Rare diseases are also called. Orphans? According to the definition of the World Health Organization (WHO), rare diseases account for 0.65% of the total population. ~ 1? Illness.
There may be several rare diseases, such as albinism, phenylketonuria and mitochondrial diseases. Everyone has heard of this, but most rare diseases, even I have never heard of them, and the number of cases is relatively small, but the cumulative number of patients with these diseases must be 20 million+.
For example, albinism is a genetic disease characterized by the lack of pigments in skin, eyes and hair due to mutations in different genes, and there is no effective treatment at present.
Until now, most rare diseases are incurable, and patients can only die in pain. Only a few rare diseases have drugs available, but they need long-term or even lifelong medication.
In the past, there was no medicine for rare diseases, which once made patients see no hope. It's unfair to them.
The biggest difficulty faced by patients with rare diseases is the accessibility of therapeutic drugs. According to statistics, only 5% of rare diseases can be cured, and these 5% people are fortunate in misfortune. Whether they can afford it or not, at least there is a cure, but what about the remaining 95%?
Therefore, the difficulties faced by patients with rare diseases should first ask if there is any medicine, and then we can talk about whether they can afford it.
One: pharmaceutical companies are enthusiastic and have no motivation. Compared with other diseases, it is more difficult to develop drugs for rare diseases and there are fewer patients. It is more difficult to determine the safety and effectiveness of drugs. Few companies want to be? Living Buddha? After hard work, research and development, and a series of quality control, it was successfully listed, but few patients used it, and even fewer patients could afford it. Everything sold in a year can't recover all the expenses of that year, let alone the profits invested in the previous period. Therefore, in the absence of considerable benefits, enterprises are usually reluctant to invest huge costs in the research and development and production of new drugs for rare diseases.
2. How to alleviate the problem of unaffordability? For the innovation of drug research and development, the State Administration of Pharmaceutical Products has issued a number of documents, which clearly formulated some incentive measures for drug research and development of rare diseases, including giving priority to review and approval, reducing or exempting clinical trials, indicating that it may have clinical value, giving priority to conditional approval for listing, and so on.
Secondly, speed up the pace of introduction, and introduce some rare disease drugs that are available overseas and not at home as soon as possible. At present, there are a number of rare disease drugs that are urgently needed in clinic and accept overseas clinical data.
For drugs with inflated prices in China, it is necessary to intensify centralized drug procurement, reduce inflated prices in intermediate links and gray areas, and make concessions. Do you have any medicine? Become? Can you afford medicine? . On the one hand, it reduces the drug price, on the other hand, it can let more rare drugs enter the medical insurance and let more patients with rare diseases use the rest assured drugs.
Doctor of medicine, licensed pharmacist, senior nutritionist, protect life and health, refuse fake health preservation, and personally care for a healthy you!