By Hélène Mauduit at the Forum-Institut für Management market access & reimbursement oncology conference
BERLIN, Jan 26 (APM) - The added benefit assessment of chimeric antigen T cell receptor (CAR-T) therapies by Germany's health technology assessment (HTA) body G-BA will be challenging, G-BA said.
When they are filed in Germany, G-BA will assess CAR-T therapies as new drugs, head of pharmaceuticals at G-BA Thomas Mueller said at the Forum-Institut für Management "market access & reimbursement oncology" conference in Berlin on Thursday.
No CAR-T therapy has yet been approved in the European Union, but G-BA carries out reviews before approval at a company's request or automatically when the European Medicines Agency (EMA)'s advisory committee CHMP has issued a positive opinion, Mueller said.
The two most advanced CAR-T therapies are already approved in the U.S. (APMHE 54476
, TRanAPMHE 55223
). Novartis filed Kymriah (tisagenlecleucel) for approval in the EU in November 2017 for leukaemia and lymphoma (APMHE 55459
), while Gilead/Kite is expected to file Yescarta (axicabtagene ciloleucel) in the region in the coming months.
No randomised clinical trials (RCT) - the gold standard for G-BA assessment - have been carried out on these first products. "We need high-quality data from historical comparisons and registries," Mueller said.
In paediatric indications, this should be possible since Germany has good registries and a large amount of data on children's cancer treatment, he continued.
But many questions remain unanswered on CAR-T therapies, he added. How long does remission last? What are the long-term effects? What is the survival rate after five years ? "Data are lacking for the moment," he said.
"We have to find a pattern, to determine what data need to be collected in order to have a balanced view," Mueller said.
"A panel discussion is necessary with scientific experts, the specialised medical teams which will treat patients, G-BA and health insurance companies to see how to handle this," he continued.
"G-BA's recommendation is that the HTA rating may include indications on data that must be provided subsequently," Mueller said.
Pricing questions also remain, in particular whether pay-for-performance, risk-sharing models or deferred payments should be used for these highly expensive therapies, he said.
Major German health insurance companies are most probably working on that issue already, he added.
Under Germany's drug pricing law AMNOG, the launch price of a new drug is freely determined by the manufacturer and is maintained for one year while HTA procedures are completed and the manufacturer and umbrella payer group GKV-Spitzenverband (GKV-SV) negotiate a new reimbursed price.
Categorised as drugs
G-BA has already made a first review on how to assess CAR-T therapies and has categorised them as drugs and not as 'methods of treatment', Mueller said
CAR-T therapies belong to the category of advanced therapy medicinal products (ATMP), which consists of gene therapies, somatic cell therapy medicinal products (sCTMP), tissue engineered products (TEP) and combined ATMPs.
The decisive criterion for a product to be assessed as a drug is that medical acts necessary for treatment are "established", Mueller said.
If classified as a 'method of treatment', the HTA and pricing path is beyond the scope of the AMNOG drug law, he added.