RARE DISEASES AND ORPHAN DRUGS
The activities were conducted according to the actions determined in line with the priorities within the scope of the 2019-2020 Strategic Plan. A stakeholder map was created in order to determine the needs of all stakeholders in this field. A position document was created summarising the views of AIFD member companies in this field and the status of orphan drugs in our country. The following opinions were highlighted in the position paper.
- Combating rare diseases should be one of the priority issues of public health policies. These policies should include elements such as the creation and support of national plans for improving diagnosis and treatment processes. It is necessary to step up Clinical trials, patient / disease registry studies, promotion of national epidemiological data creation and development, establishment of national databases and national reports on rare diseases, disease screening studies and medical education specific to rare diseases.
- A local legislation in line with international standards including the definition of orphan drugs should be prepared. Existing legislation should be updated to include orphan drugs.
- In GMP inspections and licensing processes, the number of samples required should be kept at minimum due to the high costs and restrained usage of such drugs; the products should be on high priority evaluation list, in analysis phase these products should be given high priority.
- Priority evaluation / shortened commission processes, parallel licensing and reimbursement processes should be improved; a provisional supply system should be established for products that apply for reimbursement.
- Indication-based evaluation should be made for rare diseases. In relevant institutions scientific advisory commissions specific to orphan drugs should be established. The methods of rapid access to drugs applied by internationally reputable authorities should be included in the agenda of cooperation of the health authorities.
- Diagnostic and treatment centers specialized in rare diseases should be developed. Reimbursement processes of diagnostic methods specific to rare diseases should be improved. The scope of newborn screenings should be increased.
- In order to strengthen cooperation and coordination to improve diagnosis and treatment processes, it is necessary to support the formation and operation of rare disease specialist committees and of non-governmental organizations, and to establish reference communication networks between diagnosis and treatment centers. Programs to provide the right treatment to the right patient at the right time should be developed and supported.
- Ethical committees specific to rare diseases should be established, accelerated ethical committee processes and national epidemiological data production should be encouraged, cooperation with specialized associations and expert centers, electronic identification and monitoring systems should be put in practice.
- When there are treatment options, all stakeholders should work in collaboration to ensure that patients can access the treatments they need in a sustainable way. Considering the small number of patients and the restricted number of specialists, these collaborations are primordial.
Orphan Drugs Survey
To establish a reference to the position paper prepared in 2020 by the Rare Diseases Core Committee, a survey was conducted to collect up-to-date data in this field in Turkey and in the world on the licensing and market access status of products.
A total of 17 AIFD member companies provided data to the survey where data on products with ORPHA code that were licensed or given rare diesease indication by EMA, FDA or TITCK between 01.01.2014 and 31.12.2019.
In the questionnaire, the data of 84 active substances and 120 new indication applications were analyzed. According to the data collected, the following results were seen about the access status of the products for which a license application was made at TITCK between 01.01.2014 and 31.12.2019.
- Out of 63 products with license / indication applications, 45 (71%) were approved by TITCK, 21 (33%) received reimbursement approval.
- The median of the approval periods, when 2018 and 2019 application are not included, the access rate reaches 41% (21 approvals in 51 applications) in this timeframe.
Communication and Cooperation with Stakeholders
AIFD's views and suggestions on rare diseases and orphan drugs were conveyed to key stakeholders and decision-makers operating in this field at the meetings held in 2020.
03.02.2020
We participated to the Stakeholder Network Meeting of the “İSTisNA-Istanbul Undiagnosed and Rare Diseases Solution Platform Feasibility Project” organized with the partnership of Acıbadem University, Istanbul University and TUSEB with the support of Istanbul Development Agency.
18.02.2020
A congratulation and introductory visit was made to Assoc. Prof. Dr. Onur Burak DURSUN, recently appointed as the Director to the Otism, Special Mental Needs and Rare Diseases Section that was established in January 2020 in the Health Services General Directorate
28.02.2020
Core Committee participation to the “Rare Dieases Panel” together with Mr. Mehmet Gündüz, Deputy General Manager of Health Services General Directorate of the Ministry of Health and Assoc. Prof. Dr. Onur Burak DURSUN, Director to the Otism, Special Mental Needs and Rare Diseases Section
Contribution to the Policies on Rare Diseases and Orphan Drugs
A report has been prepared within the scope of the Strategic Plan in order to elevate the awareness of the public services on the content of our position paper on rare diseases and orphan drugs and to increase its impact and to meet the need for a reliable information on these areas in our country.
A series of meetings were held during the report preparation phase with consultancy firms that have qualitative and quantitative research competence in this field. A concrete project proposal was prepared taking into account the content recommendations obtained from all interviews. The project proposal will be evaluated by the AIFD Board of Directors at the beginning of 2021. It is aimed to initiate the project with the field research phase after the project approval.
