The need for efficient and timely development of both new genomic and cellular therapies, as well as innovative treatments for multiple serious conditions, will drive efforts to modernize the conduct of clinical research for new drugs and medical products in the coming year. The nation’s focus on high drug costs that can limit patient access to vital new therapies speaks to the need for policies that enable less costly and more productive research programs that eliminate redundant and unnecessary clinical studies and protect the rights and privacy of subjects. 

FDA will continue to issue advisories to inform testing and production of needed treatments, including cutting-edge therapies that raise tricky research and quality control challenges. The agency recently finalized guidance on adaptive clinical trial designs, noting that it expects to see more than 200 study plans utilizing adaptive approaches in the coming year. FDA officials similarly support the development of more natural history studies that can better describe diseases, define external controls, and identify patients for clinical trials. And there will be continued acceptance of real-world evidence (RWE) and real-world data (RWD) to inform protocol development, define clinical endpoints, identify patients for studies, and track product safety and efficacy following approval.

A continuing priority is the validation of novel study endpoints as well as appropriate surrogate and clinical endpoints able to reflect disease manifestations. To maintain momentum, patient advocates will continue to provide important voices for shaping risk-benefit equations for promising therapies, while manufacturers will invest more in modern operations able to produce quality products more reliably and efficiently. At the same time, all parties will be watching closely to detect any safety problems or signs of limited effectiveness that could raise concerns about the long-term value of a new treatment. This will support efforts to better define and support patient registries, mobile health applications, and other remote tools to collect patient-reported outcomes following initial treatment. 

Global harmonization of standards for preclinical and clinical testing will advance, along with agreements among regulatory authorities to share and accept reports on plant inspections and on certain testing programs. The Project Orbis collaboration involving regulatory authorities in the U.S., Australia, and Canada looks to move forward from its recent joint approval of additional indications for a cancer therapy based on information in market applications submitted to the three agencies. FDA and the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) are holding a joint symposium in February on the importance of data integrity in global clinical trials. A main theme for the coming year will be to reduce extraneous requirements and promote more streamlined, risk-based approaches to drug development and production around the world. 

Politics and access

At home, innovative approaches to clinical research will be affected by the politicking and legislative activity ahead of the national elections in November 2020. Healthcare policy and pharmaceutical costs will remain leading issues for all candidates as the future of the nation’s healthcare system and shape of public and private drug coverage and reimbursement remains in the spotlight. Industry will challenge legislation authorizing drug importing and international reference pricing as a threat to continued investment innovation, but the full ramifications of election-year politicking remain to be seen. 

The ability of FDA’s new leadership to advance new regulatory initiatives may be limited by the imperative to address the nation’s devastating opioid epidemic and to halt teen vaping of e-cigarettes, which has been linked to dozens of deaths. FDA also faces pressure to clarify its rules for producing and marketing cannabidiol products and to address concerns about the rise in antibiotic resistance and need for new treatments to combat infectious diseases. 

The importance of reliable systems for producing safe and high quality pharmaceuticals will remain in the spotlight as part of efforts to assure access to needed medicines. Congressional leaders are angry about continued shortages in critical therapies, as well as increased reliance on drug ingredients imported from China, India, and other countries. 

Despite this long list of priorities, Congress may launch an effort to further support biopharmaceutical R&D programs by advancing legislation to further initiatives authorized by the 21st Century Cures Act five years ago. This time, the legislators indicate an interest in promoting wider utilization of digital health technologies to improve access to new products and health services; on modernizing coverage policies for innovative, life-saving drugs; and on utilizing RWE more broadly in drug development.