In the last week of September 2021, the Polish Cabinet finally published the Plan for Rare Diseases for 2021-2023 (the "Plan"). We reported about the Plan being submitted for public pre-consultation earlier this year.

The Plan presents a list of actions to be implemented with the aim of improving medical care for patients suffering from rare diseases. It describes the manner and timeframes for their implementation, as well as the financing model for some of them.

There are six core areas of the Plan:

• setting criteria for the establishment and functioning of Centres of Expertise for Rare Diseases (Ośrodki Ekspercie Chorób Rzadkich (OECR)), which will play a key role in meeting patients' needs in terms of diagnosis and treatment;
• defining guidelines for improving the diagnosis of rare diseases, including access to modern diagnostic methods using large-scale genomic technologies;
• improving access to medicines, medical devices and foodstuffs for particular nutritional uses for rare diseases;
• creating the Polish Register of Rare Diseases, which would act as a tool for monitoring rare diseases and health issues on a large scale;
• introducing a Rare Disease Patient Passport – a medical document for patients with rare diseases containing clinical data; and
• creating the Information Platform: "Rare Diseases" – a platform containing clinical, scientific and organisational information on rare diseases.

The Plan considers the following as priority legislative tasks:

Task 1. Declaring rare diseases as a public health priority by the Minister of Health.

Task 2. Adopting the definition of rare diseases and of medicines intended for the treatment of rare diseases as set out in EU law, by way of a resolution of the Cabinet.

Task 3. Establishing a Council on Rare Diseases (under the Minister of Health) to support the implementation of specific solutions adopted in the Plan.

In terms of improving access to medicines, medical devices and foodstuffs for particular nutritional uses for rare diseases, the Plan aims to create mechanisms within the limited resources available. Improved access should cover both innovative medicines, irrespective of their orphan drug status, and medicines that have been available for a long time, including those used outside the registered indications. The objective is to improve access by:

• analysing possible modifications to the health technology assessment (HTA) in order to develop a HTA for rare diseases by introducing a multi-criteria decision analysis (MCDA);
• defining the cost threshold of QALY (quality-adjusted life year) for technologies approved for rare diseases;
• supporting the development and wider use of risk-sharing instruments;
• changing the reimbursement application procedure for technologies with low impact on the financial plan of the National Health Fund (Narodowy Fundusz Zdrowia (NFZ)).

The Plan also proposes establishing reimbursement rules for access to off-label medicines for rare diseases, and improving access to medicines for rare diseases prior to their authorisation and reimbursement.