Special exceptions for orphan medicines to treat rare diseases

Orphan medicines, which are drugs that specifically treat rare diseases, can be categorised as either emergency or lifetime therapies. — AFP

For rare diseases, time can often be of the essence when it comes to treatment.

This is as many rare diseases can become fatal if not treated quickly.

The progression of symptoms can also occur quickly, resulting in increasing disability for the patient.

So what happens if a patient quickly needs a treatment that is not yet registered in Malaysia?

The National Pharmaceutical Regulatory Agency (NPRA), under the Health Ministry, is the body in charge of regulating and registering all pharmaceutical products, including traditional medicine and health supplements, as well as cosmetic products, in the country.

Health Ministry Pharmaceutical Services senior director Norhaliza A Halim, who oversees the NPRA, shares that there is indeed a mechanism to allow for such an unregistered treatment to be brought into Malaysia.

She says: “In a situation where a medicine is not available in Malaysia and is needed for the purpose of treatment of any person suffering from a life-threatening illness, whereby there are no other suitable options or alternative treatments available, the importation of an unregistered medicine can be applied for.

“In such cases, the healthcare facility treating the patient may submit an Import Permit application for the importation and use of an unregistered medicine.”

She notes that the Application for Medicines of Special Approval or Application to Import Product for the Treatment of Life-Threatening Illnesses provides a pathway to ensure continuous access to medicines needed for the treatment of patients in cases where there are no other treatment options available locally.

“The processing and evaluation of such applications will be expedited by the Pharmaceutical Services Programme in order to ensure the timely access to the medicine needed for the treatment of the patient’s condition,” she says.

Drugs for rare diseases are designated as orphan medicines.

They can be categorised as emergency treatment, which is required immediately to save the patient’s life or prevent permanent disability, or lifetime treatment, which is required for long-term or maintenance therapy for the disease.

However, just because a drug has been designated as an orphan medicine in another country, does not necessarily mean it will be automatically recognised as such in Malaysia.

Says Norhaliza: “The designation of orphan medicine is subjected to NPRA’s decision with input from the Drug Evaluation Committee (DEC).

“NPRA may seek advice from relevant experts, respective rare disease society or patient groups, or key opinion leaders, when deemed necessary.”

Another important point to note is that rare diseases also have a specific definition in Malaysia.

According to the Health Ministry, a rare disease is defined as “a life-threatening and/or chronically debilitating rare condition as listed in the Malaysian Rare Disease List”.

Inclusion in the list requires that:

• There are confirmed patients with the disease in the country.
• The disease affects fewer than one in 4,000 people in Malaysia.
• The disease is a severe condition.
• Approval is given by the National Rare Disease Committee.

There are over 500 diseases currently on the list, according to the Malaysian Orphan Medicines Guideline.

Norhaliza shares that any local, registered health- or pharmaceutical-related company or legal entity can apply for a drug to be designated as an orphan medicine through the NPRA.

“The application must be submitted before a product is registered as a New Chemical Entity or a Biologic product.

“The decision of the DEC to grant the designation of orphan medicine or otherwise will be made within 45 working days upon receipt of application.

“The Product Registration Holder (i.e. the company or legal entity making the application) is required to provide the product information, the proposed rare disease and condition, as well as the scientific rationale for the orphan medicine use when applying for the orphan medicine designation,” she says.

She adds that a drug that has been granted the designation of orphan medicine will be automatically granted priority review status for registration.

The review process will then be completed within 120 working days.

She also notes that the Product Registration Holder is responsible for post-marketing activities.

These include monitoring for any drug-related adverse effects, submitting regular safety reports and alerting the NPRA on any global safety issues relating to the drug.

Orphan medicines registered with the Drug Control Authority can be found on the NPRA website.

This article is part of the third package of a short series written as part of the US National Press Foundation’s 2022 Covering Rare Diseases: Journalism Fellowship & Global Reporting Grant. The next article will be published this Sunday (Jan 15, 2023) in StarHealth.