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Jill Wechsler is Pharmaceutical Technology's Washington Editor, email@example.com.
President Trump calls for faster FDA approvals and lower drug prices. Jill Wechsler reports.
Pharmaceuticals were not a main topic in President Donald Trump’s first formal address to a joint session of Congress on February 28. He did, however, call for more “miracle” cures for rare disorders, amidst cutting remarks about excessive FDA regulation of medical products and too-high prescription drug prices.
The President drew loud applause on all sides with his promise to “bring down the artificially high price of drugs ... immediately.” He considers such action important for achieving the larger goal of repealing Obamacare and replacing it with a program that will ensure access to coverage, lower costs, and improve care.
Without providing many specifics, Trump said that the way to make health coverage “available to everyone is to lower the cost of health insurance, and that is what we are going to do.” Some key strategies are to give states more control over the Medicaid program, limit medical lawsuits, ensure access to coverage for people with pre-existing medical conditions, and permit insurers to sell coverage across state lines.
Trump also highlighted the importance of discovering more therapies for critical rare diseases, citing the discovery of a life-saving treatment for Pompe disease a decade ago. But he linked prospects for developing more such “miracles” to efforts to reform FDA’s “slow and burdensome approval process.” The President called for slashing the “restraints” at FDA and across the government to speed the approval of more drugs.
While it’s great for the White House to recognize the importance of treating serious rare conditions, the National Organization for Rare Disorders (NORD) fired back that FDA is doing a terrific job of speeding new treatments through the approval process and supporting flexible R&D approaches in evaluating innovative therapies. What is really needed to expedite orphan drug development, said NORD, is adequate FDA funding to build staff expertise on rare diseases and reauthorization of drug user fees, with provisions to integrate rare disease experts into the drug review process and to further consider patient perspectives and preferences in orphan drug development.
In fact, NORD stated, strong safety and effectiveness standards for drugs and biologics are “crucial” to ensuring that individuals with rare diseases obtain the same high-quality therapies as do other patients; to “weaken the standards will only threaten our population with unsafe, ineffective therapies.” FDA already shows “an incredible amount of flexibility in reviewing and regulating orphan drugs,” but the process can be improved with adequate resources for the agency and positive reforms.
Observers speculated that Trump’s criticism of FDA may be setting the stage for a new FDA commissioner with a mandate to limit regulations and reduce the scope of clinical evidence required to gain market approval for new medical products.