reposted from Health Affairs Blog
“Orphan drug” is a bit of misnomer, or at least verbal shorthand. It’s not the drug that’s “orphan” or rare; rather, the disease is the orphan, meaning that it might not affect enough people, and provide enough of a market, to incentivize a drug company to look for therapies. That’s the premise behind the 1983 Orphan Drug Act (ODA), which offers incentives for companies to develop therapies for diseases that affect less than 200,000 people in the United States.
In a recent Knowledge@Wharton blog post, LDI Senior Fellows Daniel Polsky and Scott Harrington weigh in on the fate of “Big Pharma” under the Trump Administration. This issue will take center stage as the confirmation hearings begin for Scott Gottlieb, the nominee to head the Food and Drug Administration (FDA).
“Just in case,” the oral surgeon said, as he prescribed the opioid hydrocodone for my 17-year-old son, who just had his wisdom teeth out. “But you might try Motrin first,” he added. Not knowing what the next hours, or days, would bring, we filled the prescription for 20 pills. We’d heard that the pain on the second day might be worse than the first.
To address the crisis of opioid misuse, abuse, and overdose, every state but Missouri has implemented a prescription drug monitoring program (PDMP) that collects data from retail pharmacies on all dispensed controlled substances. PDMPs can help prescribers identify patients who might misuse or divert controlled substances, as well as patients who need substance abuse treatment.
Developing a value framework for cancer drugs can sound like an arcane exercise without much relevance to clinical care. Restate it as a question of how, and how much, to pay for cancer drugs, and you’ve got everyone’s attention.
It seems self-evident: one way to address the epidemic of opioid deaths is to make prescription opioids harder to misuse. OxyContin, for example, is especially dangerous when it is crushed for ingestion, inhalation, or injection. In 2010, the FDA approved a reformulated, abuse-deterrent version of OxyContin that made the pill difficult to crush or dissolve. The new version immediately replaced the old one, marking a substantial reduction in the supply of abusable prescription pain relievers.
“Pay more for drugs that do more.” Although few would argue with the concept of paying for value, the mechanism for doing so has thus far eluded our multi-payer, market-based system. The Gant Precision Cancer Medicine Consortium at the University of Pennsylvania looked past US borders to learn about mechanisms in other countries, in its quest to recommend sustainable frameworks for valuing precision cancer drugs.
Biomarker-Defined Subsets of Common Diseases: Policy and Economic Implications of Orphan Drug Act Coverage
In PLOS Medicine, Aaron Kesselheim and colleagues, including LDI senior fellow Steven Joffe, investigate the policy and economic implications of the Orphan Drug Act of 1983, and examine the circumstances surrounding a drug’s discovery and development, secondary approvals, off label uses, subsequent revenues, and the reported monthly cost of biomarker-defined disease subsets. The Orphan Drug Act of 1983 was intended to incentivize the development of pharmaceutical products for rare diseases by providing manufacturers with the opportunity to earn grants, tax credits, free waivers,...
In the Journal of Health Economics, Abby Alpert examines anticipation effects of Medicare Part D, which went into effect two years after it was signed into law. This potentially gave forward-looking individuals time to change their drug consumption behavior in anticipation of future subsidized drug coverage. This approach contrasts with prior studies of Part D, which do not take the possibility of behavioral responses to the announcement into consideration, thus...
Medicare Part D beneficiaries can face as much as 33% coinsurance for some drugs listed in a “specialty tier,” which can result in thousands of dollars in out-of-pocket costs. The concern, of course, is that this level of cost-sharing creates a barrier that may put patients at risk for poor outcomes because they cannot afford the drugs they need.
A few weeks ago, I was chatting with another physician in the emergency department after finishing my shift. He had thrown a birthday party for his nine-year-old son Jake, who had recently recovered from a sports-related ankle fracture. “During the party, a parent pulled me aside and said he had recently injured his shoulder doing yard work,” my colleague said. “I assumed he wanted me to examine his shoulder or give him advice – but instead he explained that he didn’t have time to see his physician, and he asked whether we had any Vicodin left over from Jake’s surgery.
[cross-posted from the Health Cents blog on philly.com]
Prescribers are drawing a lot of attention as a key target of initiatives to combat the opioid crisis. This week, the US Surgeon General, Vivek Murthy, took the unprecedented step of sending 2.3 million clinicians a letter calling for a national movement to turn the tide on the opioid crisis.