The British organization that negotiates drug prices for the National Health Service has recommended that a cell therapy for leukemia be made available for children and young adults.
The National Institute for Health and Care Excellence, or NICE, said Friday in a final draft guidance that it had recommended Swiss drugmaker Novartis’ CAR-T therapy Kymriah (tisagenlecleucel) to the Cancer Drugs Fund for pediatric acute lymphoblastic leukemia. NICE had also recommended Kymriah for the CDF in an earlier draft guidance, while a September agreement between Novartis and the NHS would also allow up to 30 patients in England to receive the drug.
The European Medicines Agency approved Kymriah in August for pediatric ALL and adult diffuse large B-cell lymphoma, the US Food and Drug Administration having approved it for ALL in August 2017 and DLBCL in May. The EMA also approved another CAR-T, Gilead Sciences’ Yescarta (axicabtagene ciloleucel), for DLBCL in August, while the FDA had approved it last October.
NICE said it recommended Kymriah to the CDF due to some uncertainties in the evidence used to support it. The CDF, founded in 2011, provides a means for patients to obtain many drugs for cancers that have been rejected by NICE. In order to recommend drugs as cost-effective for routine use by the NHS, NICE typically requires that they carry an incremental cost-effectiveness ratio, or ICER, within the range of 20,000-30,000 British pounds, or $25,689-$38,534. ICER, also known as cost per quality-adjusted life year, or QALY, means the cost for a year of good health and are calculated based on a therapy’s list price and clinical benefit. NICE typically allows drugs to go through, even if it has rejected them previously, in exchange for confidential discounts and rebates that bring the cost per QALY into an acceptable range.
However, the agency said it recognizes Kymriah as a “highly innovative treatment” and is of the opinion that further evidence will likely resolve the current uncertainties. The CDF approval, it said, will enable young people to have access to the therapy as more data are gathered. The list price for Kymriah is 282,000 pounds, but the company has agreed to provide a confidential discount. The NICE-quoted list price is equal to $361,986, while in the US Novartis charges $373,000 for DLBCL and $475,000 for ALL.
However, NICE rejected Kymriah in September for adult lymphoma, citing the cost as too great. Yescarta was rejected on similar grounds the month before. Nevertheless, it stated that it recognizes the therapy’s clinical benefits and would welcome further discussions on its cost effectiveness.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
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