The US should set a goal of doubling the output of innovative new medicines that meet critical public health needs over the next 10-15 years, while continuing to increase drug safety, the President's Council of Advisors on Science and Technology (PCAST) has urged. 

Challenges remain in translating the "stunning" scientific progress made by basic biomedical science in past decades, says a new report from PCAST, in which it assesses the reasons for this long-term trend.

The study makes a number of detailed recommendations aimed at: - bolstering the discovery and development of new therapeutic compounds; - optimising processes used by the Food and Drug Administration (FDA) to evaluate the safety and efficacy of candidate drugs; - enhancing long-term monitoring of approved medicines; and - enhancing public understanding about the benefits and risks of medicines.

While heart disease and stroke remain leading causes of mortality, many common cancers are still incurable unless they are caught in the earliest stages, and the vast majority of rare diseases lack effective therapies altogether, the report notes. Infectious diseases, including those caused by antibiotic-resistant bacteria and viruses with pandemic potential, pose a constant threat of large-scale mortality. And treatments for psychiatric diseases, which impose a tremendous burden on society, are frustratingly limited in their efficacy, as are treatments for neurodegenerative diseases such as Alzheimer's, it adds.

All three major components of the drug development ecosystem - basic biomedical research in universities and research institutes, clinical research in hospitals, and drug discovery and development in the biopharmaceutical industry - are facing growing challenges as the time, complexity and cost of developing drugs have gone up, says PCAST. It points out that rates of New Drug Applications (NDAs) submitted by industry to the FDA, as well as new drug approvals, have remained relatively constant for 20 years, but says it is encouraged by the fact that the FDA approved 35 new medicines in the past year, among the highest totals in the last decade.

The panel sees two critical needs related to drug discovery and development that must be addressed to advance innovation.

First, it says, scientists need better methodologies and tools for translating basic biological insights into validated therapeutic targets and leads - a gap in the drug discovery and development pipeline that academic scientists often view as "too applied."

Second, pharmaceutical developers and regulators need to incorporate new efficiencies into clinical trials of candidate medicines - complex and costly human studies that current constitute fully 40% of the biopharmaceutical industry's R&D budget.

To achieve some of the report's broader goals, PCAST recommends the creation of a public-private Partnership to Accelerate Therapeutics, to help identify and plan collaborative actions to speed drug development while balancing competing stakeholder interests and minimising duplication of effort. The Partnership would involve representatives of the biopharmaceutical industry, the academic biomedical research and ethics community, physician societies and pharmacists, patient-focused research foundations and advocacy groups, healthcare providers and insurers and the federal government.

The report also concludes that the return on investment (ROI) in certain disease domains may be too low to justify their pursuit by companies, even though the potential benefits for public health in these domains may be large. Therefore, it says, the Department of Health and Human Services (HHS) should commission a study to assess potential mechanisms to encourage companies to tackle important medical challenges that may be financially unattractive.

"With improved collaboration among all the participants in the drug development ecosystem and optimising of drug evaluation pathways, American researchers and companies should be able to accelerate the development of safe and effective drugs while also strengthening the US economy," said Eric Lander, president of the Board Institute at MIT and co-chair of PCAST.