China's promise of an accelerated approval pathway for a list of 48 drugs approved elsewhere to treat life-threatening or rare diseases could be a good opportunity for the companies that make the drugs, but once again the devil will be in the details, Hogan Lovells partner Philip Katz told BioWorld Asia.
In releasing the list last week, China's Center for Drug Evaluation (CDE) said it was speeding up the review and approval process for the drugs, which are urgently needed in China. Although some of the drugs on the list have been approved in the EU, Japan or the U.S. for several years, none of them are registered or in clinical trials in China. (See BioWorld, Aug. 15, 2018.)
The CDE notice suggests that makers of the listed drugs can bypass clinical trials in China if they submit overseas clinical data and supporting evidence that shows no racial/ethnic differences. The drugs also will be granted priority review.
"China is not exporting its approval process," but it's trying to make it less burdensome for the badly needed drugs, Katz said. That's good for patients in China, and it could be good for the companies with drugs on the list, he added. And should the CDE expand the list in the future, it could be good for innovators with other therapies that might be needed in China, given the size of the market.
But before they rush into China, Katz is advising drug companies to have a serious conversation with Chinese regulators about what the path will entail and how much market exclusivity they would be granted. Then the companies would have to weigh government pricing constraints – and the potential risks to their intellectual property and trade secrets.
Earlier this year when the U.S. Trade Representative (USTR) issued its annual report to Congress on China's compliance with World Trade Organization principles, it highlighted China's intent, expressed in its Made in China 2025 initiative, to dominate global markets in key sectors, including biopharma and medical devices. (See BioWorld, Jan. 24, 2018.)
The overriding aim of the 2025 initiative "is to replace foreign technology, products and services with Chinese technology, products and services in the China market through any means possible so as to ready Chinese companies for dominating international markets," the USTR said. According to the report, those means include theft of trade secrets to provide commercial advantages to Chinese enterprises, incentives for an applicant to shift its manufacturing capacity to China, pricing concessions as a pre-condition for marketing approval and technology transfer requirements.
"If a skeptic would say this is just a ploy [to advance the 2025 initiative], that strikes me as pretty harsh," Katz said of the CDE's list. "But if a Pollyanna would say there's no risk, I would say that's a bit naïve."
Another risk to consider, especially for U.S.-based companies, is the ongoing tariff war between China and the U.S. and retaliatory steps that could be taken in the future. "Who knows what's going to happen. . . . But it's a complicating factor you have to think about," Katz said. (See BioWorld, April 5, 2018.)
Many of the drugs on China's list are biologics, which are more difficult to copy than small-molecule drugs. Thirteen, or 27 percent, of the 48 drugs are monoclonal antibodies. The list also includes a shingles vaccine, a recombinant human nerve growth factor, a number of enzyme replacements and a gene therapy.
As far as therapeutic space, 13 of the drugs treat various forms of cancer, including two for pediatric neuroblastomas. Eight of the listed drugs treat conditions related to cardiovascular health, five are for psoriasis and three are for multiple sclerosis. Also listed are drugs to treat hepatitis C, HIV, Gaucher disease, Huntington's, inherited retinal disease, ulcerative colitis and cryopyrin-associated periodic syndromes.
More than half of the drugs were first approved in the U.S., while six were first approved in the EU and three were first approved in Japan. The oldest drug on the list is Alexion Pharmaceuticals Inc.'s Soliris (eculizumab), which was approved in both the EU and U.S. in 2007 to treat paroxysmal nocturnal hemoglobinuria, which is considered a rare disease. However, most of the drugs on China's shopping list were approved within the past eight years.
Nine were approved as recently as last year – Celgene Corp.'s cancer drug Idhifa (enasidenib), Teva Pharmaceuticals Industries Ltd.'s Huntington's drug Austedo (deutetrabenazine), Eusa Pharma Inc.'s pediatric neuroblastoma drug Qarziba (dinutuximab beta), Johnson & Johnson's (J&J) psoriasis treatment Tremfya (guselkumab), Ultragenyx Pharmaceutical Inc.'s Mepsevii (vestronidase alfa) enzyme replacement therapy, Glaxosmithkline plc's Shingrix vaccine, Spark Therapeutics Inc.'s gene therapy Luxturna (voretigene neparvovec), Dompe Farmaceutici SpA's recombinant human nerve growth factor and Gilead Sciences Inc.'s combination hepatitis C treatment Vosevi (sofosbuvir, velpatasvir and voxilaprevir).
The companies that make the wanted drugs are as varied as the products themselves. Novartis AG has four drugs on the list; Gilead and Pfizer Inc. each have three listed drugs. Amgen Inc., Celgene Corp., Eli Lilly and Co., J&J, and Merck & Co. Inc. had two drugs each.