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Big Pharma Handout or Drug Development Boon?

Solnick, Rachel MD

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doi: 10.1097/01.EEM.0000513594.18273.2d

    The flurry of activity around “repeal and replace” obscured one of the most important pieces of health care legislation since the Affordable Care Act snuck into law in the last days of President Obama's second term. Generally regarded as a bipartisan victory, the 21st Century Cures Act received unusually enthusiastic support across parties in an era of extreme political polarization.

    Supporters applauded the bill's focus on mental health, the opioid crisis, the National Institutes of Health, and the potential to introduce new drugs to the market more quickly. The act's complexity and the untested nature of several major elements, including the regulatory relaxation on drug approvals, have made the benefits of these reforms a bit murky.

    EPs are well aware of the pathologies of America's mental health system. Dealing with psychiatric emergencies is a small part of our training, but psychotic disorders like schizophrenia make up the highest proportion of ED visits (2080.8 per 1,000 Medicare beneficiaries) for chronic conditions. (Physician's Money Digest. Oct. 3, 2014;

    Several provisions may help tame the surge of mental health emergencies. The act establishes the new position of an assistant secretary for Mental Health and Substance Abuse to oversee these efforts. It also addresses underfunding through regulatory changes and funds avenues to increase outpatient capacity and community alternatives. It enforces existing parity laws that mandate insurance companies to provide the same coverage for mental illness as they would for physical conditions. The act also has criminal justice reforms, such as alternatives to incarceration for nonviolent offenders who are mentally ill.

    These reforms are welcome, but they do nothing to address the dearth of inpatient psychiatric beds, which decreased from 34 to 22 beds per 100,000 population (35%) between 1998 and 2013. (JAMA 2016;316[24]:2591.)

    The Cures Act is laudable in its support of the NIH, but the scope of its impact is small and limited to only a few projects, including the Cancer Moonshot, the Brain Research through Advancing Innovative Neurotechnologies (BRAIN) initiative, and the Precision Medicine Initiative.

    The act authorizes $4.8 billion on top of the current NIH budget to be doled out over 10 years. The NIH's annual budget is currently $32.3 billion, so the Cures Act's proposed contribution is a mere 1.6 percent increase to the budget per year, a number far lower than the five percent that was cut during the 2013 budget sequestration.

    Most discoveries of transformative drugs from the past two decades were made possible through public funding. Industry exclusive research produced the fewest breakthrough drugs. (Health Aff [Millwood] 2015;34[2]:286.) Despite the importance of basic science research, its funding is now under assault. Since 2003, the inflation-adjusted NIH budget has declined by over 20 percent, and scientists are about half as likely to receive research grants now compared with 1994. (Urban Wire. May 6, 2015;

    Further maligning this contribution, the money for this pledge robs preventive medicine by gutting 30 percent from the ACA's Prevention and Public Health Fund, which had funded diabetes prevention and immunization infrastructure among others. (Regulatory Affairs Professional Society. Nov. 28, 2016;

    The Cures Act is presented as a mechanism to accelerate drug discovery and availability, but critics are concerned this comes at the cost of the safeguards traditionally associated with FDA approval. Given the FDA's funding constraints, it is remarkably efficient at drug approval: All new drug applications are evaluated within six to 10 months, and a third of the drugs are approved based on trials with a median size of 760 patients. (N Engl J Med 2015; 372:2473.)

    It should give pause that the thrust for change came not from the agency charged with protecting public safety but from Washington legislators under the influence of 1,300 lobbyists, mostly representing pharmaceutical companies with much to gain from this bill. (PBS. Dec. 6, 2016; The primary bill sponsor, Rep. Fred Upton (R-MI), was the top recipient of contributions from pharma in the year prior to the bill's initial House vote, and he received more than $1.3 million in donations from health care political action committees. (Forbes. Sept. 22, 2016;

    Key provisions in the legislation allow readily available data to be a substitute for more rigorous research. Drug companies may seek approval for new indications of existing drugs using “data summaries” to support their claims instead of clinical trials. This method could obscure information about a drug's effectiveness for the new indication, allowing drug companies to milk products for new uses without investing in new research. They can also get approval through real-world evidence from a variety of sources, including observational studies, registries, claims, and patient-centered outcomes research activities.

    The act also increases the use of surrogate endpoints such as biomarkers and tumor progression indicators. Supporters hope this encourages trials of smaller sizes and durations, incentivizing investment. But this approach has created drugs that improved biomarkers but worsened clinically significant endpoints such as rosiglitazone, which lowered HbA1c but increased cardiovascular mortality. This is also a major issue for expensive oncology drugs. One study found that 74 percent of cancer therapies of the last decade were approved based on surrogate endpoints, but most did not increase patient survival. (MedPage Today. Oct. 26, 2014;

    The medical establishment relies on the public's trust to practice medicine. Numerous cases could undermine that trust, such as a recent increase in device recalls for dangerous defects, or the increase in the pure commercialism of pharmaceuticals. ( Nov. 18, 2016; Expedited drug approval appears to be a boon to research, mental health, the opioid crisis, and drug development, but it should prompt inquiry into the complex balance between fiduciary responsibility, industry influence, and patient empowerment.

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