Novartis AG (NOVN) Earnings Call Transcript & Summary

Hello. This is Jeff Sturchio. I'd like to welcome you today's Novartis Materiality Assessment series of webinars. Today, the topic is rethinking health care system strengthening to reduce the burden of sickle cell disease in underserved populations. The next slide, please. Here's our agenda. We'll be having a brief scene setting by reviewing how Novartis has come to certain priorities in their materiality assessment and why we're going to be discussing health system strengthening today. Then we'll hear from 2 speakers on how Novartis is tackling sickle cell and how they're helping to reach underserved populations. And then I'll be moderating this discussion with our 2 speakers about how we can rethink approaches to strengthening health care systems. And then we'll have time to hear from all of you. The next slide, please. Just a few housekeeping details before we start. We'll be spending an hour together. And if the questions are still coming and people still have things they'd like to ask about, we'll extend until 15 minutes after the hour. So that would be 10:15 on the East Coast here and 16:15 in Central European Time. [Operator Instructions] The next slide. I wanted to just spend a minute on feedback from the last webinar that some of you may have joined. That was at the end of April, just a month ago. There were more than 200 participants. And overall, the feedback was very positive. So we wanted to thank those of you who were on that webinar and who gave feedback, and I hope that everyone on today's webinar will share their thoughts with us in a brief survey that will be sent out afterward. But more importantly, we wanted to tell you that we also paid careful attention to some of the critical comments that were received. And one of the things that we proposed to do to adjust these webinars is to, for one thing, extend the time available for Q&A, as I mentioned a moment ago. We'll try to have more open-ended questions, and that will encourage our speakers to go into more depth. And also, there were a number of questions about why didn't we talk about COVID-19 since that's on everybody's mind. And in response to that, we've planned a separate webinar next month that will focus on the response to COVID-19. And also, Novartis plans to provide responses off-line to questions that we don't have time to get to during the webinar itself. So I just wanted to share with you how Novartis is responding to that background and critique from those of you who responded. So thank you for that. The next slide. Let me introduce our speakers. As I said, I'm Jeff Sturchio. I'm the Chief Executive of Rabin Martin, a global health strategy firm. And we have 2 very distinguished speakers joining us today who have really deep experience in this area. The first is Dr. Lutz Hegemann, who's the Chief Operating Officer of Global Health at Novartis. He -- in this role, he has responsibility for Novartis' business in sub-Saharan Africa and 4 flagship programs in malaria, leprosy, sickle cell disease and Chagas disease. He also has, in his organization, the Novartis Foundation and Novartis Social Business. He joined Novartis in 2005 and has had a series of leadership positions, including, most recently, the development unit head for established medicines and anti-infectives in the pharma division. He trained at the University of Van and the University of Utrecht. He has both an MD and a PhD in molecular pharmacology. And as a dermatologist, he's done considerable clinical research, with a special interest in inflammatory and tropical skin diseases. And he conducted that research in Cone, at Thomas Jefferson University in Philadelphia and also at the University of Vienna. And he's a fellow of the Royal Society of Tropical Medicine and Hygiene and an Executive Board member of the Swiss Alliance Against Tropical -- Neglected Tropical Diseases. Also, we have with us today from Ghana, Dr. Kwaku Ohene-Frempong. Dr. Frempong -- Ohene-Frempong, who's known widely as Kof, so I'll refer to him as Kof for the rest of the webinar, was trained in medicine as a pediatrician and a specialist in pediatric hematology/oncology at Yale Weill Cornell Medical Center and the University of Pennsylvania. He established the Southern Louisiana Sickle Cell Center at Tulane University in the early '80s and returned to Philadelphia, where at the Children's Hospital of Philadelphia, he led the comprehensive sickle cell center from 1990 to 2010. He's, in fact, still a Professor Emeritus of Pediatrics at the University of Pennsylvania. While he was in Philadelphia, beginning in 1993, he and collaborators in Ghana developed the first public health program for sickle cell screening of newborns in Africa. And he also established the Sickle Cell Foundation of Ghana in 2004 and currently serves as President. Ghana founded its national newborn screening program for sickle cell disease in 2010, and Dr. Ohene-Frempong is the program coordinator. So we'll hear more from both of them in a moment. But first, let me set the stage by reminding us of priorities emerging from Novartis' 2017 Materiality Assessment. So if I could just see the next slide. And this is a summary of those results at a glance. And this shows that the relative importance of 30 topics were ranked from surveys of both internal and external stakeholders, and that led to 4 material issue clusters that were high priorities: access to health care, patient health and safety, ethical business practices and innovation. The priority topics were identified for these webinars to help understand the managerial relevance of those clusters. And within the access cluster, in addition to pricing and availability of medicines, health system strengthening was a key topic. And so the 2020 webinars are going to focus on the outcomes and activities and changes in these priority topics. And we'll be focusing, as I said before, on health care system strengthening. So let's turn now to our first speaker, and I'll hand over now to Dr. Hegemann. Lutz, all yours.

Yes. Thank you very much, Jeff, and hello to everyone on this webinar. Thank you for your interest and for joining us this morning, this afternoon. Let me start by reminding ourselves of the vision that Novartis has [indiscernible]. That is to discover new ways to improve and extend people's lives. We discover and develop breakthrough treatment, find new ways to deliver them to as many people as possible. And the second half of the sentence that is displayed here in bold is something that we feel is truly meaningful and therefore differentiates us, also to some extent, from other members in the industry and has given rise, in the next slide, please, to defining the excess principles as a framework to put this into action. The Access Principles were launched in 2017 has been a topic of the previous webinar and really guides our approach along the value chain, starting from R&D, where we assess our portfolio against unmet needs; considers affordability; but also which will be focus of today's discussion, looks at system strengthening in a way how we can lower local barriers to health care delivery. And with that, we are bringing corporate responsibility at global health into our core business rather than as many, including ourselves, have done in the past, meeting those 2 areas independent from each other. Now with respect to sickle cell disease, next slide, please. Let me just spend a few moments to introduce the disease to those who are maybe not familiar with the condition. The name sickle cell disease indicates that the red blood cells, the erythrocytes, takes the shape of a sickle and lose their nice rounded shape, as you see here on the right side of the slide. And it is a monogenetic disease, so it's being passed on with -- from generation to generation and was initially developed by evolution to protect people against severe malaria. But when you have 2 copies of this mutant gene, then you have a significant disease in its own right. There is significant unmet needs for treatment of sickle cell disease in Africa. And we see that if not treated properly, we see about 50% to 90% of infants born in Africa with sickle cell disease will die before their fifth birthday. The disease not only has a significant impact on patient's life and well-being, but it's also very extensive in that we expect 40 million newborns that will be affected by sickle cell disease over 40 years. And the vast majority of these patients are here. We -- it's being assumed that the link to malaria comes back into [indiscernible] in Sub-Saharan Africa. Other geographies that are being affected are Brazil, and certainly, also India. And sickle cell disease truly has a tremendous impact on patient's life, well-being, but also on families and the society at large. And I'm sure that Kof will allude to that in his presentation. But suffice it to say that it is the #1 cause of stroke in children in Sub-Saharan Africa. You see here a picture of [ Alev ], 11-year old boy, who is bound to a wheelchair after having suffered from stroke with his caring father, [ Ron ]. And we were able to meet this family, which still has a lot of will to live and a lot of positive spirit despite the disease and can be truly inspiring to interact with. But what do we need to be in order to prevent dire situations like this here affecting [ Karli ], please go back to the former slide. We first need to understand the epidemiology of sickle cell disease. And I would say, not only in Sub-Saharan Africa, but even if you go into well advanced systems like the United States, the epidemiology is not fully understood yet. We need to identify those patients very, very early on so that we can screen these patients that we can start with disease-modifying treatment as early as possible before first sequela, like stroke, heart attack or kidney failure may affect those young patients. And we need access to interventional treatment guidelines. This includes fairly basic interventions like pneumococcal vaccinations, penicillin, folic acid. And then fourth, a very old medicine called hydroxyurea, which is being used in cancer treatment, has been proven over and over again to have benefits in sickle cell disease in a disease-modifying fashion that is sort of changes back the shape of the erythrocytes into something that looks more like in a normal person. And then last but not least, we need to bring advanced care to these patients that could be monoclonal antibodies, could be stem cell transplantation; or ultimately, this is our final vision for this disease: curative gene therapy, where you would exchange this monogenetic part that is diseased which could essentially restore the genome of this disease [indiscernible] fully cures the disease. And there is efforts ongoing, including in our own laboratories to develop that. The next slide, please. If we now apply the Access Principles that I just outlined to this disease, what have we learned here? And what is the road map that we are applying? If we start with research and development, I have mentioned the importance of hydroxyurea to stabilize these patients. And there is nowhere in the world in pediatric formulation that would allow each appropriate dosing to those patients in a ready-made pharmaceutical format. So we've embarked on a journey to develop such a formulation and expect to be able to submit this for regulatory approval within the next few months. We've also been working on a disease-modifying monoclonal antibody, crizanlizumab, that has received FDA clearance in the U.S. and under regulatory review around the world. And that in itself can help with crises, which are very, very painful episodes in patients and eventually lead to these clotting factors and to end-organ damages. And we need to research and conduct clinical trials, particularly to advance care and ultimately develop the therapy. Then on the affordability part, as I have mentioned earlier, basic care is truly important here. And as you know, Novartis and our generic Sandoz portfolio has a large chest of interventional medicines that can be used where we have to find affordable mechanisms to bring those to these patients. We have, for instance, delivered 60,000 packs of hydroxyurea already to Ghana to start with the program here. And I'm sure that Kof will talk more about how this is being used in; clinical practice. But this needs to be sustainable. That's why we have been collaborating very closely with government to secure affordability, ideally seeking the product to be listed on the national reimbursement. And then on the systems strengthening part, we are working with partners. This is not an area of our own focus but with partners to enhance newborn screening. We have just forged a collaboration with a diagnostic company at the U.S. West Coast called Amex, which has a point-of-care diagnostic that is currently in field testing in Ghana. We need to work with the Sickle Cell Disease Foundation or treatment guidelines and to register hydroxyurea for sickle cell disease, where Ghana was the first country to register this medicine for sickle cell disease. Training, education, very important. But then also leverage technology in data and digital or drone delivery of medicines in order to overcome infrastructural areas that we see in the countries. Next slide, please. But what is also important on the country side is that there is a political will to introduce a change in the management of these diseases. And you see here on the billboard, his Excellency is the President of the Republic of Ghana, who started a campaign as we launched the sickle cell public-private partnership to show his support and to draw attention to this disease. It also takes commitment. And you see here on this photo, our CEO, Vas Narasimhan, who joined the local team for the launch. You see a few CEOs of partner companies that we work with who all came to Ghana for the launch, our Global Head of Corporate Responsibility and Global Health, Patrice Matchaba, whom you have met in previous webinars was there and many others. So it also shows that level of commitment, which is very important. And last, but not least, shows cross-functionality and a critical role that a cross-functional, cross-sector approach we have place. At the next slide, it also takes, very importantly, dedicated physicians who care for patients, lead in science, are happy to collaborate and are tirelessly championing the goals. With that, I would like to hand it over to Professor Ohene-Frempong, who, as you know, is the President of the Sickle Cell Foundation of Ghana, who is not only a remarkable individual but also a trusted collaborator and the mentor to many of us. Over to you, Kof.

Kof, you might want to see if you're not on mute.

Hello, can you hear me?

Yes. Perfect.

Thank you very much, Lutz. And I hope I can join and continue here from Ghana. We have unstable Internet, but we'll do the best we can. Can I have my first slide, please? So Lutz has described sickle cell disease in a general way. And this is a disease that has been very well studied over the last 100 years or so when it was discovered in the early part of last century. But 2 events happened in the United States in the 1960s that truly changed the trajectory of the disease. First, it was discovered that many children with sickle cell disease died at a very young age. And that, in fact, most of them were dying from bacterial infection at the time when the symptoms of the disease that had been described were not very well known. There was a lot of debate within the medical community until the United States National Institutes of Health organized a large-scale study to truly understand the natural history of sickle cell disease in the modern era. And a very important study that was done was to see whether ordinary penicillin given to young children with sickle cell disease could protect them from the infection that killed most of them. And that infection was pneumococcus. And still, pneumococcus is a bacterial infection that is largely susceptible to penicillin. So this small study, penicillin prophylaxis study, was done in the 1980s and show clearly that if these young children would take penicillin twice daily, it would protect them from frequent infections from pneumococcus and, more importantly, save their lives. Once that information became available, it became important then to think about how do we discover these children. And so in 1987, after the paper had been published in 1986, 1987, the NIH in the U.S. organized a consensus development conference to answer the question as to whether newborn screening for sickle cell disease be instituted so that these children with sickle cell disease can be diagnosed early and protected with penicillin prophylaxis. The consensus was that this should be instituted. And so the plan was adopted throughout the public health systems in the U.S. that all children born in the U.S. be screened for sickle cell disease so that those with the disease can be started on this life-saving and very inexpensive intervention. So that launched sickle cell disease in the mainstream public health arena in the United States. And it has stayed there since then, with more improvements in the way that we take care of people with the disease. Next slide, please. So now we understand is really had a disease distributed around the world. And it's become very clear that whilst sickle cell disease is common in the Americas, both North America, South America, throughout the Mediterranean and Middle East countries and in Western Europe, that Sub-Saharan Africa bore the highest burden of the disease. And studies from many different hospitals were put together in this publication that show clearly that we are losing 50% to 90% of young children with sickle cell disease within the first 5 years of life. Most of these children are undiagnosed and never treated for sickle cell disease. So all of a sudden, sickle cell disease became identified as a major cause of under-5 mortality. We challenged all African governments especially to try to address this problem. Next slide. So experience gained in Jamaica and then in the United States and more recently in Europe clearly had shown that instituting newborn screening to diagnose sickle cell disease early and instituting this inexpensive, simple interventional penicillin prophylaxis could actually change the nature of this disease and especially save the lives of these children. These graphs here show the experiences in Jamaica. The bottom curve was Jamaica in 1973 to 1975, when by screening young babies and establishing that pneumococcal infection, in fact, was a major cause of death with more than 25% of the children dying in the first 15 years of life, instituting penicillin prophylaxis, it improved the survival to about 15 -- about 75%, 85% of them surviving these first 10 years of life. So clearly, even in a developing country like Jamaica, one could have a very, very significant impact on survival of these children. In the United States now and in London, in France, more than 95% of young children born with sickle cell disease survive the first 2 decades of life. So this became a challenge for those of us in Sub-Saharan Africa also to try to adopt these interventions. Next slide. So in Ghana, with the help of the National Institutes of Health in the United States, we also instituted a research study in the beginning just to show that we could also adopt newborn screening for sickle cell disease. So we started in 1993 to set up a program, as I said, with funding from the U.S. in collaboration with the Ministry of Health of Ghana and eventually the Ghana Health Service when it became developed. And so we started this small project, which eventually became part of the Ministry of Health program. And in fact, it is now national policy that babies born with sickle cell disease should be offered newborn screening. But unfortunately, the government hasn't had the resources to carry out this program throughout the country. Next slide. So there are some major interventions that have truly changed the trajectory of sickle cell disease around the world. And unfortunately, we're still having difficulties adopting some of these in Sub-Saharan Africa. Newborn screening that I just mentioned, several countries have piloted newborn screening programs, but no country in Africa at the moment screens all the babies that are born in the country, which is what is recommended. Penicillin prophylaxis, penicillin is inexpensive. And so for most programs that are developed in Africa, the patients are able to either be provided penicillin by government as it is in Ghana or the families can afford it. Anti-pneumococcal vaccine has also become standard, but it's not in all countries at the moment. In Ghana, for instance, pneumococcal vaccine is now part of the extended program of immunization. So both healthy children and those with sickle cell disease now have access to another life-saving intervention, especially for those with sickle cell disease. Lutz mentioned that stroke is one of the major problems that we have to deal with in children with sickle cell disease. The best way to handle stroke is to provide blood transfusions about once every month so that these children will maintain about 70% of their blood cells as normal red cells that are donated. But unfortunately, chronic transfusion programs, while they have proven themselves very well in the more developed systems in the U.S. and Europe, very few programs in Africa are able to have chronic transfusion to protect children who have had a stroke and especially to treat those that we have detected through ultrasonography studies that they had high risk for stroke. So this is one area where we are still quite behind the rest of the world. Lutz mentioned also hydroxyurea. This old drug that has truly transformed the lives of many people who have survived the early mortality of sickle cell disease but still face the disease. Hydroxyurea has reduced the frequent severe pain episodes that people with sickle cell disease have. The complications -- special complication of their lungs that we refer to as an acute chest syndrome has been reduced by hydroxyurea. And in older patients, hydroxyurea is actually prolonging their lives. So this is a very important medication for us. And I'm happy to say that Ghana is one of the first countries in Africa to be able to adopt hydroxyurea therapy on a large scale, thanks to our partnership with Novartis. Sickle cell disease used to be known as an incurable disease. But in the last 20, 25 years, it's possible to cure the disease through hematopoietic stem cell transplantation or bone marrow transplantation. And as you can imagine, this is a very sophisticated and expensive intervention. So stem cell transplantations have been developed in the advanced countries, and a few programs are now beginning to get developed in Sub-Saharan Africa also. So at the moment, we cannot say that this is something we have adopted as widely as it's been adopted elsewhere. Next slide. So here, we are quite fortunate in Ghana to have developed a partnership with Novartis, which is really changing the clinical course of sickle cell disease for many patients, we hope. We had worked on guidelines for managing sickle cell disease to adopt it through the government health system and to teach all the doctors and nurses how to modernize the way they care for sickle cell disease. But in Africa, as you can imagine, we have many different levels of capability in our health institutions. So the Ghana guidelines that we wrote were unique in the sense that they were written at 6 different levels of health institutions. And we want to be sure that these guidelines make sense. So part of our partnership initiative is to do a field study, field testing of these guidelines at the different levels of health care. We hope that within the next 12 months, we will complete the field testing and adopt these guidelines widely through the country. Also, we don't have enough places for people with sickle cell disease to go for treatment. I will refer to them as centers of excellence for treatment of sickle cell disease. And part of the partnership is to expand these treatment centers to all the regions in Ghana, and we hope to accomplish that also within the next 8 to 12 months. Hydroxyurea that we mentioned, very fortunate that Novartis decided, in response to requests from families in Ghana, to actually produce hydroxyurea specifically indicated for sickle cell disease in Ghana, has been licensed in Ghana. And we have embarked on a program, we call it [ I Hold You ] program, which is a program that gives comfort to people who are otherwise in severe pain. This program is being expanded. And again, we are lucky to have initially free hydroxyurea through this partnership, so we can get thousands of people take this medication. We are developing education and training programs for our health workers and also for our patients and parents as part of this partnership. And we are also hoping that through this partnership, we will also bring in some other partners to help us to expand their newborn screening program. I think that was my final slide. Thank you very much.

Well, thank you, Kof and also Lutz, for those really clear and informative presentations. It's so interesting to learn what progress has been made in fighting sickle cell disease in Ghana. And it really shows us the power of bringing together all of these different interventions as you can evolve and approach to this disease that really has had a major impact on the lives of patients.

So let me start off our -- the next stage of our program with a fireside chat with both Lutz and Kof. And let me start by asking, Lutz, one theme that's run through both of your presentations is the importance of the challenge of priority setting, whether it's in R&D or service delivery, every company, every health care system faces the same fundamental question, there are always more problems to solve than you can manage within available resources. So I wonder if you'd give us some insight into how you and your colleagues at Novartis think about these choices in the context of your Access Principles. And in particular, can you tell us about your decision-making with respect to tackling sickle cell disease?

Yes. Thank you, Jeff. I can only agree that the challenges in global health, by far, exceed the abilities of any given organization. And one approach that is very important for us in global health is to seek partnerships where we bring complementary skills to bear and ultimately make a bigger impact. And this starts already in R&D. We have very active drug discovery partnerships developed, which is across all of our flagship programs that you had mentioned before, and that takes us all the way to programmatic delivery. But then within Novartis, also we need to prioritize, of course. And here, we are looking for the sweet spot of a high unmet medical need in which we can make a unique contribution. So the medical need alone is not enough for us to act, but it also needs to speak to our abilities that could be based on the focus areas as we have in drug discovery and development being a science-based company, would be the existing portfolio of our medicines; or it could be the setup with more than 100,000 employees in -- across the geographies, specifically now in Sub-Saharan Africa, where our business model has changed to exclusively focus on patient reach rather than the classical financial KPIs. And then we bring other players, for instance, diagnostic companies, which is not a core competency of Novartis, on board and hope to create a momentum. I think we have shown it now with sickle cell disease in Ghana that ultimately makes an impact on [indiscernible].

Well, thanks, Lutz. Kof, let me ask this -- a similar question about priority setting in your case. As you've told us, sickle cell disease is clearly a major public health problem in Ghana. I believe there are about 1 in 50 babies are born every year with the condition. But this has been the case for a long time. And so I wanted to ask you, why has Ghana decided to address this challenge now? And what were the factors that brought together the right partners to move sickle cell disease up the agenda to become a priority for health care in Ghana?

Yes. Jeff, you're very right. Sickle cell disease has been known for a long time as a major problem in Ghana. This is a disease that, while we lose many young children to it without a proper diagnosis, for those who survive childhood, because of the frequency of the disease, it's been known as a disease in Africa for thousands of years. Almost every African language has an expression for sickle cell disease. But it was always known as a disease about which you couldn't do much of anything. Patients suffer and suffer, and most of them die at young ages. But I think the efforts that started in the United States that show that if you could diagnose this disease early, you could actually do something to reduce the mortality and the suffering from the disease, was an impetus for us. Ghana actually had been fortunate that the first -- one of the first sickle cell clinics to be established in Africa was established in Ghana in the early '70s. And so through that program, I think people at least believe that now we could do something about this disease, even though we didn't have the resources to embark on large-scale programs, that initial clinic at the major hospital, the flagship hospital in the country, in Accra, truly established that something can be done about this disease. The recent efforts have all grown out of that initial work. When newborn screening became a public health activity in the U.S., I think we felt that we needed to see if we could extend it to Africa for that's where most of the babies are born. So we approached initially a research project to demonstrate that we could do it. We couldn't do it just as scientists. We had to do it within the public health service. So our initial work actually involved the Ministry of Health with the idea that what we would learn from screening babies in Ghana would, in fact, eventually become adopted as a national public health service program. That was the initial hope. And to begin a newborn screening, we decided to also start the second clinic in Ghana and use that clinic as the model for it. So already, we were thinking about expanding the services for people with sickle cell disease. And now I can tell you, we have now 18 clinics in Ghana that all of them have sprung up based on the work that we've been doing, particularly with the newborn screening. And I think governments has seen that this is an important problem with every family in Ghana and will be the same throughout most of Sub-Saharan Africa. Know somebody or have a relative who has sickle cell disease. But our attention in public health had always been towards the infectious diseases, malaria, TB, and those things that have killed so many children especially. But now that most children are surviving malaria, mortality has been going down. Other nutritional and infectious disease are going down in their incidents and mortality rate. I think countries are beginning to pay attention to noncommunicable diseases. And that include genetic diseases and one as common as sickle cell disease. So both the changing spectrum of major diseases and the fact that something can be done about sickle cell disease has made this the right time for us to embark on what we're doing. And as sickle cell disease gain attention around the world, I think the major companies such as Novartis also began to see how they could also become partners in developing drugs and development programs that can support people with sickle cell disease around the world. And I think in Ghana, we are a bit fortunate that at least some of us leading the effort had worked with Novartis in the United States. And so when we started our programs in Ghana, we extended a request for continued partnership in Africa to deal with sickle cell disease. And we are fortunate that Novartis answered the call. And the call didn't just come from us as professional, but it came also from the patients and their families directly. So the partnership that involves government, involves the health workers themselves, involves the community and involves scientists outside Africa, I think it's a model that we hope can sustain us. We are quite fortunate that now the new medications that have been developed for sickle cell disease, a major one, again, by Novartis and some others, but rather than waiting for decades before those medications become available to us, because we are organizing treatment programs, we can also very quickly take advantage of these new developments. We still need to get government attention. I think that for most of us, we know that government can do a little more than they're doing. So we have to educate our governments' leaders to pay attention to sickle cell disease as a major cause of morbidity and mortality in our countries. But I think that the future looks bright through these partnerships.

Thank you, Kof, and that was a very comprehensive response to that question. I wanted to just build on that with a question from one of our listeners, [ Koaldip Sami ], who observed that West Africa and the African-American communities in the U.S. have been sharing good practices and resources and that's been an important development in helping to strengthen health systems in West Africa. I mean you, yourself, are an example of someone who is in the diaspora and has come back to Ghana now to apply what you learned in the decades you spent in the U.S. Would you comment on that phenomenon more generally and how that applies to the kind of work that you and your colleagues are doing in Ghana?

I think that, that is true. I think if I look around Africa right now, those who are leading sickle cell disease efforts, almost everyone either trained or part of their professional training outside Africa and with a strong commitment came back to Africa to work in sickle cell disease or that they have partners through health institutions outside with whom they are collaborating in research and program development. Clearly, the knowledge there has been developed in sickle cell disease. It's not been restricted to any part of the world. We have colleagues in Brazil. We have colleagues in Jamaica. We have colleagues throughout Europe. And I think that those types of partnerships are strengthening the programs at home and also bringing attention to our governments to maybe do more for people with sickle cell disease than they had been doing. So the partnerships are very important. But many of us have come back home because we know that the task at home is much larger than the task outside Africa. And I think we all are a bit dismayed by the fact that so much can be done for sickle cell disease elsewhere but not as much at home. So I think the commitment is well by the need.

Yes. Well, thank you again for that answer. Just staying with this, and Lutz, I'll come back to you in a moment with another question. A couple of questions were raised about -- by listeners about hydroxyurea and crizanlizumab. But Kof, just to finish off on this theme of health system strengthening. Another one of our listeners observed that the WHO model for health system strengthening has 6 pillars of: service delivery, the health workforce, health information systems, access to medicines and diagnostics, adequate financing and leadership and governance. You've already referred to most of those pillars, but I wonder if you could talk a little bit more about the health workforce and health information systems. Those are 2 that you haven't spent as much time on. And how are you and your colleagues tackling those elements to make sure that those living with sickle cell disease get the care and treatment they need?

Thank you so much, Jeff. Sickle cell disease is so common in the country and in other countries in Africa that you couldn't just have a few specialists around to manage the disease. We knew from the start that -- especially through newborn screening, that we would have to train health workers in Ghana to become more aware of the modern management of this disease and also to develop treatment modalities that may be more suitable for our part of the world. And so at least from our research projects, training of the health workers is very important. One of the extensive training programs that we did in Ghana actually involved every parliamentary district. At the time, there were 170 of them. And we asked the Ghana Health Service to select 3 health workers from every district. And we did workshops in each of the regions for these workshops -- I mean for these health workers. Altogether, we had about 550 health workers that we trained. It is from that training exercise that we identified the gaps in knowledge. And that is what informed our development of guidelines in order to fill the gaps. And now we have -- with some help from Novartis, we have finished developing the guidelines. And here, we are now on the brink of testing them in the field to see that they make sense. And we'll finalize them so that they will become part of the standard of care for sickle cell disease throughout the country with the backing of the Ministry of Health and the Ghana Health Service. So we think that, that will build up the health worker force in Ghana so that, hopefully, they can deliver improved and modernized care for sickle cell disease in the country. As far as the leadership of the country goes, I have met with every President of Ghana since we started our projects. I can't say that it's always ended up with more funding, but we have been able to carry our story to the highest levels of government. So hoping that government will make us an important part of the health budget so that we can continue to build up on our programs.

Well, thank you, Kof. And your response is just -- remind me that what you've been doing for the past several decades in trying to establish a new standard of care for sickle cell disease and to erase the disparity in outcomes between children in Ghana and children in the U.S., for instance. To change the knowledge you have into action, as you've been discussing, another element that we might say is required is persistence and you've certainly had persistence as well. So Lutz, let me turn to you. One of the questions that came up about hydroxyurea from [ Olan ] is that there's been a reluctance to use hydroxyurea in some cases because of the challenge of managing toxicity and the lack that some physicians have of how to use it appropriately. What's your view, and Kof may have a point of view on this as well, about overcoming those barriers in resource-limited settings?

Yes. Thanks for the question. And of course, we have to acknowledge that hydroxyurea does have its toxicities that need to be managed carefully. I think on the efficacy side, we are beyond any doubt that hydroxyurea does have a positive effect in patients with sickle cell disease. It has been shown very much over and over again, there's enough evidence out there. But when it comes to managing those toxicities in resource-constrained settings, then that is where, of course, training and education becomes extremely important. But also the question, how can we leverage technology in order to guide clinical decision making? And for instance, we have developed an application that works on mobile phones that helps physicians and health care workers with those adjustments calculating the dose. We are capturing now the patient records electronically, so that we can get a longitudinal view on how patients respond and can dose adjust accordingly that we can report potential side effects in a very easy way. So I think you see the power that technology brings to reduce barriers that otherwise would potentially work against the safe and effective use of a medicine.

Thank you for those insights. I want to just stay with hydroxyurea and come back to the points you made about the 3 pillars of Novartis' Access Principles, which are: R&D, you just were talking about that; affordability; and health system strengthening. Can we just talk about affordability? You mentioned that you've been collaborating with the government in Ghana to secure affordability of hydroxyurea. Could you tell us a little bit more about how you're approaching pricing in Ghana for that drug? And then I had some questions about crizanlizumab as well.

Yes. Before I get specific to the affordability of hydroxyurea, if we just put that into the broader perspective again of the Access Principles. Plus, I think in the public debate, affordability is very often singled out. But we have learned, for instance, from our leprosy program, that even if you donate medicine, we've been doing this for leprosy for the last 20 years, that you don't necessarily make through the donations the impact that you would like to make towards disease elimination or disease management. And that it does take those elements of system strengthening and of R&D in order to truly make an impact on patients. But coming back to the affordability of hydroxyurea. When we learned together with Kof and through the analyses that we had done to better understand the disease how critically important hydroxyurea is and how difficult it is for patients in Ghana and other Sub-Saharan African countries to access it, we decided to repurpose a factory to make hydroxyurea, which was not one of our core medicines, albeit we have done it before. Of course, we felt that we could make contribution here. In fact, we were able to reduce the current price for hydroxyurea by a factor of 5. So it's about 20% of what patients used to pay previously, which now makes this much more affordable. And the approach that we are taking across all of Africa is that we are really focusing on patient reach and impact. And if that means that at some point in time we have to offer medicines at cost, we'll certainly do that because those build the foundation for the very important innovations then in the health care system.

Well, thanks, Lutz. It's good to remind us that access to medicines is more than just what is the price and is it affordable. Because, in fact, what Kof has been telling us about the program to address sickle cell disease in Ghana points to all of those other factors about availability, about uptake and adoption, about training of physicians and the other elements that are required in the health system in order to take advantage of a new medicine to address a public health condition like sickle cell disease. And that brings us to your new monoclonal antibody, crizanlizumab. We had a question from [ Emmanuel Chastenet ] who says -- or who asks, "Is Novartis really considering introducing such a modern biologic treatment when more conventional or generic options are already a challenge to implement? And if you launch crizanlizumab in Ghana or elsewhere in Africa, how will you provide access to what's likely to be a more expensive drug? Is there a viable model for marketing such a drug or other monoclonal antibodies in Africa?"

Yes. No, thanks for the question. And I would say there is no easy answer to that. But we believe that we first have to fix the basics. And what we are now doing in Ghana really helps, already moves the needle for patients and people with sickle cell disease very significantly. And that to me -- but then also forms the foundation on which we can bring further innovation into the country. And then, of course, the country is not homogeneous in that all centers operate the same way. And if we can bring crizanlizumab, for instance, initially into tertiary care centers, where there's a very advanced infrastructure, very advanced knowledge in how to use those medicines, then that would be a very important step. And maybe the initial step means that patients don't have to fly to other continents in order to get advanced medicine but bring it into the continent, which doesn't mean that we'll initially very, very broadly cover everyone but also in those treatment approaches, normally you have sort of a cascading effect where you control most patients with very conventional medicines, and that's what we need to focus on. Then those who still have breakthrough episodes who qualify for more advanced treatments, we will bring crizanlizumab to [indiscernible]. We are now starting clinical trials in Ghana with crizanlizumab, and that in itself always means a commitment for us to also register and launch the medicine there. And we haven't figured out yet everything about the pricing and how we make this sustainable from a commercial perspective, but there is a lot more that we can do in order to prepare the ground for these advanced medicines. And typically, you see that when you have more volume, cost of goods come down. I mean 20, 30 years ago, it was inconceivable that anti-retroviral therapy would be widely available on the African continent in HIV/AIDS, and it is now sort of readily available. So I think we are -- these problems can be solved, but they cannot just be solved by having a debate on the pricing but really looking holistically at the health.

Well, I think that's an important insight. And it also shows what Novartis and other companies are trying to do. This is certainly true now in -- with COVID-19, as we'll explore further in the next webinar, that there's really an interest in tackling these tough problems of how to bring new innovations to Africa or other underserved areas of the world without having to wait decades and without having to increase the gap between the medical haves and the medical have-not. And I think that what the work that Kof's been talking about that is happening on the ground in Ghana shows that it is possible to bring the highest standards of care to addressing conditions like this. I wanted to turn back to Kof for a couple of minutes. And you've painted a powerful picture of the impact that your partnership with Novartis has had on the possibility of addressing sickle cell disease more sustainably in Ghana. But I wonder if you could help us understand the human impact for the children and families affected. Can you tell us a little bit more about how does sickle cell disease shape the lives of those who live with it and how access to care and treatment helps them to live longer and healthier lives?

Thank you very much, Jeff. When we started the newborn screening program, most of the families, when they were told that their child has sickle cell disease, a very emotional time, would burst into -- mothers would burst into tears. Some of them would even pass out because they really thought that for all that they had heard about the disease, their children were facing an early death sentence. And as we educated these families to anticipate some of the problems and to learn about the solutions, and they actually saw the problems developing, first, they believe that we actually know about this. We know about this disease. It's not a hopeless situation here. We have told them that when your child gets a little older, their eyes may look yellow. Their hands may swell, their feet may swell. And they actually see it coming. So what used to be just a mystery, now they began to believe that people know about this disease and can do something about it. Part of our education has always to be able to tell families that if you have a child with sickle cell disease, a chronic disease, that you worry about raising your child and we would try and worry about what to do for the child's health care. Over time, these families have grown to accept the fact that this is a disease that they can do something about. They become our strongest advocates. And I think also within the medical community itself, the idea that we just don't abandon people to the fate when it comes to chronic diseases is also developed. Everybody has developed a sense of hope. The hydroxyurea program, and I tell people in Ghana that when -- in our partnership, we knew that Novartis was not making hydroxyurea. It wasn't in my mind that we should ask Novartis to make hydroxyurea. It was at a meeting with families at one hospital, where they just wanted to meet some patients, that mothers and fathers started talking about the fact that hydroxyurea had changed the lives of their children but that it was too expensive for them and that sometimes they couldn't afford it. And that meeting did a lot more to get the attention and the need for hydroxyurea than anything that I could have said. So the impact of our interventions, how that's been received by the families and how it's affected the communities, drives what we do, drives what we always hope we can bring to them more. And I think that, not just the Sickle Cell Foundation of Ghana but other organizations that are working in sickle cell disease now all speak a different language. We teach about hope. We teach against stigma. And we try to raise the standards of expectations of these patients that, in fact, their children and themselves can do a lot more than they thought. So I think it's important to put a lot of the credit to the families themselves is that they have rallied to now develop a lot more of a hopeful approach towards the disease. And I'm not surprised that new medications that we are talking about would also become available in Ghana. An analogy I always make is that I've never heard the government comping about people owning mobile phones. When we started our project, less than 5% of families had phones. Now about 80% have phones, and that's not because anybody really forced them to do so. If there are new medications available that can help children especially and adults with sickle cell disease, people will find a way to get it. I truly believe that people have that sense of hope.

No. That's -- I think that's really an important point. Regarding to -- just to our listeners, we're going to run a few minutes past the hour because there are still some questions coming in. And I wanted to then turn to 2 more of the questions from our listeners. One was about this question of putting in place a complete value chain from diagnostic to psychosocial support. And they raised a question about what role do communities play in helping to ensure that new -- or new treatments for sickle cell disease are adopted by the community. They asked about faith groups, for instance, and about using social enterprise models to expand the program. I wonder if either of you would like to comment on that.

Lutz, do you want to take it first?

Yes. No, I think the work with the communities is critically important. We've seen in the past companies, but also, to some extent, NGOs essentially imposing solution on communities that were not adopted and just didn't resonate with the community. So I think we have, as companies, to take a very humble approach and listen, work with the local leaders to really understand what matters to the patients that we want to help. And then the other element that I think from our perspective is critically important is making a mid- to long-term commitment. I think also here in the past, what particularly, I would say, organizations from the global laws have done is to make one intervention and believe it's a one-and-done situation. And this we have all learned is not sustainable and doesn't lead to the desired impact. And that's why we are interested in forging partnerships that last years or decades in order to really be a reliable and continued partner on the ground rather than just doing some, I would say, social initiatives when we feel we should or when the balance sheet allows us to do it. So that's why we need to turn this into a long-term commitment.

And Kof, did you want to comment on that?

Yes. Just quickly. When we formed the Sickle Cell Foundation, we thought of it as more of an advocacy program development organization. And so at that time, there had existed in Ghana for a few decades the largest patient support group that is called Sickle Cell Association of Ghana. So we actually considered the Sickle Cell Association of Ghana as our sister organization. And whoever is leader of that organization is a member of the Board of the Sickle Cell Foundation. Because we had always felt that whatever we would do needed to have the voice of the patients and their families in it. And so their partners with us in all the upfront that we do. And all the different factions, faith communities, different cultural groups in the country, we get invitations all the time to attend events where we can either talk about screening for sickle cell conditions. We had developed a consular certification program in Ghana basically to serve the communities to explain this genetic disease to our communities. Ultimately, we hope that is the voice of the communities and members of the communities that is actually going to get government to respond to the request that we, the professionals, tend to make repeatedly. I think that once the voters and the people who elect people to parliaments believe that something can be done for sickle cell disease, our pleas will be listened to more because the politicians will also pay attention to what we're doing.

Yes. That's certainly critical. Well, I have one last question for you both, and that is this -- the partnership that you've created among the government of Ghana, the Sickle Cell Foundation and Novartis was announced in November 2018. And public-private partnerships like this bring together complementary skills and resources, as Lutz observed, to accomplish ambitious goals. I just wanted to ask you both to reflect for a minute on how would you assess your progress so far. What challenges surprised you? Were there any bumps in the road? And what are some of the key lessons or critical success factors that you've seen so far?

Well, I think...

Well -- go ahead.

Go ahead, Kof. No, please, go ahead.

Well, I just want to say briefly that I think we anticipated that this is not something that was just going to roll out very smoothly. And I'll use the hydroxyurea program as an example. Here was a very ambitious program. We have these 18 clinics. 7 of them were now well organized enough. We thought to begin the program. But 11 of them had been brought together. We trained for each program a doctor, a nurse and a pharmacist as part of the core specialists to develop the program with. And we are learning that even when you have a program that is providing services, you still have to get people to buy into it: the institutions where they are, the communities to be able to come forward and participate in the program. And none of this, in my mind, is a surprise. We have had more support from Novartis than even I anticipated. We just -- a couple of days ago, we had a meeting of the steering committee of this program. And Novartis, on our request, request from the sites, is going to help support even the laboratory tests that needed to be done to monitor the hydroxyurea. So I think that things are moving even better than I anticipated. And we hope that by the end of the year, we would have added a few thousand more patients to those who are benefiting from this growth. So I think things are moving quite well. I guess, organizationally, nothing is very easy. Another thing that we've always done is we try to modernize what we do to use technology to advance our work. And the app that Lutz mentioned, we now have the app. The -- all the sites have the phones, and we are training them on how to use the app on the phone. This will bring efficiency to what we're doing. So I'm happy with the pace of the progress we are making.

Well, thanks, Kof, and I can only echo that. I think the partnership and the ability to bring complementary resources and expertise to bear has proven to be really powerful. And progress that we have made in such short time altogether with the people in Ghana is truly remarkable. I mean operating in Sub-Saharan Africa always has its bumps in the road in a very literal way and also kind of has some of its challenges, which you don't expect when you work in a different environment. And I think what we need to do is to keep -- continue keeping a close eye on the goal. Sometimes those bumps drive additional creativity. And just one other point -- case in point here, for instance, the operations that we have with Zipline, with the drone delivery company, that really overcomes, which is notorious in Africa, issues around supply chain. And we have the drones now deliver hydroxyurea and other life-saving medicines through the last mile to be applied there. So sometimes I would say these bumps can spark innovation that then has benefits in its own right. And I think what we, as partners here from the pharmaceutical sector, can do is to be listening to take a humble approach and try to help where we can. And then it takes partners like Kof or also the government who sort of point us to the right -- in the right directions where we can help. On a personal level and even having known as a physician about sickle cell disease since medical school, visiting Ghana, I was really impressed how present sickle cell disease is in the public, but also how its perception and stigma still exist. And Kof had alluded to that earlier, but we were having a symposium in a hotel. And the hotel staff approached us with questions around sickle cell disease because it was in their family or affected them personally when they saw the signpost it's a meeting room. So I think I have certainly underestimated how impactful this condition is in the public and how much impact it has on people living with the disease.

Yes. Lutz, it's so important that we have to remember that the work we do in global health is really all about improving people's lives. There were just 2 final quick questions that our listeners had as they were listening to you respond to this last one. One was, do you have plans to expand this work to other countries in Africa? And secondly, and this may be -- well, to both of you, are there plans to -- you must be collecting safety and efficacy data as you continue to expand the number of people who are getting treatment with hydroxyurea and ultimately through the clinical trials with crizanlizumab. Are there plans to publish the results of what you've been doing in Ghana?

Yes. Absolutely. I mean in Ghana now, we are setting up under the auspices of Kof, a patient registry that can really capture all of these real-life data how hydroxyurea is being used in the African setting, in the African target population. And to the best of my knowledge, that would be one of the first data collections of that magnitude from real-life, real-world evidence. And we are certainly committed to publish all our data as we do in areas of research and development. So that's certainly a given, and we'll let you know as soon as we have data that could be realized [indiscernible]. On the question of geographic venture, my theme would be to replicate the Ghana-like program all across Sub-Saharan Africa. But we have to start with one country sort of at a time. And the learnings that we will gain in Ghana are incredibly important to them, also inform our approach in other countries. We've just signed a memorandum of understanding, respectively, with the government of Tanzania and with the government of Uganda. Unfortunately, the plan to roll this out is being impacted by the current pandemic. But to the extent we can, we work remotely. We work through computer videoconferencing to advance this agenda and hope to be able to launch the programs in those 2 countries before the end of the year.

That's really, really encouraging. Kof, did you have any comment on publication, for instance?

Definitely. So one of the reasons why we wanted to do this through a mobile app was really to be able to capture all the details of the management. Many of the staff that we're using around the country in this program are not in academic institutions. And it would have been very difficult for us to just use paper records in hospitals to track the patients very well. And the app really makes it easy for us to be able to collect data and analyze them very quickly. So that is really our goal. And we are developing by itself a monetary and evaluation program now going to be in collaboration with the University of Ghana School of Public Health. So that not only the medical aspects but also the psychosocial aspects of this program can also be tracked and also reported on. Because it wouldn't just be measuring lab tests but just how this program fares, how it was received as an implementation science activity and also how you transform people's lives, we want to be able to report on it.

No. That's so important. And I think that -- just one of the comments is that what you've just been talking about in the last few minutes about other -- you've seen innovations in how you're collecting data on the digital tools that patients have, the relationship with Zipline on distribution of hydroxyurea, it gets to the point the actual overall theme of this session about rethinking strengthening of health care systems and how has the kind of impact that you're having on sickle cell disease in Ghana, there's innovation at all levels, not just in new medicines like crizanlizumab, but also in the way that you develop implementation programs, the way that you monitor and evaluate them, the way that you get data and make it possible for patients to take advantage of the work that you're doing. I think that's one of the critical issues that strengthening health care systems means that we have to think creatively about how to deal with all of the problems that come up. I'm afraid we've run out of time. So I wanted to just wrap up by thanking both Lutz and Kof for a very thoughtful and thorough discussion that I'm sure all of our listeners found fascinating. And it shows us that -- I want to go back to something that Kof said early on, that it seemed impossible to actually address sickle cell disease in Ghana when you started. But what you've shown us today is with the right knowledge, with the right partners, with the right champions, you can actually do something about something that seemed impossible and make it very possible indeed. And the other point that you made toward the end that I think is so important is this gives everybody involved a sense of hope about what we can achieve in global health when we bring the right knowledge and the right resources together. So again, I wanted to thank you both. And if we can go to the next slide. I just wanted to thank the audience as well for your attention. For more information, you can check out novartis.com, the CR Materiality Assessment Results Report for 2017 and Novartis' -- Novartis in Society Report for 2019. For any feedback, there's the number -- or excuse me, the e-mail, [email protected]. And please keep your eye out for a notice about the next webinar in the series to -- which will be on COVID-19 next month. So again, thank you all, and it's really been a pleasure to moderate this. And once again, on behalf of Novartis and all of us, I want to thank Lutz and Kof for really sparking so many ideas and giving us such an interesting overview of the work that they've been doing together on sickle cell disease in Novartis. Thank you, and goodbye.