Vol. 21 • Issue 4 • Page 16
State of the Profession: HOME CARE
Scrutiny over the cost of caring for the most medically fragile patients is driving increased interest in getting and keeping patients healthy at home. Clinicians are keeping pace with drug developments to treat cystic fibrosis, partnerships in respiratory care home services, and calls for best practices in mechanical ventilation.
Nearly 25 years after scientists identified the gene defect that causes cystic fibrosis, a medication has been approved by the Food and Drug Administration that markedly improves lung function for 4 percent of people with the disease.
Ivacaftor (kalydeco, Vertex Pharmaceuticals Inc.) helps defective CF transmembrane conductance regulators work at the surface of the cell to move salt and fluid into the airways, clearing away the sticky, thick mucus that builds in cystic lungs. Trials in patients affected by the G551D mutation showed lower sweat chloride test numbers, fewer pulmonary exacerbations requiring antibiotics and hospitalizations, and reduced number and duration of exacerbations.1
“The foremost thought on the minds of parents who have a child with CF is to find a cure,” said Mary K. Lester, RRT, department of respiratory therapy, medicine, and pediatrics in the Medical University of South Carolina in Charleston. “Up until now, we didn’t have anything to tell them.” The twice-daily pill is approved only for patients 6 and older affected by the G551D mutation.
Studies are underway in people with other gating mechanism defects, with several trials targeting the most common mutation, F508del, which affects 80 percent of CF patients.2,3 Final results from a Phase 2 randomized, double-blinded, placebo-controlled trial of 109 patients testing a combined therapy of ivacaftor and VX-809 showed statistically significant lung function improvements in patients with two copies of F508del, and some improvement in people with a single copy of the mutation. A pivotal trial is planned for 2013.4
Researchers don’t know what long-term effects the medication will have on patients, but clinicians are emboldened. “Families ask, ‘Are airway clearance therapies going to be necessary?'” said Lester. “We really hope not.” For now, patients are adhering to all prescribed medications and therapies, with the possible exception of hypertonic saline spray.
While the discovery itself has the CF community atwitter, researchers are buzzing over the drug’s three-month passage through the FDA review process. New legislation advancing through the U.S. House Energy and Commerce Committee aims to change clinical trial design so more rare disease therapies can follow this expedited path. The EXPERRT Act would authorize reviewers to consult rare disease experts and patient advocates about the severity of the disease the drug will treat and risks patients may be willing to take.
“On behalf of the millions of Americans with cystic fibrosis and other rare diseases, we are heartened that the voice of patients and experts will be heard as more sophisticated therapies for complex rare diseases are advanced,” said Cystic Fibrosis Foundation President Robert J. Beall, PhD, in a prepared statement.
With competitive bidding’s second round taking effect in 90 additional metropolitan areas in July 2013, home health providers are racing to disprove the Center for Medicaid and Medicare Services’ assertion that the program saved $202 million in its first year without affecting patient care. AAHomecare is collecting data about quality, patient comorbidities, business closures, and equipment used in the first round’s nine markets. But the data may be clouded by practices companies used to stay in business. “Suppliers had the ability to revenue-shift in those competitive bid areas,” said Walt Gorski, AAHomecare vice president of government relations. “They were able to offset the steep price reductions by receiving the current allowable in non-bid areas.” He predicts the program’s expansion will magnify existing problems, leaving some providers financially unstable.
AAHomecare is lobbying to replace competitive bidding with the market pricing program it designed in conjunction with auction experts. The program would break bidding categories into smaller bundles of equipment, so that, for example, suppliers aren’t bidding on oxygen therapy and continuous positive airway pressure equipment. Reimbursement for products not included in bidding categories would be set using prices from comparable markets. Companies submitting the lowest bids would be contracted to provide services for that area, but a market clearing price would be set so that any supplier could service Medicare patients. The bill also would require that at least 30 percent of contracts be awarded to businesses earning less than $3.5 million annually.
The association reports strong support from lawmakers and they expect their proposal to gain momentum in late 2012. “What we are doing now is laying the foundation so that when Congress does turn its attention after the election to these issues, we are prepared and ready to go,” said Gorski.
However, some home care experts doubt the likelihood of the proposal’s success. “The horse is way far gone,” said Bob McCoy, BS, RRT, FAARC, former chair of the American Association for Respiratory Care home care section. “To close the barn door now is too late.”
Instead, he foresees home care companies developing pay-for-performance partnerships with hospitals, which, beginning next fall, face penalties for patients readmitted within 30 days of hospital discharge. In this model, a home care company could provide respiratory therapists who assess patients, determine that best equipment from them, and order it from the hospital’s central processing department or the manufacturer. It only would be paid for patients who remain healthy at home.
More research into which devices best serve patients, considering factors like patient mobility and oxygen titration levels, will be needed to make this arrangement feasible, said McCoy.
Additionally, hospital-based clinicians will need to deepen their understanding of issues such as chronic respiratory insufficiency or failure. “There is a very great difference between the experience, knowledge, and understanding that critical care professionals and home care professionals have of patients who require home mechanical ventilation,” said Allen I. Goldberg, MD, MBA, Master FCCP, a past president of the American College of Chest Physicians.
Smaller, lighter, and more advanced home mechanical ventilators are enabling an estimated 100,000 vent-dependent patients to live at home. But best practices for the clinicians caring for them still are coming to light.
Guidelines issued by the American Association for Respiratory Care in 1995 discussed issues in long-term invasive mechanical ventilation such as discharge considerations, resources that patients need at home, and monitoring of outcomes. A 1998 consensus statement from the American College of Chest Physicians narrowed the focus, giving physicians framework to discharge patients home and determine optimal techniques and equipment for long-term ventilatory management.
“There were no specific guidelines for home mechanical ventilation” that addressed differences between groups of patients until now, said Eduardo Mireles-Cabodevila, MD, director of the medical intensive care unit, University of Arkansas for Medical Sciences Medical Center in Little Rock.
The recently issued Canadian Thoracic Society standardized practices, which were reviewed by the ACCP, now delineate practices for choosing home mechanical ventilation modes for patients with stable chronic obstructive pulmonary disease, post-polio syndrome, obesity hypoventilation syndrome, and other diseases; and provides guidance for discharge and ethical considerations in patient care.5 n
1. Ramsey BW, Davies J, McElvaney NG, et al. A CFTR Potentiator in Patients With Cystic Fibrosis and the G551D Mutation. N Engl J Med. 2011;365(18):1663-72.
2. Clinicaltrials.gov. Bethesda, MD: U.S. National Library of Medicine. 2012. Study of Ivacaftor in Subjects With Cystic Fibrosis Who Have the R117H-CFTR Mutation (KONDUCT)
Available from: http://clinicaltrials.gov/ct2/show/NCT01614457.
3. Clinicaltrials.gov. Bethesda, MD: U.S. National Library of Medicine. 2012. Study of Ivacaftor in Subjects With Cystic Fibrosis Who Have a Non-G551D CFTR Gating Mutation (KONNECTION).
Available from: http://clinicaltrials.gov/ct2/show/NCT01614470.
4. Vertex Pharmaceuticals Inc. Final Data from Phase 2 Combination Study of VX-809 and KALYDECO™ (ivacaftor) Showed Statistically Significant Improvements in Lung Function in People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation. 2012 June. Available from: http://investors.vrtx.com/releasedetail.cfm?releaseid=687394.
5. McKim DA, Road J, Avedano M, et al; Canadian Thoracic Society Home Mechanical Ventilation Committee. Home mechanical ventilation: A Canadian Thoracic Society clinical practice guideline. 2011. Available from: www.respiratoryguidelines.ca/sites/all/files/2011_CTS_HMV_Executive_Summary.pdf
Kristen Ziegler can be reached at [email protected].