Introduction

Rare diseases are defined as diseases that occur infrequently. Even if such diseases are considered rare, it is estimated that they affect approximately 350 million people around the world. Moreover, there are an estimated 7,000 rare diseases worldwide.(1)

Rare diseases usually stem from genetic factors and their effects are not difficult to see. Those suffering from such diseases often have chronic ailments and permanent physical issues. As a result, their lives and those of their close relatives become increasingly difficult.

Orphan medicinal products are used to prevent and treat rare diseases. However, approximately 95% of all rare diseases are yet to be treated effectively.(2) Although access to medicinal products has improved, this remains an issue in the treatment of rare diseases. These difficulties start at the diagnosis stage.

Rare disease rates

Research suggests that the rate of consanguineous marriages in Turkey is between 22% and 27%. According to Turkish Statistical Institute data, in 2016 23.2% of marriages were consanguineous.(3) The high rate of consanguineous marriage increases the incidence of rare genetic diseases. It is estimated that approximately 6 to 7 million people in Turkey are affected by rare diseases.(4)

Orphan medicinal products

Orphan medicinal products used in the treatment of rare diseases often result from challenging R&D processes. R&D in this field requires investment because these products appeal to a limited number of patient populations and R&D processes are long and costly.

Rare diseases, which present serious obstacles to human life and for which the necessary medicinal products are undeveloped, constitute a social and an economic issue. Not only people who suffer from these diseases, but also their caregivers are deprived of their social life. For this reason, providing incentives and support to scientists studying rare diseases is vital. Government incentives to pharmaceutical manufacturers and researchers to research, improve and launch orphan medicinal products for use by patients are important in this regard.

Pursuant to Article 25 of the Universal Declaration of Human Rights, the right to food, clothing, housing and medical care is universal. Accordingly, contracting parties to the declaration must provide the necessary support and funding for the provision of medical treatment. Articles 5, 56 and 166 of the Turkish Constitution set out that the state is responsible for the removal of obstacles that affect the fundamental rights and freedoms of citizens and the establishment of health and social institutions. These articles set out that the state must make the necessary arrangements to provide healthcare to its citizens. Therefore, the law requires that the necessary measures are taken to ensure a healthy life for all citizens.

Comment

The issue of rare diseases and orphan medicinal products requires special regulations and policies. The US Orphan Drug Act 1983 and the EU Regulation on Orphan Medicinal Products (141/2000) have helped to foster research into rare diseases and orphan medicinal products. Establishing similar regulations in Turkey would be a valuable step in the fight against rare diseases. A balanced strategy in this regard would require a forum where sector stakeholders and relevant experts can express themselves. The need to develop common strategies with all related stakeholders is key in the battle against rare diseases.

For further information on this topic please contact E Sevi Firat at Firat Izgi Attorney Partnership by telephone (+90 212 235 25 25) or email (sevi.firat@firatizgi.com). The Firat Izgi Attorney Partnership website can be accessed at www.firatizgi.com.

Endnotes

(1) For more information see here.

(2) For more information see here.

(3) For more information see here.

(4) For more information see here.

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