New Regulation for Rare or Infrequent Serious Diseases

24 Jun 2013

ANMAT Provision N°4622/20121

ANMAT 2 understood the need to establish in the country a scientifically supported standard operating procedure for the applications that will be filed for the registration of drug products or medicines for the prevention, diagnosis and treatment of rare or serious diseases.
Diseases with a high prevalence within the population have always had a significant investment in research and development for a variety of drugs. A quite different situation can be found for the diseases with low prevalence within the population or the so called “rare diseases” that cause serious consequences for patients and their families, for which there are only few safe and effective treatments available.
According to Provision 4622/2012, ANMAT establishes a procedure for the registration of drug products and medicines developed for the prevention, diagnosis and treatment of rare or serious diseases that will be assigned to the category of products authorized “under special conditions”. This Provision also creates the Commission that will be Responsible for the Assignment and Evaluation of drug products and medicines to be registered “under special conditions”.
The main objectives and responsibilities of this Commission are the analysis of the information and documentation provided for a drug that is submitted for its registration and inclusion in the REM3 as a drug product approved “under special conditions”; the assignment of the registration category of “drug under special conditions”; the analysis of the information and documentation that will support the application of a drug product “under special conditions”; and finally the evaluation of the opportunity, merit and suitability of the inclusion in the REM of a drug “under special conditions”.
The characteristics of the diseases considered in this Provision are mentioned in Annex I, which are the following:

  • Rare diseases (or EPF in Spanish for Enfermedades Poco Frecuentes) defined as those whose prevalence in the population is equal to or less than one in two thousand (1 in 2000) people, referring to the national epidemiological situation.
  • Serious and life threatening illnesses as well as serious disabilities, among which are the following:
    1. Chronic and/or debilitating diseases for which there are no effective or only very inadequate treatments available.
    2. Severe illnesses or life threatening diseases for which there are no effective or only very inadequate treatments available.
    3. The appearance of a broad resistance (refractory) during the treatment of a disease with agents or drugs currently available
    4. The outbreak (occurrence) of a new disease with severe effects or a new life threatening disease for which no effective or only very inadequate treatments are currently available.

Annex II makes reference to the Requirements and Documentation that have to be included in the application of an “Under Special Conditions” drug product registration file, such as:

  • Information on early stages of research: Preclinical and Phase I, Phase II of clinical pharmacology studies and the results of clinical trials.
  • Submission of a credible and reliable evidence that the product that will be submitted to registration has been categorized as an “orphan drug”, or as a drug that is intended for the diagnosis, prevention and treatment of rare or serious disease for which no effective and safe treatment is currently available.
  • Monitoring Plan for the determination of the efficacy, effectiveness and safety of the drug in order to evaluate the benefit and risk of the treatment. The Department of Medicines of ANMAT must approve the Plan, which shall include the recording of the patients treated as well as the prescription and dispensing procedure that will be followed in the country.
  • A Guide for drug product administration for health professionals, patients or patient assistants.

Within the current Provision, ANMAT makes also reference to further issues that shall be required when evaluating drug products or medicines that apply for the registration as “Under Special Conditions”, as the following:

  • The possibility of including the medicine or drug product in an Intensive Sanitary Surveillance Plan, depending on the type of drug.
  • To provide Periodic Safety Update Reports that could be required as an annual Update or according to a frequency that the authority will determine in each case.
  • It also mentions that it will be necessary to obtain a written informed consent of the patient or his/her legal representative with regards to the benefits and risks of the treatment involved.

Article 4 establishes that the holders of marketing authorizations of drug products and medicines covered by this provision must include the legend “AUTHORIZED UNDER SPECIAL CONDITIONS” on the labels, labeling, patient and physician leaflets, brochures and on whichever information released to the professional body, with the same size and enhancement as the brand name, the non-proprietary name of Argentina (DCA) or the International Nonproprietary Name (INN).
Concluding, Provision 4622/2012 became effective on September 10th, 2012 and from that date, the approval that will be granted as well as the time a certificate of a drug product authorized “under special conditions” will be valid, will be considered by ANMAT on a case by case evaluation. For doing so, the health authority will take into consideration, the particular characteristics of the drug involved, the complexity of the disease being treated and the information submitted according to research and development phases.
The new registration process has to be monitored during some time in order to provide additional comments on the pros and cons of this new regulation.

1 Author: Marta Pentz, Head of Regulatory Affairs Department
2 ANMAT: National Drugs, Food and Medical Technology Administration
3 REM: Repository of Medicinal Products of ANMAT

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