Study confirms Herceptin’s promise of extra years of living cancer free

The Breast International Group (BIG) in collaboration with Roche announced that women with HER2 positive early breast cancer continue to benefit from Herceptin (trastuzumab) several years after treatment completion and as a result enjoy a longer life disease free. The patients were treated for one year with Herceptin and followed up for four years. These data from the HERA study were presented at the Primary Therapy in Early Breast Cancer conference in St. Gallen, Switzerland.

The HERA (HERceptin Adjuvant) study showed that women treated with Herceptin had a 25% reduction in the risk of their cancer coming back compared to women who did not receive Herceptin, and after four years of medical observation on average, almost 90% of the Herceptin-treated women were still alive. In addition to the significant treatment benefit, this analysis confirmed the long-term safety profile of Herceptin, with good cardiac safety and tolerability maintained throughout the four-year follow-up period.

“These data are extremely important for the treatment of breast cancer” commented Dr Martine Piccart, lead investigator of the HERA study and Chair of BIG. “HERA is the first of the four large Herceptin studies in early HER2 positive breast cancer to substantiate the long-term benefit derived from one year of treatment”.

“These important long-term results from the HERA trial reinforce that women with this aggressive type of cancer have the best chance of cure with Herceptin”, said William M. Burns, CEO of Roche’s Pharmaceuticals Division.

Historically, HER2 positive breast cancer has been associated with a poor prognosis, but the first analysis of the HERA trial, released in 2005, established unprecedented benefits in terms of lowering the risk of cancer returning (disease-free survival). “It is rewarding to see that women with HER- 2 positive early breast cancer can be confident about their future with Herceptin as the foundation of their treatment” said Dr Luca Gianni from the Istituto Nazionale Tumori Milano, Italy, lead investigator of the HERA study.

To date, four large studies HERA, NSABP B-31, NCCTG N9831 and BCIRG 006 have consistently demonstrated that Herceptin prolongs survival in women with HER2 positive early breast cancer.

About the HERA study

HERA is a large international phase III study conducted as collaboration between BIG and Roche. The study, with over 5000 patients enrolled, is assessing the benefits of adjuvant Herceptin treatment in women with HER2 positive early breast cancer. The primary endpoint is disease-free survival (DFS), the secondary endpoint is overall survival (OS) and cardiac safety.

Previously, with 2 years of median follow up, the HERA study demonstrated that one year of treatment with Herceptin given at three-weekly intervals after the completion of adjuvant chemotherapy and/or radiotherapy achieved a highly significant improvement in DFS versus the observation group (no Herceptin), reducing the relative risk of relapse by 36% (hazard ratio [HR]: 0.64; 95% confidence interval [CI]: 0.54, 0.76; p=0.0001).1 Herceptin also reduced the risk of death by 34% compared to observation (HR: 0.66; 95% CI: 0.47, 0.91; p=0.0115). Upon publication of these unprecedented results in 2005, more than 50% of the patients in the observation arm opted to receive Herceptin (‘crossed-over’ to Herceptin treatment).

The focus of the current analysis was to evaluate the efficacy and safety of one year of Herceptin treatment versus no Herceptin at a median of four years follow-up after entry onto the study. The results of the analysis including all women involved in the trial (intent to treat, ITT) showed a 25% reduction in risk of cancer recurrence for women receiving Herceptin compared to those on observation (no Herceptin) (HR 0.76; p=0.0001). At four years of follow-up, nearly 79% of women receiving Herceptin remained cancer-free, a significant increase compared to 73% of women in the observation arm. With regard to safety, it was shown that the incidence of severe cardiac dysfunction associated with adjuvant Herceptin-based therapy was low (0.8%). These results confirm the benefit and safety of one year of Herceptin treatment in women with HER2 positive tumors despite the substantive crossover of patients from the observation group to active treatment. The present analysis also suggests that women who crossed over derived benefit from Herceptin even if they started Herceptin therapy late after completion of adjuvant chemotherapy.

The HERA study is ongoing and final results are expected in 2011.

About breast cancer

Breast cancer is the most common cancer among women worldwide.2 Each year more than one million new cases of breast cancer are diagnosed worldwide, and nearly 400,000 people will die of the disease annually.3

In HER2 positive breast cancer, increased quantities of the HER2 protein are present on the surface of the tumour cells. This is known as ‘HER2 positivity’ and affects approximately 20-25% of women with breast cancer.

About Herceptin

Herceptin is a humanised antibody designed to target and block the function of HER2, a protein produced by a specific gene with cancer-causing potential. The mode of action of Herceptin is unique in that it activates the body’s immune system and suppresses HER2 to target and destroy the tumour. Herceptin has demonstrated unprecedented efficacy in treating both early and advanced (metastatic) HER2 positive breast cancer. Given on its own as monotherapy as well as in combination with or following standard chemotherapy, Herceptin has been shown to improve response rates, disease-free survival and overall survival while maintaining quality of life in women with HER2 positive breast cancer.

Herceptin received approval for use in the European Union for advanced (metastatic) HER2 positive breast cancer in 2000, and for early (adjuvant) HER2 positive breast cancer in 2006. In the advanced (metastatic) setting, Herceptin is approved for use as a first-line therapy in combination with paclitaxel where anthracyclines are unsuitable, as first-line therapy in combination with docetaxel, and as a single agent in third-line therapy. It is also approved for use in combination with an aromatase inhibitor for the treatment of post-menopausal patients with HER2 and hormone receptor co-positive metastatic breast cancer. In the early (adjuvant) setting, Herceptin is approved for use following standard (adjuvant) chemotherapy.

Herceptin is marketed in the United States by Genentech, in Japan by Chugai and internationally by Roche. Since 1998, Herceptin has been used to treat nearly 600,000 women with HER2 positive breast cancer worldwide.

About Roche

Headquartered in Basel, Switzerland, Roche is one of the world’s leading research-focused healthcare groups in the fields of pharmaceuticals and diagnostics. As the world’s biggest biotech company and an innovator of products and services for the early detection, prevention, diagnosis and treatment of diseases, the Group contributes on a broad range of fronts to improving people’s health and quality of life. Roche is the world leader in in-vitro diagnostics and drugs for cancer and transplantation, and is a market leader in virology. It is also active in other major therapeutic areas such as autoimmune diseases, inflammatory and metabolic disorders and diseases of the central nervous system. In 2008 sales by the Pharmaceuticals Division totaled 36.0 billion Swiss francs, and the Diagnostics Division posted sales of 9.7 billion Swiss francs. Roche has R&D agreements and strategic alliances with numerous partners, including majority ownership interests in Genentech and Chugai, and invested nearly 9 billion Swiss francs in R&D in 2008. Worldwide, the Group employs about 80,000 people. Additional information is available on the Internet at http://www.roche.com.

All trademarks used or mentioned in this release are legally protected.

About BIG

The Breast International Group (BIG) is an international non-profit organisation for academic breast cancer research groups from around the world, based in Brussels, Belgium. Created by leading European opinion leaders in 1996, BIG now constitutes a network of 44 groups based in Europe, Canada, Latin America, and the Asia-Pacific region. These research entities are tied to approximately 3000 specialised hospitals and research centres worldwide. BIG also collaborates with the U.S. National Cancer Institute (NCI) and North American collaborative groups, with BIG and the North Americans together representing an impressive integrating force in the breast cancer research arena. To make significant scientific advances in breast cancer research, reduce the wasteful duplication of effort, and optimally serve those affected by the disease, large-scale cooperation is crucial. Therefore BIG facilitates breast cancer research at international level, by stimulating cooperation between its members and other academic networks, and collaborating with, but working independently from, the pharmaceutical industry.

References

(1) Smith I, Procter M, Gelber RD, et al., Lancet 2007; 369:29-36 1

(2) World Health Organization, http://www.who.int/cancer/detection/breastcancer/en

(3) Ferlay J, et al., GLOBOCAN 2002. Cancer Incidence, Mortality and Prevalence Worldwide. IARC CancerBase No.5, Version 2.0. IARCPress, Lyon, 2004. 2004

Roche
http://www.roche.com

View drug information on Herceptin.

The University of Pittsburgh School of Nursing will host a free conference for women living with metastatic breast cancer. Adam Brufsky, M.D., Ph.D., co-director of the Magee Breast Cancer Program of UPMC Cancer Centers and medical director of the Women’s Cancer Center at Magee-Womens Hospital of UPMC, will discuss current and potential treatment options for metastatic breast cancer. The conference also will feature breakout sessions with topics including complementary and alternative therapies, physical activity, self-care strategies, nutrition and communication.

The conference is offered through the support of the Oncology Nursing Foundation Public Education Grant and the Pittsburgh Susan G. Komen Public Education Grant.

WHEN: 9 a.m. to 2 p.m., Saturday, March 21
WHERE: Gilda’s Club
2816 Smallman St.
Pittsburgh, PA 15222

R.S.V.P.: The conference is free and open to women living with advanced breast cancer. Each attendee is welcome to bring a guest.

To register, call (412) 383-8839 or e-mail mros@pitt.edu.

ACCESS PHARMACEUTICALS, INC. (OTCBB: ACCP), announced positive safety and efficacy results from its Phase 2 monotherapy clinical study of ProLindac(TM) in late-stage, heavily pretreated ovarian cancer patients. In this monotherapy study 66% of patients who received the highest dose achieved clinically meaningful disease stabilization according to RECIST criteria. No patient in any dose group exhibited any signs of acute neurotoxicity, which is a major adverse side-effect of the approved DACH platinum, Eloxatin, and ProLindac was well tolerated overall. The maximum tolerated dose of ProLindac was established as well as the recommended dose levels for future combination studies.

“We are very pleased with these results. ProLindac was well tolerated in an absolute sense and relative to commercially-available platinum therapies. We saw significant DACH platinum activity and efficacy in patients at the highest dose levels which is very encouraging given that this study involved monotherapy in a heavily pretreated patient population that typically only respond to an aggressive drug combination,” commented Dr. David Nowotnik, Access’ Senior Vice President R&D. “The DACH platinum activity level seen benchmarked favorably with published studies of monotherapy oxaliplatin in similar but less heavily pre-treated patient populations. Having achieved the recommended dose for future combination studies, we look forward to moving ahead in the clinic ourselves and with our regional partners.”

This 26 patient Phase 2 study explored 3 different dose levels and 2 dosing regimens of ProLindac as a monotherapy treatment for advanced ovarian cancer, to provide data on the monotherapy anticanceractivity and safety of ProLindac. Of patients eligible for evaluation according to standard RECIST criteria, clinically-meaningful disease stabilization was achieved in 42% of all patients, and 66% of all patients in the higher dose groups. Sustained and significant reductions in Ca-125, the established specific serum marker for ovarian cancer, were also observed in several patients.

“We are delighted that the results from this study support our belief that ProLindac is an active platinum agent with a favorable side effect profile,” stated Jeffrey B. Davis, Access’ President & CEO. “These data provide us with a strong incentive to continue the clinical development of ProLindac. As previously announced, we are currently planning a number of combination trials, looking at combining ProLindac with other cancer agents, such as taxol and gemcitabine, in multiple solid tumor indications including colorectal and ovarian.”

Access has previously announced that it has licensed ProLindac to Jiangsu Aosaikang Pharmaceutical Co., Ltd. (“ASK”) for the Greater China Region and to JCOM, Ltd for South Korea. Under these agreements both of these partners will be conducting Phase 2 combination studies with ProLindac in specific tumor types at their expense based on these results. Access is currently in discussion with potential partners for development and commercialization of ProLindac in additional territories.

About ProLindac(TM):

ProLindac is a novel DACH platinum prodrug which has been shown to be active in a wide variety of solid tumors in both preclinical models and in human trials. Access believes that ProLindac’s unique molecular design potentially could eliminate some of the toxic side effects seen in the currently marketed DACH platinum, Eloxatin, which has sales in excess of $2 billion.

About Access:

Access Pharmaceuticals, Inc. is an emerging biopharmaceutical company that develops and commercializes propriety products for the treatment and supportive care of cancer patients. Access’ products include ProLindac(TM), currently in Phase 2 clinical testing of patients with ovarian cancer, and MuGard(TM) for the management of patients with mucositis. The company also has other advanced drug delivery technologies including Cobalamin(TM)-mediated targeted delivery and oral drug delivery, its proprietary nanopolymer delivery technology based on the natural vitamin B12 uptake mechanism; Angiolix(R), a humanized monoclonal antibody which acts as an anti-angiogenesis factor and is targeted to breast cancer; Prodrax(R), a non-toxic prodrug which is activated in the hypoxic zones of solid tumors to kill cancer cells; Alchemix, a chemotherapeutic agent that combines multiple modes of action to overcome drug resistance. Access is also developing Phenylbutyrate (“PB”), an HDAC inhibitor and differentiating agent currently a Phase 2 clinical candidate. Access recently announced the acquisition of MacroChem Corporation. This acquisition provides Access with three additional late-stage product candidates. Pexiganan, a novel topical anti-infective for the treatment of diabetic foot infection, has already completed two Phase 3 trials. EcoNail is a topically applied econazole lacquer based on Access’ proprietary SEPA polymer technology, for the treatment of onychomycosis, a condition commonly known as nail fungus. Thiarabine is a new generation nucleoside analog which has demonstrated both pre-clinical and clinical activity in certain cancers. For additional information on Access Pharmaceuticals, please visit our website at http://www.accesspharma.com.

This press release contains certain statements that are forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, and that involve risks and uncertainties. These statements include those relating to: clinical trial plans and timelines and clinical results for ProLindac and product candidates acquired in the MacroChem transaction, our ability to execute licensing agreements in the future, Access’ plans to continue and initiate clinical trials, the value of its products in the market, its ability to achieve clinical and commercial success and its ability to successfully develop marketed products. These statements are subject to numerous risks, including but not limited Access’ need to obtain additional financing in order to continue the clinical trial and operations and to the risks detailed in Access’ Annual Reports on Form 10-K and other reports filed by Access with the Securities and Exchange Commission.

Also Included In: Women’s Health / Gynecology; Clinical Trials / Drug Trials; Pharma Industry / Biotech Industry

US scientists reviewing 20 years of research and expert opinion on generic versus brand name drugs in the treatment of cardiovascular diseases found no clinical evidence showing brand names were superior to generic versions even though a substantial number of experts writing editorials advised against interchanging them.

The study was the work of Dr Aaron S Kesselheim, of Brigham and Women’s Hospital, Harvard Medical School, Boston, Massachusetts, and colleagues, and is published online in the 3rd December issue of the Journal of the American Medical Association, JAMA.

When two drugs are bioequivalent it means that to all intents and purposes after they have been given to the patient they are biologically equivalent to each other, for example the composition, rate and extent to which their active ingredients are present at the target site inside the body are so similar that you can’t tell the difference between them.

And yet there appears to be a general opinion among doctors and patients that despite the fact generic drugs are bioequivalent to brand name drugs, the brand names are clinically superior. But generic drugs are much cheaper, so Kesselheim and colleagues decided to investigate the available clinical evidence on generics versus brand names and the views of editorial writers on the subject with respect to cardiovascular treatments.

For the study, the researchers systematically searched for peer reviewed studies published between 1984 and 2008 and listed in a number of well known databases, including MEDLINE, EMBASE, and International Pharmaceutical Abstracts.

They selected those studies that compared the clinical effectiveness and safety of generic versus brand name cardiovascular drugs. In a separate exercise they also identified editorials that wrote about substituting brand names with generic versions.

Kesselheim and colleagues then used techniques commonly used in research that reviews other studies, whereby the design, setting, participants, results and funding of each study is extracted and put through a test that assesses the quality of the trial, whilst the results are pooled in such a way that they can then be viewed as if they had come from one giant trial (meta-analysis).

As a separate exercise they reviewed the editorials and classified them as negative, positive or neutral, depending on the authors’ view on generic substitution.

They found a total of 47 clinical trials covering 9 subclasses of cardiovascular drugs, and established the following results:

* 38 of the 47 (81 per cent) trials were randomized controlled trials (considered to be higher quality).

* For beta-blockers, 7 out of 7 randomized controlled trials (100 per cent) found generics to be clinically equivalent to brand names.

* For diuretics, the figure was 10 out of 11 (91 per cent).

* For calcium channel blockers it was 5 out of 7 (71 per cent).

* For antiplatelet agents it was 3 of 3 (100 per cent).

* For statins it was 2 out of 2 (100 per cent).

* For angiotensin-converting enzyme inhibitors it was 1 out of 1 (100 per cent).

* And for alpha-blockers, 1 out 1 randomized controlled trial (100 per cent) found generics to be clinically equivalent to brand names.

* In drugs that have a narrow therapeutic index (where you have to be really careful to give the right dose so as not to injure the patient), clinical equivalence was found in 1 out of 1 randomized controlled trial (100 per cent) for class 1 antiarrhythmic agents, and in 5 out of 5 (100 per cent) for warfarin.

* Pooling the results of all the trials gave a total of 837 participants and an aggregate effect size of -0.03 (95 per cent confidence interval of -0.15 to 0.08), meaning that across the studies as a whole, there was no statistically significant evidence that brand names were superior to generic drugs.

Reviewing the material from 43 editorials, the researchers found that:

* 23 of them (53 per cent), expressed a negative view about whether generic drugs could replace or be used instead of brand names.

* This compared with 12 (28 per cent) that encouraged substitution.

* The other 8 editorials did not reach a conclusion on interchangeability.

* Among editorials covering narrow therapeutic index drugs, 12 (67 per cent) expressed a negative view compared with 4 (22 per cent) in favour generic substitution.

Kesselheim and colleagues concluded that:

“Whereas evidence does not support the notion that brand-name drugs used in cardiovascular disease are superior to generic drugs, a substantial number of editorials counsel against the interchangeability of generic drugs.”

They wrote that the rising cost of prescription drugs is a critical policy issue: it strains the budgets of patients and insurance providers, and leads to poorer health as it works against helping everyone to make sure patients can complete their medication schedules.

“The primary drivers of elevated drug costs are brand-name drugs, which are sold at high prices during a period of patent protection and market exclusivity after approval by the Food and Drug Administration (FDA),” they added.

The idea of generics is to help people afford drugs, and these become available after the brand names have had their period of being the only ones on the market, the so called exclusivity period. Many doctors and payers encourage this.

However, some patients and doctors have been concerned that the generic versions may not be as effective. As Kesselheim and colleagues explained:

“Brand-name manufacturers have suggested that generic drugs may be less effective and safe than their brand-name counterparts. Anecdotes have appeared in the lay press raising doubts about the efficacy and safety of certain generic drugs.”

Kesselheim and colleagues suggested that one explanation for the discordance between the clinical evidence and the opinion expressed by experts in the editorials could be that:

“Commentaries may be more likely to highlight physicians’ concerns based on anecdotal experience or other nonclinical trial settings.”

Another explanation they suggested was that the:

“Conclusions may be skewed by financial relationships of editorialists with brand-name pharmaceutical companies, which are not always disclosed.”

Nearly half the trials (23 out of 47) and nearly all the editorials and commentaries they reviewed did not reveal where the funding came from, noted Kesselheim and colleagues, who also wrote that:

“We identified numerous studies that evaluated differences in clinical outcomes with generic and brand-name medications. Our results suggest that it is reasonable for physicians and patients to rely on FDA bioequivalence rating as a proxy for clinical equivalence among a number of important cardiovascular drugs, even in higher-risk contexts such as the NTI drug warfarin.”

“These findings also support the use of formulary designs aimed at stimulating appropriate generic drug use. To limit unfounded distrust of generic medications, popular media and scientific journals could choose to be more selective about publishing perspective pieces based on anecdotal evidence of diminished clinical efficacy or greater risk of adverse effects with generic medications. Such publications may enhance barriers to appropriate generic drug use that increase unnecessary spending without improving clinical outcomes,” they added.

“Clinical Equivalence of Generic and Brand-Name Drugs Used in Cardiovascular Disease: A Systematic Review and Meta- analysis.”
Aaron S. Kesselheim; Alexander S. Misono; Joy L. Lee; Margaret R. Stedman; M. Alan Brookhart; Niteesh K. Choudhry; William H. Shrank.
JAMA. Vol 300, No 21, pp 2514-2526, December 3, 2008

Noroviruses are a group of viruses that are the most common cause of acute gastroenteritis and the HPA estimates that they affect between 600,000 and a million people in the UK each year. Infection can spread rapidly, and as Norovirus outbreaks continue to hit the headlines, the RIPH is pleased to announce it is running the first in a UK-wide programme of one-day courses on Norovirus in May.

The course is designed to provide delegates working in the hospitality industry with the skills needed to recognise the signs of Norovirus infection, and to prevent and manage outbreaks. By the end of the course, delegates will go away equipped with the skills necessary to prepare their own practical infection control strategy. The course will be particularly relevant to occupational health professionals, health and safety practitioners and environmental health officers.

Professor Rodney Cartwright, RIPH Trustee and Medical Adviser to the Federation of Tour Operators comments, “With the continuing difficulties involved in controlling Norovirus outbreaks, the RIPH is pleased to be continuing its educational programme on Norovirus, and this course will complement the three successful conferences we have already run on the issues surrounding this problem. The course will be of specific interest to those responsible for the management of infection in non-clinical outbreak settings such as hotels, cruise ships, health clubs and holiday sites.”

The course will cover:

- the clinical features of Norovirus, molecular identification and detection techniques;
- the importance of staff training;
- routine cleaning and product selection;
- procedures for declaring an outbreak;
- managing staff illness;
- reducing person to person spread;
- statistics and theory of hand hygiene (including practical demonstration and training);

Notes :

- The Royal Institute of Public Health was founded in 1886. Based in London, it is a leading independent body with an international reputation dedicated to the promotion, practice and protection of the highest standards of public health.

- It offers a wide range of public health related qualifications delivered through an extensive network of training centres nationwide.

- The Royal Institute enjoys the patronage of Her Majesty The Queen, an honour that has been conferred by the Sovereign continuously since 1897. The Royal Institute is a Registered Charity.

The Royal Institute of Public Health

Hosted by the Biotechnology Industry Organization (BIO), the BIO International Convention will take place June 17-20 at the San Diego Convention Center in San Diego, Calif. The convention will highlight all the ways that biotechnology, the life sciences, investments, policy, and the international community work together to heal, fuel, and feed the world. This year, the day pattern will change to Tuesday through Friday (previously, the Convention was held Sunday through Wednesday).

The BIO International Convention features keynote sessions from thought leaders, including policymakers, scientists, CEOs and celebrities. Former speakers include President George W. Bush, President Bill Clinton, Michael J. Fox, and Her Majesty Queen Noor of Jordan, among others. The convention will include 175 breakout sessions covering biotech trends, policy issues and technology innovations and the world’s largest biotechnology exhibition – the BIO Exhibition. The preliminary schedule is available at http://www.bio2008.org/schedule.

Recently, the BIO International Convention was named one of the Fastest 50, a list of the fastest-growing trade shows in the country, by Tradeshow Week magazine.

In 2007, the convention attracted 22,366 attendees and exhibitors with approximately one-third from countries outside the U.S. Growth of the meeting, and BIO overall, has paralleled that of the dynamic industry that BIO represents. BIO currently projects more than 20,000 attendees for the 2008 BIO International Convention in San Diego. The convention was last held in San Diego in 2001 and attracted 14,731 professionals.

Registration is complimentary for credentialed members of the news media. To register, please visit here. Reporters and editors working full-time for print, broadcast, and online news organizations may register onsite with valid media credentials. All freelancers, college and online publications are strongly encouraged to register in advance by Wednesday, June 4, 2008.

The BIO International Convention helps to support BIO programs and initiatives. BIO works throughout the year to create a policy environment that enables the industry to continue to fulfill its vision of bettering the world through biotechnology innovation. For more information on the global event for biotechnology, including program and housing information, please visit http://www.bio2008.org.

Advance media registration for the 2008 BIO International Convention, the Global Event for Biotechnology, is now available online here.

About BIO

BIO represents more than 1,100 biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and 31 other nations. BIO members are involved in the research and development of healthcare, agricultural, industrial and environmental biotechnology products. BIO also produces the annual BIO International Convention, the world’s largest gathering of the biotechnology industry.

http://www.bio.org

Backed by a government intent on promoting innovation and fuelled by the “brain gain” of talented scientists and entrepreneurs returning from abroad, China’s health biotech industry only needs a more favourable investment climate to emerge as a global force in the production of therapies and medicines – both new and low-cost generics – experts say in a new study.

Long considered a skillful product replicator, China today boasts of daring medical science innovation and stunning breakthroughs – including the world’s first commercialized gene therapy product and the sole cholera vaccine tablet. However, Chinese firms face an uphill battle in attracting high-risk venture capital needed to sustain innovative, research-driven projects, says the study published by Nature Biotechnology.

Conducted through face to face interviews with management of 22 Chinese firms, the work is the first study of China’s most innovative health biotechnology companies available in the public domain.

It says that despite substantial Chinese government funding to promote an innovative industry and entrepreneurs who will commercialize new health biotech products, the intense interest of potential international investors is typically muted by an uncertain financial system, rigid restrictions on the export of capital that limit the options for exiting investments and continuing doubts about the Chinese government’s approach to quality control and intellectual property rights.

“The Chinese biotechnology industry is like a baby dragon, which will grow quickly and soon become hard to ignore. It’s no longer the case that the industrialized world has hegemony over biotechnology innovation,” says co-author Peter A. Singer, MD, of the McLaughlin-Rotman Centre for Global Health (University Health Network and University of Toronto).

“However, for all its blossoming as an industrial and economic superpower, China still has one foot in the closed society of the past. For the sake of both national and global health, we hope China will embrace the financial and regulatory reforms needed to attract the venture capital required for sustained innovation in the health biotech sector,” he adds.

The report builds on a similar study of India’s private health biotech sector published in April 2007, opening a window on the product development capabilities and strategies used in rapidly growing economies to survive and grow amid developing country challenges. It also recommends policies that could help their biotech firms succeed.

Lead author Sarah Frew, a research associate of the McLaughlin Rotman Centre for Global Health, says the budding of biotech industries in China and India holds major implications for the global industry and for improving both health and prosperity in the developing world.

The biotech revolutions in the two countries differ significantly, she says: India’s firms are largely focussed on process innovations to improve affordability and accessibility of medicine among local and global populations; Chinese firms are striving to create novel products in such areas as gene therapy and regenerative medicine.

“Addressing the health needs of 1.3 billion people in China is global health,” says Dr. Frew. “The challenges faced by the private sector, research institutes and universities, and health care providers will be very difficult to meet unless the country’s leaders are prepared to look at new approaches.”

Drs. Singer and Frew, with co-authors Abdallah S. Daar, MD, Stephen Sammut, Alysha Shore, Joshua Ramjist, Sara Al-Bader and Rahim Rezaie, say they found a home-grown Chinese private health biotech sector motivated both financially and morally to develop innovative health products to address local and global health needs.

“The responsibility now lies with both the Chinese government and the international health community to support these companies in their ventures and ensure that these products reach their intended markets,” says Dr. Frew.

The return of the ‘sea turtles’

The report notes a strong focus on encouraging the return of Chinese scientists and entrepreneurs who left the country to study or train abroad – the so-called ‘sea turtles’ who return with scientific talent and international credibility.

“While the ‘sea turtle’ phenomenon is beneficial, the country’s industry might be better served if Chinese residents in the West built transnational companies with a footprint in both China and the West,” says co-author Stephen M. Sammut of Burrill & Company, San Francisco.

“While this practice is already common, regulations and taxation policies to encourage this approach would address many of the concerns of private and public capital, assure prospective alliance partners, and add depth to the pool of experienced managers. Such an approach would also promote China as a co-development partner rather than a purely low-cost venue to international companies to contract services.”

Population a major driving force

China’s 1.3 billion inhabitants (20% of the world’s population) create a significant demand for low-cost products with the result that biogenerics accounted for more than 90% of the $3 billion biopharmaceutical market last year. For some companies, generic products represent a low-risk entry point into the industry.

The same is true for diagnostics. Chinese companies like Beijing Wantai Biological Pharmacy Enterprise and Shanghai Huaguan Biochip Co., Ltd. play an important role in keeping local consumer prices down. Wantai has developed and marketed a large range of blood screening tests for diseases such as HIV, hepatitis B and C, sexually transmitted diseases and rotavirus. Huaguan earns most of its revenue exporting its range of fertility tests and formulations for HIV, tuberculosis, hepatitis C and STDs to other Asian, African and South American countries.

The Chinese government, however, remains intent on pushing applied research, driving Chinese firms to develop new therapies in pioneering fields such as gene therapy and stem cells.

The first commercialized gene therapy product approved anywhere in the world was Gendicine, an injection used in the treatment of head and neck cancers developed by Shenzhen SiBiono GeneTech Co., Ltd. More than 5,000 patients have been treated with Gendicine, about 400 of them from overseas. The drug is currently undergoing further clinical trials in China for several new indications, including liver, abdominal and pancreatic cancer.

Several Chinese companies are working in the field of human and animal stem cells. One of them, Beike Biotechnologies, has organized a network of satellite hospitals, clinicians and research laboratories to commercialize its stem cells therapies, which involve harvesting stem cells from the umbilical cord or amniotic membrane, in vitro expansion, and administration to patients either intravenously or by injecting directly into the spinal cord.

Beike has treated more than 1,000 patients, including 60 foreigners, for a variety of conditions including Alzheimer’s disease, autism, brain trauma, cerebral palsy, diabetic diabetic foot arteriosclerosis and spinal cord injury.

Because therapies using cells derived from the umbilical cord are considered a clinical technology in China, the State Food and Drug Administration does not require clinical trials before these treatments are approved. This absence of clinical data makes it difficult to evaluate the efficacy of these therapies.

New therapies developed for local and global health problems

The study found that the government’s focus on innovation is also driving development of new therapies to address significant local health needs, including lung fibrosis, liver cirrhosis and various problems associated with an aging population caused in part by the country’s one-child policy.

Lung fibrosis, caused by radiation treatment, is a major cause of death for the more than 275,000 Chinese who die from lung cancer every year. Inflammation and fibrosis of the liver is a side effect from hepatitis B virus infection, which afflicts over 100 million Chinese.

Shanghai Genomics Inc. has focused its efforts in this area, developing novel non-steroid anti-inflammatory therapeutics to replace currently available treatments which achieve poor results and have many side effects. The company’s first product on the market, GuBang, is a material for bone void filling that can aid bone growth and serve the growing needs of Asia’s aging citizens.

Chinese firms are also developing vaccines to address both local and global needs. They include Shanghai United Cell Biotech, which is manufacturing and marketing one of only two oral cholera vaccines available worldwide (and the only one available in tablet form). Other firms are working on an oral HIV vaccine and novel vaccines against Japanese Encephalitis, SARS and pandemic avian influenza (H5N1 strain).

Barriers to development

The study found that the lack of favourable conditions for investment in an innovative biotech sector may be actively discouraging its growth. Most Chinese biopharmaceutical companies sell generic drugs and do not invest in R&D.

“Some of the firms that are pursuing innovative R&D have incorporated hybrid business models that dilute resources to include contract services or non-innovative products, to first fund the firm’s survival and then fund R&D activities,” the report says.

These hybrid business models have fallen out of favor in the west, where venture capitalists prefer a well-defined targeted strategy over a mix of models that mitigate commercial risk. As price-based competition among domestic manufacturers continues to put pressure on profit margins, even fewer firms may be able to support in-house R&D programs.

Other barriers to growth include a lack of trust among potential international partners, particularly in discussions that involve intellectual property protection. Other barriers to international partnerships involve language, travel, culture, and differences in project management styles.

Slow-moving policy and regulatory processes hinder progress in a fast-paced research environment. Among the major issues China will need to address soon is the need to expand its capabilities in protecting intellectual property by refining civil procedures, developing a body of jurists, and accumulating a body of precedent and custom for assessment of damages.

Sales and distribution are problematic for small Chinese companies; as a result the needs of the large domestic market are unmet. The country’s health system is weak and in many cases clinicians, facilities and products are not reaching rural or isolated regions. Another challenge is the disparity between the high cost of developing an innovative health product and the price the domestic market can pay.

Other lessons learned

The government’s efforts to expand China’s innovative capacity in biotechnology are bearing fruit. According to the country’s Institute of Science and Technology Information, China in 2006 became the world’s second largest scientific research publisher (after only the U.S.), indicating improved research and innovation capacity and deeper involvement of Chinese scientists in the international academic community.

The researchers say Chinese companies need global health organizations to help them increase global access to their products.

As well, in addition to incentives to develop domestically novel products that address local health needs, more government-sponsored incentives and procurement programs are needed to ensure that innovative products reach their intended end users.

Recommendations for biotech development in China

* Reform the financial environment to facilitate exit mechanisms for entrepreneurs and investors in the health biotech sector.

* Create and promote specialty programs in biotechnology entrepreneurship and management.

* Leverage the ‘sea turtle’ phenomenon to promote transnational companies that will be attractive to western investors and strategic partners.

* Promote credibility of domestic firms to the international community by enforcing uniform financial reporting, a transparent regulatory regime and fair business practices.

* Enact timely legislation and regulations to nurture scientific and economic development.

* Stimulate rapid development of the IP infrastructure through academic and exchange programs.

* Strengthen health systems infrastructure and distribution mechanisms in concert with the development of the industry to ensure that innovative health biotech products are available to the entire domestic population.

The NPA have produced a guide to completing the Pharmacy Premises Assessment Tool for Scottish pharmacy contractors and Health Boards.

It follows the circulation of the assessment tool by the Scottish Government in September 2007. The tool must be completed by 31 March 2008 for contractors to qualify for payment.

This resource aims to help pharmacists satisfactorily complete the assessment by providing practical suggestions and signposting to further sources of information for each step of the assessment tool. A template Disability Discrimination Act assessment form is included in the document.

This document is available in electronic format only and can be downloaded from the NPA members’ website http://www.npa.co.uk/members or obtained by emailing m.mcdonald@npa.co.uk.

http://www.npa.co.uk

Epinephrine (adrenaline) is a drug that can be used to treat life-threatening allergies. The drug is stored in salt water and can be expressed as both a dose or mass concentration level (e.g. 1 mg per 1 mL of salt water) and a ratio (1 part drug for every 1000 parts of salt water).

This article appears in Annals of Internal Medicine.

A randomized, blinded, controlled study, designed to see whether labeling the drug as a dose or a ratio affected accuracy or speed in giving the drug, divided 28 doctors into two groups to handle a simulated severe allergic reaction.

The study found that all but two of the doctors in the group that used adrenaline in ampules with ratio labels gave more adrenaline (i.e. overdosed) and took longer to do so than the doctors in the group using ampules with dose labels (Article, p. 11).

http://www.annals.org

American College of Physicians

Novartis officials on Thursday announced pilot projects in three states aimed at developing closer relations with health insurers and HMOs, which increasingly are influencing what medications physicians prescribe, the Wall Street Journal reports. The pilot projects will begin in January 2008 in Minnesota, Oregon and Washington state.

According to the Journal, “The move reflects a growing reality” in the pharmaceutical industry as major payers seek to cut health care costs by influencing physician prescribing habits. In some U.S. markets, many physicians work for HMOs that decide which drugs to cover based on cost and efficacy. Physicians often are less likely to prescribe drugs if payers refuse to cover the medications. The Journal reports that the “frugality of payers is a big reason pharmaceutical companies are tightening their belts and predicting a sales slowdown.”

Joe Jimenez, CEO of the Novartis pharmaceutical division, said that sales representatives in some markets will focus more attention on payers and less on individual physicians. He added that in HMO markets, “if you are organized around the physician, you are missing the key influencer.” If the projects are successful, the initiative will be introduced to other areas of the U.S. and in Europe (Whalen, Wall Street Journal, 12/14).

Reprinted with kind permission from http://www.kaisernetwork.org. You can view the entire Kaiser Daily Health Policy Report, search the archives, or sign up for email delivery at http://www.kaisernetwork.org/dailyreports/healthpolicy. The Kaiser Daily Health Policy Report is published for kaisernetwork.org, a free service of The Henry J. Kaiser Family Foundation© 2005 Advisory Board Company and Kaiser Family Foundation. All rights reserved.