As part of Arcana Institute’s participation in the XV International Conference of the Polish Pharmacoeconomics Society (ISPOR Poland Chapter) held on 30 Nov – 1st Dec 2017 in Warsaw, Poland, our team prepared two posters to share. Click on the poster image to view full poster in original language. Translations of poster summaries are posted below.
Registration of oncological drugs by the european medicines agency based on the results of single-arm trials. Moćko P, Tomassy J, Lasota K, Augustyńska J, Walczak J; Arcana Institute, Krakow, Poland. XV International Conference of the Polish Pharmacoeconomics Society 30 Nov-1st Dec 2017, Warsaw, Poland.
OBJECTIVES: In 2016, the EMA granted marketing authorization to 23 pharmaceutical products with oncological and hemato-oncologic indications, of which 11 had orphan status [Human medicines highlights 2016]. When considering registration of drugs, the EMA takes into account studies with a control group (randomized studies) or those without a control group (single-arm studies) as scientific evidence. The aim of our analysis was to verify EMA’s registration practice for marketing authorization supported by results of single-arm studies for oncology drugs.
METHODS: Our analysis covered all EMA registration processes for oncology drugs assessed in 2006-2017. We selected registration processes for oncology drugs from the EMA database (oncological drugs with ATC codes of first level L were included). We searched for processes whose clinical efficacy evaluation was based on results from single-arm studies. We included processes with a negative or positive decision by the European Commission (EC) and excluded supportive therapies (e.g. treatment of cancer pain). Single-arm studies were defined as studies without a control group, including randomized trials that compared doses of a drug.
RESULTS: We identified 110 oncology drugs that were assessed by the EMA in the specified period. Clinical efficacy assessments based on the results of single-arm studies were performed in the case of 23 oncology drugs. Our analysis included 26 decision-making processes for 23 medicines (due to registration of the first and any following indications or extending an existing indication). From overall processes for oncology drugs receiving a positive EC decision, most were for hemato-oncologic indications (72%), followed by solid tumors (20%) and skin tumors (8%). Out of the processes evaluated based on the results of single-arm studies, 85% concluded with a positive EC decision.
CONCLUSIONS: The EMA has been registering oncology drugs supported by single-arm studies since at least 2006, with 1/5 of such drugs already registered based on results from single-arm studies. Hemato-oncologic drugs make up 72% of drugs registered based on outcomes of single-arm studies. One study was sufficient for 83% of the processes identified in order to receive a positive decision from the EC. The minimum number of study participants for registering indications based on single-arm studies is 16.
Ahtapol’s decision-making practice for onological drugs based on the results of single-arm trials. Moćko P, Tomassy J, Augustyńska J, Walczak J. Arcana Institute, Krakow, Poland. XV International Conference of the Polish Pharmacoeconomics Society 30 Nov-1st Dec 2017, Warsaw, Poland.
OBJECTIVES: In 2016, the Agency for Health Technology Assessment in Poland (AHTAPol) evaluated (recommendation was given by the President of AHTAPol) 76 new drug technologies, of which 20 had indications for oncology and hematological oncology. In the current year (until the beginning of November 2017), the President of AHTAPol gave out 53 recommendations, 19 for oncologic and hemato-oncologic indications. The aim of this analysis was to verify AHTAPol’s decision-making practice in terms of drug reimbursement based on single-arm trials in regard to oncology drugs.
METHODS: In our analysis, we included decision-making processes for oncology and hemato-oncological drugs evaluated for reimbursement (Art 35 part 1 of the Reimbursement Act), from Jan 2012 – July 2017. We also considered positive and negative decisions by the MoH for the processes. We performed the analysis based on publicly available data provided by AHTAPol, including the agency’s verification analysis, Transparency Council statements and recommendations from the agency’s president. We excluded assistive therapies for treatment of undesirable effects during the course of cancer. Single-arm studies were defined as studies without a control group, including randomized trials that compared doses of a drug.
RESULTS: We identified 103 reimbursement decision-making processes for oncology drugs assessed by AHTAPol in the specified period. From the identified processes:
- Excluded 91 processes that were based on studies with a control group
- Included 12 processes further analysis that were based on single-arm studies
- 1 process was divided into 2 separate processes, due to separate assessments conducted by AOTMiT for two indications that were included in a single application.
Out of the processes assessed that were based on results of single-arm studies, 77% resulted in a positive decision by the MoH.
CONCLUSIONS: The President of AHTAPol issues recommendations for the reimbursement of oncology drugs based on results of single-arm studies since at least 2012, with 1 out of 10 drugs being reimbursed based on the results of single-arm studies. Even if AHTAPol does not consider single-arm studies as enough for a recommendation, it is still possible to obtain a positive reimbursement decision. The lack of cost-effectiveness information on drugs evaluated based on the results of single-arm studies (23%) does not prevent issuance of a positive reimbursement decision. 1 out of 3 processes included a single-arm study. Based on results of single-arm studies, the minimum number of study participants sufficient to obtain a positive reimbursement decision from the MoH is 52. In 60% of processes that received a positive decision by the MoH, the assessment time is greater than the average time of 307 days, likewise for statutory time with a maximum of 240 days.
