• The Health Insurance Fund pays for 22 “orphan drugs”

    July 13, 2015

    During the past 2014 the NHIF allocated 72 million for rare medicines

    The innovations are crucial for achieving better treatment, but they are very expensive. Good examples of this are the so called “orphan drugs”, which treat rare diseases. Treatment of a patient for a year could exceed millions. Because of that the access is problematic for every country. The analysis of the Center for Health Technology Assessment and Analysis and the Institute for Rare Diseases shows – where are we in comparison with EU countries.

    The ensuring of access for patients to “orphans drugs” across the EU is different. Although the drugs have Marketing Authorization through the Centralized procedure (simultaneously for each country), the payment from public funds is at a different time. According to the data in the analysis of the Centre for Health Technology Assessment and Analysis and the Institute for Rare Diseases the average delay in the countries is 29.1 to 43 months from the receipt of the Marketing Authorization to funding. The analysis examines all 36 diseases that are covered by the NHIF. Among them are congenital coagulopathies (disturbances in blood clotting), beta thalassemia, idiopathic thrombocytopenic purpura, non familial hipogama-globulinaemia, selective deficit of subclasses of immunoglobulin G, severe combined immunodeficiency with low or normal content of B cells, common variable immunodeficiency, etc.

    The delay in Bulgaria for the reimbursement of “orphan drugs” is within a few years. The delay in access to medication for mucopolysaccharidosis II for example was 5 years, as was the reimbursement for another medicine for phenylketonuria. The delay in access to “orphan drug” for hereditary amyloid polyneuropathy was 2 years, while to “orphan drug” for primary pulmonary hypertension – 3 years. “This delay represents a significant obstacle to timely and adequate treatment of patients with rare diseases,” says the analysis.

    In the middle of last year registered in the EU “orphan drugs” were 72. Of them in Bulgaria the fund and the hospitals pay only 22. The remaining 50 are not funded with public money and in practice remain inaccessible to patients. For comparison, in other member states on average about 80% of the approved on European level “orphan drugs” are included in the health insurance system. Although many drugs remained inaccessible to the sick people in our country, our country has made great progress in recent years in providing access to rare drugs to patients, says the analysis. The costs of the NHIF for such medicines were increase from BGN 28 million in 2011 to over BGN 72 million in the last. The average cost of treatment per patient per month was about BGN 2 thousand

    Although some European countries cover more drugs for patients with rare diseases, they also face many challenges. The establishment of the clinical efficacy of these drugs is number one priority, because the evidence is often insufficient from the standpoint of the health authorities. This is due to the limited number of patients who use them, and the required data can not be accumulated quickly. Furthermore, it is difficult to find for comparison a competitive conventional therapy already covered by public funds and available to patients. These problems, combined with the extremely high cost of most of the treatments for rare diseases, raise the question of finding a partnership between the government and the industry in the financing of the drugs. The mechanisms for the management and sharing of financial risks are many. In Belgium, for example, public expenditure on drugs has a limit. 2/3 of the over-expenditure should be covered by the pharmaceutical industry and the rest by the National Health Insurance Institute. In Australia, as early as 2004, the state agreed to reimburse the medicine provided that a registry of patients with primary pulmonary hypertension will be created and the future price will be consistent with the results. In practice the data on the clinical efficacy of treatment locally is required by the authorities in most countries. This is the main reason for the peculiar boom of the registers for rare diseases in Europe. According to the European portal Orphanet the number of registries for rare diseases in the Member States is 641 as of 2014. In this respect Bulgaria was a kind of best performer in Eastern Europe, says the analysis. However, this is clearly not enough. According to the specialists a better control is needed over funding, reallocation of resources according to the effects of treatment and provision of monitoring network for patients with rare diseases.

    There are 8000 rare diseases

    A disease is considered rare when it affects less than 5 persons in 10 000 in the EU. According to scientists there are between 5000 and 8000 such diagnoses. They will affect between 29 and 32 million people in the EU, of which 400 000 in Bulgaria.

    They are mainly against cancer

    More than 72 “orphan drugs” are discovered so far. Only for the last two and a half years 27 new drugs have been approved for marketing, 9 of which in the first half of 2014. Most of the designated drugs are intended for cancer. Lysosomal storage disorder and primary pulmonary hypertension are emerging as the next “most preferred” areas of the industry for development of new drugs.