Orphan Drugs & Rare Diseases
Nishimura & Asahi / Japan
The ins and outs of orphan drugs & rare diseases in Japan. Prepared in association with Nishimura & Asahi, a leading law firm in Japan, this is an extract from The Pharma Legal Handbook: Japan, available to purchase here for USD 99.
1. What is the definition of Rare Diseases in your country?
In Japan, there is no fixed definition for Rare Diseases, although the concept is included in the definition of Orphan Drugs (refer to Q2). It should be noted that our country has a system to deal with diseases designated as “Intractable Diseases,” the definition of which is provided in the Act on Medical Care for Intractable Disease Patient (the “Intractable Disease Act”), but the range and definition of Rare Diseases does not necessarily correspond to those of Intractable Diseases.
2. Does the designation of ‘Orphan Drug’ exist in your country? (Does it correspond with the definition of Rare Diseases?)
An “Orphan Drug” is defined as pharmaceuticals designated as such by the Minister of Health, Labour, and Welfare (“MHLW”) under the Act on Securing Quality, Efficacy, and Safety of Products Including Pharmaceuticals and Medical Devices. More specifically, MHLW can designate a pharmaceutical as an Orphan Drug when all of the following requirements are satisfied: (i) the number of patients who need the drug is less than 50,000; (ii) there is no alternative drug and the drug has particularly excellent efficacy or safety; and (iii) the manufacturers have a possible and reasonable development plan for the drug. With regard to requirement (i), as an exception, if a disease falls under a category provided in the Intractable Disease Act, the number of the patients may defer to the provision of the said Act.
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?)
Designation of an Orphan Drug does not immediately lead to approval (authorization) of the manufacture and sale of the drug. The process of Orphan Drug authorization is essentially the same as that of ordinary drugs. However, a certain priority treatment is given in the approval review of an Orphan Drug for the purpose of expediting its development. More precisely, if a drug is designated as an Orphan Drug, its approval review is prioritized (in terms of time) over an examination or investigation into other pharmaceuticals. The Japanese regulatory framework does not consider an Orphan Drug’s foreign status.
4. Does your country have provisions for relaxed clinical trial/scientific evidence requirements in respect of Orphan Drugs as compared to other drugs?
No. Currently, Japan does not have any provisions for relaxed clinical trial/scientific evidence requirements in respect of Orphan Drugs as compared to other drugs.
5. Is there an expedited pathway for Orphan Drugs?
As stated in Q2, if a drug is designated as an Orphan Drug, a priority review process is applicable for the approval (authorization) of the manufacture and sale of the drug. To clarify, however, there is no expedited pathway for the designation of Orphan Drugs.
6. Are foreign marketing authorizations recognized in your jurisdiction for Orphan Drugs? If yes, marketing authorizations from which countries are recognized?
No foreign marketing authorizations are recognized for Orphan Drugs in Japan.
7. Can Orphan Drugs be reimbursed? If so, is there a specific reimbursement procedure for Orphan drugs?
Yes, Orphan Drugs are reimbursed under the National Health Insurance. However, there is no special reimbursement procedure for Orphan Drugs.
8. How are the prices of Orphan Drugs regulated?
In Japan, drugs are typically expected to be used under the auspices of the national health insurance system. The Japanese government determines the price of medicines by using a model prescribed under the national health insurance pricing system. The prices of Orphan Drugs are regulated in the same manner as other drugs. However, under the pricing model, Orphan Drugs will normally get additional price. In addition, Orphan Drugs receive special treatment regarding price decisions, under which the prices of Orphan Drugs are exempted from periodical price cuts for a certain period of time.
9. In case of reference price based on a basket of countries, what countries are included?
Upon determining the price of a new drug which has already been made available for sale in countries other than Japan, the government adjusts the price so that there is no significant price difference between Japan and certain other countries. The countries used for comparison are the United States, the United Kingdom, Germany, and France.
10. Have there been any significant legal/judicial developments in relation to Orphan Drugs in your country?
No, there have not been any recent significant legal/judicial developments relating to Orphan Drugs in Japan.
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?
No. There is currently no noticeable movement for reform or significant change to the regulation of Orphan Drugs.
