Next year, the National Rare Disease Plan will be implemented. The document is to regulate, inter alia, issues related to the availability of orphan drugs – extremely expensive pharmaceuticals with a very narrow effect.
By the end of the year, we will adopt the National Rare Diseases Plan to start implementing it from 2017, said Deputy Minister of Health Krzysztof Łanda at a conference on rare diseases on Tuesday. He announced a different approach to the reimbursement of the so-called orphan drugs.
As the deputy minister added, a team is to be appointed at the health ministry college on Wednesday to prepare the plan.
«We plan to finish the works in the second half of the year. It is difficult to predict in which particular month, because I do not know yet what the scope of this work will be, but (…) we will do everything in our power to complete such a plan by the end of this year, adopted by the ministry for implementation already in 2017 »Said Wanda.
He added that the basis for creating the plan will be the work started by the previous management of the ministry.
Łanda informed that the team will include 7 experts and 4-5 representatives of various ministries and central offices. “I am a supporter of small teams because they ensure us efficient operation and speed of work progress” – he explained. He added that a framework document will be prepared first, and then thematic working groups will be appointed with a specified time to prepare recommendations for the minister of health.
The deputy minister emphasized that the axis of the plan will be the needs of patients with rare diseases.
He also announced a different approach to the reimbursement of the so-called orphan drugs (i.e. drugs used in the treatment of rare diseases). “The so-called egalitarian approach in the Reimbursement Act will apply to those technologies that are the first technology proven to be clinically effective in a given ultra rare indication,” said Wanda.
He added that it would definitely not be the case so far that orphan drugs had to compete on the same terms with drugs for common diseases. He explained that this is unfair, because if the manufacturer of a regular drug invests in research and development of the drug, then with widespread use, he has a quick profit. Meanwhile, he said, the producer of the so-called orphan drug, no chance of that. “Our propensity to accept higher prices must therefore certainly be higher,” he said.
“That is why we will propose an egalitarian approach. So clinical analysis, scientific evidence will still be required; an analysis of the impact on the payer’s budget will still be required, but instead of a classic economic analysis, we will require producers to justify the price, ”said Łanda. He noted that no one should think that justifying the price is any significant facilitation for the manufacturers of these drugs. He said that it would not be an easier path, but a separate one.
The conference in the Sejm was organized by the Association of People with Mucopolysaccharidosis (MPS) and Rare Diseases, which has been operating since 1990. As its president, Teresa Matulka, said, children with rare diseases are often not accepted by their peers, they understand their problems and the consequences of the disease. «It should be remembered that a rare disease is a problem for the whole family and the whole family must receive support and help so that they do not live in loneliness and feeling hurt. No one has chosen such a disease and has never dreamed of living with such problems, ”she said. She added that, first of all, doctors, experts and state administration officials should be made aware of the social problem related to this group of citizens, and then consider health problems related to individual diseases.
According to the vice-president of the National Health Fund, Andrzej Jacyna, in 2015 the fund spent PLN 135 million for the treatment of people with rare diseases; the treatment of 164 patients was financed. «It would be good if it was financed centrally, because when we look at the implementation of drug programs, they are very unevenly distributed among voivodships. If a small provincial branch of the National Health Fund has such an item in the expenditure, it affects the entire budget and officials do not welcome such a situation. If we centralized it, this problem would cease to exist and it would be possible to avoid reprehensible situations where department directors react differently to the situation related to the reimbursement of drug programs, ”he said.
Rare diseases are most often genetically determined diseases, with a chronic and often severe course, about half of which are revealed in childhood. Due to their rarity, recognition difficulties and lack of social awareness, little is known about them.
So far, over six thousand rare diseases have been detected, in Poland about 420 (a disease is considered a rare disease in Poland if it affects no more than 5 out of 10 people).