Rare Diseases

Our scientific principles

With rare diseases it is essential to separate hope from evidence. This section is prepared according to the principles of evidence-based medicine.

1. Evidence-based medicine

Evidence-based medicine means deciding whether a treatment works on the basis of well-designed research, not personal opinion. How strong a piece of information is depends on the type of study, the number of patients, whether there was a comparison group, and whether independent experts reviewed the results.

2. We label every update by level of evidence

  1. Peer-reviewed publication

    A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.

  2. Official drug approval

    A decision by a medicines regulator (FDA, European Commission, etc.). The regulator has reviewed the clinical studies behind the drug. For accelerated or conditional approvals, the lasting benefit is still being confirmed.

  3. Preliminary result

    An early result that has not yet been published in a peer-reviewed journal, or comes from a small, uncontrolled study. Treat it with caution.

  4. Official statement or regulation

    A statement by a ministry, institution or international organisation, or a change in regulations. It is not evidence about how well a treatment works.

  5. News report

    Based on a single news source or an institutional announcement. The level of scientific evidence is low; check the details with a doctor.

An official drug approval applies to one country or region. For a treatment approved in the US or Europe to be used in Türkiye, it must go through the processes of TİTCK (Turkish Medicines and Medical Devices Agency), and reimbursement requires a decision by SGK (Social Security Institution). We point this out in every relevant update.

3. Source rules

  • Every update is based on a source page that our editors have opened and read, and we link to that page.
  • Order of priority: peer-reviewed journals, medicines regulators (FDA, EMA, TİTCK), the Turkish Ministry of Health, SGK and the Official Gazette, international organisations (WHO, EURORDIS, Orphanet), universities, then news agencies.
  • Updates based on company announcements or preliminary results are clearly marked “Preliminary result”.
  • We do not use exaggerated words such as “miracle” or “definitive cure”.

4. Independence

  • We do not publish advertising or sponsored content.
  • Because advertising prescription medicines to the public is prohibited in Türkiye, we do not name drug brands; we only use the active ingredient.
  • No specific hospital, doctor or company is promoted.

5. Medical review

The Doktorclub Rare Diseases Scientific Board is being set up. It is planned to include physicians from fields such as medical genetics, paediatric metabolism, paediatric neurology and haematology. Content reviewed by the board will show the reviewing physician’s name and the date. Detailed disease profiles will only be published after this review.

6. Corrections

If you spot a mistake, write to us via our contact page. We correct errors quickly and note important corrections on the page.

7. Not medical advice

This section is for information only. Diagnosis and treatment decisions are made by the physician who examines the patient.

The information on these pages is for general information only and does not replace medical advice. Talk to your doctor about diagnosis and treatment decisions. In an emergency in Türkiye, call 112.

Every update is based on an open source and labeled with its level of evidence. No advertising and no drug brand names. Our scientific principles

This page was translated from Turkish by the Doktorclub editorial team.