Why Some Drugs Are Hard to Access: Orphan Drugs and Rare Diseases

---

Accessing life-saving treatments can be challenging, particularly for those living with rare diseases. In Singapore, as in many other regions, orphan drugs — medications specifically created to treat these rare conditions — come with unique challenges regarding availability and cost. In this guide, we delve into the complex issues surrounding orphan drugs and offer a closer look at Singapore’s current pharma market access landscape.

Definition and Scope

According to the Medicines (Orphan Drugs) (Exemption) Order, rare diseases are life-threatening or severely debilitating conditions that affect fewer than 20,000 people. Orphan drugs in Singapore are defined as medicinal products that a doctor or dentist has identified as essential for treating a rare disease when no effective substitute is available. These drugs typically haven’t been licensed under the Medicine Act but have received approval from health authorities in their country of origin or other countries where they have been used.

While the prevalence of rare diseases in Singapore is relatively low compared to more common health issues, the impact on patients and their families can be significant. The rarity of these conditions makes rare diseases access and the development of effective treatments particularly challenging.

Research and Drug Development Challenges

Developing orphan drugs involves several significant hurdles. Pharmaceutical companies often face high costs and technical difficulties due to the small patient populations involved. Research and development (R&D) for these drugs requires substantial investment, yet there is no guarantee of a large market. The financial risks, coupled with complex clinical trial requirements, can discourage investment in orphan drug development. Consequently, many promising treatments never make it to market, leaving patients with few options.

Regulatory Framework

Singapore currently lacks orphan drug-specific registration guidance. However, under the Medicines (Orphan Drugs) (Exemption) Order, importers can bring in and supply these drugs without a product licence, provided that a doctor or dentist prescribes them for a patient under their care. These drugs must be stored in a hospital and managed by a custodian, who must be a physician, dentist, or pharmacist appointed by the hospital. Doctors or dentists needing these drugs for treatment must request them from the custodian. Additionally, while drug developers often benefit from expedited approval processes and incentives for the development and registration of pharmaceutical products, there are currently no regulatory incentives for these drugs in Singapore.

Cost and Affordability

One of the significant barriers to accessing orphan drugs is their high cost. Because these drugs are developed for small patient populations, the development expenses are spread across fewer individuals, leading to higher prices. In Singapore, like in many other countries, this places a financial burden on both patients and the healthcare system. To help address this, the Ministry of Health and SingHealth Fund established the Rare Disease Fund (RDF), a charitable fund that combines community donations with government matching contributions to support Singaporeans with rare diseases who need expensive treatments. However, the funding currently covers eight medications for five conditions.

Patient Advocacy and Support

Patient advocacy groups play a vital role in improving access to the drugs. These organisations work tirelessly to raise awareness, support affected families, and push for policy changes. In Singapore, patient groups are instrumental in bringing attention to the needs of those with rare diseases and advocating for regulatory and financial reforms. Their efforts help create a more supportive environment for accessing treatments and drive meaningful changes in policy and practice.

The challenges of accessing orphan drugs and treating rare diseases in Singapore are part of a broader global issue involving access and affordability. Overcoming the abovementioned hurdles requires collaboration among policymakers, healthcare providers, and patient advocates. For those seeking expert guidance on pharma regulatory services and market access, The Reg Consultants in Singapore provides valuable insights and support to help navigate these intricate processes. 

Get in touch with us today to discover how we can assist with your needs.

• Share This Article:

---