Orphan Drugs and Rare Diseases
An intro to the legal situation for orphan drugs and rare diseases in Thailand. Prepared in association with Tilleke & Gibbins, a leading law firm in Thailand, this is an extract from The Pharma Legal Handbook: Thailand, available to purchase here for USD 99.
1. What is the definition of Rare Diseases in your country?
Neither the Ministry of Public Health in Thailand (MoPH) nor the Thai Food and Drug Administration (Thai FDA) has prescribed a definition for “rare diseases.” However, the Unit of Clinical Genetics, Department of Pediatrics, Faculty of Medicine at Mahidol University, an autonomous research institution and one of the most prestigious universities in Thailand, has provided a definition of rare or orphan diseases as “any disease that has a disease prevalence of less than one in 2,500 people, wherein there are limited drugs available for treatment of such disease.”
2. Does the designation of ‘Orphan Drug’ exist in your country? (Does it correspond with the definition of Rare Diseases?)
Yes, the Thai FDA has defined an “orphan drug” as:
a) A drug needed for diagnosis, alleviation, treatment, prevention, or cure of:
- a rare disease,
- a seriously harmful disease, or
- a disease resulting in continuing disabilities.
b) A drug with a low usage rate, wherein there is no other drug that can be utilized as a replacement, and there is also a shortage issue.
3. What is the regulatory framework for the authorization of an Orphan Drug? (Is this regulatory framework based on Rare Disease status or can it alternatively be based on Orphan Drug foreign status?)
Unlike the orphan drug status in other countries, the regulatory framework for orphan drugs in Thailand is based on both the status of rare diseases and drug shortage issues.
In 2013, the FDA published guidelines (in Thai language) regarding the registration of orphan drugs. Through these guidelines it was determined that the Subcommittee of Orphan Drugs, under the supervision of the National Drug Committee and coordinated by the Bureau of Drug Control of the Thai FDA, would be responsible for (a) providing and updating the list of orphan drugs, (b) creating thorough guidelines for orphan drug registration, and (c) developing a mechanism for increasing the accessibility of orphan drugs.
As provided in the guidelines, the Thai FDA announced a list of approved orphan drugs through the Notification Re: List of Orphan Drugs 2016, which includes the 70 orphan drugs that have been authorized for use in Thailand.
4. Does your country have pro- visions for relaxed clinical trial/ scientific evidence requirements in respect of Orphan Drugs as compared to other drugs?
Yes. The Thai FDA has exempted some requirements for the evaluation of orphan drugs, particularly if the orphan drugs have had extensive usage in other countries or if they have been previously marketed in other countries. In such cases, the Thai FDA would allow the registration of the orphan drugs without the submission of preclinical or clinical modules.
5. Is there an expedited pathway for Orphan Drugs?
Yes. There is an expedited pathway for registration of orphan drugs in Thailand. An applicant can submit, together with a registration dossier, a cover letter requesting the Thai FDA to apply an expedited pathway for the registration of the orphan drug.
6. Are foreign marketing authorizations recognized in your jurisdiction for Orphan Drugs? If yes, marketing authorizations from which countries are recognized?
Yes. The Thai FDA allows an abridged evaluation, wherein the marketing authorizations from benchmark countries such as Australia, Canada, the EU, Japan, and the U.S. are well accepted.
7. Can Orphan Drugs be reimbursed? If so, is there a specific reimbursement procedure for Orphan Drugs?
Orphan drugs can be reimbursed in Thailand, but the reimbursement scheme for each individual patient will determine which drugs they can receive reimbursement for. Currently, there are three main reimbursement schemes related to the healthcare system in Thailand.
- Civil Servant Medical Benefit Scheme (CSMBS): a scheme administered by the Comptroller’s Department of the Ministry of Finance that covers government officials and their dependents (spouse, parents, and up to three children).
- Social Security Scheme (SSS): a scheme administered by the Social Security Office, Ministry of Labor, that is applied to non-government workers and employees.
- National Health Insurance or Universal Coverage Scheme (UC): a scheme administered by the National Health Security Office (NHSO), Ministry of Public Health, that covers the remaining population not covered under the CSMBS or the SSS.
Patients under the CSMBS are eligible for reimbursement of almost all drug items, including orphan drugs. Patients under the SSS or the UC are also eligible for drug reimbursement; however, fewer orphan drugs are covered under these schemes. For all available schemes, there is no specific procedure in order to receive reimbursement for orphan drugs.
8. How are the prices of Orphan Drugs regulated?
Prices of drugs, including orphan drugs, are regulated when they are listed in a Notification from the National Drug System Development Committee, in which the “maximum allowable purchase price” will be prescribed for each drug. If a specific drug, including an orphan drug, is listed in said Notification, a public hospital can purchase the drug if the price is less than or equal to the maximum allowable purchase price.
9. In case of reference price based on a basket of countries, what countries are included?
The prices of orphan drugs in Thailand are not based on a basket of countries.
10. Have there been any significant legal/judicial developments in relation to Orphan Drugs in your country?
On April 17, 2019, the Council of Ministers of Thailand agreed to exempt the importation tax on orphan drugs. Currently, the Ministry of Finance is preparing a Notification in relation to this matter.
11. Are there proposals for reform or significant change to the regulation of Orphan Drugs? If yes, when are they likely to come into force?
As mentioned in our answer to question 10 in Regulatory Pricing and Reimbursement Overview, the Thai FDA has requested an exemption of the importation tax on orphan drugs—a significant initiative to ensure that the supply of orphan drugs is sufficient to meet the demands.
As it stands now, the Government Pharmaceutical Organization (GPO), Thai Red Cross, and Defense Pharmaceutical Factory (DPF) all play a major role in the manufacturing and importation of orphan drugs. However, the Thai FDA has issued a request to both Thai manufacturers and importers to join in the production and importation of orphan drugs to facilitate increased supply.