Regulatory Incentives for Development of Orphan Drugs

In recent years, there is a huge leap in the pharma industry towards rare disease research and orphan drug developments due to the great support from regulatory authorities such as faster approval timelines, supportive regulatory frameworks, financial incentives, and many more. Currently, large pharmaceutical companies like Novartis, GSK, Roche, and Pfizer are extremely active in the orphan drugs market. Asian country’s regulators have also initiated providing regulatory incentives for the development of orphan drugs.

To treat patients with rare diseases and to support the pharma industries that are working on orphan drugs, a supportive regulatory framework was established long back by the regulators in the countries like, USA (1983), Japan (1993), and Europe (2000).

Check out the first article in this series Rare Diseases: Regulatory Incentives for Development of Orphan Drugs – US & EU

Now, there is an increasing trend towards orphan drug development worldwide. Many other regulatory authorities are working on coming back with new frameworks to establish great support for orphan drug developments in rare diseases.

In the previous article series, we have discussed regulatory benefits in the US, Europe, Japan, and Australia, now in this article, we will be looking through the regulatory aspects and new regulatory changes in orphan drugs for rare diseases in the countries like China, India, Russia, and South Korea.

Check out the second article is this series Rare Diseases: Regulatory Incentives for Development of Orphan Drugs – Japan & Australia

Regulatory incentives for the development of orphan drugs in China

Rare disease definition in China

A disease is considered to be rare if the prevalence is <1 patient per 500,000 people or neonatal morbidity of < 1 patient per 10,000 people.

China is currently suffering from a shortage of treatments for rare diseases. Of the 44 globally available therapies for rare diseases, only 19 are currently available in China. These drugs are priced outreach to the average Chinese patient.

On May 22, 2018, the new NHC (National Health Commission), along with the CNDA (China National Drug Administration), and the three other agencies jointly published the first edition of the Rare Disease List (RDL). The RDL includes 121 diseases and is considered a huge milestone for regulation in rare diseases.

Regulatory benefits for orphan drugs in China

In China, there are no proper guidelines and regulations for rare diseases. However, there are some special considerations, such as,

Overseas data acceptance
- The state council issued an opinion that orphan drugs and drugs for unmet medical needs can be approved with overseas clinical data only if certain conditions are met, and that the CNDA shall review the application within 3 and 6 months, respectively. This not only adds a clear review timeline to the regulatory pathway but also promises to accelerate approval significantly.
- The main takeaway is that a rare disease drug can gain approval with overseas clinical data, regardless of whether the drug has been approved in other countries or not.
Tax benefits
- China has announced a plan to slash the Value Added Tax (VAT) on 21 rare disease therapies and four active pharmaceutical ingredients, dropping it by 80% in a bid to stimulate the domestic rare disease therapy market and drop the prices of the therapies.

Criteria for orphan drug designation in China

The disease has a low prevalence or incidence in China.
There is a significant impact on the patient or family due to the disease.
There is a clear method of diagnosis.
The disease is treatable, intervention is feasible and economically accessible.
There is no currently available treatment, but the disease is investigated under a government-funded research program.

Regulatory incentives for the development of orphan drugs in Russia

Rare disease definition in Russia

Diseases are considered to be rare diseases if they have a prevalence of not more than 10 cases per 100 thousand people. Russian Ministry of Healthcare based on statistical data maintains a list of rare diseases on its official website.

Regulatory benefits for orphan drugs in Russia

Provision for clinical trial data
- Clause 3.1 art. 26 of the law on drug circulation concerning orphan drugs specifies provisions to provide the results of clinical studies of orphan drugs performed outside of the Russian Federation (provided they are following standards similar to Russian good laboratory practice and good clinical practice)
The accelerated procedure of examination
- Examination of documents in the registration dossiers, quality control, and expertise of benefit-risk balance for state registration of orphan drugs is carried out by the decision of the Russian Ministry of Healthcare.
- The Russian Ministry of Healthcare, according to the applicant’s application, examines the documents within a period not exceeding 80 business days (whereas in general, the examination takes 110 business days).

Read more about Rare Diseases: Clinical Trials & Drug Development

Regulatory incentives for the development of orphan drugs in India

Rare disease definition in India

In India, a drug intended to treat a condition that affects not more than five lakh (500,000) persons are considered an orphan drug.

Regulatory benefits for orphan drugs in India

India has started revising its orphan drug policy to benefit patients with rare diseases. Provisions made for clinical development for rare diseases in New Drugs and Clinical Trials Rules, 2019. are

Regulatory
- Fastrack approval process and special status for orphan drugs, including a complete fee waiver for filing clinical trial.
- Provision for expeditious review process even if the drug has not completed all clinical trial phases, the sponsor or applicant may apply to the Central Licensing Authority (CLA) for expedited review.
- Waiver of local clinical study and Phase IV on the satisfaction of the Central Licensing Authority (CLA).
Financial Benefits
- No application fee is charged for conducting clinical trials for orphan drugs.

Regulatory incentives for the development of orphan drugs in South Korea

Rare disease definition in South Korea

The term rare disease applies to diseases for which there are fewer than 20,000 patients and for which no appropriate treatment or substitute treatment modality is available in South Korea.

Regulatory benefits for orphan drugs in South Korea

There is a gradual increase in awareness of rare diseases. A support program for research in rare disease clinical trials was initiated by the government for patients with rare diseases in 2001.

The Ministry of Food and Drug Safety (MFDS) announced that it would amend regulations to allow orphan drug designation on approved clinical trials.

The amendments include
- Expand the scope of applicants for orphan drug designation.
- Improve disclosure methods by changing the designation procedure of orphans drugs.
- Simplify the procedure for orphan drugs.

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Way forward

Utilize these incentives to your advantage in the development of medicines for rare diseases, or orphan drugs, as they are commonly known.

Interested in knowing more details about these benefits from other countries?

Find out in the next articles in this series about incentives and support mechanisms for rare diseases in

Rare Diseases: Regulatory Incentives for Development of Orphan Drugs – Asia