How Much Can Trump’s Childhood Cancer Funding Proposal Boost Drug Approvals?

President Trump’s 2019 State of the Union Address broadly touched on the themes of funding childhood cancer research, drug pricing and eliminating HIV.

Only three of the 17 novel cancer treatments approved by the US FDA in 2018 contained a pediatric indication. But can an increase in government funding bring a boost to this area?

President Trump appears to think so. In his 2019 State of the Union address, the president said that his next budget proposal to Congress would include a request for $500m over the next 10 years to fund childhood cancer research.

"Many childhood cancers have not seen new therapies in decades," Trump said Feb. 5. "My budget will ask the Congress for $500m over the next 10 years to fund this critical life-saving research."

As with many of the pharma topics mentioned throughout the speech, the specifics of the proposal are unclear. For example, presumably much of the funding would be funneled through the National Institutes of Health, but the speech did not indicate how it might be allotted. Other missing specifics are whether the plan would emphasize novel therapies aimed at cancers prevalent among children or also include efforts to study pediatric indications for drugs approved for adults; how the funding might be distributed to research on drug treatments versus other therapies; and whether the plan might address issues such as trial enrollment. Additional information on such topics could be included in the Trump Administration’s FY 2020 budget proposal, expected to be unveiled in the next several weeks.

Even if the entire funding was earmarked for drug research, it would be unlikely to go far. While estimates of the cost to bring a drug to market are wide-ranging and sometimes contentious, they generally all peg it at least several hundred million dollars. (Also see "The Cost Of Drug Development: Can We All Just Agree It’s Expensive?" - Pink Sheet, 14 Sep, 2017.)

The US National Cancer Institute estimates that 11,060 children ages 14 and younger will be diagnosed with cancer in 2019, most commonly leukemias, brain and central nervous system tumors, and lymphomas. About 1,190 will die from cancer, the leading cause of death from disease in children.

The three novel cancer approvals in 2018 that included a pediatric indication went to Stemline Therapeutics Inc.'s Elzonris (tagraxofusp-erzs) for blastic plasmacytoid dendritic cell neoplasm, Servier SA's Asparlas (calaspargase pegol-mknl) for acute lymphoblastic leukemia and Loxo Oncology Inc.'s Vitrakvi (larotrectinib) for solid tumors with a neurotrophic receptor tyrosine kinase gene fusion. (Also see "US FDA’s Best Year Ever For New Drugs: CDER Novel Approvals Hit 59" - Pink Sheet, 1 Jan, 2019.)

The number is nevertheless an improvement in 2017, when EMD Serono Inc. and Pfizer Inc.'s Bavencio (avelumab) for the treatment of metastatic Merkel cell carcinoma was the only cancer approval that included a pediatric indication. (Also see "Keeping Track: US FDA Clears Three Novel Agents Over Three Days – Bavencio, Symproic And Xadago" - Pink Sheet, 26 Mar, 2017.)

Drug Price “Freeloaders”

The president kept up the heat on drug pricing, saying, "I am asking the Congress to pass legislation that finally takes on the problem of global freeloading and delivers fairness and price transparency for American patients," Trump said.

He did not directly cite the adminstration’s proposal to tie US prices to an International Price Index (Also see "Medicare's Foreign Price Bench-marking Will Only Hurt Bad Negotiators, HHS's Azar Argues" - Pink Sheet, 25 Oct, 2018.) But HHS Secretary Alex Azar highlighted it in tweets applauding the presidents address. "The IPI model we put forward would finally tackle foreign freeriding and lower drug prices for American patients,” he said. Republicans in Congress have so far not weighed in officially on the IPI, although the model has drawn opposition from biopharma. (Also see "Democrats’ Drug Pricing Bills Echo Trump Ideas But Broad Republican Support Unlikely" - Pink Sheet, 10 Jan, 2019.)

Among the next congressional forums on drug pricing is a Feb. 26 hearing at the Senate Finance Committee. The committee has invited representatives from AbbVie Inc., AstraZeneca PLC, Bristol-Myers Squibb Co., Johnson & Johnson, Merck & Co. Inc., Sanofi and Pfizer. Drugmakers have already drawn the ire of committee leaders by refusing to testify at the first Finance Committee hearing. (Also see "Sen. Grassley To Be "More Insistent" About Drug Firms' Testimony At Future Hearings" - Pink Sheet, 29 Jan, 2019.)

Trump also seemed to allude to the HHS proposal to compel drugmakers to disclose list prices in direct-to-consumer (DTC) advertising. (Also see "Democrat-Controlled House Will Turn Up The Volume On Drug Pricing" - Pink Sheet, 7 Nov, 2018.) He closed out his drug pricing remarks with a broad call to "require drug companies, insurance companies, and hospitals to disclose real prices to foster competition and bring costs down."

Funding To Eliminate HIV/AIDS?

The president additionally trumpeted a plan to eliminate HIV and AIDS in the United States. "No force in history has done more to advance the human condition than American freedom," Trump said. "In recent years we have made remarkable progress in the fight against HIV and AIDS. Scientific breakthroughs have brought a once-distant dream within reach.

Azar offered a bit of color on the proposal in a statement. According to the HHS secretary, the plan would fund three major areas of action:

• "Increasing investments in geographic hotspots through our existing, effective programs, such as the Ryan White HIV/AIDS Program, as well as a new program through community health centers that will provide medicine to protect persons at highest risk from getting HIV.

• Using data to identify where HIV is spreading most rapidly and guide decision-making to address prevention, care and treatment needs at the local level.

• Providing funds for the creation of a local HIV HealthForce in these targeted areas to expand HIV prevention and treatment."

"The President’s initiative will work to reduce new infections by 75% in the next five years and by 90% in the next ten years, averting more than 250,000 HIV infections in that span," Azar added.

FDA Commissioner Scott Gottlieb gave the thumbs up to Trump's remarks on the subject on Twitter.

"The commitment to ending the #HIV epidemic in America, announced by @POTUS tonight in the #SOTU, could be one of the most significant public health initiatives we undertake together," Gottlieb tweeted.