Outlook 2018: Pharma/Life Sciences Law and Policy
Will the 21st Century Cures Act further its aim to boost drug and device development in 2018? That's one of the questions the life sciences industry and attorneys are asking for the new year. The law provides extra money for research and development—including ambitious research into cancer and a landmark precision medicine project involving 1 million Americans—and spurs more use of real-world evidence to support product approvals.
21st Century Cures
Cures also adds funding for precision medicine, and the NIH's centerpiece effort in the field, the All of Us Research Project, is to begin this spring. “This is a really big positive to get more and more people engaged in taking part in clinical trials,” Mary Woolley, president of Research America, said.
Gene editing and CRISPR-based therapeutics will continue to garner attention as both the products and the intellectual property landscape relating to them evolve.
Gene editing refers to a group of technologies that give scientists the ability to change an organism's DNA. A method known as CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats) has enabled gene editing to be faster, cheaper, more accurate, and more efficient than other methods. It essentially allows scientists to cut out unwanted sections of DNA and replace them with desirable sequences.
Analysts call it one of the most important inventions ever and cite a potential value of billions of dollars. The first U.S. clinical trials applying gene editing technology to mutations causing serious diseases could be up and running in 2018.
The partisan environment during a midterm election year will make it especially difficult for Congress to follow the normal process for passing bills known as regular order, Research America's Woolley said.
“It's inescapable that next year it's going to be even more intensely political than the one we're in right now,” Woolley said. “But this year, trying to get to anything close to regular order is like climbing Mt. Kilimanjaro.”
When Congress doesn't act, it creates a ripple effect throughout the federal government, as agencies remain uncertain about whether they will be funded on endless continuing resolutions or face reorganization.
What does that mean for research? “The relatively easy stuff is not going to necessarily get done so that people can't work on the emerging challenges,” she said.
These challenges come amid some of the largest statutory and regulatory changes affecting the biomedical industries.
Few situations highlight this unpredictability better than the status of the Common Rule revisions, the biggest changes in three decades to regulations governing human research subjects. Those changes kick in Jan. 19—possibly.
A proposed rule to delay the effective date to 2019 is under White House Office of Management and Budget review. “The current effective date stands unless there is a final change to the rule,” Lisa Nichols said. She is the director of research and regulatory reform for the Council on Governmental Relations, which represents research institutions. Most institutions are preparing to comply with the revisions by January.
There's no further guidance from the HHS on the Common Rule revisions other than the revised rule itself, which includes some complex changes, such as broad consent for future research on biospecimens and a new exemption if the Health Insurance Portability and Accountability Act privacy rule applies. “There are parts of the rule that are very hard to implement without guidance,” said Heather H. Pierce, the Association of American Medical Colleges’ senior director of science policy and regulatory counsel. “That doesn't mean they can't be implemented, but they won't be implemented consistently.”
At the same time, the FDA hasn't updated its regulations. “If it does go into effect as intended on Jan.19,” Pierce said, “a federally funded clinical trial that is FDA regulated will be under [both] the old and new Common Rule because the FDA regulations are so closely aligned with the old Common Rule.”
21st Century Cures (Pub. L. 114-255) requires the HHS and the FDA to harmonize their regulations, and FDA officials have said they are working on it.
Regenerative medicine, which also has Cures money, will continue to be important as researchers are recruiting for gene therapy trials. Emergence of the CRISPR-Cas9 technology has advanced the field by making gene editing faster, cheaper, and more accurate.
Still Waiting on Diagnostics
For the medical device industry, what the FDA should do regarding laboratory-developed tests is pressing, but remains unsettled.
“The federal government, as a whole, including Congress, has been unable to come up with an appropriate regulatory pathway that both protects patients and advances innovation,” Bloomberg Law board member Bradley Merrill Thompson of Epstein Becker & Green said.
“It's rather unbelievable that such an important issue should take so long to resolve,” he said.
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