MS studies new paths to improve the treatment of rare items

Rare things (DR) do not have this name just in case. According to the World Health Organization (WHO), the definition is that it affects 65 people in every 100 thousand inhabitants, in other words, just one case for every two thousand individuals. But even though these are rare items, it is not difficult to find people who love them. Para se ter uma ideia, no mundo, 400 milhões de pessoas possuem some six a oito mil doenças catalogadas hoje. Only in Brazil, the number is around 13 million inhabitants with some DR. It is estimated that 80% of them derives from genetic factors, while the others correspond to environmental, infectious, immunological causes, among others.

In truth, back in the 80s, the rare doenças are not part of the government discussions. Meanwhile, since the beginning of 2000, the issue has been widely discussed among technicians, including genetic issues.
The Ministério da Saúde (MS), by means of portal n° 199 of January 2014, establishes guidelines for comprehensive care of people with rare cases in the Único de Saúde System (SUS). The document states that “(…) user assistance must be focused on their field of needs, seen in a broad way. Not that it refers to specialized care in rare items, it will be the purpose of Specialized Care Services and Reference Services in Rare Items as complementary structuring components to the Health Care Network.”. Even though there is care and treatment for patients with DR offered in the public network, there is still a demand for a specific official policy for these cases.
On June 30, 2016, health professionals and people linked to the government will meet in the Federal Senate for the VIII National Health Policy Forum in Brazil – Rare Doenças. The event contributed to expanding integrated action proposals for diagnosis and treatment of patients with rare diseases. Rodrigo Silvestre, director of the Department of Industrial Complex and Innovation in Health DECIIS/SCTIE of MS, emphasized during the opening that “speech excites, but for the patient it is not useful. We are focused on developing concrete proposals and actions aimed at the patient.”.
Além disso, director of the Department of Management and Incorporation of Technologies in Health/SCTIE/MS, doctor Clarice Petramale, explained that some factors need to be tightened to improve the treatment policy for patients with rare diseases, such as the granting of registration and fixing of prices together with the National Health Surveillance Agency (ANVISA) and elaboration of clinical investigations that can verify the effectiveness of the treatment.
Meanwhile, the medical team that the National Commission for the Incorporation of Technology in SUS (CONITEC) is working incisively in the implementation of new policies that meet the demands of DR patients, both in the incorporation, exclusion or alteration of technologies in health, bem as in the constitution or alteration of Clinical Protocols and Therapeutic Directives – PCDT. “We already have more than 400 endorsed claims and, in fact, approximately 60% have been incorporated in some way in SUS. It is also important that people process analysis requests so that we can verify the possibility of implementation on your behalf.”, he explains.
In accordance with current legislation, CONITEC sets a period of 180 days (extended for more than 90 days) to make a decision regarding requests for analysis of new technologies, “which included an analysis based on evidence, taking into consideration aspects such as efficiency, accuracy, effectiveness and security of technology, as well as comparative economic evaluation of the benefits and costs in relation to already existing technologies. The law also establishes the requirement of prior registration of the product at the National Health Surveillance Agency – ANVISA so that it can be validated for incorporation in SUS.”.
NEW RUMOS – It has been a year since the beginning of the development of 47 Clinical Protocols and Therapeutic Directives (PCDT) that aim to reduce mortality and improve the quality of life of patients with DR and two relatives. At the end of 2015, 12 protocols of rare doenças organized by the Ministry of Health were published. The publication of the other directors is expected to occur until 2018.
The expected investment for the policy is approximately R$ 130 million. The two procedures for diagnosing rare cases are carried out by the Fundo de Ações Estratégicas e Compensação (FAEC) and reviewed by the States, Federal District and Municipalities.
For items that do not yet have their own protocols, assistance and care for people with rare items will continue to follow the guidelines established by the National Policy for the Care of People with Rare Items in SUS.
Check the eixos/groups of teachings that have protocols for 2018 by clicking here.
EXPERIENCE - Three and a half years ago, after feeling a lot of pain in the back of the neck, pain and no signs of sensitivity on the direct side of the body, Grazielle Alves, 36 years old, who lives in Belo Horizonte, discovered that she has syringomyelia. Basically, this happens due to a tube-shaped cavity inside the spinal cord that fills with fluid and harms the spinal cord. Among the consequences of the disease are intestinal and abdominal problems, and paralysis of both arms and legs.
“Chorei muito when I discovered what he had. It is not easy to hear that you have a rare gift that does not have a cure. I was strong to oil or that it needs to be oiled to surgery”he explains. Apart from the difficulties inherent in possessing a rare condition, Grazielle tells that there are people who do not accredit our symptoms. “Falam that the body is mole, preguiça and freshness. Some people lost friends", he laments.
Currently, mine moderates a group on Facebook along with other people who can do and share their needs. “The objective is to protect the people who discover the truth and explain why the people simply do not know”, he says.
The Health Ministry does not have a specific Clinical Protocol and Therapeutic Directive (PCDT) for syringomyelia, but in these cases, patients are advised to seek Basic Health Care, in any of the Basic Health Units (UBS), and if necessary , a specialized service in medical and high complexity units, such as High Complexity Assistance Units in Neurosurgery and two Reference Centers in Neurology.
Grazielle has been regularly using two medications withdrawn through SUS for approximately one year and a month. In general, the Single Health System (SUS) makes available treatments for symptoms, through the endorsement of multidisciplinary teams. Among the procedures available are physiotherapy and the offer of medications, orthoses, prostheses and special materials. Additionally, one of three surgeries intended to drain fluid from the bone marrow was also offered for SUS.
Aline Czezacki, for the Health Blog