AOTMiT: List of highly innovative drug technologies

Highly innovative drug technologies focus on rare diseases and oncology, and the creation of the TLI list, focuses on promising therapies offered for these conditions.

AOTMiT determines the level of innovation and the conditions for the creation of this list with particular regard to the expected health effects, taking into account, among other things, the strength of the intervention, the quality of scientific data, the unmet health need, the size of the target population and health priorities. The extent of this data is extremely important because drugs with a high level of innovation that would be subject to funding are relatively short-lived on the market, and this implies a high degree of uncertainty in the application.

The creation of the TLI list is the first stage of the reimbursement process for highly innovative drug technologies. The next is the creation of the TLI list by the Minister of Health after prior consultation with the Transparency Council, National Consultants and the Patient Ombudsman. Placing a technology on the Minister of Health's TLI list allows the technology to move on to the next stage, which is the submission, in accordance with Article 24(1)(1b) of the Reimbursement Law, of applications for inclusion of the technology in reimbursement.

An innovative drug technology is granted reimbursement only if the application passes the entire procedure specified in the provisions of the Reimbursement Law.

The Transparency Council presents an assessment of drug technologies from the March 15, 2023 list of highly innovative drug technologies for inclusion in the Minister of Health's list of highly innovative drug technologies. The order of the following technologies reflects the level of innovation in the Council's assessment.

1. kimmtrak (tebentafusp) for the indication: in monotherapy for the treatment of adult patients with human leukocyte antigen (HLA)-A*02:01 unresectable or metastatic uveal melanoma;

2. padcev (enfortumab vedotin) for the indication: monotherapy of locally advanced or metastatic urothelial carcinoma in adult patients who have received prior platinum derivative-based chemotherapy and a programmed cell death receptor 1 inhibitor or a programmed cell death ligand 1 inhibitor;

3. Zokinvy (lonafarnib) for the indication: treatment of patients 12 months of age and older with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome or progeroid laminopathy with processing deficient progeroid laminopathy, which are associated with a heterozygous mutation of the LMNA gene with progerin-like protein accumulation or with either a homozygous or compound heterozygous mutation of the ZMPSTE24 gene;

4. Artesunate Amivas (artesunate) for the indication: for the initial treatment of severe malaria in adults and children;

5. Xenpozyme (olipudase alfa) for the indication: in children and adolescents and adults as enzyme replacement therapy for the treatment of symptoms of Acid Sphingomyelinase Deficiency (ASMD) type A/B or B, not related to the central nervous system (CNS); AOTMiT Transparency Board Opinion No. 114/2022, dated 19.06.2023. 2

6. nulibry (phosphenopterin) for the indication: treatment of patients with molybdenum cofactor type A deficiency;

7. vyvgart (efgartigimod alfa) for the indication: adjunctive treatment to standard therapy of adult patients with generalized myasthenia gravis (gMG) with antibodies to the acetylcholine receptor (AChR);

8 Oxbrita (voxelotor) for the indication: for the treatment of hemolytic anemia due to sickle cell disease (SCD) in adults and children 12 years of age and older as monotherapy or in combination with hydroxyurea.

When selecting technologies from among those qualified by AOTMiT to the list of highly innovative drug technologies, the Transparency Council was guided by the following criteria, which make up the assessment of technology innovation:

- Relevance of the condition (or specific indication), consisting of severity of effects and incidence in Poland,

- Meeting a health need - the availability in Poland and the effectiveness of available drug technologies for this condition,

- The strength of the proposed intervention, taking into account its effectiveness and safety (side effects) in the indication under consideration,

- Relevance of the most important endpoint,

- Quality of available scientific evidence. The Council did not take into account the economic aspects of possible reimbursement of the above technologies.

Source: AOTMiT