**6. Discussion**

In general, our questionnaires showed that the examined physicians and clinical pharmacists, as well as patients, were very interested in expressing their attitudes regarding the issue of rare diseases and their treatment in our country. This is confirmed by the high percentage of provided answers (the total response rate was 97.2% and 95.83%, respectively).

The first thing to notice here is that both the patients and medical professionals did not show sufficient knowledge regarding this topic. It is important to emphasize that both groups of our participants were directly involved in this issue (for survey, medical professionals who were in daily practice in contact with RDs patients and patients who belonged to the RDs patients' group by definition were selected).

Our respondents estimated the importance of rare diseases in our society as a problem of crucial importance. Here, we noticed a significant difference in attitudes between RDs patients and third- and sixth-year medical students that we have examined in previous research (P < 0.01). In opposite, the observed patients and examined students showed a similar opinion regarding the overall quality of health care of RDs patients, rated it as insufficient (P < 0.05) [10].

We need to reveal that the issue of pharmacotherapy of rare diseases involves different kind of ethical dilemmas and controversies. That is why we strongly believe that understanding and analyzing such a sensitive topic must also implement knowledge of basic principles of biomedical ethics [11–14].

In our survey, respondents found that the state was most responsible for the improvement of availability of orphan drugs. However, we need to be aware that national policies and activities of the state start from the initiative of those who

participate in the health system of one country (policy-makers, members of the regulatory bodies, medical doctors, pharmacists, patients, etc.). Our research has shown that patients do not participate sufficiently in these activities and do not take an active role in the fight to improve their position in society. Related to these findings, *Baker* and *Trisnadi* in their works emphasized the importance of understanding personal responsibility to address a bioethically vulnerable issue in one community [15, 16].

In the last three decades, the concept of "medicalization" of society ("a pill for every ill") has been increasingly present in numerous bioethical debates [17]. In contrast to this phenomenon, there is a real need to develop novel drugs for rare diseases and their inaccessibility in many societies, on the one hand, and the lack of concern of the pharmaceutical industry regarding this topic, on the other [18, 19].

A controversial and substantive question would be is it reasonably and ethically justified to invest large amounts of money to treat a small number of patients? In a previous study performed on third- and sixth-year medical students, we received an affirmative answer to this question in both groups (P < 0.05) [10].

The most important results of survey conducted among third- and sixth-year medical students are shown in **Table 1**.

Finally, in order to improve the pharmacotherapy of rare diseases in Serbia, medical professionals and patients similarly suggested: improvement of general and scientific knowledge, well-timed diagnostics, simplified procedures for drug provision, registration of more appropriate drugs, etc. (see **Tables 2** and **3**).

The first comprehensive study was recently published by Joldic et al. related to the needs of patients with rare diseases in Serbia. According to this paper, four different groups of needs are identified: needs for health care, needs for social care, psychological needs, and other needs. The most important problems of RDs patients and their families arise first from the insufficient information and knowledge on this topic and second from the non-recognition of rare diseases in the legislation of health care system [7, 20–24].


#### **Table 1.**

*Medical students' knowledge and attitudes concerning rare diseases.*


#### **Table 2.**

*Physicians' and pharmacists' knowledge and attitudes concerning rare diseases.*


#### **Table 3.**

*The knowledge and attitudes of patients suffering from rare diseases*
