Rare and Ultra-Rare Diseases – V HTA Symposium in Krakow

HTA Real World Data

V HTA Symposium in Krakow – Session Recap II

Arcana Institute hosted the V HTA Symposium in Krakow on 29th-30th May 2017, which provided an in-depth view of the many important topics taking place in Poland in the realm of systematic changes in healthcare related to the development of clinical registries and planned introduction of amendments to reimbursement regulation concerning rare diseases and medical devices.

Rare and Ultra-Rare Diseases

Speaker’s Panel: Krzysztof Kopeć, Dr Tomasz Bochenek, Karolina Skóra, Prof Jolanta Sykut – Cegielska, Joanna Augustyńska

Moderator: Karolina Skóra

According to the generally accepted definition, rare diseases are severe and often life-threatening, affecting about 5 individuals per 10,000 in the general population. According to the Big Amendment on Reimbursement (DNUR), a draft amendment to the Reimbursement Act, an ultra-rare disease  is defined as a clinical condition occurring in not more than 1 individual per 50,000 people residing in Poland or in the EU.

The growing number of patients affected with rare diseases poses a serious social problem and a huge challenge for the healthcare and social security systems. One of the key systematic challenges in Poland is ensuring safe and effective treatment for patients with rare and ultra-rare diseases. A lack of health policies specific to rare diseases and limited expertise in the field cause delays in diagnosis and lead to difficult and unequal access to treatment and care.

The Reimbursement Act (in its current form) does not provide a separate approach for therapies used in rare and / or ultra-rare diseases. Nonetheless, 33% of orphan drugs exceeded the cost-effectiveness threshold and then entered reimbursement. Analysis of the MoH’s decision-making practice shows that orphan drugs were treated more poorly (4% fewer positive decisions) than all medicines. The main issues concerning orphan drugs, from the point of view and of their evaluation by AHTAPol during the reimbursement process, include the high price, lack of sufficient scientific evidence and a high uncertainty of assessment.

The strategic goal of the proposed changes in the treatment of rare diseases is to implement the National Plan for Rare Diseases as a tool to ensure the execution of health policies targeting the needs of patients with rare diseases. The National Plan for Rare Diseases introduces systematic solutions for the health and social issues affecting those with rare diseases. Systematic changes make it possible to expand reimbursement given to products imported from abroad to include products registered in Poland that are not available on the market. Such change serves as an important ‘security system’ in case of drug shortages. It should be emphasized that in addition to ensuring patients with access to effective publicly funded treatments, it is also important to offer support for families of patients with rare diseases.

The draft amendment, DNUR, plans to offer a chance for reimbursement for treatments of ultra-rare diseases. Such an amendment provides changes related to the possibility of replacing an economic analysis with an HTA analysis and justification of price. The type and scope of data necessary to prepare the price justification is identified. During discussion, however, it was pointed out that in a situation of limited financial resources, there is concern that due to the increasing range of financing of costly treatments, worsening health status of patients with common diseases may occur. There is concern that the proposed approach does not guarantee the stability of health financing system in Poland. It is important to note that the chances of a draft with the above provisions coming into force are currently under question.

One of the alternative options of access to treatments for patients with rare diseases is the Emergency Access to Drug Technology (RDTL). In the case of justified and current knowledge, there is need for a patient to use a certain drug that is not financed by public funds for a given indication, and if it is necessary to save the life or health of the beneficiary in the indication occurring in individual patients, and if for the beneficiary all possible publicly available medical technologies in a given indication have already been exhausted, the MoH may, by administrative decision, agree to cover the costs of the drug under RDTL. Such a decision is issued for the duration of the therapy, but no longer than 3 months or 3 cycles of chemotherapy. However, attention has been placed on the unfavorable provisions for the patient as a result of access to RDTL, mainly when the cost of the proposed treatment is on a quarterly basis or three treatment cycles exceed one quarter of the GDP per capita. Furthermore, within DNUR are the conditions under which the MoH may refuse to authorize the use of a given drug in a specific indication.

Rare and Ultra-Rare Diseases – V HTA Symposium in Krakow