##  [Navigating the Approval Process for Rare Disease Orphan Drugs in Hong Kong](/blog/navigating-the-approval-process-for-rare-disease-orphan-drugs-in-hong-kong) 

    July 10, 2025 

  ![Navigating the Approval Process for Rare Disease Orphan Drugs in Hong Kong]()

1 min read 

- [Medicinal Products](/blog-main-categories/medicinal-products)
- [Regulatory Affairs](/blog-main-categories/regulatory-affairs)





 

In Hong Kong, the approval process for rare disease orphan drugs, particularly those containing New Chemical or Biological Entities (NCEs) for life-threatening or severely debilitating diseases, is managed by the [**Pharmacy and Poisons Board of Hong Kong**](https://www.ppbhk.org.hk/eng/index.html). Below is a step-by-step guide to navigating the process:

## Step-by-Step Guide to Rare Disease Orphan Drug Approval

1. **Preparation and Submission**
    
    Prepare the application including key documents:
    
    
    - Expert evaluation reports and CTD documents on safety, efficacy, and quality
    - Risk management plans (RMPs)
    - Product labels and information leaflets
    - Elemental impurity risk assessment reports
    - Worldwide registration record
    
    Submit your application for registration via the [Pharmaceuticals Registration System 2.0 (PRS 2.0)](https://www.drugoffice.gov.hk/eps/do/en/pharmaceutical_trade/guidelines_forms/prs_guide_main.html) on the Drug Office website. This is a critical first step for those registering orphan drugs for rare diseases in Hong Kong.
2. **Payment of Fees**
    
    Pay the application fee through:
    
    
    - PRS 2.0 (online) – via credit card or PPS
    - In-person – using cash or cheque at the Drug Office
3. **Screening and Evaluation**
    - The Drug Office screens all submitted documents before accepting the application for evaluation.
    - If documents are incomplete, a letter of deficiency will be issued.
4. **Refuse-to-File (RTF) Mechanism**
    - Effective 1 July 2024, the RTF mechanism ensures only complete and properly documented applications proceed to evaluation. A **60-day response period** is provided for applicants to address any deficiencies identified during the initial screening.
    - Incomplete applications will be refused at the screening stage. Failure to respond within 60 days will result in an automatic refusal of the application.
5. **Enhanced Procedures for NCE Products**
    
    For NCE products under the “**1+ mechanism**” (for life-threatening or severely debilitating diseases), the Board has endorsed enhancements to facilitate and expedite the registration process—particularly beneficial for orphan disease drugs.
6. **Post-Approval Requirements**
    - After product registration: 
        - Comply with pharmacovigilance requirements
        - Report serious adverse drug reactions (ADRs)
        - Implement and maintain risk management plans
7. **Appeals**
    
    If aggrieved by a committee decision, the applicant may appeal to the Pharmacy and Poisons Appeal Tribunal.
8. **Timelines**
    
    The Department of Health aims to complete the registration process around **9 months** after submission, provided all documents are complete. This helps bring drugs for rare diseases to market efficiently.
9. **Consultation and Feedback**
    
    The Board may conduct consultations with stakeholders and industry experts during the evaluation and policy implementation stages.
10. **Continuous Updates**
    
    Stay informed by Regularly checking:
    
    
    - The Board’s official website
    - Announcements from the Drug Office

## Flowchart

![Step-by-Step Guide to Rare Disease Orphan Drug Approval]()

## How Freyr Can Help 

Freyr offers end-to-end support for [registering orphan drugs in Hong Kong](https://www.freyrsolutions.com/medicinal-products/medicinal-products-regulatory-support-in-hong-kong), including dossier preparation, PRS 2.0 navigation, and post-approval compliance. With local expertise and a strategic approach, we help ensure faster, compliant market entry for orphan drugs for rare diseases.