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FDA on In Vitro Diagnostics, Vaping; Cost of Novel Treatments

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FDA issues finalized guidelines for use of in vitro diagnostics in oncology drug trials and warnings on vaping; new findings report patients bear more costs when MBC treatment is not concordant with NCCN guidelines.

October 11, 2019
FDA Issues Final Guidelines for Use of In Vitro Diagnostics in Oncology Drug Trials

As part of its ongoing efforts to reduce administrative burdens and delays related to oncology drug approvals, on October 9, 2019, the US Food and Drug Administration (FDA) released its finalized risk evaluation guidelines for the use of an investigational in vitro diagnostic (IVD) in a clinical trial where the IVD and the oncology drug are part of a co-development program.1

The guidelines outline the new process by which the sponsor may determine whether an IVD is a significant risk (SR), a nonsignificant risk, or exempt from investigational device exemption requirements.2 If it is determined to be an SR, the application is to be submitted to either the Center for Drug Evaluation and Research or the Center for Biologics Evaluation and Research for further evaluation.1

These guidelines complete the work that the agency began in 2018 under former FDA Commissioner Scott Gottlieb, MD. At that time, Dr Gottlieb stated that the goal was “a common filing for a drug and diagnostic system” in cases where a drug is being co-developed with a diagnostic test.3 Such a system could facilitate the tandem development of drugs and companion diagnostics, “enabling more efficient regulatory review.”3

Although the initial guidelines were announced in April 2018 and were met with a positive reception, the release of the final guidelines remained on hold while the FDA made changes requested by drug and diagnostic manufacturing companies.4 Among these requests were calls for clarification, such as in cases where multiple diagnostic devices would be used in a single trial. Other changes were the inclusion of language stating that sponsors will receive SR determinations within the 30-day review period for the investigational new drug (IND), and that the streamlined submission option only applies to new INDs.4 Despite the changes, the regulatory process as outlined in the original draft has not changed. Further details can be found in the Federal Register.

1. US Food and Drug Administration. Investigational invitro diagnostics in oncology trials: streamlined submission process for study risk determination guidance for industry. October 9, 2019. Accessed October 10, 2019.
2. Tayler NP. Experimental cancer IVD trial guidance finalized. October 10, 2019. Accessed October 11, 2019.
3. US Food and Drug Administration. Leveraging innovation for the treatment of cancer. April 12, 2018. Accessed October 10, 2019.
4. Brennan Z. FDA finalizes guidance on streamlined IVD reviews for cancer clinical trials. October 9, 2019. Accessed October 10, 2019.

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October 11, 2019
Study Finds Patients Bear Higher Costs with Novel Treatment Regimens

In the first study to examine the cost burden for patients with metastatic breast cancer (MBC) whose treatment is discordant with National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology for Breast Cancer, researchers at the O’Neal Comprehensive Cancer Center at the University of Alabama found that the burden of cost rose substantially when treatment was not concordant with NCCN recommendations.1 Earlier studies showed that overall costs increased when treatment veered from the NCCN guidelines, but it was not known whether patient cost would also be affected. This study is the first to demonstrate that costs for patients increased as well.

The results of the retrospective study, “Guideline Discordance and Patient Cost Responsibility in Medicare Beneficiaries With Metastatic Breast Cancer,” are published in the October 2019 issue of the Journal of the National Comprehensive Cancer Network.2

Using information from the SEER-Medicare linked database from 2000 through 2013, researchers evaluated the first year postdiagnosis for 3709 women diagnosed with MBC between 2007 and 2013. Guideline discordance was defined by the NCCN guidelines for each year, which vary because of frequent updates. Patient cost was defined as financial responsibility for deductibles, coinsurance, and copayments.

The median patient cost for the year postdiagnosis for first-line antineoplastic treatment for patients with MBC was $5171 for care that concorded with the NCCN guidelines for that year, versus $7421 for care that was discordant.1 It was found that both overtreatment and undertreatment resulted in higher patient costs.

The authors concluded that their findings are significant for patient–clinician communication regarding the value of adhering to guideline-based care and can be used to raise clinicians’ awareness of the costs associated with discordant treatment.

1. National Comprehensive Cancer Network. Following NCCN guidelines for metastatic breast cancer results in lower costs for patients, according to new study. October 10, 2019. Accessed October 11, 2019.
2. Williams CP, Azuero A, Kenzik KM, et al. Guideline discordance and patient cost responsibility in Medicare beneficiaries with metastatic breast cancer. JNCCN. 2019;17:1221-1228.

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October 11, 2019
FDA and CDC Investigate Outbreak of Vaping-Related Illness

In the past week, the US Food and Drug Administration (FDA) has released 2 public announcements related to the use of vaping products. In conjunction with the US Centers for Disease Control and Prevention (CDC) and other federal and local partners, the FDA warns that the use of tetrahydrocannabinol (THC)-containing vaping products, as well as vaping products obtained off the street, pose a significant health risk.

The agencies released 2 strongly worded statements regarding their ongoing multistate investigation prompted by >1200 incidents of severe lung injury and deaths associated with the use of vaping products.1 Lung infection does not appear to be the cause of patient-reported symptoms, which include cough, shortness of breath, or chest pain; nausea, vomiting, abdominal pain, or diarrhea; and fatigue, fever, or weight loss.2,3 These symptoms may develop over a few days or weeks.

The FDA’s Forensic Chemistry Center is analyzing hundreds of samples submitted by multiple states for the presence of a broad range of chemicals, including nicotine, THC, other cannabinoids, and opioids along with cutting agents/diluents and other additives, pesticides, poisons, heavy metals, and toxins.3 Although the FDA does not have sufficient data to identify the specific cause of the lung injuries, it is known that THC is present in most of the third-party samples in the analysis.4

Consumers are warned not to modify or add any substances such as THC or other oils to retail vaping products and should not purchase any vaping products off the street or from other illicit channels.

Healthcare professionals are advised to report possible cases of vaping-associated respiratory illness to their local or state health department for further investigation. If vaping use is suspected as the cause of the illness, a detailed history of the substances used, the sources, and the devices used should be obtained.

1. Centers for Disease Control and Prevention. Outbreak of lung injury associated with e-cigarette use, or vaping. Updated October 11, 2019. Accessed October 11, 2019.
2. US Food and Drug Administration. Lung illnesses associated with use of vaping products. October 4, 2019. Accessed October 11, 2019.
3. US Food and Drug Administration. Vaping illness update: FDA warns public to stop using tetrahydrocannabinol (THC)-containing vaping products and any vaping products obtained off the street. October 4, 2019. Accessed October 11, 2019.
4. US Food and Drug Administration. Statement on consumer warning to stop using THC vaping products amid ongoing investigation into lung illnesses. October 4, 2019. Accessed October 11, 2019.

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