Archive for the 'Global Health' Category

Ebola Outbreak Raises Ethical Issues

The development of new treatments and preventives to combat the lethal Ebola virus has been slow, marked by caution at public health agencies to approve testing of high-risk compounds, and reluctance of biopharmaceutical companies to invest in a field with limited market potential. All that has changed now, as thousands of people have been sickened by the virus, and the death rate has escalated. The risk of harm from an untested treatment now is much less alarming than the prospect of infection, and the potential benefit of any effective treatment outweighs the need for regulatory caution.

Thus international and national authorities are working hard to facilitate access to experimental drugs and vaccines, despite a long history of distrust related to studying experimental treatments in Africa and other developing regions. Sponsors often face charges of using poor patients to test products that later will be unaffordable and unavailable. Authorities also question whether severely ill patients can make informed decisions about treatment options amid community fears of medical treatment. Vaccines raise added concerns, as they require widespread testing over long periods, often in healthy individuals and children. While the possibility of severe side effects from test drugs can generate a backlash against potential treatment, withholding therapy raises even more serious questions.

An important step was for the World Health Organization (WHO) to declare it ethical to use unproven therapies to try to contain the growing Ebola outbreak in West Africa. FDA similarly permitted a small biopharma company to distribute an experimental product that had been put on clinical hold due to concerns about a possible serious side effect.

These actions, though, raise similarly difficult questions about how to distribute the very limited supply of an experimental drug. Some experts cite the importance of treating children first. Others believe that patients with access to effective care are more likely to recover, making that a factor in using scarce medical resources.

Healthcare workers often get top priority, on the basis that doctors and nurses are needed to care for the sick, and that the prospect of treatment may encourage critical professionals to volunteer for service. But the treatment of two Westerners with the potentially effective drug Zmapp raised protests that the therapy was not available to sick Africans.

Data critical
One rationale for treating health professionals is that they are familiar with protocols and can understand the risks and the informed consent process. Treatment in a hospital setting also is more likely to support scientific assessment of an experimental regimen. One area of agreement is the importance of collecting and evaluating data on the safety and efficacy of experimental drugs used during the outbreak, and to share that data with all affected parties.

Researchers are continuing to assess factors related to the recovery of the two health workers who received Zmapp, acknowledging that they don’t know if that good result is are due to the drug, to effective care, or to other health factors. Treatment with this monoclonal antibody therapy produced by tiny Mapp Biopharmaceutical of San Diego may facilitate FDA approval of initial clinical trials, once the company can rebuild its exhausted supply.

Tekmira Pharmaceuticals of Canada may gain important safety information on its test product, TKM-Ebola, now that FDA converted a “full clinical hold” to a “partial hold” on its phase 1 trial, which permits the company to provide the drug to infected patients – but not to healthy volunteers — due to concerns about a potentially serious side effect.

While US and international authorities grapple with these ethical and logistical issues, manufacturers are struggling to ramp up production. Three centers in the US have capacity to quickly scale up production of a vaccine or therapy in response to an epidemic or biological threat and may be tapped to produce candidate vaccines.

A low-priority Ebola vaccine development project at GlaxoSmithKline’s Okairos unit now expects to move from preclinical development to clinical trials this year. Several small biotech firms are looking to gain needed funding to revive abandoned Ebola vaccine development programs. But optimistic statements from biopharma companies raise the risk of appearing “opportunistic” and  “self-promoting,” noted biotech commentator Adam Feuerstein in his “The Street” blog. A lethal outbreak of contagious disease provides an opportunity for biopharma companies to demonstrate their capacity to respond with innovative therapies, based on ethical and scientific factors, as opposed to the potential for financial gain.

by Jill Wechsler

A New Strategy to Tackle Antibiotic Resistance

VLUU L110  / Samsung L110We all know that the discovery of penicillin by Alexander Fleming was a turning point in medical history, which led to the development of lifesaving antibiotics. But who would have thought that decades down the road, we would be battling antibiotic resistance on a global scale. Data from the European Centre for Disease Prevention and Control (ECDC), released at the end of last year, showed a marked increase in carbapenem-resistant infections across Europe. Mortality rates have increased as well due to the limited options of antibiotics available to treat these infections. According to the European Commission, the widespread prevalence of antibiotic resistance has been estimated to cause approximately 25,000 deaths annually and more than €1.5 billion in healthcare expenses and productivity losses in Europe alone. A June 2013 report by IMS Health, “Avoidable Costs in US Healthcare,” noted that in 2012, there were 900,000 hospital admissions for drug-resistant conditions. According to IMS Health, the avoidable cost from antibiotic misuse ranges between $27 billion to $42 billion. Read more »

Where Is the Vaccine for Meningitis B?

The bacterial meningitis outbreak at Princeton University in recent weeks has raised questions about why there is no vaccine in the United States to prevent this deadly disease, when such a therapy is approved in Europe and Australia. The current Princeton health danger prompted the Centers for Disease Control and Prevention (CDC) to request emergency import of the vaccine, and the Food and Drug Administration approved that move, authorizing an investigational new drug application (IND) for Novartis’ Bexsero vaccine. New reports of additional meningitis B cases in California may spark further discussion about ensuring US access to protection against this dangerous infection.

The situation illustrates the complexities of vaccine development, approval and reimbursement at home and abroad. Studies to document vaccine safety usually require testing in thousands of healthy individuals, plus certain high-risk populations. There are four meningitis vaccines licensed in the US, but they protect against four other meningococcal disease strains, and not B, which has proven more difficult to attack.

Moreover, meningitis B is not that prevalent in the US, so Novartis and other vaccine makers have concentrated clinical trials for B preventives in Europe, Australia, and other regions. The resulting study data supported market approval for Bexsero in the European Union in January 2013 and more recently in Australia. More trials have been launched in Latin America and even Canada, but not in the US where Novartis did run a small, early trial on adolescents several years ago. Meningitis is most deadly in infants and very young children, and testing in those populations raises difficult risk-benefit calculations for a relatively uncommon condition.

In April 2011, the FDA advisory committee on vaccines and related biological products discussed the effectiveness of meningococcal vaccines in young children and approaches for demonstrating effectiveness in serogroup B vaccines. Representatives from Novartis and Pfizer made presentations on new but differing approaches for developing effective therapies and new surrogates for demonstrating protection.

Novartis says it is discussing a “pathway to licensure” with FDA and “evaluating its phase 3 plans” for the US and other regions. But market approval of a meningitis B vaccine in the US may take much longer because Novartis and other vaccine makers now appear more interested in developing a combination vaccine that will cover all five meningitis strains.

To some extent, that decision reflects problems with reimbursement for new vaccines, which largely comes from public health agencies. Novartis received a blow in July when the UK’s Joint Committee on Vaccination and Immunisation advised against adding Bexsero to its routine vaccination program. The Committee is reconsidering that recommendation, which was based largely on cost-benefit considerations: whether it is worth the money to vaccinate some 800,000 infants a year when only 1800 cases of the disease are found in children in the UK each year, and there are concerns that the vaccine may be only 73% effective.

These developments have spurred more talk about Novartis selling its vaccine business, which has struggled to be profitable, according to market analysts. Novartis recently announced the sale of its blood transfusion diagnostics unit, which is housed with its vaccines unit.

Researchers and patient advocates for a more effective meningitis vaccine point out that cost-effectiveness studies often fail to assess prevention appropriately. They also fear that a final UK non-coverage decision for Bexsero could discourage vaccine research in general. Coverage certainly would be easier to justify for an all-strain meningitis vaccine, but that may involve years of more testing.

Health Reform May Boost Illicit Drug Sales

Health plans that limit drug converge may encourage consumers to obtain medicines illegally, according to pharmacy experts. Marv Shepherd, director of the Center for Pharmacoeconomic Studies at the University of Texas College of Pharmacy and others noted at the Partnership for Safe Medicines Interchange in October in Washington that an increase in narrow health plan formularies that carry only one or two drugs per class or category will boost purchases of substandard, counterfeit and diverted prescription medicines through illicit operators. Patients accustomed to treatment with a certain drug may seek out other sources of supply if a streamlined plan fails to provide coverage for that medicine, explained Bryan Liang, anesthesiologist and law professor at the University of California San Diego.

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A World Without Polio by 2018

More than 400 eminent scientists and public health experts from around the world have come together to endorse a new initiative to eradicate polio by 2018. The Scientific Declaration on Polio Eradication, published on 11 April, stated that an end to polio is scientifically feasible and achievable. Read more »

Rare Disease Day: Progress and Potential

The US Orphan Drug Act of 1983 defines a rare disease as one that affects fewer than 200,000.  There are nearly 7000 rare diseases affecting nearly 30 million—or nearly one in 10—Americans, according to the National Organization for Rare Disorders (NORD).

The National Institutes of Health (NIH) reports that approximately 80% of rare diseases are genetic in origin; about half affect children. The challenges of facing a chronic, disabling, severe, or even life-threatening disease is compounded by difficulties in receiving an accurate diagnosis, and the lack of therapies to address the condition.

For the past six years, the last day in February has been designated Rare Disease Day. First observed in Europe in 2008 by the European Rare Disease Organization, the initiative expanded to the US in 2009. In 2012, more than 60 countries participated.

This year, events will be held worldwide on Feb. 28 to bring the cause of rare diseases to the attention of patients, the public, the medical profession, and policy makers. For example, the NIH is sponsoring a two-day conference for researchers and policymakers, including FDA representatives, to raise awareness of rare diseases, bring stakeholders closer together, coordinate policy actions between nations, and emphasize rare disease research and the search for new therapeutics.

The Orphan Drug Act provides tax credits and marketing incentives for sponsors to develop products for rare diseases. Since 1983, the number of applications submitted and products designated as orphan drugs has continued to grow. More than 400 drugs and biologic products for rare diseases have been approved.  In contrast, fewer than 10 such products supported by industry came to market between 1973 and 1983.

Drug makers have financial incentives to develop orphan drugs. A 2012 report from Thomson Reuters, The Economic Power of Orphan Drugs, estimated the orphan-drug market at the end of 2011 at $50 billion globally, or 6% of total pharmaceutical sales. However, a compound annual growth rate (CAGR) of 25.8% for 2001 to 2010, compared to a 20.1% CAGR for a matched group of non-orphan control drugs, and an increasing number of orphan-drug approvals, suggests that the growth rate of orphan drugs will exceed that of non-orphan control drugs over the next 30 years, according to the report. Because many orphan drugs are biologics, they are less susceptible to generic competition than small-molecule drugs.

The impact of the smaller population needing the orphan drug is offset by the high cost of the drugs. In fact, the Thomson Reuters study found that 29% of orphan drugs have annual sales greater than $1 billion, about the same percentage as non-orphan drugs.

While the Orphan Drug Act has spurred the development of life-saving and life-improving therapies for individuals with rare diseases, it also has provided financial incentives for drug companies to invest in these narrow specialties. However, as the sponsors of Rare Disease Day point out, there is still much work to be done.

President Obama’s Plans for Healthcare Reform and Job Growth

President Obama’s State of the Union Address on Feb. 12, 2013 touched on some issues that may directly impact the pharmaceutical industry: healthcare reform, innovation, and job creation. So how has the pharmaceutical industry responded?

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UN Pact Scuttles Anti-Vaccine Provision

Jill Wechsler Washington EditorA new international agreement to reduce mercury contamination of air and water was recently adopted by 140 countries, without a proposal that threatened to limit access to vaccines in much of the world. The credit goes to public health authorities and medical experts who challenged a provision blocking production of vaccines with the preservative thimerosal, the formulation necessary for efficient vaccine distribution in developing countries. Banning thimerosal in vaccines “would be a tragedy” that put millions of children at risk, stated GAVI (Global Alliance for Vaccines & Immunization) director Seth Berkley in the New York Times (Jan. 17, 2013). Read more »

Microchips and Video Games: Innovations in Healthcare

Stephanie Sutton Pharm Tech EuropeOnce upon a time in the world of healthcare, the only competition for a pharmaceutical company was other pharmaceutical companies.

Times have changed. Pharma companies are now competing with many nontraditional players in the healthcare arena, including electronic and mobile health firms, retailers, financial services companies and IT firms. Healthcare is no longer about just making a new medicine, but about creating greater value for patients, providers and payers, and encouraging a more active and healthy lifestyle.

In PharmTech, we often talk about manufacturing innovations, such as new dosage forms or more efficient manufacturing techniques, but today I’m going to look at some of the innovations that have been made in the healthcare industry as a whole. Read more »

ABPI Issues Call to Improve UK Pharma

Stephanie Sutton Pharm Tech EuropeThe head of the Association of the British Pharmaceutical Industry has issued a stark warning about the state of the UK’s pharmaceutical industry. In particular, concerns have been raised about the lack of new medicines being used by the UK’s National Health Service (NHS), which has affected the profits of the pharmaceutical industry and led to a decline of R&D in the country. Read more »

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