“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.
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.
“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.
Is cancer “special” in terms of the public view and the value placed on potential treatment and cures? The multidisciplinary Penn Precision Cancer Medicine Consortium discussed whether cancer is treated differently from other diseases, and then considered the more normative question of whether it should be treated differently.
The eye-popping price tags on some new cancer drugs pose two fundamental questions: are the drugs worth their cost, and if they are, how can we afford them? The Penn Precision Cancer Medicine Consortium considered cost trends and drivers in the second of its conference calls leading up to an in-person conference in May.
A new multidisciplinary consortium of more than 20 experts and stakeholders has come together at Penn to address the promise and challenges of precision cancer medicine. Through multiple conference calls culminating in a conference in May 2017, the group will tackle the hard questions that precision medicine raises for patients, providers, and payers. This is the first in a series of posts on the consortium’s work.
Adjuvant Chemotherapy Use and Health Care Costs After Introduction of Genomic Testing in Breast Cancer
The promise of personalized genomic testing is that it can reduce unnecessary care and costs by predicting which patients are most likely to benefit from a treatment. In this study of actual treatment patterns, LDI Senior Fellows Andrew Epstein and Peter Groeneveld and colleagues investigate how genomic testing of women with early-stage breast cancer affects subsequent chemotherapy use and medical spending in the year after diagnosis. After surgery, women with early-stage breast cancer face the decision of whether to undergo expensive and potentially toxic chemotherapy to prevent recurrence, although most will not have a recurrence. The 21-gene recurrence score test (RS) was developed in 2004 to predict this risk, and its use in clinical medicine is increasing. Epstein and Groeneveld find that genomic testing is associated with decreased use of chemotherapy and lower costs in younger patients, and slightly increased use of chemotherapy and higher costs in older patients. Genomic testing in actual practice may “rule out” chemotherapy in younger women, and “rule in” chemotherapy in older women.