January 10, 2011
By Leah Voigt*
The Teaching Hospitals and Academic Medical Centers Practice Group (TH/AMC PG) distributes a compilation of updates of interest to their members on a weekly basis. We thought last week's edition would be of special interest to our members and wanted to thank the TH/AMC PG leadership for sharing it with us.
Industry Says FDA Should Exercise Caution With Biosimilar Interchangeability
In comment letters submitted to the U.S. Food and Drug Administration (FDA), stakeholders said the abbreviated approval pathway for biosimilars should require clinical trials and unique product names, and the agency should exercise caution with respect to interchangeability determinations.
The Biologics Price Competition and Innovation (BPCI) Act of 2009 establishes an abbreviated approval pathway for biologic drugs that are demonstrated to be highly similar (biosimilar) to, or interchangeable with an FDA-licensed biological product. The act was part of the Patient Protection and Affordable Care Act (PPACA, Pub. L. No. 111-148), signed into law in March. FDA held a public meeting in November to seek comments on the abbreviated pathway. Written comments on the pathway were due December 31, 2010.
The Pharmaceutical Research and Manufacturers of America (PhRMA) said in a December 23, 2010, comment letter that FDA should exercise caution when making determinations on whether a biosimilar is interchangeable with a brand product. If FDA decides to make interchangeability determinations, the agency needs to consider the possibility that the interchangeable biosimilar could diverge from the brand product after it is approved, the group said. Biologics can change over time as a result of manufacturing variations. Additionally, FDA should require unique product names for biosimilars so that patients or healthcare professionals can report adverse events associated with biosimilars, PhRMA said.
The Biotechnology Industry Organization (BIO) said in comments submitted December 23, 2010, that clinical trials "are fundamental for evaluating and demonstrating the safety and effectiveness of a biosimilar" and biosimilars, "must be properly evaluated through post-marketing surveillance and post-marketing clinical studies as needed."
BIO agreed with PhRMA that biosimilars should be assigned unique names to distinguish them from brand products. "Assigning the same name to a product that is not the same would be confusing and misleading to patients, physicians, and pharmacists, and could result in inadvertent substitution of the products, and would make it difficult to quickly tract and address adverse events that may be attributable to either the innovator or biosimilar product," BIO said. Additionally, prescribers should be involved in decisions to switch among biological products, not just pharmacists, BIO said.
The American Medical Association (AMA) said in a December 29, 2010, comment letter that it "supports a science-driven approval process that prioritizes product efficacy and patient safety and empowers [FDA] to use its expertise to determine on a case-by-case basis what data are needed for the approval" of a biosimilar. AMA said interchangeability determinations "require adequate pre-market clinical studies in patients to establish safety and efficacy and in some cases also may require targeted post-market surveillance studies."
Bronwyn Mixter, Industry Says FDA Should Exercise Caution With Biosimilar Interchangeability, BNA's Health Care Daily Report
(Jan. 4, 2011) (note: registration is required to view this content).
Simple Question Aids Parkinson's Diagnosis
Dutch researchers said they have found a way to distinguish atypical Parkinson's disease from the more common standard form: asking patients if they can still ride a bicycle.
When the question was put to 111 consecutive patients with Parkinson's disease, nearly all those who said it had become impossible were subsequently determined to have the atypical form, according to researchers at Nijmegen Medical Center in the Netherlands. "Simply asking about cycling abilities could be added to the list of red flags that can assist clinicians in their early differential diagnosis of parkinsonism," they reported in the January 8, 2011, issue of The Lancet.
The researchers noted that it is often clinically difficult to determine whether patients with clear symptoms of Parkinson's disease have an atypical form that, as the disease progresses, includes symptoms not normally seen in the typical presentation. Whereas "regular" Parkinson's disease is caused by loss of dopaminergic neurons, patients with atypical disease also show degeneration in the striatum. The etiologies of atypical versus typical disease may also differ.
The difference is important both for advising patients on what to expect in the future and for recruitment in clinical trials of Parkinson's disease therapies, noted the researchers, who conducted structured interviews with 156 Parkinson's patients who had not yet received a definitive diagnosis. At study entry, patients were asked if they had ridden a bicycle before developing symptoms and, if so, whether, when, and why they no longer could. The forty-five patients who said they were not bike riders were excluded from the analysis.
The final determination of whether patients had atypical or typical Parkinson's disease was made three years after the initial interview, on the basis of response to treatment, neurological exams, and MRI studies. Typical disease was diagnosed in forty-five patients and atypical syndromes in sixty-six. During the baseline interview, thirty-six patients said they were no longer able to ride a bicycle. Of those, only two were later diagnosed with typical Parkinson's disease.
John Gever, Simple Question Aids Parkinson's Diagnosis, MedPage Today (Jan. 6, 2011).
Study Linking Vaccine to Autism Was Fraud, Journal Reports
The first study to link a childhood vaccine to autism was based on doctored information about the children involved, according to a new report on the widely discredited research. The conclusions of the 1998 paper by Andrew Wakefield and colleagues was renounced by ten of its thirteen authors and later retracted by the The Lancet, where it was published. Still, the suggestion that the MMR shot was connected to autism spooked parents worldwide and immunization rates for measles, mumps, and rubella have never fully recovered.
A new examination found that Wakefield and colleagues altered facts about patients in their study by comparing the reported diagnoses in the paper to hospital records. The analysis, by British journalist Brian Deer, found that despite the claim in Wakefield's paper that the twelve children studied were normal until they had the MMR shot, five had previously documented developmental problems. Deer also found that all the cases were somehow misrepresented when he compared data from medical records and the children's parents.
Wakefield could not be reached for comment despite repeated calls and requests to the publisher of his recent book, which claims there is a connection between vaccines and autism that has been ignored by the medical establishment. Wakefield now lives in the United States where he enjoys a vocal following including celebrity supporters like Jenny McCarthy.
Last May, Wakefield was stripped of his right to practice medicine in Britain. Many other published studies have shown no connection between the MMR vaccination and autism. But measles has surged since Wakefield's paper was published and there are sporadic outbreaks in Europe and the United States. In 2008, measles was deemed endemic in England and Wales.
Associated Press, Study Linking Vaccine to Autism Was Fraud, Journal Reports, The New York Times (Jan. 5, 2011).
Health System Reform Expected to Boost House Calls
For a time during his seventeen years of running a house, call-based practice, the pay was so bad for Tom Cornwell, MD, that his family lived off his wife's income. But physicians like Dr. Cornwell say they are finding themselves much more in demand. During the last decade, Medicare has increased its rates for house call visits as an aging population has necessitated more of them. The program paid for more than 2.3 million house calls in 2009, compared with more than 1.5 million in 1995.
"I started my practice at a time when many people doing this were going bankrupt, and we were being paid half what we are being paid now," said Dr. Cornwell, a family physician in Wheaton, IL. "Medicare has realized the value of this."
But experts say several aspects of healthcare reform will make house call practices more common. "The home as a venue to provide care is becoming more important," said Steven Landers, MD, MPH, director of the Center for Home Care and Community Rehabilitation at the Cleveland Clinic. "And physician leadership in home care is essential. First and foremost, physicians will have to take on more of the responsibility for leading the team."
One program mandated by the health system reform law signed into law in March 2010, Independence at Home, supports expanding house call medicine. The rules and regulations are being written, but some important aspects already are spelled out. Independence at Home, which will launch January 1, 2012, provides payment incentives for primary care teams providing house calls. Physicians and healthcare practitioners would receive a portion of the money saved by improving health outcomes and reducing preventable hospitalizations, readmissions, and emergency department visits. Decreasing the number of duplicative diagnostic and laboratory tests and improving patient and caregiver satisfaction will be taken into account.
Because it is a demonstration project meant to reduce expenses related to care of the highest-cost Medicare beneficiaries, the program will be limited initially to 10,000 patients who live in parts of the country with high healthcare costs.
Experts say smaller practices or those focused on providing house calls are more likely to take part in the program. Only 200 patients per practice are needed, considered the average patient load for a team comprised of a physician and a nurse practitioner that makes only house calls, with no in-office care. The patients may be split among several medical professionals at the same practice.
Many expect institutions to incorporate house calls within the accountable care organizations (ACOs) that are due to launch on January 1, 2012. House calls could be used as one part of an ACO's efforts to lower costs as part of the Medicare Shared Savings Program. Participation in ACOs will provide financial bonuses to groups of health institutions and medical professionals that work together to manage and coordinate care for Medicare fee-for-service beneficiaries. There is no upper limit on the number of patients who eventually will be enrolled in various ACOs across the country, but each qualifying ACO needs to enroll a minimum of 5,000 patients.
Victoria Stagg Elliott, Health System Reform Expected to Boost House Calls, AMA News (Jan. 3, 2011).
AHLA Teaching Hospital Updates are intended to provide quick summaries of cutting edge issues of interest to teaching hospitals and their counsel. Additional information and more in-depth coverage on these topics may be available from AHLA Health Lawyers Weekly and appropriate AHLA Practice Groups.
*We would like to thank Leah Voigt, Esquire (Squire Sanders & Dempsey LLP, Washington, DC), for providing this week's update.