Top Cancer Stories of 2014
By Nancy Ciancaglini
2014 was an exciting year for cancer developments — from first-in-class FDA approvals for immune checkpoint inhibitors to obesity being identified as a leading cause of cancer. With no shortage of news, here is a recap of some of the headlines from the past year that generated a tsunami of media coverage.
1) First FDA Approvals for PD-1 Inhibitors—Expansion into Blood Cancers
Forbes named Keytruda and Opdivo “the most important drugs of 2014.” Keytruda (pembrolizumab, Merck) won an accelerated approval from the FDA on Sept. 4, 2014 for advanced melanoma in patients who no longer respond to other drugs, becoming the first anti-PD-1 immune checkpoint inhibitor to be approved by U.S. regulators. Opdivo (nivolumab, Bristol-Myers Squibb) followed with an FDA accelerated approval for the same indication on Dec. 22, 2014. These new immunotherapies act by releasing an immune system brake called PD-1 (programmed death receptor-1) that tumors use to fend off an immune system attack.
Researchers are excited because in 2014 both therapies demonstrated their clinical potential beyond solid tumors for treating blood cancers like Hodgkin lymphoma (HL). In early-stage data reported for the first time at ASH in December, patients with advanced HL who failed to respond to other currently approved therapies including Adcetris (brentuximab vedotin, Seattle Genetics) and stem cell transplants achieved partial or complete remissions of HL when treated with either Keytruda or Opdivo based on two separate small studies. The results were called ‘profound’ by investigators. Nivolumab has already been granted a “breakthrough therapy” designation in HL by the agency, which could speed its approval for the disease. Stay tuned for what’s hoped to be more good news on the clinical effectiveness of PD-1 therapies in treating a wide range of cancers, including difficult-to-treat blood cancers.
2) CAR-T Cell Therapies Are Hot
New data at ASH from a pediatric study in leukemia of Novartis’ investigational CAR therapy, CTL019, might signal a near-future sea change in the treatment of hematological malignancies. Researchers from the Children's Hospital of Philadelphia and the University of Pennsylvania provided longer-term and updated data from a small study of patients ages 5 to 22 with relapsed/refractory acute lymphocytic leukemia (ALL). Ninety-two percent, or 36 of 39 patients, had complete remissions of the disease after treatment with CTL019. Investigators reported that more than two-thirds of the patients were cancer free six months following treatment and 75% of patients were still alive. In a Lancet study published in October by a National Cancer Institute (NCI) team, researchers said that six out of 21 young patients with primary chemorefractory ALL who had never had a remission after multiple chemotherapy regimens showed a complete response after one infusion of CAR T-cell therapy. Clinicians think CAR-T cell therapy could replace stem cell transplant for some patients with blood cancers.
3) Power Morcellators and Hidden Cancer Risk
The backlash against the use of laparoscopic power morcellators was intense in 2014 and it divided doctors. After nearly a year of debate about the hidden cancer risk that the use of the tools posed to women during common gynecological procedures like uterine fibroid removal and hysterectomies in spreading undetected cancers like uterine sarcoma, the FDA took decisive action in November and issued an immediate “black box” warning for the devices. In April, the agency estimated that 1 in 350 women undergoing surgery for fibroids were at risk for an undetected cancer that the tool could spread and discouraged its use. But despite the FDA’s initial warning, reports surfaced in September that gynecologists continued to use the surgical tools. Johnson and Johnson (J&J), the U.S.’s biggest manufacturer of the devices, directed surgeons to stop using them and pulled them from the market, followed by a voluntary J&J worldwide recall in July. Hospitals, including Boston's Brigham and Women's Hospital and Philadelphia's Temple University Hospital, banned the use of the tools, and some insurers like Highmark stopped covering them. The FDA’s strongest warning in November came nearly a year after a review of the surgical cutting tools and is expected to sharply curtail their use.
4) The Continued Spiralling Costs of Cancer Drugs
“Financial toxicity” was a familiar phrase this past year, a side effect of the high cost of cancer care that leaves patients having to decide between either receiving life-saving treatments or personal bankruptcy. In a CBS “60 Minutes” interview in October, noted oncologist Leonard Saltz, MD, of Memorial Sloan-Kettering Cancer Center, pointed out that the cost of a new cancer drug is now well over $100K a year. Newly approved drugs proved the point: Blincyto (blinatumomab, Amgen) for ALL will cost around $178,000 a year, making it one of the world’s most expensive cancer drugs. Keytruda and Opdivo, both newly approved for advanced melanoma, will each carry a price tag of $150,000 per year. After the CBS piece aired, ASCO issued a statement saying that the organization was committed and working on several fronts to ensure that all patients would have access to high-quality, high-value cancer care.
5) New Payment Models for Cancer Care
Driven by the arrival of accountable care, alternative payment models to the traditional fee-for-service model in cancer care evolved in 2014. In August, the Center for Medicare and Medicaid Innovation (CMMI) released a preliminary design for an Oncology Care Model. ASCO has an initiative underway called the Consolidated Payments for Oncology Care, which will restructure how oncologists are paid. Wellpoint said it would pay oncologists a bonus of $350 per month per patient for following specified treatment regimens or “pathways,” saving 3% on overall cancer costs in the process. Florida Blue is teaming up with physician practices to create cancer-specific “accountable care organizations” that reward doctors if they save money and hit quality targets. And, in an expansion of a pilot project using flat payments for certain cancers, including drug costs, United Healthcare announced in December a pilot program with MD Anderson Cancer Center that would provide a “bundled payment” for head and neck cancers.
Top Cancer Stories of 2014 (continued)
6) A New Option in Colorectal Cancer Screening
Although not as accurate as the more traditional colonoscopy, U.S. regulators approved Exact Sciences’ Cologuard® on Aug. 11, 2014 as the first and only FDA approved, noninvasive stool-based DNA screening test for colorectal cancer. In March, an FDA advisory panel unanimously backed the self-administered test for premarket approval as safe and effective and said that its benefits outweighed its risks. In a clinical trial that enrolled more than 10,000 patients, the test found 92% of colorectal cancers in average risk patients, with 87% specificity. In August, the Mayo Clinic became the first healthcare system to offer it to patients. In November, the American Cancer Society updated its national guidelines to include the at-home test among its recommended list of screening options for colorectal cancer. Physicians praised Cologuard as an alternative for people who refused or were unable to tolerate the more invasive colonoscopy. Any screening method to detect colorectal cancer was better than none, most doctors agreed. In another coup for Exact, CMS agreed to reimburse the test and issued a final payment decision set at $502.00.
7) CMS Likely to Cover Lung Cancer Screening
In November, the Centers for Medicare and Medicaid Services (CMS) made a preliminary decision to cover lung cancer screening with low-dose computed tomography (LDCT) for eligible patients. In April, the Medicare Evidence Development & Coverage Advisory Committee (MEDCAC) voted against recommending national Medicare coverage for annual screening for lung cancer with low-dose CT in high-risk individuals. The MEDCAC ruling, which isn’t binding, had screening supporters up in arms. Forty-plus medical societies and members of the US House and Senate have urged CMS to reimburse for screening in older adults. CMS is expected to issue a final decision in February 2015. As of Jan. 1, 2015, the Affordable Care Act requires private insurers to cover lung cancer screening without cost-sharing.
8) E-Cigarettes, Regulation and Kids
In April, the FDA proposed for the first time a rule to regulate the growing market for electronic cigarettes (e-cigarettes) as well as cigars, pipe tobacco, and hookahs. But the agency stopped short of broader restrictions for marketing practices saying that rigorous scientific research was first needed to prove that such products posed a significant public health risk, especially to young people. The fierce debate over risks versus benefits of e-cigarettes raged as tobacco-control advocates argued that the use of the battery-operated devices was a gateway to smoking tobacco and especially appealing to youth, while supporters said that e-cigarettes might help smokers quit the more deadly habit as a tobacco cessation tool. The confusion only intensified in August when the Centers for Disease Control and Prevention (CDC) released a new study showing that adolescents who “vaped” said they were much more likely to take up smoking conventional cigarettes. In August, ASCO and the American Association for Cancer Research (AACR) in a joint letter urged the FDA to extend its regulatory authority over all tobacco products including e-cigarettes and called on the agency to do more, especially in the area of aggressive youth-oriented advertising and marketing.
Update: On January 12, 2015, ASCO and AACR issued a joint policy statement calling for further research and tighter state and federal regulations regarding the use and sale of e-cigarettes and other electronic nicotine delivery systems (ENDS).
9) An Increased Emphasis on Palliative Care
An inaugural Palliative Care in Oncology symposium in October was the first major meeting to bring together cancer care specialists and health care professionals who focus on the relief of symptoms in cancer patients. The conference reflected an increased awareness of the critical role that palliative care plays in ensuring quality cancer care for patients, many of whom are coping with their disease as a chronic illness rather than a death sentence. Topics covered at the meeting included the value of Patient Reported Outcomes (PRO’s), allowing physicians to gauge the side effects of medications on patients and alleviate suffering if possible, the importance of advance care planning, and the use of ‘smart’ technology to monitor symptom management. One positive shift in the oncology treatment landscape noted by attendees is the fact that oncologists and palliative care specialists are now consulting together earlier than ever before during the treatment continuum to improve the well-being and quality of life of both patients and caregivers.
10) Obesity: A Major Cancer Risk
In October, ASCO released its first-ever policy statement on cancer and obesity, identifying it as a leading cause of cancer and a complication in caring for cancer patients, and calling for increased education, research, and advocacy to reduce its toll. Obesity, the organization said, is projected to outpace tobacco as the leading preventable cause of cancer in the US and for those already living with cancer, being overweight increases the chances of cancer recurrence and lower survival. An interesting study in 2014 found that obese cancer patients often suffer from malnutrition.