Sept. 29, 2015
Snake Oil Alert: U.S. Senate Ready to Roll Out 21st Century Cures Bill
Senate HELP Committee Prepares to Release Its FDA Overhaul Proposal; Beware of Talk of Bipartisanship
The U.S. Senate’s Health Education Labor and Pensions (HELP) Committee is expected to release its version of the 21st Century Cures Act soon. If the bill resembles the version approved in July by the U.S. House of Representatives (H.R. 6), the public should be very concerned.
The House measure – touted as an overhaul of the way the U.S. Food and Drug Administration (FDA) approves pharmaceutical products and high-risk medical devices – is being sold as enabling scientific innovation and medical breakthroughs for America. But it actually would undermine efforts to approve safe and effective drugs and medical devices.
Those pushing it – U.S. Reps. Fred Upton (R-Mich.) and Denise DeGette (D-Colo.) – have ransomed critical funding for medical research in exchange for the bill’s passage.
Still worse, the bill has been reported as a commonsense, bipartisan compromise as our nation searches for cures to treat rare conditions and diseases, rather than the grab bag of goodies for Big Pharma and medical device companies that it is.
In fact, Democratic opposition surfaced during the House negotiations, but weakened when specific exclusive drug marketing perks for the pharmaceutical industry were scaled back (although some problematic provisions remain). Another selling point for Democrats was the very popular increase of $8.75 billion over five years to fund the National Institutes of Health (NIH), currently funded at $30.3 billion per year.
Industry interests continue to mount campaigns to push this initiative. Supporters of the House legislation received hundreds of thousands of dollars in contributions from the pharmaceutical industry and the medical device industry last year, according to the nonpartisan Center for Responsive Politics. The Pharmaceutical Research & Manufacturers of America (PhRMA) spent $16.6 million in 2014 lobbying on 21st Century Cures and other legislation, and more than $5.4 million in 2015, much directed toward this legislation.
What will the Senate version contain? It’s too soon to tell, but earlier this year, HELP Committee Chair Sen. Lamar Alexander (R-Tenn.) and Sen. Richard Burr (R-N.C.) released a report, “Innovations for Healthier Americans,” to launch their own effort to revamp the FDA. The Senate version of 21st Century Cures will stem from this report and initiative.
The report implies that the senators support deregulation of a vast scope of American medicine and health care. For example, it implicitly calls for lowering the FDA’s standards for drug approval.
Senate leaders aim to follow an ambitious timeline – they want to enact it into law by the end of the calendar year.
As release of the Senate version nears, it’s important that your readers understand the potential consequences for public health if specific House provisions become part of this fast-moving piece of legislation.
The House bill calls for dangerous regulatory changes and financial incentives for pharmaceutical and medical device companies. These changes and incentives would put patient safety at risk and threaten public health. They are detailed below.
1. 21st Century Cures would further undermine the FDA’s ability to ensure the safety and efficacy of medical devices (Sections 2222 and 2221).
Section 2222 would pressure the FDA to approve new high-risk medical devices based on case studies or medical journal articles alone. High-risk devices should not be approved on the basis of uncontrolled case studies of just one, two or even a series of patients (in essence, clinical anecdotes). Medical journal articles often omit critical information because of space limitations or concerns that admitting shortcomings in study design or conduct will make it difficult to get the article published. FDA reliance on journal articles as the sole basis for device approvals, as outlined under Section 2222, could prevent the FDA from learning of important problems with clinical testing, which could lead to serious patient harm.
Section 2221 would establish a mechanism for device manufacturers to make changes to even the highest-risk devices (such as artificial heart valves) without first notifying the FDA or documenting that the modified device remains safe and effective.
2. 21st Century Cures would effectively lower FDA approval standards for antibiotics and antifungals (Section 2121).
Included in the legislation is the Antibiotic Development to Advance Patient Treatment (ADAPT) Act (Section 2121), which presents a fast-track pathway for FDA drug approval based on data from animals, test tubes, mathematical modeling and small, early-stage clinical trials in humans with diseases – rather than larger, later-stage “Phase III” trials that have long been the gold standard for drug approval. Well-designed randomized controlled Phase III clinical trials are critical for weeding out bad drugs; more than a third of the drugs that enter Phase III testing fail to gain FDA approval. Approval standards already are dangerously low for antibiotics, and many risky antibiotics are being rushed through the approval process without strong evidence of safety and effectiveness. Two of the antibiotics approved by the FDA in the past decade, bedaquiline (Sirturo) and tigecycline (Tygacil), carry black-box warnings because they increased the risk of death in clinical testing. Congress should be looking for ways to strengthen approval standards for antibiotics, not chip away at them further.
While ADAPT provisions fail to truly address antibiotic resistance and lower FDA standards for approving antibiotics and antifungals, more effective approaches to address these problems are contained in the Helping Effective Antibiotics Last (HEAL) Act (H.R. 931), sponsored by U.S. Reps. Rosa DeLauro (D-Conn.), Louise Slaughter (D-N.Y.) and Grace Meng (D-N.Y.).
3. 21st Century Cures would weaken reporting requirements of the Physician Payment Sunshine Act, allowing for secret influence by pharmaceutical and medical device companies over the practice of medicine and medical education (Section 3041).
Section 3041 of the bill would create an exemption under the Physician Payment Sunshine Act for drug and medical device manufacturers, allowing them to keep secret any speaker fees or gifts they give to doctors that are intended for “continuing medical education” purposes, regardless of cost. Speaking fees can be a lucrative source of income for physicians, and under the bill, gifts intended for medical education may include lavish items, such as admission to an expensive conference at a fancy resort. An additional provision would exempt expensive medical textbooks and journals from reporting requirements by classifying them as “educational materials that directly benefit patients.” These gifts should be reported under the Physician Payment Sunshine Act.
4. 21st Century Cures would hasten the rise of resistant superbugs by giving hospitals incentives to use new antibiotics rather than conserving them for appropriate use (Section 2123).
Section 2123 of the bill would give hospitals a Medicare reimbursement incentive to use new antibiotics. Such a provision would encourage the overuse of antibiotics by giving hospitals a financial bonus each time these drugs were prescribed, rather than encouraging hospitals to use older, effective antibiotics before using new ones that may not be medically necessary. This practice would only speed the rise of antibiotic-resistant infections, as bacteria will increasingly become resistant to these new drugs as they are used more often.
5. 21st Century Cures would bar generic entry of medicines into the market for a longer period and would deny patients access to affordable, life-saving medicines (Section 2151).
Section 2151 provides six months of patent-like market exclusivity in addition to a drug’s existing patent and other exclusivity periods if a new “orphan” indication is approved that involves treatment of a rare disease. This provision would extend the exclusivity period for all conditions the drug is FDA-approved to treat, not just the rare condition, thereby increasing health care costs and limiting patient access to new drugs for a potentially broad range of diseases.
As written, this provision would delay patients’ access to affordable, life-saving medicines. The Congressional Budget Office estimates that this six-month extension of marketing exclusivity for certain pharmaceutical products would cost the federal government about $869 million between 2016 and 2025 (this does not include additional costs to private insurance and individual patients). We believe that costs to the federal budget would increase sharply over time as drug manufacturers identify more ways that blockbuster drugs can be tailored for rare diseases.
Senate lawmakers should stay far away from the toxic House bill and instead put forward language that restores federal investments in basic science and research while upholding standards to ensure safe and effective treatments are approved for patient use.
There is much to be done to both protect public health and advance medicine and research in the 21st century. For example, Congress could improve the transparency of clinical trials data and ensure medical device regulations are based on science, not simply industry assertions that their products are safe and effective. We could appropriately invest in the NIH and FDA to ensure they have the necessary resources to pursue their purpose. We hope these proposals will continue to be addressed during the next phase of this initiative.
As you cover the next phase of the 21st Century Cures Act, we look forward to the opportunity to speak with you.