Could a Decision by NICE not to Approve Jakavi Undermine Investment in Orphan Drugs?

| 14 February 2013 |  By 

A new draft decision by the UK's cost containment agency, the National Institute of health and Clinical Excellence (NICE), not to recommend reimbursement for Novartis' first-in-class new drug for blood cancer (myelofibrosis) related splenomegaly could serve as a disincentive for future research and development on therapies intended for rare diseases, critics say.

Those critics contend that if a company is unable to expect a return on the substantial investment that is needed to bring a new drug to market, then there is no incentive to undertake that investment. Similar concerns were the driving force behind the US's Orphan Drug Act of 1983, which provided sponsors of rare disease therapies market-based and research incentives aimed at incentivizing investment.

At issue in the UK is not the efficacy of the Novartis' drug, Jakavi (ruxolitinib), which NICE concedes is effective in reducing spleen size and in treating symptoms of myelofibrosis, such as itch and fatigue. NICE even believes the drug may improve survival in blood cancer patients.

But the regulator has determined that the drug is not cost-effective when compared with other available therapies-a common finding by regulators that has been the bane of many therapies before it, including those for rare diseases.

But critics say the denial of Jakavi ignored the fact that no other currently available treatment does what Jakvi does: target the underlying molecular defect that drives blood cancer. Further, they contend that denying this drug could be a powerful signal to other developers of rare diseases that investment costs aren't worth the uncertain return, denying patients of potential therapies.

In the UK, the annual incidence of myelofibrosis is estimated to be in the range of 0.34-0.76 people in every 100,000, with those over the age of sixty most at risk. Myelofibrosis is an uncommon, life-threatening blood cancer characterized by bone marrow failure, enlarged spleen (splenomegaly) and debilitating symptoms such as; extreme fatigue, night sweats and intractable pruritus (itching), poor quality of life and weight loss, as well as shortened survival.

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