Novartis: Seeking a Step Change in Global Health Outcomes

Recent research from the Novartis Foundation-initiated Leprosy Post-Exposure Prophylaxis (LPEP) program suggests that contact tracing with prophylactic treatment could near-eliminate leprosy in a single generation.

More than 200,000 people receive a diagnosis of leprosy every year. The LPEP program covered more than 150,000 people across seven countries and highlighted that post-exposure prophylaxis with a single dose of the antibiotic rifampicin is safe, can be integrated into different leprosy control programs, and is generally well accepted by patients, their contacts, and healthcare workers. The results, Novartis reported, “mean we can finally imagine a world without leprosy.”

Novartis has been committed to eliminating leprosy for over 30 years, with its multi-drug therapy for the disease, donated for free to over 7 million patients since 2000. Pharm Exec caught up with Novartis’ Rosanne Rotondo to discuss how LPEP and the company’s other global health flagship programs are addressing major, unresolved health challenges around the world.

PharmExec: We tend to think of leprosy as something from the bygone era. What is the reality of the burden of this disease in 2021?

Rosanne Rotondo: People often hear “leprosy” and think of something Biblical or maybe remember a story from the nineteenth century about Father Damien in Hawaii, but there are still over 200,000 new cases globally every year and an estimated 2-3 million people living with leprosy related disabilities.1 For a disease which is both curable and preventable, that is far too many.

Until the 1980s, millions of people were affected by leprosy. The introduction worldwide of freely available multi-drug therapy (MDT) in that decade marked the start of a massive shift in the fight against the disease. In the past four decades, cases have dropped by 95%. Many countries have eliminated the disease, but evidently, there is still a lot to do in the last mile of elimination.

Novartis has just announced that it is renewing its leprosy medicines donation program with WHO. Why has the company decided to do this?

Novartis donation of MDT has been a great success, treating well over seven million patients since it started in 2000. MDT has been the backbone of the drive to eliminate leprosy over the past four decades. The new agreement with the WHO will build on and sustain that success by extending the donation program up to the end of 2025.

What is Novartis’ perspective on supporting global efforts of eliminating leprosy beyond donating MDT?

As mentioned above, despite the progress in reducing leprosy, we started to see a stagnation in new leprosy cases and child cases, indicating that MDT donation alone may not be enough to fully eliminate the disease.

Therefore, the world needs to fight leprosy using a broader range of tools such as ensuring universal access to treatment, enhancing early screening and diagnosis of the disease and exploring the implementation of new prophylactic measures to contacts.For instance, we plan to work with partners on in-country ”last mile” solutions to increase prompt treatment in remote and under-served communities where leprosy often lingers. The Novartis Foundation is also working with Microsoft and the Oswaldo Cruz Foundation (Fiocruz) to assess the feasibility of developing an AI-enabled screening tool that can analyze a photo of a skin lesion and suggest whether it is due to leprosy. Such a tool would significantly support frontline worker’s screening capacity and promote earlier diagnosis.

Finally, we need to counter the extreme stigma still associated with this disease, which stops people who think they may have leprosy coming forward for treatment. We know we can do this through targeted awareness-raising and engagement with patient groups. It was very encouraging to see India’s Minister of Health, Dr. Harsh Vardhan, committing to get rid of more than 100 laws that discriminate against people living with leprosy in India.

Overall, we believe scalable, self-sustaining social business approaches, with their roots in communities and national health systems, have the greatest potential to address global public health needs. Then there are the other nuts and bolts which are critical to any elimination program: better diagnostic tools and strict epidemiological surveillance and response systems to track progress.

What is exciting about the recent research of Leprosy Post-Exposure Prophylaxis program in relation to the WHO announcement?

The global community now has another promising route to the elimination of leprosy — which has the potential to accelerate progress by decades. The Leprosy Post-Exposure Prophylaxis program (LPEP) works to reduce the risk of leprosy among those potentially exposed, who are identified through contact tracing. I’m incredibly proud that the Novartis Foundation was able to work with our partners and governments in affected countries to conceive and support the research which underpins it.

LPEP was based on a massive operational research program over five years and reported at the end of last year: it traced 170,000 people in seven countries who had been in contact with individuals newly diagnosed with leprosy.The program treated 150,000 of them with a single dose of rifampicin (SDR) to prevent the disease. This multi-country research was built on earlier observational data from single-country studies. We now have a simple, highly effective tool to lower the risk of people from becoming ill with leprosy. Novartis will work in collaboration with global partners and governments to explore the operational requirements to embed contract tracing and administration of SDR as a routine activity.

Novartis has been strongly supportive of leprosy elimination for over two decades. With declining progress towards elimination in recent years, our commitment has shifted from a single MDR-based strategy to a multi-pronged approach which explores introducing new tools and optimizing existing ones. We remain deeply committed to leprosy elimination, remaining agile in our focus and vigilant for patient impact.

How are Novartis Global Health’s three other flagship programs advancing in comparison?

At Novartis, we apply our expertise and full organizational capability to address major, unresolved global health challenges. In line with this commitment, we identified four disease areas where we have the knowledge, capacity and experience to be a uniquely useful partner. We call these our flagship programs, and they cover leprosy, malaria, sickle cell disease and Chagas disease.

Malaria has significant burden of illness, and we have worrying recent news that strains of malaria partially resistant to the current artemisinin-based gold standard treatment are reported in Rwanda and in parts of Asia. Emerging resistance means we need a new generation of anti-malarials. With financial support from the Bill and Melinda Gates Foundation and the Wellcome Trust, Novartis is working with the Medicines for Malaria Venture to develop two promising candidate treatments that are in late-stage clinical trials. As part of the PAMAfrica research consortium led by Medicines for Malaria Venture, Novartis also initiated the development of a new formulation of our ACT for infants weighing less than 5 kilograms, one of the most vulnerable groups affected by malaria. Meanwhile we continue to support in-country programs to combat malaria, many of which have been badly affected by the COVID pandemic.

Although it is on very few priority lists, in some areas of Africa the sickle cell disease (SCD) trait may affect one child in five.2 The great majority of those born with SCD in Africa die in early childhood. We are signing agreements with ministries of health for SCD programs to improve screening, diagnosis, treatment and care. So far, we have signed agreements with Ghana, Uganda, Kenya and Tanzania and plan to reach a total of 10 countries in the near future. We are also conducting multi-country clinical trials in Africa for crizanlizumab, our innovative medicine recently licensed in the U.S. for prevention of the vaso-occlusive crises seen in SCD. This is part of Novartis’ commitment to get innovative medicines to lower-income countries where there is a clear clinical need — certainly the case in SCD as over three-quarters of those with the condition are born in sub-Saharan Africa.3 In addition, as announced recently, Novartis is collaborating with the Bill & Melinda Gates Foundation to discover and develop an accessible in vivo gene therapy for SCD — this is at a very early stage, and is a very tough scientific challenge, but would transform treatment of the disease in low-income settings if it could be achieved.

Chagas is another disease that sometimes gets overlooked because it mainly affects people in remote areas of Latin America with poor access to healthcare. Chagas slowly debilitates patients over many years — often serious symptoms such as cardiovascular complications appear 20 years after someone has been infected through an insect bite. These cardiovascular complications are severe and life-threatening if left untreated — and often never get diagnosed. We are investigating the use of one of our heart failure drugs to treat patients with chronic Chagas cardiomyopathy, the most important clinical manifestation of Chagas disease, which results in the majority of morbidity and mortality. As with all our four flagship programs, it is not just about providing treatment, but a holistic program is needed in Chagas-affected communities. This includes working with committed partners to improve access to health and to develop better diagnostic tools. There is also an urgent need to educate healthcare workers worldwide on the symptoms and management of late-stage Chagas: it might be a cardiologist in Manila or Milan who encounters a patient who has been infected 25 years earlier somewhere in a rural place in Latin America.

In all these flagship programs, partnerships are vital to bring complementary expertise and integrated solutions. We work with multi-laterals like WHO, governments, civil society and the private sector to ensure we maximize our impact. With all four flagships, we hope we can achieve a step change in health outcomes.

Rosanne Rotondo is Head of Global Health Flagship Programs at Novartis.

Notes

1. https://www.cdc.gov/leprosy/world-leprosy-day/index.html

2. https://core.ac.uk/download/pdf/82547186.pdf

3. https://www.nature.com/articles/515S10a