Trump's new prescription drug plan is incomplete -- here are two ways to make it better
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On Friday President Trump in the White House Rose Garden briefly outlined the four key aspirational strategies of his "blueprint to lower drug prices": "improved competition, better negotiation, incentives for lower list prices, and lowering out-of-pocket costs."
Overall, we agree with law professor Rachel Sachs, who was quoted in the Washington Post saying this about the administration’s 39-page American Patients First plan: “With all the buildup the administration has given it, the president’s speech was deeply underwhelming. There is very little new in the administration’s plan, and little if anything that will make a difference in the near future, as the president has promised.”
Conspicuously absent were the two commitments for immediate action in the president's campaign speeches -- government negotiation of Medicare drug prices and importation of drugs from oversees.
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We have two proposals that would increase the number of different drugs in the marketplace, which could increase both competition and patients’ access to new medicines, and also alleviate critical shortages of old drugs in the bargain.
First, the Food and Drug Administration should modernize its drug review process to approve drugs more rapidly in order to increase the pool of available treatments and vaccines. One way that could be accomplished is by greater use of “accelerated approvals,” which permit the FDA to issue what amounts to a limited, or “conditional approval" of a new drug that is intended for a "serious or life-threatening disease" and for which there is an "unmet medical need."
Fluad had been in use in Italy since 1997 and approved in more than three dozen countries. That 18-year delay in availability in the United States likely resulted in many avoidable hospitalizations and deaths.
Such an approval can be achieved more rapidly because it is based on clinical trials that show improvement in a "surrogate endpoint" that is believed to correlate with clinical benefit but have not yet shown efficacy on a “definitive” health endpoint such as increased longevity or reduction in the incidence of heart attacks. Examples of surrogate endpoints are the shrinking of a tumor or improvement in a laboratory value such as blood urea nitrogen, or BUN, a measure of kidney function.
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Following accelerated approval, the drug sponsor (company) must perform confirmatory trials to prove to the FDA that the medicine is effective in meeting a definitive clinical endpoint (such as greater longevity), at which time the approval is converted to a standard, unconditional approval. If the studies are not done or fail to provide such confirmation, the FDA can withdraw the drug from the market.
Our second proposal – reciprocity of drug approvals based on approval by foreign regulatory agencies that have approval processes comparable to the FDA's -- is another way to lower regulatory costs and increase competition and access to a greater number of drugs on the market in the United States. It would also benefit patients directly, because the detrimental effects of FDA delays in approving certain new drugs already available in other industrialized countries are well documented.
An example was the FDA’s approval in 2015 of Fluad, a flu vaccine that contains an adjuvant that boosts the immune response. It is intended for use in the elderly, whose immune response to flu vaccines is typically poor. According to the CDC, “It has been estimated that between 71 percent and 85 percent of seasonal flu-related deaths have occurred in people 65 years and older and between 54 percent and 70 percent of seasonal flu-related hospitalizations have occurred among people in that age group."
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Fluad had been in use in Italy since 1997 and approved in more than three dozen countries. That 18-year delay in availability in the United States likely resulted in many avoidable hospitalizations and deaths.
Reciprocity of drug approvals with certain of the FDA’s foreign counterparts with comparable drug approval regimes would allow an approval in one such country to trigger approval in the U.S. upon application by the foreign drug manufacturer or licensee (subject to the creation of approved labeling in appropriate format, etc.). That would make more drugs available sooner in the United States, increase competition and put downward pressure on prices. This would fulfill Mr. Trump's campaign promise to allow the importation of foreign drugs.
Reciprocity of foreign approvals would also help to alleviate the pressing problem in the United States of shortages of certain critical drugs, many of which have been essential in medical practice for decades. The majority are generic injectable medications commonly used by EMTs and in hospitals, including analgesics, cancer drugs, anesthetics, antipsychotics for psychiatric emergencies, and electrolytes needed for patients on IV supplementation.
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Hospitals are scrambling to assure adequate supplies of drugs that are in short supply, or to find substitutes for them. The FDA is severely limited in what it can do to address shortages. The agency’s app to enable health care providers to keep current on shortages informs them about the problem but doesn’t actually remedy it. Reciprocity of approvals would make numerous needed alternative drugs available. It could have been in place decades ago if only the FDA had met its long-standing commitment to pursue it through the International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH).
The ICH’s agenda (supposedly) includes reciprocity of drug approvals among certain governments, but generations of FDA officials have resisted any such “delegation” of their responsibilities. When a senior European regulator was asked about the extent of the FDA’s cooperation on this issue, she quipped, “It’s like discussing the Thanksgiving dinner menu with the turkeys.”
The FDA has improvised procedures for importing drugs approved and marketed abroad that have not been approved in the U.S., but that “enforcement discretion” approach – a kind of ad hoc reciprocity -- is legally questionable. In a footnote to the agency’s October 2013 Strategic Plan for Preventing and Mitigating Drug Shortages, FDA acknowledged its awareness of a relevant recent court decision, Cook v. FDA (D.C. Circuit, Case No. 12-5176), in which the court prohibited FDA from using enforcement discretion to permit the importation of an unapproved drug for capital punishment execution, because the law is clear that an unapproved drug cannot come through U.S. Customs for marketing. The FDA’s terse comment, “We are currently reviewing the decision in the context of our drug shortages program,” belies the existential importance of that decision.
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The White House Domestic Policy Council's Katy Talento said on Wednesday about the forthcoming drug-pricing plan, “This is a fearless president and he doesn't know or care why things have always been done…” Well, we do know and care. The bottom line is that we need quicker and affordable access to medical treatments. More accelerated approvals and reciprocity of medical-product regulatory decisions would move us in that direction -- and also make good on an important campaign promise.
John J. Cohrssen is an attorney who has served in a number of government posts in the executive and legislative branches of the federal government, including as associate director of the President’s Council on Competitiveness (Bush 41), and counsel for the House Energy and Commerce Committee.