SINGAPORE – The days of blockbuster drugs may be coming to an end, but Harish Dave, chief medical officer at Singapore-based Aum Biosciences Pte. Ltd., offered companies a new term. During the second day of the Phar-East Pharma and Biotech Festival biopharma conference, Dave said companies should focus on "niche-busters" and finding new sectors to dominate instead.

Noting that most of the low hanging fruit in health care have been picked, Dave said it "is no longer acceptable to get 20 to 30 percent response rates now. The economics of the field also no longer allow us to chase blockbusters." He cited factors such as the high cost of development (larger trials and longer timelines), demand for better outcomes, general economic pressures (state of economy and budgetary constraints) and the fact that most treatments that address large audiences have already been developed.

The niche-buster model basically calls for companies to "take a disease and slice it into pieces, then expand into multiple indications," Dave said. That could involve a stratification-based dimension or a mutation-specific strategy – it's an effort that has already proved successful in oncology, which boasts a number of therapies that target specific mutations.

Those niche-busters also can be made to fit within orphan disease regulatory frameworks, allowing companies to tap the benefits given to treatments for such indications. "Niche or orphan drugs get priority treatment from most regulatory bodies now. The market exclusivity is also longer, being up to 10 years in the U.S.," said Dave.

Other benefits include fast-tracked approval, tax credits, clinical trial subsidies and reduced regulatory fees.

The economic reception for novel treatments also makes sense.

"Last year, orphan drug sales grew over 11 percent growth rate annually. Overall pharma, in comparison, grew just 6.4 percent," said Dave.

China focus

And since innovations for rare diseases have been mostly Western-driven so far, there is also a geographic niche.

"Fifty percent of Chinese rare disease patients do not receive timely [or] adequate treatment," said Dave.

In a separate talk, Ying Pan, the director of translational science at Cstone Pharmaceuticals Co. Ltd., said that China still has a way to go for treatments of both its rare and prevalent diseases.

"China is in great need of innovative medicines. It is still largely a country of generic drugs," she said.

Pan said government efforts in recent years have been trying to change that, including the multiple revamps of its drug regulatory body.

"It's a new era of innovative drug development in China. The review timeline acceleration brings new horizons," she said, comparing the 25-month wait to approval in 2015 with the mere nine months in 2018. "But the regulators still need better clarity, especially in terms of what innovative drugs can be reimbursed."

She said the R&D and expertise gaps also remain. "We [China] still haven't done much in terms of designing clinical trials so there is a talent gap there. And the R&D gap with the U.S. is still significant," said Pan.

She said she hopes that China's growing innovative treatments could mean great news for China-relevant cancers like hepatocellular carcinoma and gastric cancer.

Founded in 2015, Cstone develops immuno-oncology and molecularly targeted drugs. It has 14 drug candidates in the pipeline, with eight of them at the clinical stage. The Chinese drugmaker raised $410 million from investors such as Singapore's sovereign wealth fund GIC Private Ltd., Sequoia China and a slew of others before it filed for an IPO in Hong Kong in November.

By focusing on those unmet areas and more Asian-specific diseases, pharmaceutical companies could tap into the benefits afforded to novel drugs and a captive market.

Aum's Dave noted that regulators are becoming aware that strategy and regulations could change in the next five to 10 years. But for now, the door is wide open, and companies should take advantage of that.

He said there are certain criteria for what a niche-buster should be. It needs to be aimed at a targeted population; be science-driven and focused on disease pathways; utilize a parallel cell line-derived xenograft (CDx) development model to target multiple indications; and be a niche creation that could potentially tackle the 1,000-plus rare or orphan diseases in oncology.

"A niche-buster can become a blockbuster, but [only] taking a single niche won't make [it] a blockbuster," said Dave, citing the successful example of Merck & Co. Inc.'s Keytruda (pembrolizumab).

"Take a niche approach, then extend it horizontally across all barriers to make it become a blockbuster," he added.

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