Now the world and Ukrainian public is paying close attention, in particular, to everything related to medicine. UA-Times spoke with “Rosh Ukraine” Medical Director Oksana Surtseva. This company, among other things, is looking for innovative solutions in areas of medicine where the possibilities of effective and safe therapy are limited or absent, as well as clinical trials of new drugs.
– Tell us what innovative medicines mean?
-Innovative drugs are drugs that radically change the approach to patient treatment. This is something that did not exist before – a new molecule, new technologies.
Many years of search and efforts of many people – scientists, government agencies, pharmaceutical companies invested in innovative developments. Sometimes patient’s organizations when they initiate the creation of new drugs. For example, a drug for the treatment of spinal muscular atrophy (SMA), a genetic disorder in which no muscle protein is produced to maintain muscle tone. The patient’s organization has asked to develop a fundamentally new technology that will replace muscle protein and, if not completely cure, at least stop the progression of this disease.
Today, treatment of the cause of the disease (pathogenesis) exists for only 10% of diseases. In 90%, it is a symptomatic treatment. For instance, in rare diseases with a huge impact on patients and their families, treatment is available for only 200 of them.
The search for innovative solutions that bring new healthcare systems to patients is always based on an in-depth study of the needs that exist in society. It is a partnership with the medical community, patients, independent research centres and the state.
– How long does research to create drugs take? Are inventions expensive?
-The research process is very long, time consuming and costly, as there are always several teams of scientists, many laboratories around the world, sometimes competing with each other to find the molecule that will be the hope for the patient. On average, the study takes 12-15 years. If you look at the statistics, for example, of our company, only 5% of molecules out of 100% go all the way through clinical trials and get to the patient. The remaining 95% do not reach the finish line. The development of innovative drugs involves several important stages.
Molecule development – imagine 40,000 molecules were studied to select one candidate molecule for clinical trials that would mimic the work of 8th coagulation factors in patients with haemophilia. To optimize the process and reduce costs, pharmaceutical companies actively cooperate with digital companies. With the help of artificial intelligence, it is possible to reduce the number of losses at the search stage and increase the number of successful molecules for clinical trials.
Clinical trials include three phases. The first phase is a test for safety, tolerability and availability of therapeutic actions. The second is to evaluate the effectiveness and safety to the patients with this disease. At this stage, evidence of clinical efficacy and therapeutic doses are determined. And the third phase is research on large groups of patients of different ages, when risk and benefit indicators are assessed. There is a comparison with the existing standard of treatment. However, when it comes to new molecules, it is often compared to nothing. Successful completion of this stage guarantees a positive decision on drug registration. This is the most expensive stage. This explains the high price of innovative drugs.
Production is also an important part of creating an innovative drug. Since all processes are high-tech, the companies that produce innovative drugs have very high requirements for location, environment, staff. Not every country meets them, so such companies are mainly located in economically developed countries such as Italy, USA, Switzerland, Germany, Japan.
– Which countries are leaders in this direction and why?
-There are several companies in the world that can afford to invest in new molecules. One of the reasons is the cost of failure, the 95% of molecules that do not reach the stage of clinical trials. To make it clear how much money you need to invest in the development of new molecules, I will give an example of our company. The amount of investment spent on the development of innovative drugs in 2019 amounted to 11.7 billion Swiss francs, which is several times higher than Ukraine’s GDP. ‘Rosh’ is the number one pharmaceutical company in the world in terms of investment in innovation and ranks eighth among all industries in the world. With us in the top ten are such giants as Amazon, Alphabet, Volkswagen and Apple.
Laboratories are needed to develop new molecules. We have such laboratories in the USA, Japan, Switzerland. We buy 50% of our drugs from other major laboratories that specialize in developing new molecules. Then such ones undergo clinical trials and, if successfully passed, become drugs for treatment.
“Rosh” is a leader in personalized medicine. The unique combination of two areas – diagnostics and pharmacy – allows you to develop new solutions – unique to each patient. There is no treatment solution that will suit every patient. That is why personalized medicine is our present and future.
–Tell us, which diseases are now a priority in terms of studying and creating innovative drugs?
-Today, cancer is the leading cause of death in developed countries, and the number of cancer incidents continues to rise. Research shows that many forms of cancer can be effectively treated with early diagnosis.
‘Rosh’ is a leader in oncology both in the world and in Ukraine. We are proud of the widest portfolio in oncology and in the next 5 years the company plans to double its investment in the development of innovative molecules in this area.
Neurology – new developments in the treatment of multiple sclerosis, such rare diseases in neurology as autism, Huntington’s disease and the most common, such as Parkinson’s disease, Alzheimer’s. In the last two directions the molecules in the final stage of research.
New directions – ophthalmology and gastroenterology.
These are also orphan (rare) diseases – such as SMA. These diseases require lifelong, mostly expensive treatment for the patient, but there may be 200 people with such diseases nationwide.
As already mentioned, today only 10% of existing diseases have a solution to treat the cause or the main mechanisms of development, rather than symptoms. When a pharmaceutical company, as a leader in innovation, is committed to finding new molecules that never existed before, it is always a big challenge and a big risk. To some extent, it is much easier to be the twenty-first drug, where there are many patients, than the only one for a small number of patients, where there have never been solutions. This requires very large investments for development and research to change the approach to treatment in these areas.
A very small percentage of rare diseases have treatment, because such patients are few (compared to other pathologies) and not all pharmaceutical companies are willing to invest in the development of new drugs for orphan diseases. After all, here the development of the search for molecules often begins with a review of the very cause of the disease. The work begins with the study of the mechanism of development and only then proceeds to the search for molecules.
The needs of society, the quality of diagnosis, the quality of understanding of many diseases have changed, the need for treatment has expanded.
– Can you give examples of which innovative medicines are now a real breakthrough in the treatment of a disease?
-In oncology in several areas. For example, for each of the four types of breast cancer there is an approach in therapy. Lung cancer has various types of mutations that occur in less than 1% of cases. Our company is looking for innovations in areas where 0.3% of the entire cancer population has such a mutation. To find it, you need to examine all patients. When such a patient is found, the therapy is personalized for him, i.e., he receives treatment that is suitable only for him. There is no patient with an identical disease and the same diagnosis.
In the treatment of SMA, the drug acts on a key link in the pathogenesis (protein production throughout the body). We hope that the drug will significantly improve the quality of life of the patient.
In haemophilia, the drug mimics the work of 8th coagulation factors, which changes the emphasis in the treatment of the disease – provides an opportunity to live not the life of a patient, but the life of a person with medical peculiarities.
– Probably one of the most relevant trends in recent months is the development of a vaccine against coronavirus. How do you assess the efforts of the whole world to create such a drug? Is the process going right? Are there any mistakes in accelerating development?
– There is worldwide experience. On average, it takes 15-18 months from the start of vaccine development to its launch. This is an expensive and time-consuming process that includes development, research, registration. Many companies around the world are now working to find a vaccine. There are large international corporations that specialise in vaccine development. Some have stated this publicly, some have not. It is wrong to make any predictions and estimations. There are encouraging results in the search for a vaccine, but we need to wait for the official results of clinical trials.
–According to your estimates, when can we expect the arrival of the first drugs for mass vaccination? Which countries can be the first on this path, why?
-First of all, these are countries where vaccination policy is of strategic importance and equates to the security of the nation. And if you look at vaccine manufacturers, they prefer countries that make government orders.
According to UNICEF, the situation with vaccination of the population in Ukraine deserves special attention, both from the state and outreach among the population, support from the media and international institutions.
– The WHO estimates that $ 31 billion will be needed next year to create the vaccine. Are these realistic estimates?
-WHO focuses on its statistics and data provided by vaccine companies. If the research is delayed over time, the costs may increase accordingly.
– Based on this cost of development, will the coronavirus vaccine be available to poor countries? According to the Ministry of Health, Ukraine has queued up to buy a coronavirus vaccine. Judging by the current situation in the world, according to your calculations, when should we expect the import of the SARS-CoV-2 coronavirus vaccine to Ukraine?
– The issue is not in the country’s poverty and priority. The attitude of the state to its citizens is important. Ukraine has a low level of vaccination compared to other countries. And there are two reasons for this – the state and the population. In many countries, vaccination of citizens is a matter of national security. The pandemic plan is being discussed and adopted at the state level and is being strictly implemented. Ukrainians themselves do not always treat vaccination with due responsibility: there are many cases when parents refuse to vaccinate their children, including not vaccinating against the flu, and themselves as well. And this is also the responsibility of the state, which does not take measures to solve this problem. Understanding the situation with vaccination against other infectious diseases, can we expect that the majority Ukrainians will be vaccinated against COVID-19?
– According to your experts, was it possible to get this vaccine faster and cheaper in Ukraine?
– There are cases when vaccine companies are willing to consider changing the price for countries with low GDP. If they follow this path, then Ukraine may have a chance to buy the vaccine at a special price.
– As mentioned earlier, the production of innovative drugs is an expensive pleasure. What steps should the Government of Ukraine take to reduce the cost of innovative drugs imported from abroad for Ukrainian consumers in general, and how can their cost in Ukraine be reduced?
-It is often asked that some drugs in Ukraine are more expensive than in countries with more developed health care systems. There is a simple explanation for this: Ukraine buys drugs at a list price. This price is open to the whole world, it is formed by the laws of supply and demand in the market. But there are other prices under “access agreements” at which the state can purchase drugs on completely different terms. These conditions are discussed in the dialogue between the authorised state body and drug manufacturers, fixed in access agreements and are not public. Under such agreements, the state can receive either a financial discount on drugs, or pay only for effective treatment for the patient.
With this approach, the government can provide 100% of patients (for example, with multiple sclerosis) “top” drug in the required amount – free of charge for patients. It is impossible to get the drug “from under the floor”. If the drug does not work as prescribed in the relevant documents for public procurement, its cost is partially or fully covered by the manufacturer.
The experience of our company and other large pharmaceutical companies that develop innovative drugs shows that dialogue with the manufacturer always allows the state to purchase drugs cheaper to ensure access for patients in this country to innovative drugs. Our company has many initiatives that work successfully in Europe and Asia, and our closest neighbours – such as Poland, Belarus, Turkey. Fortunately, the Ministry of Health, the Verkhovna Rada Health Committee, and regional authorities have recently begun discussing such initiatives. And I very much hope that this dialogue will bring positive decisions first of all to patients, healthcare structures and Ukraine as a whole.
–What innovative drugs do Ukrainian patients lack today and is it possible to develop and produce them in Ukraine?
-In Ukraine, according to statistics, only 8% of the budget allocated to the health care system is spent on medicines. Patients lack access to modern effective evidence-based treatment. For example, for oncology, multiple sclerosis, cystic fibrosis, rheumatoid arthritis, haemophilia access is available in the form of national and regional programs, but not 100% of patients are covered by such programs. For multiple sclerosis, the program covers only 10% of the need.
Nomenclature lists for public procurement for some programs in which our company operates include only basic drugs. As for spinal muscular atrophy and metastatic cancer, there are no drugs in the nomenclature lists at all.
In the nomenclature lists for public procurement there are no innovative quality drugs that are critically necessary for patients and meet modern standards of treatment of serious nosologies (oncology, multiple sclerosis, orphan diseases), whose effectiveness has been proven by international clinical trials and medical practice. This means that the state cannot purchase these drugs from the budget.
In our therapeutic areas, the drug is only part of the effective treatment of the patient. Of great importance is the quality of health infrastructure (equipment, laboratories, conditions in medical institutions) and the level of knowledge of employees and professionals.