A wider debate on “rare diseases public policies” took place on July 30 at the Third Permanent Forum on Public Policies for persons with disabilities, coordinated by the Brazilian Ministry of Women, Family and Human Rights.
The purpose of this Forum was to identify the major challenges and needs of persons with rare diseases and hear suggestions and opinions on how to meet such challenges and needs, as a first step towards making public policies aimed at assisting persons with rare diseases.
According to the World Health Organization (WHO), any disease affecting fewer than 65 people in 100,000 is considered rare. More than twelve million people are estimated to suffer from any kind of rare disease in Brazil, which is worthy of attention.
In April this year, an important medication used in the treatment of Spinal Muscular Atrophy (SMA) – type 1 was added to the list of medications for which the universal healthcare program (SUS) provides coverage. The SMA type 1 is the most frequent form of SMA in Brazil. Patients can now get such a costly medication for free.
The Ministry of Health is also considering allowing for the use of the same medication in the treatment of other subtypes of the disease. In this case, however, coverage is given on a risk sharing basis, which means the Brazilian government will only pay for the medication if there is proof that patient condition has improved.
In Brazil, the regulation that deals with the approval of clinical trials and the registration of new drugs for rare diseases is the Plenary Board Resolution No. 205 (RDC 205) issued by Brazilian health surveillance agency (ANVISA) in 2017.
Some rules have been relaxed without compromising the quality, safety and efficiency of the drugs. By signing a Commitment Letter with ANVISA, the registration applicant is now allowed to submit additional data and proof after the registration is complete.
Under the RDC 205/2017, imported drugs are no longer required to undergo quality control inspection in Brazil, provided that: (i) such quality control is conducted by the drug manufacturer, and that (ii) evidence is produced proving that the drug was kept in the appropriate conditions during transportation.
ANVISA’s Plenary Board Resolution No. 204 (RDC 204), of December 27, 2017, in turn, provides the terms under which a request is considered a priority, lays down the priority criteria for the review of petitions for post-registration amendments – such as in case of new therapeutic indication or new purposes – with respect to rare disease drugs if no therapeutic alternative is available or when it shows a significant improvement in safety or efficiency.
With respect to health technology, the Brazilian patent and trademark office (INPI) gives priority to patent applications involving pharmaceutical products and processes as well as to equipment and/or material for use in the health industry for prophylaxis diagnosis and treatment of rare and neglected diseases.
