It is worth noting that drug technologies with a high level of innovation 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 to include 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.
In accordance with the wording of Article 40a (4) of the Law of May 12, 2011 on the Reimbursement of Drugs, Foodstuffs for Special Dietary Purposes and Medical Devices[1] (the Reimbursement Law), the Agency for Health Technology Assessment and Tarification (the Agency), as part of its work on the next list of highly innovative drug technologies (the TLI list), conducted an assessment of expected health effects guided by, among other things:
- The strength of the analyzed intervention,
- quality of scientific data,
- an unmet health need,
- The size of the target population,
- health priorities.
The process of creating a TLI list consists of the following steps:
- Identification of drug technologies for detailed evaluation,
- Analysis in accordance with HTA expertise,
- Preparation and publication of the TLI list.
The drugs evaluated were those for which the decision on marketing authorization in the European Union (EU) central procedure was issued from November 27, 2021 to December 31, 2022, for which marketing authorization decisions were published by 02/01/2023). According to a statement by the European Medicines Agency (EMA), 103 medicinal products were authorized in the EU during the aforementioned period.
Drugs that received marketing authorization based on documentation of bioequivalence or biosimilarity were excluded from the process. The remaining 76 drug technologies are original drugs.
Of the 76 original medicines that qualified for further selection, products for which the marketing authorization decision covers use in oncology or rare diseases and for which no application for reimbursement and setting the official selling price had been submitted by the date of publication of the TLI list were evaluated.
A total of 31 drug products met the selection criteria for evaluation, most of which are drugs with an indication in rare diseases (about 64%). Some of the drugs are products registered in oncological disease with concomitant orphan drug status (about 10%).
Of the products that met all the selection criteria together, one drug had more than one indication to be evaluated and was evaluated separately in each of the three registered indications. As a result, 33 drug technologies were evaluated.
During the evaluation, special attention was paid to the quality of the available scientific evidence, which is related to the reliability of the results analyzed and affects the (in)certainty of the inference. And the health effects of the therapies were evaluated with a view to whether the endpoints are primary or surrogate endpoints, as well as whether the difference in health effect size between the intervention and the comparator is significant.
According to the analysis, some of the assessed technologies do not have an alternative technology funded in the assessed indications, which can be taken as the existence of an unmet medical need, although it should also be emphasized that the mere availability of a technology in a given indication is not tantamount to meeting the medical need in a given disease entity.
Based on the assessment of drug technologies, taking into account the statutory criteria, a TLI list was created, which includes 8 drug technologies.
Nazwa leku |
Substancja czynna | Oceniane wskazanie |
| Artesunate Amivas | artezunat | do stosowania w leczeniu początkowym ciężkiej malarii u osób dorosłych i dzieci |
| Kimmtrak | tebentafusp | w monoterapii w leczeniu dorosłych pacjentów, u których występuje ludzki antygen leukocytarny (HLA)-A*02:01, z nieresekcyjnym lub przerzutowym czerniakiem błony naczyniowej oka |
| Nulibry | fosdenopteryna | leczenie pacjentów z niedoborem kofaktora molibdenowego typu A |
| Oxbryta | wokselotor | w leczeniu niedokrwistości hemolitycznej spowodowanej niedokrwistością sierpowatokrwinkową (ang. sickle cell disease, SCD) u dorosłych i dzieci w wieku 12 lat i starszych w monoterapii lub w skojarzeniu z hydroksymocznikiem |
| Padcev | enfortumab vedotin | monoterapia raka urotelialnego miejscowo zaawansowanego lub z przerzutami u dorosłych pacjentów, którzy otrzymali wcześniej chemioterapię opartą na pochodnych platyny i inhibitor receptora programowanej śmierci komórki 1 lub inhibitor ligandu programowanej śmierci komórki 1 |
| Vyvgart | efgartigimod alfa | leczenie uzupełniające do standardowej terapii dorosłych pacjentów z uogólnioną miastenią rzekomoporaźną (gMG), u których stwierdzono obecność przeciwciał przeciwko receptorowi acetylocholiny (AChR) |
| Xenpozyme | olipudaza alfa | u dzieci i młodzieży oraz dorosłych jako enzymatyczna terapia zastępcza w leczeniu objawów niedoboru kwaśnej sfingomielinazy (ang. Acid Sphingomyelinase Deficiency, ASMD) typu A/B lub B, niezwiązanych z ośrodkowym układem nerwowym (OUN) |
| Zokinvy | lonafarnib | leczenie pacjentów w wieku 12 miesięcy i starszych z genetycznie potwierdzonym rozpoznaniem zespołu progerii Hutchinsona-Gilforda lub progeroidowej laminopatii z wadliwą obróbką prelamin (ang. processing deficient progeroid laminopathy), które są związane z heterozygotyczną mutacją genu LMNA z gromadzeniem białka podobnego do progeryny lub z homozygotyczną albo złożoną heterozygotyczną mutacją genu ZMPSTE24 |
It is estimated that the above-mentioned drug technologies with a high level of innovation can be applied to a population of about 2,000 patients, with an estimated total cost of more than PLN 607 million.
Source: AOTMiT
