Progress in delivery science, manufacturing technologies, and commercialization are playing critical roles in advancing the development of complex parenteral drug formulations for new drug substances having a variety of formulation challenges. Unique formulation strategies and solutions are being investigated to extend the release of target peptides, proteins, nucleic acids, and small molecules for systemic, local, or cellular delivery. Pharmaceutical Technology will hold a live educational webcast, “Complex Parenteral Formulations for the Delivery of Peptides, Proteins, Nucleic Acids and Small Molecules,” on Thursday Nov. 21 from 10:00 to 11:00 AM EST, to examine the delivery science and product development of complex parenteral formulations. Read more »
Archive for the 'R&D' Category
A team of scientists recently reported on the first study of interactions between the antiretroviral drug, amprenavir, and the HIV-1 protease enzyme. The findings provide the first true picture of how the protease inhibitor blocks viral replication. More importantly, the study reveal how drug design can be improved to enhance performance, combat resistance and reduce dosage of antiretroviral medications to treat HIV. Read more »
We’re already embroiled in the annual speculation game about whether FDA approvals this year will keep pace with last year’s near-record of 39 new molecular entities (NMEs) brought to market. The tally is closely watched as a sign of the state of biopharmaceutical innovation and the health of the pharmaceutical industry and biomedical research enterprise.
But this focus on the quantity of new drug approvals is misleading, according to FDA analysts, because it fails to distinguish between truly innovative new therapies and those that are similar to medicines already on the market, explains Mike Lanthier, operations research analyst on the economics staff of FDA’s Office of Planning. While all NMEs offer some therapeutic advantage, those that are “first-in-class” and “advance-in-class” medicines represent important advances, he explains. Alternatively, “addition-to-class” therapies may provide useful options for patients, but not substantial advances over existing products. And it is this last category that has experienced the much-hyped decline in approvals in recent years, while more vital therapies are holding steady or increasing in number, Lanthier points out in an FDA Voice blog posted Aug. 6, 2013. This “more nuanced and informative” assessment of NME categories thus refutes fears of an “innovation gap” that threatens drug discovery.
One factor may be a rise in small biopharma companies developing more innovative drugs, while large drug companies have focused on refining blockbuster drugs for large patient populations. NMEs from small companies have increased notably since 1996 and now account for 50% of approvals, compared to roughly one-third in the past, Lanthier and colleagues explain more fully in an article in the August 2013 issue of Health Affairs.
FDA initiatives also may support these developments. Most innovative NMEs have benefited from priority review treatment, and the new breakthrough drug program and added incentives for new antibiotics and pediatric treatments promise to expedite the development of innovative therapies. It’s also possible, the authors say, that the increased influence of large pharmacy benefit management firms reduces reimbursement for pricey drugs that lack proven benefits.
Another measure of biopharma innovation may be the number of new drug applications filed with FDA, although the quantity-over-quality measure also may apply here.
Risk and reward. It is a balance that has to be achieved in any business endeavor and is of utmost importance for pharmaceutical and biopharmaceutical companies managing their growth and manufacturing in emerging markets. Emerging markets are a crucial part of pharmaceutical companies’ growth strategies, but in serving those markets, pharmaceutical and biopharmaceutical manufacturers must align that strategy with partners that can facilitate access to local markets, manage complex supply chains, meet global and national regulatory standards for quality, and secure production for local as well as established markets in North America and Western Europe. Read more »
Pharmaceutical Research and Manufacturers of America (PhRMA) International Vice-President Jay Taylor has expressed the organization’s concerns over the Office of the United States Trade Representative’s (USTR) 2013 Special 301 Report. The Special 301 Report, released in May 2013, is an annual review of the state of intellectual property (IP) rights protection and enforcement in trading partners around the world and reflects the US Administration’s resolve to maintain IP protection worldwide.
Expressing appreciation for USTR’s efforts to ensure IP protection, Taylor expressed dismay that an out-of-cycle review was not granted for India. “The deteriorating protections for patented medicines in India have become increasingly concerning,” Taylor said in a PhRMA blog. “Over the past year, the Government of India has issued several intellectual property decisions that have disproportionately impacted US biopharmaceutical companies and a number of other innovative sectors. The IP regime in India has been structured and applied in ways that prop up local industries to the detriment of US jobs and the worlds patients.”
Ernst & Young recently released its annual biotechnology industry report, Beyond Borders: Matters of Evidence, stating that while the major players are performing well, it is essential that small- to mid-size biotech companies focus on demonstrating the value of products in their pipelines instead of just creating a drug that works. If not, they will lose out in a challenging environment, especially now that there is a global shift towards evidence-based healthcare. Read more »
Stem cells are being developed to treat a diverse set of conditions, including spinal cord injury, amyotrophic lateral sclerosis, macular degeneration, Parkinsons disease, and Type I diabetes. But the challenges in moving from the laboratory to the clinic are formidable. The California-based biotech company, Geron, pioneered clinical trials using embryonic stem cells, with a Phase I trial using oligodendrocyte precursors derived from embryonic stem cells to treat spinal cord injury approved in 2009. In November 2011, however, Geron announced it would be discontinuing all of its stem-cell development work, citing cost and regulatory complexity as factors in that decision.
At the BIO International Convention, a panel will convene to discuss the current landscape for developing stem cell therapies in a breakout session titled Opportunities and Challenges in Developing Innovative Stem Cell Therapies. BioPharm International spoke with one of the session’s presenters, Dr. Armand Keating, Epstein Chair in Cell Therapy and Transplantation, University of Toronto about the challenges facing developers. According to Keating, funding remains the biggest challenge in moving from laboratory to clinic. He points out that unlike small-molecule development, stem-cell development is still very much rooted in academia. The traditional sources of funding available to academics are ill-suited or inadequate to fund preclinical validation studies, manufacturing scale-up studies, or the clinical trials themselves. While an organization such as the California Institute for Regenerative Medicine is able to provide some funding for early-stage clinical trials, later-stage trials will be far too expensive to be carried out at academic research centers, he says.
Clarification of statement in podcast: There have been approximately one million bone marrow transplants performed worldwide since 1959.
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 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?
Developing and maintaining the right labor pool is an ongoing challenge for any industry, and it is one that the pharmaceutical industry also is facing. Recent attention in the pharmaceutical industry has focused on the restructuring that has occurred and that is still occurring among the large pharmaceutical companies. And while re-allocating resources, including labor resources, is always a crucial task for companies, it is a challenge made even more difficult amidst shifting industry fundamentals and a demand for higher productivity from scientific talent.
The pharmaceutical industry requires employees with a high-end scientific skill set, and PwC’s 2012 Global CEO Survey indicates that pharmaceutical companies are having a hard time finding the right people. A recent report by PwC’s Health Research Institute (HRI) takes a look at how changes in R&D models have affected the pharmaceutical workforce.