PhRMA companies push hard on House bill to ease testing of new drugs

(U.S. Food and Drug Administration)

Pharmaceutical interests are pushing hard in favor of a bill that could speed up Food and Drug Administration (FDA) approval of new drugs. But the bipartisan 21st Century Cures Act, which was approved last month by the the House Energy and Commerce Committee (the members of which have all sponsored the bill) and could reach the House floor by the end of June, has some health advocates up in arms.

A primary concern is that the bill would encourage the FDA to move away from its reliance on controlled trials — and specifically those using randomization — as the main marker for determining whether a drug is safe and effective enough to go on the market. Instead, it suggests that “clinical experience,” which includes observational study, patient anecdotes, and other such methods, be given more consideration. Clinical experience is and has been used as supplemental evidence by the FDA, but not as the primary method for determining a drug’s safety and efficacy.

Dr. John Powers, the former lead medical officer for antimicrobial development and resistance issues at the FDA and a member of the National Physicians Alliance’s FDA task force, said that such a method was akin to using “Star Trek medicine mentality” that relies on unsubstantiated belief instead of valid scientific evidence.

“The number of ways patients can be different from each other that can influence patient-centered outcomes independent of treatment is innumerable,” Powers told OpenSecrets Blog, adding that with clinical experience, one “can’t take into account all complexities that happen in the human body.”

The 21st Century Cures initiative was launched on April 30, 2014 by House Energy and Commerce Committee Chairman Fred Upton (R-Mich.) and committee member Diana DeGette (D-Colo.), and a white paper on the initiative was released the following day. The legislation, which was introduced by Upton last month (with a discussion document released in January), currently has 138 sponsors — most of whom boast the pharmaceuticals/health products industry as one of their top 20 contributors in the 2014 election cycle.

That includes all of the bill’s original sponsors: $302,700 for Upton, $184,500 for Joe Pitts (R-Pa.), $174,072 for Frank Pallone (D-N.J.) — the Committee’s ranking member, $112,949 for DeGette and $72,044 for Gene Green (D-Texas).

Pallone’s district contains the headquarters of pharmaceutical giant Johnson & Johnson, whose PAC gave him $10,000 in the last election cycle.

The company has lobbied on the initiative as well — as have numerous pharmaceutical companies and trade groups, which bring vast resources to bear on anything going on in Washington that might affect their interests. In fact, the pharmaceuticals/health products industry consistently spends more on lobbying than any other interest group. Big spenders over the past two years that have mentioned the initiative most frequently in lobbying reports include drug companies Sanofi (which spent nearly $6 million on lobbying in 2014, $1.8 million in the first quarter of 2015), AstraZeneca PLC ($2.9 million in 2014, $503,000 in 2015) and GlaxoSmithKline ($4.4 million in 2014, $1.7 million in 2015) — as well as industry associations such as the Biotechnology Industry Organization ($8.3 million in 2014, $2 million in 2015) and the Pharmaceutical Research & Manufacturers of America (PhRMA) ($16.6 million in 2014, over $5.4 million in 2015).

Both name brand and generic drug companies, often at odds, want the bill to go through. The Generic Pharmaceutical Association‘s President and CEO Ralph G. Neas referred to it in a press release as a “bipartisan achievement that should enhance the drug approval process and significantly boost critical research and development efforts.”

In the first quarter of 2015, 208 entities lobbied on the initiative, a list with countless pharmaceutical and biotechnology-centered companies.

A spokeswoman from the House Energy and Commerce Committee maintains that safety will not be compromised by the bill. “Modernizing health care innovation and maintaining the highest standards of safety and efficacy are not mutually exclusive,” she said. “This is why the committee brought every key player to the table — including the FDA, the NIH, top scientists, researchers, and thought leaders — for more than a year to carefully develop this vital legislation.”

Former FDA commissioner Andrew von Eschenbach, who served under former President George W. Bush, along with Manhattan Institute scholar Paul Howard, wrote in support of the bill in the Morning Consult this week. “Armed with massive computing power and modern analytics, advanced diagnostics like functional MRIs, and electronic medical records, researchers can now compile massive databases containing genomic variants and patient health outcomes that can allow them to rapidly identify, test, and validate hypothesis about effective disease fighting strategies,” von Eschenbach and Howard write. “With this information in hand, it is unnecessary and perhaps even unethical to demand that we conduct traditional large, blinded and randomized controlled trials for precision medicines supported by a variety of mechanistic and pre-clinical data.”

But two other former FDA commissioners have concerns with 21st Century Cures — even though it does allot over $10 billion to the National Institutes of Health (NIH) and $550 million to the FDA over five years. Margaret Hamburg, a recently departed commissioner, said  in a March speech, that “there’s a misperception that you might be able to speed up innovation by lowering the standards for safety and efficacy, and I think that would be a terrible mistake that would not only just damage patients, but industry as well.” David Kessler, who served as commissioner during both the George H.W. Bush and Bill Clinton administrations, wrote this month in a joint New York Times op-ed that the act “could substantially lower the standards for approval of many medical products, potentially placing patients at unnecessary risk of injury or death.”

And the National Center for Health Research, a think tank involved in research “used to encourage new, more effective programs, policies, and medical treatments,” is also worried about the legislation. In addition to uneasiness about relying on clinical experience, other concerns include parts of the Act that would allow new drugs to be approved for select individuals based on preliminary data, create financial incentives for the use of new antibiotics, and could allow companies to use limited evidence in order to get FDA approval for new medical devices.

So far, it’s only in the House where the two sides are tussling over whether the bill is a salutary prescription; no companion measure is yet pending in the Senate.

Correction, 6/16: This post has been corrected to reflect the fact that formal 21st Century Cures legislation was not introduced until the second quarter of 2015; the companies mentioned lobbied on the initiative.