US Capitol Capsule: Measuring obesity prevention would improve efforts, IOM says

Obesity, diet and weight management are Americans' greatest personal health concerns, according to a new survey sponsored by the Pharmaceutical Research and Manufacturers of America (PhRMA), which reported that 74% of the responders insisted addressing the obesity epidemic, which costs the US close to $200 billion annually, should be a priority for the nation.

But the US lags behind international efforts in providing common guidance, support and the appropriate infrastructure to nurture evaluation of obesity prevention activities, a panel of experts from the Institute of Medicine (IOM) said in a separate report.

According to the Centers for Disease Control and Prevention, more than one-third of adults in the US are obese, while the rate in children and adolescents has skyrocketed from 5.5% in 1980 to 17% in 2013.

In June, the American Medical Association's House of Delegates at the group's annual meeting declared obesity a disease. A bipartisan group of lawmakers responded days later by introducing legislation, known as the Treat and Reduce Obesity Act, aimed at providing better access to counseling and new prescription drugs for chronic weight management for Medicare beneficiaries (scripintelligence.com, 24 June 2013).

So far, two new prescription weight-loss medicines have won FDA approval –Arena Pharmaceuticals' and Eisai' Belviq (lorcaserin) and Vivus' Qsymia (phentermine/topiramate) (scripintelligence, 28 June 2012, 18 July 2012, 19 July 2012).

Orexigen Therapeutics is expected to soon resubmit its new drug application to the FDA for the company’s antiobesity medicine Contrave (naltrexone SR/bupropion SR), which initially was rejected by the agency in 2011 (scripintelligence.com, 7 January 2013).

The two reports from the IOM and PhRMA brought praise from drug makers.

"The IOM's report further underscores that a variety of solutions are needed to address obesity, including effective pharmacological interventions," Vivus told Scrip in a 2 August statement.

Being obese, Vivus said, "significantly increases the risk of developing many different diseases and health conditions, including type 2 diabetes, hypertension, metabolic syndrome, cardiovascular disease, some cancers and osteoarthritis," which the company noted are other concerns expressed by Americans in PhRMA's survey.

"With obesity affecting over 35% of American adults and contributing over $200 billion annually to healthcare spending, it is encouraging to see increased attention to preventative behaviors along with heightened concern about weight management," Orexigen said in a 2 August statement to Scrip.

Orexigen CEO and PhRMA board member Michael Narachi said the new research "highlights the growing awareness for the significant health consequences of obesity."

"Fortunately, we are seeing an inevitable change in the nation's concern for this disease and would agree with patient opinion from the survey that dealing with obesity should be in the top two or three health priorities for the country," Mr Narachi said.

The recognition of the health, economic and social consequences of the obesity epidemic has accelerated efforts to characterize and understand the problem more fully, to take evidence-based and innovative actions and to assure progress in prevention, the IOM's experts said in their new report, Evaluating Obesity Prevention Efforts: A Plan for Measuring Progress, released on 2 August.

But there are gaps in the certainty of the effectiveness or mixture of obesity prevention actions being implemented across the US, the IOM panelists declared.

Systematic and comprehensive evaluations along with more routine assessments, monitoring and surveillance offer valuable guidance for improving the quality and outcomes of the actions being implemented and for defining the direction of further basic and implementation research, the IOM's experts contended.

But information generated from current obesity prevention evaluation efforts does not always address the needs and interests of its users, often because of limited or outdated data, especially at the community level, and few presentations of the information are being made in useful and timely formats, the IOM panelists asserted.

The committee also said the current data monitoring systems do not adequately track progress of environmental and policy-related obesity prevention actions or systems changes recommended by the IOM in a 8 May 2012 report, Accelerating Progress in Obesity Prevention: Solving the Weight of the Nation, which found that America's progress in arresting its obesity epidemic was too slow, with the condition continuing to erode productivity and causing millions of Americans to experience potentially debilitating and deadly chronic illnesses.

In last year's report, the IOM said solving the complex and stubborn problem of obesity would require a comprehensive set of solutions that work together to spur across-the-board societal change.

It identified strategies with the greatest potential to accelerate success of obesity prevention, which included making healthy foods and beverages and opportunities for physical activity easy, routine and appealing aspects of daily life for Americans.

But in the new IOM report, the committee complained that current investment in measuring obesity prevention efforts is "too low and sporadic, presenting serious barriers to understanding the impact of and need for future investments in implementing interventions."

The panelists recommended that a systems science approach to evaluation, such as interactions and connectedness of components in a whole system, could help identify the necessary combinations of actions and strategies needed in multiple sectors and at multiple levels with available resources.

The committee outlined a framework for obtaining end-user input, identifying indicators and measures for data collection and analysis and improving the evaluation structure.

But the IOM panelists said the evaluation plans outlined in their report would not fully be realized without coordinated changes across multiple federal, state and local government agencies and departments in collaboration with other nongovernmental partners responsible for obesity prevention-related activities.

The committee recommended creating an obesity task force or other entity to oversee and lead the implementation of the IOM's proposed national plan evaluating prevention efforts and provide support to the community plan.

That task force, the IOM panelists added, could be a newly organized panel consisting of various public and private-sector stakeholders, or it could be part of an existing committee, such as the Health and Human Services' Healthy Weight Task Force or the National Prevention Council.

In other Washington news:

FDA data proposal irks PhRMA

While responsible data sharing could enhance the public health and accelerate development of new medicines by allowing for independent reanalysis of the rich datasets compiled by drug manufacturers in clinical testing, PhRMA said the FDA should not be the party releasing that information.

The FDA in June proposed making "de-identified" and "masked" study data derived from drug company applications submitted to the agency available to non-FDA experts and other interested parties for the purpose of generating new knowledge aimed at facilitating innovation in new medical products (scripintelligence.com, 5 June 2013).

But in public comments submitted to the FDA, PhRMA insisted data sharing arrangements are "more appropriately implemented" directly through "data generators" than through agency data disclosure.

PhRMA argued that focusing on processing trial data for outside analysis "risks distraction and additional burdens on FDA's valuable resources."

US regulators would need to ensure the FDA's de-identification and masking methods would prevent re-identification and unmasking for now and in the future – a task PhRMA said would present "considerable practical challenges."

The group noted that in recent studies, researchers were able to use computational methods and publicly available information to deduce subject identities from supposedly de-identified data, even though re-identification had been thought impossible just a few years earlier.

PhRMA said it was concerned similar strategies would be used to unmask data and identify the drugs to which that information corresponds.

The widespread public release of non-summary data, PhRMA added, could have unintended consequences, including increasing the risks to patient privacy and unjustified second-guessing by non-experts of FDA's regulatory decisions, which may prompt patients to forego medically appropriate treatments and vaccinations.

The group also raised concerns that masking may not prevent competitive harm to the submitting sponsor on widespread release of the data.

PhRMA said it was worried a sponsor's competitors could use masked data to "address key hurdles in drug development" or to develop patentable inventions, and exclude the original submitter from using them.

If a competitor is able to unmask the data, that firm could obtain approval of competing products with "little effort or investment," PhRMA contended.

The trade group asserted the FDA would need to secure statutory changes and modify its regulations before it could implement its proposal because the trial data submitted in applications would continue to have competitive value after masking, and therefore, would remain trade secret or confidential commercial information.

PhRMA insisted implementation of a new set of data-sharing principles it released jointly last month with the European Federation of Pharmaceutical Industries and Associations, which take effect 1 January 2014, and clinicaltrials.gov would fulfill the FDA's goals without creating policy and legal issues (scripintelligence.com, 26 July 2013, 24 July 2013).

FDA lacks compounding data

The FDA lacks timely and reliable information to oversee compounding pharmacies, although the agency has nonetheless been able to discover problems through its limited oversight of those entities, the Government Accountability Office (GAO) – the watchdog for Congress – reported last week.

Specifically, the GAO said in a 31 July report, the FDA's inspection database is inadequate for identifying all of the agency's investigations into compounders or the final classification of results of all of federal regulators' probes of pharmacies, which are licensed through their respective states.

Until this year, the FDA limited its inspections of compounding pharmacies to those conducted in response to complaints or adverse events.

But the agency stepped up its inspections of compounding pharmacies after a nationwide meningitis outbreak was triggered by contaminated compounded injectable products made by Framingham, Massachusetts-based New England Compounding Center (NECC), in which more than 60 Americans have died, with nearly 800 sickened (scripintelligence.com, 10 June 2013, 30 May 2013, 10 May 2013, 17 April 2013).

Through those probes, the FDA identified problems at some other pharmacies, such as a lack of sterility, which resulted in recalls of compounded drugs, with one of those compounders being linked to a second outbreak of infections, in which 26 people in four states have been sickened (scripintelligence.com, 1 June 2013, 29 May 2013, 27 March 2013, 22 March 2013, 19 March 2013).

Nonetheless, the GAO concluded the FDA's authority to oversee compounders is unclear, resulting in gaps of oversight of those entities.

Senator Lamar Alexander (Republican-Tennessee) said he hopes the GAO's report will spur adoption by the full Senate of bipartisan legislation advanced in May by the chamber's Health, Education, Labor and Pensions Committee, which aims to make the FDA the "flagpole" agency over compounding (scripintelligence.com, 23 May 2013, 17 July 2013).

Groups urge new antibiotic pathway

A coalition of drug makers – Cempra, Durata Therapeutics, Rib-X Pharmaceuticals, Theravance, The Medicines Company, Optimer Pharmaceuticals and Trius – known as the Antibiotics Working Group (AWG), joined the Infectious Diseases Society of America (IDSA) and several other groups last week in again urging lawmakers to create an expedited pathway for the FDA to approve antibiotics intended to treat serious or life-threatening infections for which there are currently few or no satisfactory treatment options (scripintelligence.com, 8 July 2013).

The limited population antibacterial drugs (LPAD) pathway would encourage the development of antibiotics to treat highly resistant infections by allowing the FDA to OK the medicines based on trials using smaller subpopulations consisting of the most serious illnesses, the groups said in their 31 July letters to House and Senate lawmakers.

Regulators have acknowledged that a limited-use designation like LPAD could be used to help reduce the development of resistance to antibacterials by restricting the medicines to the most appropriate and necessary patients (scripintelligence.com, 15 January 2013).

The FDA held a public hearing in February to gain input on whether an LPAD or another pathway for serious or life-threatening conditions with unmet medical needs was necessary in light of the agency's other expedited pathways, including the new breakthrough therapy designation – with regulators receiving mixed feedback at the meeting (scripintelligence.com, 5 February 2013, 26 June 2013).

PhRMA was among those raising concerns that adding yet another regulatory pathway to FDA's plate could result in confusion of expectations or a diversion of resources from existing mechanisms (scripintelligence.com, 1 March 2013).

The AWG, IDSA and the other groups asserted the LPAD's streamlined process would provide patients more rapid access to potentially life-saving therapies by lowering regulatory barriers to the development of antibiotics they most need, providing an incentive for companies that otherwise would be discouraged by prohibitively high development costs and lengthy testing timelines.

Narrow indications for LPAD drugs could create conditions for value-based or premium pricing for high-need antibiotics, which the groups said would encourage greater research and development investment, the groups said.

The LPAD designation and labeling also would help encourage judicious use of products approved under the pathway, which the groups said would be "an important goal if we are to preserve the effectiveness and promote stewardship of these vital medicines over time."

This and past US Capitol Capsule columns are available at http://www.scripintelligence.com/capitolcapsule/