by Hélène Mauduit
BERLIN, Nov 17 (APM) - Bayer is satisfied with pilot assessments provided by the European health technology assessment (HTA) body network EUnetHTA on two of its cancer drugs and hopes that national HTA bodies will follow up, the company told APM.
EUnetHTA published in mid-October a 'relative effectiveness assessment' (REA
) on Bayer's Stivarga (regorafenib) in its latest indication in liver cancer.
This is the second time Bayer has submitted drugs for a REA, after a first one published in March 2015 on Nexavar (sorafenib) in thyroid carcinoma.
The REA for Stivarga confirmed that Bayer had "selected the right trial design" and provided "a good understanding on how the clinical endpoints will be assessed", Bayer's head of communications EMEA, Giusy Chiovato Rambaldo, told APM in an emailed statement on Wednesday.
"The design of our pivotal trial was fully accepted with a low risk of bias and the chosen comparator considered as adequate," said Chiovato Rambaldo. During the procedure, additional data were requested and provided on biomarkers and the latest standard Stivarga pharmacovigilance safety report, she added.
On endpoint assessment, "we highly appreciate EUnetHTA’s statement that progression-free survival (PFS) is considered as an 'important' efficacy outcome," Chiovato Rambaldo said.
"Surrogate endpoints such as PFS are not always accepted or evaluated consistently across HTA bodies. In our pivotal trial we have implemented overall survival (OS) and quality of life, both valued as 'critical' endpoints, however it is not always feasible to collect OS data, especially in cancer indications with long post-progression survival."
Bayer volunteered for the REA, conducted from February to October, for Stivarga's latest indication in adult patients with hepatocellular carcinoma (HCC) previously treated with its Nexavar (sorafenib). Bayer filed Stivarga in this indication in November 2016, and the extension was granted by the European commission in August (APMHE 54213
Bayer's experience on the first REA, for Nexavar, was "positive in terms of the learnings we got from the entire process".
"Some countries reused the EU REA report in their national decision-making process," said Chiovato Rambaldo.
Using REA conclusions in national HTA
Bayer supports EU cooperation on HTA and "EUnetHTA's efforts to address divergences in HTA methodologies, with the overall goal to accelerate patient access to innovative medicines", Chiovato Rambaldo said.
"The added value of having a REA conducted at EU level is that the EU assessment is reused by national HTA bodies, which should avoid duplicating the work that has been carried out at European level".
"Based on the experiences for both Nexavar and Stivarga, we encourage EUnetHTA to ensure that conclusions are aligned across HTA participants, with EMA, and with available external medical standards," Chiovato Rambaldo said.
Currently national HTA bodies remain entirely "responsible to conduct the country context-specific assessments and provide recommendations to local government decision-makers for respective appraisals".
HTA bodies can decide if and how to use REA conclusions, EUNetHTA confirmed to APM.
"The intention of the joint work is that the HTA bodies could use the information in the assessments when preparing their national assessments. They can use the assessments as [they are], add a summary in their own language, or use parts of the assessments relevant for their national work," EUnetHTA said in an emailed statement on Monday.
"Hopefully, the assessments are also useful for the companies since they can base their national submission on the work already done with EUnetHTA," it added.
Since 2010, EUnetHTA has published nine pilot assessments on drugs. It is now using the third version of its "HTA Core model".
One REA is ongoing on Roches' Alecensa (alectinib) as monotherapy for the first-line treatment of adult patients with ALK-positive advanced non-small cell lung cancer (NSCLC), for which it received a positive recommendation for approval from EMA's advisory committee CHMP in October (APMHE 55152