GlaxoSmithKline (GSK) has signed a pay-for-performance deal with the Italian Medicines Agency (AIFA) to boost usage of Strimvelis, the pharmaceutical company’s gene therapy treatment developed for ADA-SCID, a rare genetic disorder in children. The key to the contract, however, is that the drug must work. If treatment doesn’t provide a cure, GSK must return a portion of the funds paid by AIFA based on the terms of the agreement.

Luca Pani, director general, AIFA, which set the price, said, “Ethical considerations are an integral part of these agreements because the monitoring of treatments and data collection allows us to verify any side effects of the drug and to take action on the appropriateness of prescriptions.

“The drug has to deliver … or we don’t pay,” said Pani. “If it does not work, they will return the money.”

The deal with GSK is not the first time AIFA has established financial safeguards to ensure a treatment works. Risk-sharing agreements—particularly for specialty medicines—are a common element of the Italian healthcare system. Using patient registries, the company tracks how well several drugs—including cancer therapies—succeed in helping patients. Pharmaceutical companies have refunded hundreds of millions of dollars already for therapies unsuccessful in treating disease.

“The industry will need to adapt the way in which medicines are priced and funded to support healthcare systems around the world to plan for and support access to new cell and gene therapy medicines,” said Mary Anne Rhyne, director, External Communications, GSK.

GSK hopes that Strimvelis is the first of several innovative gene therapy medicines that will become available to patients during the next few years. The company consulted with multiple reimbursement groups across Europe before submitting a formal dossier to AIFA.

ADA-SCID occurs when both parents pass on a defective gene that prohibits children from producing a necessary enzyme for survival. As a result, toxins collect that kill lymphocytes designed to guard against infection. The traditional methods for treating the disease are a bone marrow transplant, which can cost over $1 million, or injectable enzyme replacement with an annual cost of approximately $250,000. In comparison, treatment with Strimvelis is between $650,000 and $700,000, and is expected to provide a cure. “To date, there is a 100% survival rate at three years post-treatment with Strimvelis observed for all children in the pivotal study,” said Rhyne.

“Even with advances in stem cell transplants, there remains a large unmet need in ADA-SCID,” said Rhyne. “Four out of five children do not find a suitable stem cell donor match and it is estimated that there is a 66% survival rate at one year with a bone marrow transplant from a matched, non-family donor, a 43% survival rate at one year with a transplant from a half-matched family (or haploidentical) donor and a 29% survival rate at one year with an unmatched, non-family donor.”

But treatment still boils down to cost, and few patients contract the disorder. In all of Europe, approximately 12 children in total are afflicted each year, which makes return on investment for the drug’s development a long-term process. For now, GSK is focused on ensuring how to best make a one-time treatment like Strimvelis cost-affordable to healthcare systems through established payer mechanisms.   

Patients will continue to require new cures and cutting-edge therapies. In turn, pharmaceutical companies must leverage incentives to bolster innovation and speed up development time.

Novel treatments like Strimvelis can provide patients with a new approach to treatment while enabling companies to increase their experience and knowledge base. By standardizing the production of a gene therapy and the methods of evaluating its effectiveness, organizations can apply successes to other diseases and treatment approaches.

Cells as medicine have a strong future. “They harness the power of the individual’s own cells to provide treatments with highly durable outcomes,” said Rhyne. “In the long-term, they may sit alongside our existing areas of vaccines (for prevention), small molecules and biopharmaceuticals.”

Deals like the one struck between GSK and AIFA are a critical aspect of the new development paradigm. 

This article was reprinted from Volume 20, Issue 34, of CWWeekly, a leading clinical research industry newsletter providing expanded analysis on breaking news, study leads, trial results and more. Subscribe »