The drug discovery, development, and regulatory approval process typically takes up to 15 years. This lengthy time frame makes it hard to respond to emergencies requiring new therapies, deprives patients of fast access to life-saving medicines, decreases the profitability period of pharmaceutical companies, and contributes to the high cost of new medicines. Moreover, the COVID-19 pandemic has made the need for faster drug development and regulatory evaluation process clear.

To address this critical issue of national importance, NIPTE is conducting a series of Pathfinding Workshops focused on two major goals:

1. To identify scientific, technological, and regulatory mechanisms capable of reducing injectable drug product development time frame without increasing patient risk, and

2. To integrate these mechanisms into a coherent strategy for accelerating the discovery, development, and regulatory approval process of these important drug products.

The proposed workshop will bring together a focused group of thought leaders from academia, industry, and regulatory agencies, to discuss the critical elements needed to accelerate drug development, and regulatory evaluation based on recent scientific and technical advances in medicine, biology, chemistry, materials science, biostatistics, and advanced manufacturing technologies. These discussions will be led by pre-selected panelists with an established track record in their respective fields. Acceleration mechanisms will be examined from multiple perspectives, including the strength of the underlying science, technical feasibility, and regulatory acceptability. Brainstorming and analysis with the participation of experts across the relevant disciplines will enable significant outcomes, which wouldn’t be achieved in any existing scientific communication forum or conferences.

Acceleration mechanisms deemed to be potentially effective will be selected for further analysis by a focused multidisciplinary team of workshop participants. Workshop participants will be tasked with integrating these mechanisms into a strategic plan and developing an implementation pathway. These documents, which will be the main output of the workshop, will serve as a blueprint for the Federal Government, industry, and academia to direct efforts in this field.

PROBLEM STATEMENT

During the past three decades, entire manufacturing sectors have largely moved overseas. This is particularly prevalent in the manufacturing of Active Pharmaceutical Ingredients (APIs) and also, to a considerable degree, finished pharmaceutical products. By recent estimates, more than 80% of all APIs consumed in the US are currently manufactured overseas, predominantly in China. There is a rapidly growing national consensus that such dependence on imports of critical medical materials places the American population at risk.

It’s difficult to imagine or quantify the impact on the health of the US population if a major disruption of international commerce were to disrupt the United States’ ability to import APIs and finished pharmaceutical products. Since the Pharmaceutical industry only keeps about 75 days of inventory at hand3, the domestic supply of many life-saving drugs would be exhausted in a matter of weeks, grinding the pharmaceutical industry to a standstill. Such an event could cause massive loss of life, as millions of US citizens would lack access to drugs required to keep them alive, including hundreds of substances that are critical to maintaining the lives of many of our citizens afflicted by heart disease, metabolic conditions, HIV, cancer, infectious diseases, and many other potentially fatal conditions.

Importantly, at present, we not only lack much of the industrial capacity to replace API imports, but we also have lost much of the know-how needed to make these substances. Recreating this capacity from scratch would require several years of sustained efforts, a process that would be further delayed by our current lack of an implementation and prioritization plan.

Over the past year, NIPTE has undertaken efforts to grow awareness and make inroads towards a systematic solution to this crisis and has articulated a four-pronged strategy that was presented to and discussed with the White House’s Office for Science and Technology Office:

• Initiative 1: Create a prioritized list of critical APIs and excipients.

• Initiative 2: Retrosynthetic analysis to determine feasible alternative pathways to critical APIs and excipients using domestically sourced key starting materials

• Initiative 3: Demonstrate, scale-up, and transfer to Industry the synthetic pathways developed in

• Initiative 2.

• Initiative 4: Create a platform for rapid development, scale-up, manufacturing, and technology transfer.

The workshop objective is to focus on these initiatives related to the Strategy for Mitigating the Risk to the US Pharmaceutical Supply Chain.