Saudi Arabia is introducing a new accelerated route for orphan drugs.
The Saudi Food & Drug Authority (SFDA) has published a new regulation for orphan drugs – the Regulatory Framework for Rare Diseases Drugs – and announced the launch of a new accelerated programme for rare disease therapies: the New Accelerator Program for Drugs in Rare Diseases (NADR).
The document was published on 5 August 2026 and enters into force on 5 September 2026.
🧬 Orphan Designation: What Matters
Orphan Drug Designation (ODD) is granted not to a drug in general, but to a specific drug for a specific orphan indication.
Each new indication requires a separate application, so a single drug may hold multiple orphan designations.
It is important to note that obtaining ODD does not constitute marketing authorisation: the drug must still undergo assessment of quality, efficacy, and safety.
An ODD application may be submitted at any stage of development, but prior to submission of the marketing authorisation dossier. The designation may also be granted to a drug already registered in Saudi Arabia – for a new orphan indication, a new dosage form, or a significant modification.
📌 Key ODD Criteria
To obtain orphan designation, a drug must meet several conditions:
— the disease is serious, life-threatening, or leads to significant disability;
— the prevalence is fewer than 5 cases per 10,000 individuals in Saudi Arabia;
— the drug is being developed for the corresponding orphan indication;
— there is no satisfactory method of prevention, diagnosis, or treatment, or the drug offers a significant advantage in terms of efficacy, safety, or patient outcomes.
There is no fixed validity period for ODD. The SFDA may suspend or revoke the designation if the drug no longer meets the criteria, or if the designation was obtained based on incomplete or inaccurate data.
⚡ What the NADR Programme Changes
NADR does not replace ODD but creates an additional accelerated pathway for orphan drugs that address a high unmet medical need.
If the drug does not yet have ODD, both applications may be submitted simultaneously.
To be eligible for NADR, the drug must be intended for the prevention or treatment of a rare, life-threatening, or seriously disabling disease, meet the prevalence threshold of fewer than 5 cases per 10,000 individuals, and be used in the absence of satisfactory treatment or provide significant clinical advantage.
The programme may apply to drugs at various stages of development – from preclinical stage to Phase III – as well as to drugs already registered in another country but not yet authorised in Saudi Arabia for the claimed indication.
⏱️ Timeframes and Accelerated Tools
Applications for NADR and the parallel ODD submission will be reviewed within 20 working days.
Once NADR is granted, the marketing authorisation dossier in eCTD format must be submitted within six months.
The programme provides:
— priority review;
— rolling submission;
— conditional marketing authorisation;
— regular consultations with the regulator;
— assignment of a dedicated SFDA manager to support the drug.
One of the most important provisions of the new NADR programme is the expansion of the acceptable evidence base for orphan drugs.
The SFDA indicates that for the development of rare disease therapies, not only traditional clinical trials may be used, but also more flexible methodological approaches.
📊 Types of Data That May Be Considered
Under the new regulatory model, the following may be used:
— adaptive Bayesian trials;
— N-of-1 trials;
— basket, umbrella, and platform trials;
— natural history data as external controls;
— surrogate endpoints and biomarkers;
— pharmacokinetic and pharmacodynamic modelling;
— artificial intelligence and machine learning;
— in silico modelling;
— organ-on-a-chip models;
— data from the Saudi Human Genome Programme and national biobanks.
For ultra-rare diseases, the use of previously accumulated data on the same molecule, pharmacological class, vector, or technological platform is permitted.
⚖️ Proportional Approach to Evidence
The SFDA is introducing a risk-based approach taking into account the severity of the disease.
This means that for a rapidly progressive fatal paediatric disease in the absence of treatment, the regulator may accept a smaller volume of pre-registration data – subject to enhanced post-marketing monitoring.
This approach is particularly important for rare diseases, where conducting large randomised trials is often impossible due to small patient populations, disease severity, and ethical constraints.
🔬 Flexibility in Nonclinical and Manufacturing Aspects
The document also allows for reduction of the preclinical programme, toxicity studies in a single relevant animal species, use of data from similar platforms, and deferral of certain chronic, carcinogenicity, and paediatric studies to the post-registration period.
Applicants may request exemption from certain Chemistry, Manufacturing, and Controls (CMC) requirements. Such a request must be reviewed within 20 working days.
Flexibility is also provided with regard to GMP inspections: the SFDA may take into account certificates and inspection reports from regulators of countries with well-established control systems.
📌 Why This Matters
Thus, Saudi Arabia is moving from simple expedited review to adaptive regulation of the entire orphan drug lifecycle. At the same time, the document does not yet link NADR with health technology assessment, pricing, reimbursement, or public procurement, so accelerated authorisation alone does not guarantee actual patient access to treatment.
