approach called adoptive cell transfer (ACT) collects and uses a patient’s own immune cells to treat their cancer and one of the most advanced forms of ACT is CAR-T cell therapy. CAR-T cell therapy uses T-cells, which play a critical role in orchestrating the immune response and killing cells infected by pathogens.

Decades of cancer R&D have also benefited the development of the latest covid-19 vaccines. The most talked about are mRNA vaccines, which carry instructions to tell the body to make proteins that attack viruses. This breakthrough was built on the back of more than a decade of research into mRNA in the field of oncology.

Q: What are some of the major challenges in delivering these new NCD treatments to those who need them?

A: Biopharmaceutical innovation is behind some of the greatest achievements in modern medicine. Unfortunately, not all communities have fully benefited yet from these medical advances. Addressing the reasons for this is a complex challenge that requires long-term commitment from governments, society, and the private sector.

Our industry understands the responsibility it has to people living with NCDs and has a strong track re- cord of sustaining programmes to improve the health of patients in low-and-middle-income countries. These initiatives strengthen local healthcare capacity, educate patients and populations at risk, and conduct R&D in diseases of the developing world.

A resilient health system with well-trained and well-equipped health professionals is foundational to achieving UHC. Innovative medicines have a major role to play in this regard, as they can put healthcare systems on a more sustainable path by reducing costs in other parts of the system such as hospitalisations and healthcare practitioners’ time. For example, per capita expenditure on cardiovascular hospitalisations would have been 70% higher in 2004 had new cardiovascular medicines not been introduced between 1995 and 2004.

Having said this, medicines are still only one part of the overall access-to-healthcare equation. As we see in diabetes, strong primary healthcare systems, secure supply chains, health literacy, good public health and prevention policies, access to diagnostics and devices—all need to be in place to achieve good health outcomes.

The work of Access Accelerated, our industry-wide initiative to tackle NCDs, is exemplary in showing how even when treatments for chronic diseases are widely available and affordable, the barriers to access to early diagnosis and ongoing treatment can only be addressed holistically, involving governments, society, as well as biopharmaceutical companies

Q: What has the Covid 19 vaccine development programme taught us?

A: Covid-19 has genuinely taught us that we need to ensure all players that can contribute to positive change have a seat at the table. In this light, there are many positives to take away from the covid-19 experience in terms of collaboration and solidarity that will be useful for “building back better” as a society; we have also learned the hard way that the linkages be- tween NCDs and covid-19 cannot be ignored.

Seeing people living with NCDs at greater risk of complications from covid-19, greater risk of hospitalisation and higher risk of death just re-emphasises the need to ensure we invest more in NCD prevention and control as a means of building back a global population that will be more resilient to the next pandemic.

The academic

“We must not let opioid-phobia deter countries from ensuring access to opioids for palliative care”

Dr Afsan Bhadelia, research associate, Department of Global Health and Population at Harvard T. H. Chan School of Public Health, United States

Q: How important is palliative care in treating NCDs?

A: In much of the world, palliative care falls at the bottom of the priority-setting list, if it makes it on there at all. It is one of the most neglected areas in global health. Policies and resources to strengthen health systems and improve health outcomes tend to focus on prevention and treatment, as well as on pro- longing life and increasing productivity. This focus has also resulted in the overall neglect of quality of life and of reducing suffering. Palliative care is a core part of the care continuum and is important to ensuring dignity in life and death. Health systems need to be designed to ensure care throughout the care continuum, prioritising suffering alleviation, which is of immense value to patients and families who don’t want to see their relatives in pain or experiencing other forms of distress.

I was one of the lead authors of the 2017 Lancet Commission on Global Access to Palliative Care and Pain Relief report, which introduced the concept of serious health-related suffering (SHS). The report quantified the burden of SHS that can be addressed with adequate access to palliative care. We found that over 25.5 million people of the 56.2 million who died in 2015 and more than 35.5 million people who did not die in 2015 experienced SHS, a large proportion of which is associated with non-communicable diseases. More than 80% of people with SHS lived in low-and- middle-income countries with severely limited access to any palliative care, even oral morphine for pain re- lief, demonstrating a major health inequity.

Misconceptions around palliative [care] include that it is only relevant at the end of life—during the last months and even hours—when it is actually essential from the point of diagnosis through to death. Another is that palliative care is only required for cancer patients, while in fact it is essential to alleviating suffering associated with other conditions such as HIV, TB, dementia, cerebrovascular disease, and lung disease. Suffering can also include other physical symptoms such as shortness of breath and psychological symptoms, including depression. The burden of NCDs is projected to rise and this will also increase demand for palliative care.

Q: Is access to medicines for palliative care an issue globally?

A: We have the medicines we need to address SHS through palliative care and these are off-patent and cheap. While there is room for development of new formulations of opioids and of creating opioid alternatives, safe and effective options are currently available. The main issue is over access, particularly to both injectable and oral immediate-release morphine, in most of the world. The Lancet commission found that 95% of distributed opioid morphine-equivalent, an indicator used to examine the morphine available to prescribe to patients, is within the US, Canada, western and central Europe and Oceania, which together represent only 9% of the population globally.

The Commission designed an essential package of palliative care services, including medicines, basic equipment and human resources that could alleviate much of the avoidable suffering, at a cost of only $2.16 per capita per year. Most of the medicines in the package make up only two to three per cent of the total cost of the essential universal health coverage package.

Q: What are the barriers to palliative care becoming more widely available?

A: There are various barriers to addressing the huge inequities in access to palliative care. One is “opioid-phobia”, which stems mainly from misinformation on the medical use of opioids by policymakers and healthcare professionals alike. This is due to a focus on preventing non-medical use of opioids, particularly in response to the opioid epidemic in the US, instead of a balanced approach that simultaneously ensures access to the benefits of opioids while minimising risk of non-medical use. As such, regulatory barriers are a major hurdle. Issues of access for medical use exist even within the US, where impoverished and minority communities are less likely to have access to evidence-based palliative care and pain relief. However, we can take appropriate steps to ensure safe and evidence-based access. Greater awareness of policymakers on the issue is critical.

Further, a lot of countries in the world don’t have palliative care experts, but specialised palliative care is not necessary in most contexts. Training of health care professionals such as primary care physicians and nurses on core palliative competencies, through integration in the medical and nursing curriculum, could vastly improve access to palliative care.



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