Want to know more about orphan drugs & rare diseases in India? Read on! Prepared in association with Nishith Desai & Associates, a leading law firm in India, this is an extract from The Pharma Legal Handbook: India, available to purchase here for USD 99.

1. What is the definition of Rare Diseases in your country?

India does not have a definition of rare diseases. The National Policy on Treatment of Rare Diseases 2017 (“NPTRD 2017”), which is currently in abeyance, highlighted the necessity of coming up with a definition of rare diseases that fits India’s needs but did not provide one.

It may be noted that according to representations made by the Ministry of Health and Family Welfare (“Health Ministry”) before the Delhi High Court in the case of Mohd. Kalim v. Employees State Insurance Corporation & Ors. (W.P.(C) 8445/2014) (“Mohd. Kalim Case”), the NPTRD 2017 was held in abeyance as the policy was framed without the concurrence of most states in India. The NPTRD 2017 allocated work to the Public Health Division specific to each state without obtaining the consent of such state. Since then, the Health Ministry has reportedly transferred the work of rare diseases of the National Health Division set up under the National Health Mission (“NHM”).

2. Does the designation of ‘Orphan Drug’ exist in your country? (Does it correspond with the definition of Rare Diseases?)

Yes. Orphan Drugs have been defined in the New Drugs & Clinical Trial Rules 2019 (“New Drugs & CT Rules”) as a drug “intended to treat a condition which affects not more than five lakh (500,000) persons in India”. There is no definition of rare diseases in India.

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?)

Clinical Trials

The regulatory framework for the clinical trial of an Orphan Drug is similar to the clinical trial process of other drugs apart from a few relaxations that are granted to Orphan Drugs. The relaxations are as follows:

1. The Central Drugs Control Standards Organization (“CDSCO”), India’s apex drug regulatory agency, can dispense with the requirement of conducting local clinical trials in the case of Orphan Drugs.
2. The sponsor of a clinical trial of an orphan drug can request the CDSCO to expedite the review process for approval of such Orphan Drug.
3. No application fee is required to be paid for the conduct of a clinical trial of an Orphan Drug.

Authorization

The entity seeking to market any new drug in India, including an orphan drug, must obtain authorization from the CDSCO to import or manufacture such drug. A new drug is a drug whose safety and therapeutic efficacy has not been tested on the Indian population.

4. Does your country have provisions for relaxed clinical trial/scientific evidence requirements in respect of Orphan Drugs as compared to other drugs?

Please see response to Question 3.

5. Is there an expedited pathway for Orphan Drugs?

Yes, there is an expedited review process for approval of Orphan Drugs after clinical development.

6. Are foreign marketing authorizations recognized in your jurisdiction for Orphan Drugs? If yes, marketing authorizations from which countries are recognized?

India recognizes foreign marketing authorizations of new drugs (including orphan drugs) from countries recognized by the CDSCO (under the New Drugs and Clinical Trial Rules, 2019 (“New Drugs & CT Rules”)). The CDSCO is yet to notify such countries under the New Drugs & CT Rules. In the event the CDSCO recognizes marketing authorizers from a particular country, the drug will not be required to undergo local clinical trials for obtaining marketing authorization. However, unlike other drugs, requirements for post-marketing surveillance for Orphan Drugs may be relaxed at the discretion of the CDSCO.

7. Can Orphan Drugs be reimbursed? If so, is there a specific reimbursement procedure for Orphan drugs?

India has a financial assistance program in place for treatment of rare diseases.

The Union Health Ministry as well as the various state health ministries have set up technical committees known as the Central Technical Committee (“CTC”) and the State Technical Committee (“STC”) respectively, to review applications by patients requesting financial assistance for treatment of rare diseases. The relevant patient or caregiver can apply to the STC or the CTC directly to request assistance. It may be noted that the CTC will convey its decision only to the relevant STC. In the event the CTC considers the case favorably, the relevant State can claim 60% of the approved cost of treatment from the CTC.

8. How are the prices of Orphan Drugs regulated?

Orphan Drugs, as identified by the Health Ministry from to time, are exempt from price regulation.

9. In case of reference price based on a basket of countries, what countries are included?

India does not follow a system requiring a reference price based on a basket of countries.

10. Have there been any significant legal/judicial developments in relation to Orphan Drugs in your country?

The constitutional obligation of the state to ensure access to life saving drugs has been formally recognized by the Delhi High Court in Mohd. Ahmed (Minor) v. Union of India & Ors W.P.(C) No. 7279 of 2013 (“Mohd. Ahmed Case”). In the Mohd. Ahmed Case, the Delhi High Court directed the Delhi Government to provide the Mohd. Ahmed (minor patient in question) with enzyme replacement therapy worth INR 600,000 (USD 8,334) per month as a treatment to gaucher. The petitioner was represented through his father who was a rickshaw-puller and could not afford the exorbitant cost of treating his minor child Mohd. Ahmed. Therefore, the parents of the minor patient had approached the Delhi High Court to request the Court to direct the Delhi Government to offer financial assistance with respect to the treatment of the rare disease.

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?

The Delhi High Court in the Mohd. Kalim case had directed the Health Ministry to frame a new National Policy for Treatment of Rare Diseases, following the discontinuation of the NPTRD 2017. Moreover, the Delhi High Court has also directed the Health Ministry to disburse funds to patients from the INR 1 billion fund that was created under the NPTRD 2017 for the treatment of rare diseases.