Orphan Drugs and Rare Diseases
Important legal info for orphan drugs and rare diseases in Mexican pharma. Prepared in association with Olivares, a leading global law firm, this is an extract from The Pharma Legal Handbook: Mexico, available to purchase here for GBP 99.
1. What is the definition of Rare Diseases in your country?
In accordance with the General health law, rare diseases are those that have a prevalence of not more than 5 people for every 10,000 inhabitants.
2. Does the designation of ‘Orphan Drug’ exist in your country? (Does it correspond with the definition of Rare Diseases?)
Yes, in Mexico there is a designation of Orphan Drugs contemplated in the General Health Law and these drugs are intended for prevention, diagnosis or treatment of rare diseases.
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?)
The regulatory framework for the authorization of an Orphan Drug are:
- General Health Law (Ley General de Salud).
- General Health Law Regulations for Healthcare Products (Reglamento de Insumos para la Salud).
- Official Mexicans Standards (NOMs).
- Mexican Pharmacopoeia;
Foreign marketing authorizations are not valid in Mexico.
4. Does your country have provisions for relaxed clinical trial/scientific evidence requirements in respect of Orphan Drugs as compared to other drugs?
Orphan drugs were introduced into the General Health Law and the Mexican Pharmacopeia some years ago. However, they follow the main principles and general rules as other drugs.
5. Is there an expedited pathway for Orphan Drugs?
Orphan drugs were introduced into the General Health Law and the Mexican Pharmacopeia some years ago. In practice, they are approved by a particular procedure, following some of the rules for new molecules when applicable and appropriate, yet it is not necessary to go through the New Molecules Committee as a requirement to submit an approval application. Despite that this procedure has worked reasonably well, specific rules would be welcomed.
6. Are foreign marketing authorizations recognized in your jurisdiction for Orphan Drugs? If yes, marketing authorizations from which countries are recognized?
Foreign marketing authorizations are not valid in Mexico. However, COFEPRIS has set a special procedure for drugs to be approved for the first time in Mexico, already approved by equivalent regulatory authorities abroad. In this procedure, the requirements for approval of these agencies are recognized as equivalent to those in Mexico. According to the equivalence agreement, marketing authorizations which have been approved by the following agencies:
- The European Medicines Agency;
- The US Drug and Food Administration;
- Health Canada;
- The Swiss Agency for Therapeutic Products (Swissmedic); and
- The Therapeutic Goods Administration in Australia.
Yet, concerning Orphan Drugs, there are no express provisions to consider as reference these foreign approvals, thus in these cases it would be under the authority’s discretion, to consider them in the evaluation process.
7. Can Orphan Drugs be reimbursed? If so, is there a specific reimbursement procedure for Orphan Drugs?
The primary legislation for the reimbursement of these kind of drugs, is the General Health Law (Ley General de Salud) (HL), its Regulations. These law and regulations are supplemented by Guidelines and Official Norms (NOMS) published by COFEPRIS.
In private sector, there is no reimbursement in Mexico. For the ISSSTE, a prescribed medicinal product can be dispensed in a private drug store registered with a public insurer, provided that this is not available within ISSSTE facilities and under certain conditions. The ISSSTE reimburses the cost of that product according to previous agreements.
8. How are the prices of Orphan Drugs regulated?
Price control in the private sector is based on a scheme of self-regulated maximum retail price (MRP) only covering patented products, overseen by the Ministry of Economy. Pharmaceutical companies’ participation is voluntary. Under the price control each product’s MRP must not exceed an international reference price, estimated as the average price in six major markets, plus a market factor. There are no established sanctions for violations of the MRP.
In the private sector, most payments are made on an out-of-pocket basis. Private insurers are improving the level of pharmaceutical coverage as the private market in medicines has grown considerably.
Public acquisitions are supported by the Committee for the Negotiation of Drug Prices (CNDP).
For direct purchasing of patented products, the CNDP analyses the effectiveness of the drugs and relevant therapeutic alternatives and the feasibility and implications of an eventual substitution with equivalent medicines. The CNDP also conducts an economic evaluation of the cost-effectiveness of patented medicines compared with potential substitutes.
For the ISSSTE, a prescribed medicinal product can be dispensed in a private drug store registered with a public insurer, provided that this is not available within ISSSTE facilities and under certain conditions. The ISSSTE reimburses the cost of that product according to previous agreements.
The political party currently governing in Mexico (MORENA) is promoting an amendment to the scheme of self-regulated maximum retail price (MRP), which consist, in general terms, that the Ministry of Economy in collaboration with the Ministry of Health shall guarantee, through a transparent process and taking into consideration differentiated policies, the access to medications and inputs to people in situations of poverty. In addition, the price control would be regulated and annually reviewed by these Authorities.
On August 11, 2020, the Mexican Congress published the Decree amending Article 1 of the Public Sector Procurement, Leasing and Services Law, through which the Mexican Government is now empowered to acquire medicines through intergovernmental organizations, such as UNOPS, without having to observe the procedures set forth in the applicable Law and Regulation on public procurement.
According to UNOPS’ guidelines, the process is made up of different stages grouped within three phases identified as:
- Pre-Bidding Process (market research, requirements definition and identification of potential suppliers).
- Bidding Process (publication of the bases and call for bids and/or invitations for negotiation, bid management, evaluation and award).
- Post-Bidding Process (contract management and logistics). The distribution to the final destination will be in charge of INSABI.
Therefore, during these proceedings UNOPS is entitled to negotiate the prices of the supplies.
9. In case of reference price based on a basket of countries, what countries are included?
As mention in answer 8, under the price control each product’s MRP must not exceed an international reference price, estimated as the average price in six major markets, plus a market factor.
10. Have there been any significant legal/judicial developments in relation to Orphan Drugs in your country?
In 2014, the Mexican Supreme Court of Justice ruled that the refusal to supply orphan drugs is not unconstitutional if they have not been approved, prior examination and analysis, by the inter-institutional commission of the so-called National formulary, which is the document containing all medicines, medical supplies, instruments used by the public health institutions in Mexico.
The Second Chamber of the Supreme Court issued a partial favorable decision in this case. First, such decision recognized the right to health is a constitutional right of primary importance. Yet secondary laws shape the access to health services. Therefore, the denial of providing basic health services, such as medical care for conditions that require “orphan” drugs for their treatment, directly affect the fundamental right to protection of health.
On the other hand, the Chamber considered it was necessary to follow the procedure to include the product in the National Formulary, as this is a mechanism through which the State guarantees that the drugs necessary to treat the diseases of the population are safe, efficient and effective.
Thus, the Supreme Court recognizes that the health institution that should be providing these to the patients can and should request the evaluation and analysis of the drug to be included in such list, in order to be able to provide the orphan drug.
In conclusion, the Supreme Court mainly stated that the applicable procedures should be observed in order to be able to supply the drug. Yet, the Second Chamber may have omitted to consider that it would take a while, which would eventually put in danger the health of the patients.
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 are no proposals to reform or significantly change the regulation. Nevertheless, specific rules would be welcomed.