According to a recent publication from Express Digest, England’s National Health Service (NHS) has reversed its earlier position on a rare disease drug after lengthy public debate, now agreeing to provide cerliponase alfa (branded as Brineura) to Batten disease patients who might benefit.
Initially, English health officials had balked at the idea of providing the drug through the state healthcare system, citing high individual costs and limited health benefit. The policy was hugely unpopular with Batten disease patients and their families, some of who had already seen benefit with the treatment in studies or elsewhere in Europe.
About Batten Disease
Batten disease is actually a group of related neurological conditions that are also called neuronal ceroid lipofuscinosis. The conditions are almost universally characterized by a number of serious neurologically-rooted symptoms that include seizures, vision loss, and intellectual disability. Eventually, patients experience further physical and cognitive decline, losing both muscle mass and previously acquired skills like speech. Ultimately, the disorders are considered to be terminal.
The effects of Batten disease are caused by mutations to a wide variety of genes associated with the production of important enzymes in lysosomes. Lysosomes act kind of like the stomach of a cell, “digesting” fats, waste proteins, and other by-products created during normal cell function. Mutations to just a few genes can disrupt the body’s ability to create functioning versions of certain lysosomal enzymes. Without these enzymes, waste products accumulate in lysosomes in cells throughout the body, eventually severely impairing basic cell function.
Types of Batten disease are differentiated by their genetic origins and varied expressions of similar symptoms. Certain forms may progress slowly, not leading to noticeable symptoms until adulthood. Other forms are highly aggressive, becoming apparent in early childhood.
Reversal of Earlier Policy
Brineura (generic name cerliponase alfa) was approved for certain forms of Batten disease by the American Food and Drug Administration and the European Commission in 2017. The drug is intended to slow the loss of patient mobility. Not long ago, clinical trial sites existed in major cities in Europe, including London and Hamburg. Many families in the UK participated in these trials and saw positive results with Brineura.
Regardless, in February 2018, NICE, one of the organizations responsible for creating English healthcare policy, officially recommended that the NHS not provide cerliponase alfa to Batten disease patients. Citing insufficient evidence of long-term effectiveness and a lofty price tag (about $700,000 annually), the NICE report kicked off what would be over a year and a half of discourse.
The recent change in policy came about only after negotiating a “fair price” with manufacturer BioMarin. Even without a negotiated drug price, however, the rarity of Batten disease in the UK would limit the annual cost of covering the drug to about 0.0012% of the annual NHS budget.
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