A boost for authorized funding for the National Institutes of Health (NIH), a new $10 billion innovation fund at NIH and a paring back of many drug development incentives are among the many changes unveiled in the second draft of the 21st Century Cures legislation.
Released on April 29, 2015, by the U.S. House Energy & Commerce Committee, the latest iteration slims down to 200 pages, or about half of the nearly 400 pages of the first cut produced in late January. In a bid to gain Democrat support, a number of provisions are gone and some new ones have appeared, including a reauthorization of the NIH for three years — Fiscal Years 2016 through 2018 — that would encourage an increase in appropriations funding for each of those years. The bill, however, still does not contain any budget offsets for the new spending and includes a number of “placeholder” and bracketed provisions in several key areas, indicating that much work lies ahead.
The committee released the draft the day before it is scheduled to hold a hearing of Administration witnesses to discuss themes of Cures. It comes out nearly a year to the date since Committee Chairman Fred Upton (R-MI) and Rep. Diana DeGette (D-CO) launched the effort on April 30, 2014.
Supporting Biomedical Research
The bill authorizes funding of $31.8 billion for NIH in FY 16, $33.3 billion in FY 17 and $34.85 billion in FY 18 — a significant change from the flat lines that have defined NIH appropriations now for several years. It also establishes an innovation fund and appropriates $2 billion annually for five years as long as NIH base appropriations do not dip behind a “to-be-inserted” level. The bill also names precision medicine and programs focused on emerging sciences as those eligible for support via the fund, but contains a glaring “other” that will shine as a beacon to many parties interested in tapping such a fund.
The updated draft retains provisions to allow the National Center for Advancing Translational Sciences (NCATS) to fund more later-stage projects (today its authorities are limited to Phase 1 and 2A trials) and to provide NCATS with “DARPA-like” transactional authorities it can use to operate in a more nimble and flexible manner, a reference to the innovative Defense Advanced Research Projects Agency. It also includes some ideas likely to continue to rattle some in Bethesda, such as requirements for NIH to develop a strategic plan (including areas of prioritization) as well as term limits — now five years from four in the first draft — for Institute and Center directors.
True to the spirit that launched Cures a year ago, many provisions focus on accelerating the pace of innovation by requiring greater levels of collaboration and data sharing as well as uses of technology to streamline current processes. Sections in this space include ones to collect de-identified clinical trials data and to standardize the format of such data, as well as a proposal to create a public-private partnership to “collect, maintain, analyze, and interpret data on the natural history of diseases, with a particular focus on rare diseases.”
The new draft contains the provision included in the original version that would support greater use of central Institutional Review Boards (IRBs) and other similar reforms with the aim of shortening the clinical research timeline and reducing development costs. It also retains the provision to develop a biomarker qualification pathway at FDA and a direction for guidance on precision medicine, though both sections are bracketed indicating that significant discussions remain ahead. Another heavily bracketed section is focused on updating FDA regulation of health IT software.
In a nod to FDA calls for increased resources, the latest draft updates existing law to provide FDA with greater flexibility and resources to hire review staff.
Proposals to further study and advance alternative clinical trials designs and to streamline the data review process are included in the updated draft, though the latest version is noticeably lighter around incentive provisions that drew some stakeholder and Democrat resistance earlier in the year. However, the legislation contains a provision on “repurposing drugs for serious and life-threatening diseases and conditions” that would suggest a potential slot for some of these missing provisions to return in some form later, such as the dormant therapies concept.
Access to Therapies
In a nod to concerns about patient access to investigational drugs, the draft includes a slimmed down and bracketed provision that would require industry sponsors to disclose their expanded access/compassion use policies, including the company’s point of contact, its procedures to request a product, the length of time to process requests and the criteria a sponsor will use to evaluate requests.
The draft would create a Medicare Pharmaceutical and Technology Ombudsman office to respond to industry grievances. And in recognition of the growing threat of prescription drug abuse, it would require the Medicare Part D programs to establish a Safe Pharmacy Access Program to identify potential abusers and to restrict their access to excess medication.
Politics and Path Forward
One marked difference between the release of the update and the early draft in January is the bipartisan support behind the latest version. While Democrats sat on the sidelines in January, Committee Ranking Member Frank Pallone (D-NJ), Health Subcommittee Ranking Member Gene Green (D-TX) and Rep. DeGette joined Chairman Upton and Health Subcommittee Chairman Joe Pitts (R-PA) in the April 29, 2015, release.
But while bipartisan spirit is back, the reality is that the number of gaps and unresolved issues — including how the proposed increased spending will be offset or paid for and the need to ultimately reconcile any House-passed bill with what may come from Senate counterparts — means that much work remains ahead on the pathway to 21st Century Cures.