From 12 Expanded-Access Drug Programs 50 Million in Savings for the Italian National Health Service

How much can the NHS save by using expanded-access drugs? We had no answer to this question until the documented analysis conducted by SDA Bocconi’s CERGAS in partnership with Roche Italia, the pharma multinational that is in the top positions worldwide for its research activities. This study measured the net financial effect of Compassionate Use Programs (CUPs) on our National Health Service. Directly funded by pharma companies, CUPs involve patients suffering from serious conditions who do not meet the criteria to access clinical trials.

These Programs offer an important opportunity to patients, clinicians and the entire system in that they allow for early access to new treatment options. CUPs can be used to treat patients with serious, rare or life-threatening pathologies when no effective therapeutic alternatives are available.

CERCAS research involved 12 CUPs activated by Roche from 2016 up to the present (9 in cancer-related areas), treating 2,713 patients. Their net financial effect was estimated based on the difference between what the NHS would have paid for alternative treatments and what it actually paid in CUPs for other drugs used in combination with CUP ones, diagnostic procedures not covered by CUP sponsors and the management of the collateral effects of these drugs.

The researchers in charge of CERGAS project, Marianna Cavazza and Claudio Jommi, went into more detail about the results during a meeting online: “Saved average spending for alternative treatments is between 11,400 and 20,300 euros per patient per year, depending on the type of treatment, while extra costs generated by CUPs are 1,600 euro. Therefore the net saving effect is between 9,800 and 18,700 euros per patient per year”.

“Overall savings,” the researchers said, “range from 26,5 to 50,6 million euros per year, based on the drugs potentially used in existing clinical practice. These savings could well be underestimated due to the fact that the cost of collateral effects of alternative drugs to those used in CUPs are not considered.”

“This first evidence,” Jommi and Cavazza concluded, “provides an objective picture of the financial effects for our NHS. Estimated financial impact could be integrated by the fallout of different CUP and non-CUP drug efficacy. Measuring such impact could be possible by enriching the data gathered in CUPs and applying perspective simulation models.”

“As a company that views research as an imperative, ranking first in the pharma industry and among the top 10 worldwide, Roche strongly believes it has not only a scientific value, but also an ethical, economic and social one,” said Anna Maria Porrini, Medical Affairs & Clinical Operations Director Roche Italia. “Our mission is to foster progress in medicine, bringing drugs to areas with unmatched needs in as short a time as possible, thus enabling early access to the most innovative treatments by thousands of patients. This is an extremely valuable opportunity for patients, clinicians and the entire Service. Cooperating with CERGAS is the beginning of an ongoing effort to improve the opportunities for gathering data from these Programs, thanks to the work of all the actors involved.”



SDA Bocconi School of Management.