New research unit boosts hope for patients with rare diseases

On Monday, Pfizer announced the creation of a new research unit which will specifically focus on the treatment of orphan diseases — those that affect fewer than 200,000 patients worldwide.

Of the more than 6000 diseases classified as orphan diseases, fewer than ten percent have therapies that directly address the underlying disease.  Pfizer stated that they intend to work closely with patient advocacy groups as it develops and advances the unit’s research strategy.

The new unit got the thumbs-up from National Organization for Rare Disorders in the USA. Its chief executive, Peter Saltonstall, noted that “30 million Americans, 30 million Europeans and millions more around the world have rare diseases” and applauded the development of new treatments “for this medically-underserved population.”

Revolutionizing immunization campaigns? New technology keeps vaccines stable at tropical temperatures

British scientists have found a cheap and simple way of keeping vaccines stable, even at tropical temperatures, which could transform immunization campaigns in the developing world (Reuters).

The ability to transport vaccines at normal temperatures would reduce cost and greatly improve access to vaccines.

The newly discovered method involves mixing the vaccine with the sugars trehalose and sucrose and leaving it to dry out on a filter or membrane.  As the water evaporates, the vaccine mixture turns into a syrup and solidifies on the membrane, preserving the active part of the vaccine in a kind of suspended animation and protecting it from harm even at high temperatures.  Flushing the membrane with water rehydrates the vaccine in a few seconds,

The researchers managed to store two different virus-based vaccines on sugar-stabilized membranes for 4 to 6 months at 45 degrees Celsius (113F) without the medicines being damaged.

The next step in the research is to demonstrate that the process can be scaled up for large production with standard or newly-licensed human vaccines.  The work was funded by the Bill and Melinda Gates Foundation and the Wellcome Trust.

Increase in innovation, access to orphan drugs

The number of orphan drug designations in the US more than doubled in the last decade, growing from 208 during 2000-02 to 425 in 2006-08, says a new study.

Since the US Orphan Drug Act of 1983 was signed into law, more than 2,000 products in development have been designated as orphan drugs, while the Food and Drug Administration (FDA) has granted market approval to 350 drugs and biologics, according to the study, which was conducted by the Tufts Center for the Study of Drug Development (CSDD).

Orphan drugs are products developed for a rare disease or condition affecting fewer than 200,000 people in the US, while in the European Union (EU) they are defined as treatments for diseases or conditions affecting five people out of every 10,000, or fewer.

The National Institutes of Health (NIH) estimates that 25 million Americans have a rare disease.

Malaria drugs may help fight lupus

Drugs used to treat malaria may be useful for patients with lupus, a chronic debilitating “autoimmune” disease, according to according to a new report published in the journal Arthritis and Rheumatism.

“The data presented, taken in conjunction with the data from the published literature, suggest that antimalarials should be used in all lupus patients regardless of their disease manifestation or disease duration,” the authors concluded.

Pons-Estel and his team studied nearly 1,500 patients with lupus from 9 countries.  About 12 percent of the patients who did not use the drugs died during the follow-up period, compared to about 4 percent of those who did.  The difference was even higher for patients who used the drugs for more than two years.  After the team accounted for various factors, using antimalarial drugs appeared to reduce the risk of death during the study by almost 40 percent.

Lupus is a chronic disease in which the immune system confuses its own healthy tissues with foreign tissues and sometimes attacks both. The condition can manifest as a skin rash or arthritis and may lead to damage to the kidneys, heart, lungs and brain to varying degrees. The disorder disproportionately affects women.

Pharmaceutical company opens access to malaria research and compounds

Pharmaceutical company GlaxoSmithKline has outlined a series of initiatives aimed at attacking neglected diseases and says that its experimental malaria vaccine is just a couple of years away from being approved.

GlaxoSmithKline (GSK) is to reveal previously confidential data on thousands of potential anti-malaria compounds. The company has 13,500 molecules which have been tested against the parasite which causes malaria.

In addition to this, the company is to pump millions into an ‘Open Lab’ for independent research teams.  One expert said more sharing of data could trigger advances like those that came from the human genome project.

Dr Timothy Wells, Chief Scientific Officer of the Medicines for Malaria Venture, which has worked with GSK on the project, said it had the potential to “dramatically alter” the way the world approached malaria research.

GSK also outlined what it calls a “sustainable approach” it has developed to price RTS,S, the world’s most advanced malaria candidate vaccine, which is currently in late-stage trials across seven African countries.  The pricing model “will cover the cost of the vaccine together with a small return” which will be “fully reinvested into R&D for second-generation malaria vaccines, or vaccines for other neglected tropical diseases”.

More positive news in the fight against HIV/AIDS – close to 100 new products in testing for treatment/prevention

97 new products for the prevention or treatment of HIV/AIDS and related conditions are currently in human clinical trials, or awaiting approval by the FDA, according to the Pharmaceutical Research and Manufacturers of America.  These products in development include 23 vaccines and 54 antivirals.

While not all these products will be approved, it’s more encouraging news in the fight against HIV/AIDS following from yesterday’s announcement by UNAIDS of a global decline in HIV infection rates (UNAIDS report: 33.4 million worldwide have HIV, but infections slow).  UNAIDS estimates that new HIV infections worldwide have been reduced by 17% over the past eight years.

Since the introduction of anti-retroviral therapy in 1995, the US Centers for Disease Control and Prevention (CDC) estimates that the annual number of deaths in the US due to AIDS has dropped by more than 70%.

Innovative vaccine venture offers hope to less-developed countries

Britain’s largest charity, the Wellcome Trust, and pharmaceutical company Merck & Co. have joined together to create a non-profit, £90-million (about $150 million) research center in India to facilitate the development of new vaccines, “including [those for] neglected diseases for which inadequate or no vaccines exist.”  According to Nature News, “the move marks the first time that a major medical-research charity and a pharmaceutical company have directly partnered to create vaccines aimed at low-income countries.”

Wellcome Trust Director Mark Walport said, “Linking the ingenuity of academic research with the know-how of industry is vital if we are to produce a new generation of vaccines to reduce the burden of infectious diseases in low-income countries.”

The joint venture has yet to decide which diseases it will tackle, but will base the decisions on criteria such as scientific and technical feasibility, affordability and whether vaccine formulations will meet the field and other needs of the large procurement agencies such as the WHO, the United Nations Children’s Fund (UNICEF) and the GAVI Alliance.

“Affordability will be key in the technical and other choices all along the product design and development path,” said Mark Feinberg, Merck’s vice president of medical affairs and policy. “We really want to lower a lot of the barriers that exist for developing promising products.”  The center is expected to open by the end of the year.

Drug development for TB: an innovative collaboration

A landmark collaboration could see the development of the first new-action tuberculosis drug in 40 years.  Global Alliance for TB Drug Development (TB Alliance), a not-for-profit, product development partnership, and Tibotec, a Johnson & Johnson company, have joined forces to focus on the development of the new experimental drug TMC207.

Forbes reports that if the drug is approved, it could be the first of its kind on the market and the first tuberculosis drug in more than four decades to take a new approach to treating the condition. A cocktail of antibiotics is the mainstay of treatment and there has been no major advancements since the antibiotic Rifampicin hit the market in the 1960s.

The drug candidate, now in midstage clinical trials, works by inhibiting an enzyme responsible for fueling tuberculosis cells, thereby cutting off the cell’s energy supply. Midstage study results have so far shown the drug is effective as part of a combination treatment when compared with a placebo. Results from a second stage of the midstage program are expected in 2010.

If it were approved, the parties involved will establish an access program for developing countries. The TB Alliance will have a royalty-free license on the drug for drug-susceptible forms of tuberculosis.

Tuberculosis remains one of the world’s deadliest diseases and has harshly impacted developing countries as the drug-resistant form of the condition gains ground. The World Health Organization estimates approximately one-third of the world’s population is infected with bacillus that causes tuberculosis and the disease is responsible for nearly 5,000 deaths each day worldwide.

WHO and 30 vaccine manufacturers meet: commit to meeting public health needs re H1N1

WHO Director-General Dr Margaret Chan and United Nations Secretary-General Ban Ki-moon met with over 30 vaccine manufacturers from developing and developed countries at WHO headquarters yesterday.  Industry representatives affirmed their wish to cooperate in making supplies of any eventual vaccine for Influenza A(H1N1) available to developing countries, and said they stood ready to produce the vaccine when requested.

At a press conference later in the day, Dr Chan said, “We have a very serious commitment from companies in the North and in the South to work with WHO.”  She said the Secretary-General’s ability to mobilize resources “is extremely vital when the world is under threat of an imminent pandemic.” 

Together with the UN and other members of the global community, Dr. Chan stated that the WHO would work to find innovative funding mechanisms to ensure that developing countries were not denied access to vaccines because of lack of means.  Both the Director-General and Secretary-General expressed their appreciation of the efforts of the manufacturers, with support from governments, to further increase their production capacity.

While the development and production of a vaccine for Influenza A(H1N1) is certainly essential, there has been some debate recently regarding whether production efforts should be diverted to provide for a potential swine flu pandemic or remain focused on creating vaccines for traditional seasonal viruses.  This is of particular concern in areas of Latin America and Africa which are approaching their main flu seasons.

New Pharmaceutical Price Regulation Scheme in the U.K. to Encourage Innovation and Access

A new Pharmaceutical Price Regulation Scheme (PPRS) came into effect in the U.K. on January 1, 2009.  The PPRS represents an attempt to balance incentives for innovation with cost and price restraint.  The objectives of the 2009 PPRS are:

• To deliver value for money; securing the provision of safe and effective medicines at reasonable prices;
• To encourage innovation by promoting a strong and profitable pharmaceutical industry;
• To promote access and uptake for new medicines; and
• To provide a stable and predictable market environment.

“The health departments of the UK and the Association of the British Pharmaceutical Industry have a common interest in ensuring that safe and effective medicines are available on reasonable terms to the NHS, and in a strong, efficient and profitable pharmaceutical industry. Such an industry must be capable of sustained research and development leading to the future availability of new and improved medicines in this and other countries.”
              — Department of Health Site

Some of the important new features are provisions aimed at encouraging and rewarding innovation and encouraging the uptake of cost-effective medicines.