20 years of innovation: challenges and opportunities

This year, Amgen celebrates the 20th anniversary of its presence in Poland. What areas are you currently focusing on?

Amgen remains focused on helping patients. We develop innovative therapies used to treat diseases that pose a challenge to modern medicine. So far, we have focused primarily on lifestyle diseases, such as osteoporosis, cardiac diseases, cancer and hematological diseases. Now we are entering a new area – rare diseases. In Poland, almost 3 million people struggle with them. Therefore, despite their definition, rare diseases are not that rare after all and remain a challenge for the health care system. And the problems start at the diagnostic level.

You raised an important topic – diagnostics. What priorities do you see in this regard?

First of all, prevention is definitely better than fixing the consequences. It is primarily about patients, but also about the health care system. Therefore, educational screening programs should be promoted among the general public, thanks to which we can detect the disease at an early stage and reduce the risk of its development and consequences. A good example is osteoporosis. One of its symptoms may be loss of height resulting from a spine fracture. This can be detected with a very simple test: measuring your height. The next step is densitometry, i.e. a bone density test – it should be performed in every patient over 60 years of age who has suffered a low-energy fracture.

PLN 500 million is spent annually on treating the effects of osteoporosis, which affects over 2 million Poles, while preventing this disease would be much cheaper. Meanwhile, over 90 percent patients do not start treatment at the right time, i.e. at an early stage of the disease. A very high risk of osteoporotic fractures may lead to severe osteoporosis, resulting in disability or threatening the patient's life. The mortality rate in the first year after a hip fracture is over 20%.

So what should be done to change these disturbing statistics?

It is necessary to improve the health care system in such a way that access to diagnostics and treatment does not encounter barriers, because the social cost of lack of intervention is huge. Today, it is actually difficult to imagine a therapeutic area in which early diagnostics would not be important. Let's take cardiovascular diseases, for example. One of the risk factors for cardiovascular events, such as heart attack or stroke, is high LDL cholesterol levels. It may be the result of familial hypercholesterolemia, which is a genetic disease usually associated with a mutation in one of the three genes responsible for LDL cholesterol metabolism. There are two forms of familial hypercholesterolemia – heterozygous, occurring in one in 250 people, and homozygous, which is a rare disease.

The detection rate of familial hypercholesterolemia in Poland is estimated at approximately 3%. Meanwhile, according to studies, the relative risk of a fatal coronary event in women with familial hypercholesterolemia aged 20 to 39 years increases 125-fold despite treatment, and in affected men aged 20 to 39 years it increases almost 50-fold, compared to the general population. Patients with heterozygous familial hypercholesterolemia experience, on average, almost four times as many cardiovascular events compared to patients with a similar risk profile without genetic risk. In turn, in the case of homozygous hypercholesterolemia, failure to provide effective treatment means that the patient with this form usually does not live to the age of 30.

The occurrence of familial hypercholesterolemia in one of the parents causes a 50% chance of likelihood of passing it on to children. If we add to this the low detection rate of the disease in Poland and the risks it poses, it seems necessary to introduce screening among children in Poland, i.e. perform a lipid profile, at least during a six-year-old's examination.

What about access to modern diagnostics in oncological diseases?

The process could also be improved here. It would be important to speed up the diagnostic path. There is no simple solution, such as cascade referral, which would enable pathologists to transfer material for molecular diagnostics. Currently, lung cancer diagnosis takes an average of 4.5 months from the moment the patient appears in the system to the initiation of treatment. It is crucial to take actions that will help start optimal treatment faster.

It seems that this is a relatively easy problem to solve – it would be enough to immediately obtain the patient's consent for molecular diagnostics and issue a cascade referral, speeding up the diagnostic process.

It sounds very optimistic indeed, but how to implement these changes?

The implementation of any solutions, of course, requires coordination with various stakeholders and the implementation of a sustainable spending plan. As Amgen, we are open to cooperation with all stakeholders of the health care system to respond to patients' needs with even greater effectiveness.

The partnership model between the pharmaceutical community and the government should be based on building a dialogue with decision-makers, which would include, among others: to seek solutions to problems in access to therapy, shorten the period from registration to reimbursement and from the reimbursement administrative decision of the Minister of Health to the actual availability of the drug to the patient. It is also important to create a good, predictable legal ecosystem, so important in this highly regulated business environment. It is important to remember that at the end of all these activities is the patient. Or rather, it's at the very beginning. That is why we are constantly working to equalize opportunities for Polish patients in access to innovative therapies in accordance with clinical guidelines and recommendations of scientific societies.

Interviewed by: Bartosz Uchański