OBR Daily Commentary

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FDA: 2019 Continues Uptick in Orphan Drug Approvals

(Regulatory Focus) Jan 6, 2020 - Similar to 2017 and 2018, the US Food and Drug Administration (FDA) in 2019 approved an increasing number of orphan drugs and biologics, as well as drugs targeted at specific cancers.

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William McGivney, PhD (Posted: January 13, 2020)

quotesIn Oncology, drug/biologic development is clearly in a world of orphan diseases. The FDA defines an orphan disease as one that involves less than 200,000 patients diagnosed or afflicted with the disease in the United States. As such. In general terms, all but the major tumor types can be considered to be orphans. As we progress deeper and deeper into targeted therapies, almost all tumor types and specific indications will be considered to be orphan diseases. The advancement in our knowledge about targets and biomarkers that predict responsiveness of a patient’s cancer will be major determinants of specific diagnoses. As patient populations become smaller and smaller, the pricing pressures on the cost of agents that will be approved for such indications will be great. We will have important increases in the number of targeted therapies in the coming months and years. As such indications will become more "orphan-orphan". quotes

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Why The Cancer ‘Moonshot’ Has Been So Disappointing

(Washington Post) Sept 24, 2019 - Rather than a concerted effort to cure cancer, pharmaceutical companies pour resources into “orphan drugs.” Cancer therapeutics have become a big business for the pharmaceutical industry. Since 2013, cancer drug expenditures in the United States as a percentage of total U.S. prescription spending has increased by almost 60 percent, and the number of cancer drugs in late-stage trials increased more than 60 percent during the same time frame. Major pharmaceutical houses such as Pfizer, GlaxoSmithKline and AstraZeneca are reportedly “pivoting to cancer.”

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William McGivney, PhD (Posted: September 30, 2019)

quotesI am somewhat “dazed and confused” by the article entitled “Why the Cancer Moonshot Has Been So Disappointing”. I guess the article should have been entitled “Why the Orphan Drug Designation Blocks the MoonShot!”. Making sure that I understood the definition of a cancer “death rate”: “the number of cancer deaths (the number of people who die from cancer) out of 100,000 people in one 1 year”, I can see that as one succeeds in prolonging survival one may not affect the death rate substantially in an aging population. Hence the statement in the article that “the cancer death rate has fallen only 5 percent since 1950” seems to be obscuring the significant gains from our scientific advances (improvements in survival). There is no question that public health measures such as no smoking have substantial effects. Next, the statement that the federal government has not exactly “invested large sums or directed research to focus on cancer” belies the fact that the federal government provides the 27 Institutes and Centers of the NIH with annual funding of $31.3 Billion of which $5.74 Billion (18.3 %) is allocated to the NCI. I guess the other Institutes might argue against the lack of investment in Cancer point. As I make my way through this article and meander through this commentary, things are becoming foggier and more obfuscated. I think it better (than meandering further) for the reader for me to close with two points. Firstly, in regard to Cancer drug/biologic development, the Orphan Drug Act might be viewed as “Prophetic” as the discovery of genomic aberrations and other biomarkers define target patient populations as being smaller and smaller. Secondly, the one clear statement in the article is that “curing cancer is an enormously difficult technical and medical problem”. It was good to find my way out of this article with one beacon of simple truth. quotes

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Congress Must Improve Prior Authorization to Ensure People With Cancer Have Timely Access to Care

(ASCO) Sept 10, 2019 - The American Society of Clinical Oncology (ASCO) joined approximately 350 health care groups in urging members of the U.S. House of Representatives to support the Improving Seniors’ Timely Access to Care Act (H.R. 3107). The bipartisan bill would help protect patients from unnecessary care delays by streamlining and standardizing prior authorization under Medicare Advantage (MA) and providing program oversight and transparency for MA beneficiaries.

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William McGivney, PhD (Posted: September 17, 2019)

quotesAny opportunity to limit the adverse effects associated with payer-driven, resource-wasting, patient-harming preauthorization requirements and review is important to take advantage of. The proposed federal legislation, Improving Seniors’ Timely Access to Care Act, represents one such opportunity. A key provision is the requirement for plans to report the “results” of their precertification processes. Plans will be required to report on the percentage of precert reviews that result in approvals. Also, importantly, plans must report on the percentage of precert denials that are reversed, overturned and approved. In the end, these statistics will show an egregious waste of time. More importantly, these statistics will show that such a waste of time, often delays treatment for patients whose cancers are progressing. Plans will be required to report on the length of time it takes to reach a final decision once a precert request is filed. One can be sure that an insurance company C-Suite executive would not stand for such delay or denial for themselves or a loved one. I know that from experience during my days at a major payer. Also, I have recounted the story here of a payer cardiology nurse (by training) lecturing a world-renown oncologist on what the NCCN Guidelines recommend. Being patient, the oncologist finally interpreted the NCCN Guideline recommendation for the cardiology nurse and closed by saying that she/he (the oncologist) was the Chairperson for that NCCN Guideline Panel. The key is for the 300 plus organizations supporting the bill to not relent until it passes and then to drive precertification processes further into the ground. For those working on this, I am happy to testify given my experience as VP for Coverage Policy at Aetna and service on the Medicare Coverage Advisory Committee. quotes

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Tomasz M. Beer, MD, FACP

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