Japan Orphan Drug Update 2017


Japan has some of the highest healthcare standards in the world, with the Japanese government providing health insurance for approximately 99% of its citizens. After the United States, Japan was the second country to establish regulations for the development of orphan drugs, benefitting orphan drug developers. Incentives include tax exemptions, waived or reduced fees, expedited approval, and market exclusivity for up to 10 years.

New Regulations

Japan’s MHLW expands orphan drug criteria from diseases with less than 50,000 patients to less than 180,000 patients if the disease is a “nanbyou” in 2015. A list of all designated “nanbyou” exists on the Japan Intractable Diseases Information Center.

New Orphan Drugs in Japan

  • 3/22/2017 Alexion announced it has submitted an application to Japan’s Ministry of Labour and Welfare (MHLW) to extend the indication for Soliris (eculizumab) as a potential treatment for patients with refractory generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive.
  • 12/29/2016 Japan Tobacco and Torii Pharmaceutical receives marketing approval for its combination HIV-1 treatment, Descovy combination tablets. (Emtricitabine/ Tenofovir alafenamide fumarate)
  • 12/19/2016 Biogen receives marketing approval for Tecfidera (dimethyl fumarate) capsules for multiple sclerosis.
  • 11/22/2016 Jansenn Pharmaceutical receives marketing approval for Prezcobix combination tablets (Darunavir ethanolate/ Cobicistat) for HIV-1 treatment.
  • 5/24/2016 Chugai Pharmaceutical, majority-owned by Roche, announces that the Japanese MHLW has approved marketing of Avastin (bevacizumab) for the treatment of advanced or recurrent cervical cancer.
  • 2/26/2016 Japanese MHLW grants Orphan Drug Designation to Takeda’s oral proteasome inhibitor Ixazomib for patients with Relapsed/Refractory Multiple Myeloma
  • 7/6/2015 Alexion’s Strensiq (asfotase alfa) receives marketing approval in Japan for treatment of patients with Hypophosphatasia (HPP)
  • 6/30/2015 Mitsubishi Tanabe Pharma Corporation announced it has received approval to market Radicut® for treatment of ALS in Japan.

Recent Developments

The Japanese government under the Abe administration is pursuing a bold policy of re-pricing expensive drugs in Japan. This was triggered by the launch of Opdivo, initially approved in July 2014 as an orphan drug for the treatment of unresectable malignant melanoma, a rare cancer in Japan. It was priced at $320,000 per patient per year. After initial approval for melanoma, Ono Pharmaceutical sought and received two non-orphan indications, non-small cell lung cancer and renal cell carcinoma. The expansion of indications for Opdivo to these two non-orphan cancers significantly increased the patient population for this expensive drug, raising healthcare costs dramatically. In retaliation, Japanese regulators introduced the “ultra-expensive drug repricing rule” in early 2016 that allowed for drug prices to be slashed if they exceeded revenue forecasts provided at the initial approval. Opdivo was ineligible for repricing under this rule as its rapidly growing revenues had yet to surpass its revenue forecast, so it was singled out for a one-off 50% price cut in November of 2016.

In December 2016, Japanese officials announced that all drug prices will be reviewed annually instead of biennially, and quarterly for the newest and most expensive medications. According to the Pharmaceutical Research and Manufacturers of America, pharmaceutical sales are expected to drop 30% to about $62 billion through 2025 because of lower drug prices.