Is 3D Printing The Next Big Thing In Pharma?

Posted by Tom Penrice on Wed,Feb 10, 2016 @ 06:00 PM

A Brief History of 3D Printing in the Medical Industry

Are 3D printed drugs the next big thing for the pharmaceutical industry? 3D printing has been making waves since 1984 when its inventor, Charles Hull, used the technology to make a 3D model from a picture, demonstrating that this technology would be a revolutionary way to test a product design before sinking big money into a large manufacturing program. The power of 3D printing limited innovators only to their imagination and took the world by storm in the intervening years- everything from fashion to firearms was transformed.

The medical industry is just one of the industries revolutionized by 3D printing. In 1999, 3D printing allowed scientist to implant a lab grown organ into patients undergoing urinary bladder augmentation. In the early 2000’s, researchers engineered a functioning kidney, giving scientists the ability to “print” organs and tissues. In 2009, a 3D printer was used to “print” the first blood vessel.

3D-Printing, the FDA, and the Pharma Industry

A few years later, headway is still being made in the medical industry. In early August 2015, the U.S. Food and Drug Administration (FDA) approved the first 3D- printed pill. SPRITAM LEVETIRACETAM, manufactured by the pharmaceutical company Aprecia, is used for reducing seizures in epileptics. The manufacturing of SPRITAM LEVETIRACETAM is done through the 3D printing process of layering the active and inactive ingredients layer by layer. The unique process of 3D printing is beneficial to its patients, as it can make each pill more potent than the traditional tableting machine can produce.

The FDA’s approval of SPRITAM LEVETIRACETAM means that Aprecia is the first major pharmaceutical company to “print” drugs, and they now own the rights to their successful technique. Aprecia’s ZipDoseâ Technology is a combination of science and 3D printing, protected by numerous patents through 2033.

The Future of 3D Printing in the Pharma Industry

No one is certain of how the concept of 3D printing will continue to change the pharmaceutical/ medical industry. But the concept of 3D pill printing brings the process closer to patients in need and may allow for hospitals to adjust doses to individual patients. In addition, the effective layer by layer printing concept in SPRITAM LEVETIRACETAM could be beneficial to other similar drugs where potency and precise dose release are important.

The FDA approval of SPRITAM LEVETIRACETAM is likely to lead to more novel 3D-printed therapies. So what’s next? Pharma companies in the UK are already experimenting with everything from pyramid to donut shaped pills, and many other companies are taking a look at the best way to structure pills for the patients who need them.

The above post is a condensed and edited summary of a Brief History of 3D printing , the Business Insider article: 3-D printing is revolutionizing the pharmaceutical industry and TheGuardians: The first 3D-printed pill opens up a world of downloaded medicine


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They are headquartered in the United States, the United Kingdom and Switzerland.

Tags: FDA, Drug Development, 3D Printing Pharmaceuticals, 3D Printed Drugs

Drug Development Clinical Trial Data – Public or Private?

Posted by Judi Space on Thu,Jan 23, 2014 @ 06:00 PM

Should drug development information from clinical trials be made public or kept private? In the past, data from clinical trials of new drugs and medical devices have been the property of the companies that designed them, and they were allowed to control how and what data was released. The FDA and international equivalents research new products before granting approval, so data the company generated during drug development was up to the company to disseminate as it wished. Now, with the widely held perception that companies have only been releasing results that favored their products, the pendulum is swinging toward regulation making all data on approved drugs and devices available to the public.

The Transparency Campaigners

In Britain, the transparency initiative has been headed by the British Medical Journal, which now requires all authors of drug or device clinical trials who publish in the Journal to provide full scientific study data to anyone with a reasonable request. Also, the Cochrane Collaboration (an international non-profit) began campaigning for increased transparency when it was attempting to verify the efficacy of TamiFlu and was unsatisfied with the available data. 

These major players are joined by doctors, researchers, medical journals, and scientific societies who advocate more transparency in publishing detailed results of clinical trials and as much data about the specifics of the trials as possible. They have been pressing for this data not only going forward, but also for all medicines currently on the market.

The Drug Regulators: EU, EMA, FDA

In December 2013, the EU drafted a clinical trials regulation that will require clinical trials to be registered and results uploaded to a database that can be accessed by the public. This legislation is expected to be formally approved this year. Also, the Public Accounts Committee (of British Parliament), has issued a report this monththat calls for “full methods and results” to be released from all clinical trials, future and past.

Meanwhile, the European Medicines Agency (EMA), which is the European equivalent of the FDA, is considering a new policy on proactive disclosure of trial results, but further discussion of the issue has been tabled until March. Last year, The EMA received injunctions from both AbbVie and InterMune received injunctions against the EMA to prevent it from releasing trial data to competitors, but these injunctions were lifted last month.

In the United States, basic results of all clinical trials must now be registered in a national database that is accessible to the public (www.clinicaltrials.gov). The FDA also publishes staff reviews and other documents when it approves a new drug. However, federal laws restrict what kinds of information can be released, especially data that could reveal personal or commercially confidential information.

Self-regulation by the Pharmaceutical Industry

The European Federation of Pharmaceutical Industries and Associations (EFPIA) and the Pharmaceutical Research and Manufacturers of America (PhRMA) are fighting back by drawing up “principles for responsible data sharing” for pharmaceutical companies to voluntarily abide by. Some companies have already promised to release years of detailed clinical data upon request, in response to public relations issues.

The ABPI (Association of the British Pharmaceutical Industry) has pointed out that a peer-reviewed study it commissioned in 2013 showed a “positive trend” of increasing levels of disclosure – almost nine out of ten of all industry-sponsored trials were disclosed as of January 31, 2013.

Balancing Economic Interests with the Public’s “Right to Know”

PhRMA has argued that proposals for mandates to release all trial information go too far. “If you dump onto the sidewalk all the data, and you include commercially protected information,” he said, “then you’re essentially giving to competitors what we invested billions of dollars in.”

PhARMA claims that if the medical research industry is forced to disclose data that allows competitors to replicate their products, companies will be deterred from developing new drugs and devices that could improve quality of life for many people. On the other hand, patent protection prevents this kind of product replication. At the same time, publication bias can be used by unscrupulous members of the industry to mislead the public.

When the EFPIA/PhRMA pact was announced last year, EFPIA acknowledged that the industry was slow in affecting change. Given the exposure this topic has received recently, efforts to self-regulate may be too late to affect the groundswell of opinion in favor of legislating more transparency in drug development.

This post is based on an article by Ian Schofield – “The rising tide of trial transparency.” 


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They are headquartered in the United States, the United Kingdom and Switzerland.

Tags: FDA, Drug Developers, EMA, Drug Development, Clinical Drug Trials

New Cancer Drugs Fuel Debate over Drug Testing Process

Posted by Judi Space on Thu,Oct 17, 2013 @ 06:00 PM

Question:
If the results of initial testing on a drug are amazing – and could mean the difference between life and death – should pharmaceutical companies be required to go through the current long and costly process of drug testing before making it available to the public? 

Is the FDA's quick drug approval process better, or is NICE's cost/benefit approach the right way to go? Read and decide.If a drug is fine-tuned to treat a specific genetic profile, does it make sense to do randomized controlled trials, even if it means delaying the availability of the drug and possibly increasing the cost, thereby rendering it unattainable to certain patients?  

The Renewed Debate over Drug Testing

Two drugs that were recently approved by the United States Food and Drug Administration (FDA) have helped renew this debate. In 2011, Zelboraf, a drug that targets specific mutations in cancer cells, was approved by the FDA after an early-stage trial showed eight out of ten patients experienced significant tumor shrinkage.  Earlier this year, another drug – Tafinlar, a drug targeting the same mutant genes as Zelboraf - was approved by the FDA based on a single clinical trial of just 250 patients. 

Tafinlar is available at 30 percent less the cost of Zelboraf. 

Are Randomized Controlled Trials Becoming Obsolete?

For many years, randomized controlled trials have been the most effective way of seeing if a drug works.  However, according to Richard Pazdur, M.D., the FDA expert who wanted to shorten the Zelboraf trial, “The types of drugs that we’re seeing now are different.  They are just simply better in terms of efficacy.”    Scientists have a better understanding of molecular changes that promote cancer growth.  For example, an estimated 50-60 percent of melanoma patients - the people Zelboraf and Tafinlar target - have a specific genetic mutation. For drugs that focus on patients with specific genetic mutations, randomized controlled trials may not make sense anymore.

Will Faster Drug Approval Impact Pricing?

Eleven of the twelve cancer drugs launched in the United States last year cost more than $100,000 a year per patient.  In April, more than 100 leukemia specialists from around the world spoke up in the American Society of Hematology’s journal Blood that cancer drug prices were “too high, unsustainable, may compromise access of needy patents to highly effective therapy, and are harmful to the sustainability of our national healthcare systems.”

Accounting for 36 percent of the total research expenditure in 2012, clinical trials are the biggest single cost in drug company R&D.  Many contend that if researchers were permitted to test certain treatments on just people with a specific targeted mutation, researchers could work more quickly, and with fewer patients, to determine if the treatment is effective - bringing these treatments to patients more quickly and, possibly, more cheaply.  Unfortunately, there has not been a significant decrease in medications with the most recent changes in testing procedures.  Paul Workman, head of drug discovery for Britain’s Institute of Cancer Research says, “The costs should be coming down tremendously.  What’s disappointing is that we haven’t seen it happen yet.”

European Drug Regulators Vs. FDA

Part of the issue is that European drug regulators and particularly government funding agencies, such as the National Institute for Health and Clincial Excellence (NICE), may not be willing to accept products based on the FDA’s more flexible clinical trial standards.  In Europe, even if a drug is approved by the European regulatory agencies, if the governments don’t approve funding for it, it will still not be available to many people.

It will be interesting to see what happens.  As Workman said, “We are in a fascinating but frustrating period of transition.”

This post is based on Insight: How new cancer drugs can skip randomized trials by Julie Steenhuysen and Ben Hirschler published in Reuters on September 26, 2013.


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They are headquartered in the United States, the United Kingdom and Switzerland.

Tags: FDA, Cancer Drug Development, Drug Development, NICE, Clinical Drug Trials

Defining Success in Clinical Trials: Is Learning Something Enough?

Posted by Judi Space on Thu,Jul 25, 2013 @ 06:00 PM

Avastin discussed at ASCOAt last month’s ASCO conference, rapt attention was given to a presentation on the clinical trial results for the drug Avastin for patients recently diagnosed with an aggressive form of brain cancer, glioblastoma multiforme (GBM), according to an article by Clinton Leaf appearing in the New York Times, which is summarized below, with our comments added. The presentation by Dr. Mark R. Gilbert, professor of neuro-oncology at the University of Texas M. D. Anderson Cancer Center, revealed earlier clinical trial results that were encouraging:  tumors shrank and the disease appeared to stall for months in two smaller single-arm studies.

No Difference

However, Dr. Gilbert’s larger double blind study of over 600 brain cancer patients found no difference in the survival rate for patients given a placebo with standard treatment vs. those given Avastin with standard treatment.  In effect, the larger, more reliable double blind study eliminated doubt as to the general effectiveness of the drug.   In other words, the trial was “a success” because it had a clear result, but we learned that the drug has no statistically significant impact on the disease as a whole. But is that enough for the trial to truly be deemed a success?

It depends on how you define success. On one hand, doctors have no additional information on how to successfully treat brain cancer as a result of the study. On the other, one possible solution has been eliminated, so doctors and pharmaceutical companies can continue their quest for an effective drug armed with the knowledge from the study.

Not so Fast

The problem is that it is not quite as cut and dried as all that. Some patients who take Avastin do significantly better than those who do not. However, the trial failed to reveal the responders that make this result possible. Despite 400 clinical trials over 16 years, no correlation has been made as to why Avastin works or fails to work in certain patients. Again, given that we don’t know, can we deem the trial a success?  

Individualized Factors May Hold the Key

Researchers are learning that individualized pathology and physiology may be significant factors in developing effective drugs for treating certain diseases.  The variability of tumors and cancers at the genetic level can be great, as can the way our genes affect our response to drugs. So the solution to some diseases may lie in identifying the drugs that best align with the individualized factors associated with a given disease.

Failure rates in clinical trials tend to increase significantly as the drug moves from phase I to phase II. In the final phase it is not unusual for trials to wholly contradict earlier trials, or for the benefits to be far less than what was first reported, according to a 2005 paper published in the Journal of the American Medical Association by Dr. Ioannidis, an authority on statistical analysis.

The Difficulty of Matching Clinical Trial Populations to Patients

Rules governing statistical trial enrollment typically produce trial populations of younger and healthier members than those likely to use the drug. In addition, trial populations typically have had far less medical treatment. These factors combine to form somewhat of a mismatch between the trial population and patients. Given that the vast majority of drugs that enter clinical testing fail to get approved (95%), finding ways to match clinical trial participants with the appropriate genetic or molecular signature may be paramount to finding drugs that work.  

That’s the approach taken by Genentech in developing Herceptin, a breast cancer drug that targets tumor cells with an abundance of the HER2 protein. Small clinical trials were designed that enrolled only those with the required molecular or genetic signature. 60% of the new drugs in development at Genentech/Roche are being developed in concert with a diagnostic test to determine patients most likely to benefit. They did not go that route when they developed Avastin and tested it in patients with GBM.

Back to the Question at Hand

So, is it enough to simply learn something from a clinical trial, even if we don’t find a solution to treat a disease? Given that there is so much riding on finding drugs that improve and extend the lives of those suffering, anything that moves us forward, even if only eliminating what was thought to be a potential solution, is a step in the right direction.


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They are headquartered in the United States, the United Kingdom and Switzerland.

Tags: FDA, Oncology, Targeted Therapy, Cancer Drug Development, Oncology Clinical Trial Design, Breast Cancer, Breast Cancer Drug Development, Breast Cancer Oncology, Glioblastoma, ASCO

Breakthrough Therapy Opens Door to Streamlined Drug Development

Posted by Rebecca Levine on Wed,May 15, 2013 @ 07:51 AM

The FDA's New Breakthrough Therapy Designation

What does the FDA's new Breakthrough Therapy designation mean for pharma and biotech firms? Commentary by Pennside Partners, consultants to the pharmaceutical and biotechnology industries.The FDA recently added a new designation, ‘Breakthrough Therapy’, to its roster of pharmaceutical regulatory terminology in order to ensure that truly innovative compounds get to the patients who need them most as soon as possible.  This new designation was enacted as part of the 2012 FDA Safety and Innovation Act (FDASIA).  The Breakthrough Therapy designation will provide companies with the opportunity for a streamlined developmental process, which could greatly shorten the development timeline. 

How the Breakthrough Therapy Request Process Works

A company can request the designation from the FDA at any time after the original IND submission, but is under no obligation to publicly announce the request.  Once the FDA grants the compound the Breakthrough Therapy designation, the process for approval becomes abbreviated.  The FDA representatives effectively form an advisory panel with the company, in order to determine the most efficient trial designs.  The review time will also be shortened due to the rolling NDA / BLA submission. 

Oncology Likely to Benefit Most

While the language of the bill does not specifically refer to oncology as a primary source of Breakthrough therapies, it is likely that a majority of these designations will apply to oncology compounds.  Other diseases that have already received Breakthrough Therapy status include the rare genetic disease epidermolysis bullosa, hepatitis C and cystic fibrosis. 

Flexible Approval Standards

These new breakthrough therapies must show early clinical evidence of a substantial improvement over existing therapies for serious or life-threatening diseases.  The bill purposefully left the approval standards vague, so that a judgment call can be made by the reviewers at the FDA.  There is no efficacy bar set; however officials warn that if a company wonders if their drug is in a grey area, it is unlikely to be a true breakthrough. 

Rigorous Data Requirements Remain

The data requirements for Breakthrough Therapies will be as rigorous as other submissions, but other faster trial designs may be possible for these compounds.  The options could include a phase Ib expansion or single-arm pivotal trial for diseases states where there is no standard of care.  A phase IIb randomized controlled trial with a modest-sized population might also be an option for some of these compounds where substantial efficacy benefit is seen over the standard of care.  Both these options and others could lead to either full or accelerated approval.

50% Breakthrough Therapy Approval Rate So Far

Over a dozen companies have already sought the Breakthrough Therapy designation, with approvals being granted to approximately half.  One of the compounds recently granted Breakthrough Therapy status is Pfizer’s palbociclib, an oral inhibitor of cyclin-dependent kinases (CDK) 4 and 6.  The designation was granted based on preliminary data from their phase II trial in first-line post-menopausal patients with ER+, HER2- locally advanced or metastatic breast cancer.  Interim data presented at the 2012 San Antonio Breast Cancer Symposium showed that women treated with the combination of palbociclib plus letrozole achieved a statistically significant improvement in median progression free survival (PFS) compared to women who received letrozole alone (26.1 months and 7.5 months, respectively).

Official FDA Guidance Set for July 2013

The official master guidance will be released by the FDA in July of 2013; however it is clear that this new designation will provide a significant benefit to qualifying extraordinary therapies. 

Pharmaceutical Industry Hurdles

What remains unclear at this time is how the already underfunded FDA will manage to provide even more time and attention to these compounds and how much the designation will actually streamline the regulatory pathway. 


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They are headquartered in the United States, the United Kingdom and Switzerland.

Tags: FDA, Oncology, Cancer Drug Development, Drug Development, Breakthrough Therapy, FDASIA

Momentum for Biosimilars is Building. What’s Next?

Posted by Tom Penrice on Tue,Apr 23, 2013 @ 06:00 PM

Overview:
Both the FDA and the EMA are showing increasing levels of support for biosimilars, while physician resistance is declining in both the US and Europe. Cost is a big driver, among other factors.[1]

Biosimilar Drug Development Trends in Europe – Timing is Everything

Biosimilar drug development trends in US and European pharma drug development industryResistance to biosimilars in Europe is declining just as patents are expiring on many highly lucrative biologics, among them Herceptin (trastuzumab), Remicade (infliximab), and Rituxan (rituximab).  Significant cost advantages, important regulatory changes, and a growing openness to new methods of scientific analysis have combined to drive the trend.  Although the financial performance of biosimilars has been disappointing to date, nevertheless the perceived opportunities are attracting innovators and new players.

Why the Resistance to Lower Cost Biosimilars?

Resistance to date has been due primarily to two factors: pushback from physicians reluctant to switch patients to a biosimilar drug, and the lack of a systematic way for pharmacists to easily substitute a biosimilar for an originator drug.  As a result, the returns from biosimilars in Europe have been unimpressive to date. 

Meanwhile, in the US the FDA has yet to receive its first biosimilar application, despite the fact that for two years a regulatory pathway has been provided.  Innovator lobbying at the state level is a major factor contributing to the quagmire, whereby bills are being pushed to make it more difficult for automatic substitution of biosimilars for branded biologics to take place.

Doctors Signing On – Slowly

Unlike the FDA in America which has decision making power for all 50 states for such issues,  the EMA must leave it to each national government to make their own rulings on biosimilars.

In Norway, physicians are slowly growing more positive toward biosimilars partly due to price, a growing factor given sluggish European economies and rising health care costs, according to a cardiologist and medical director at Norway’s regulatory agency.

Economic incentives to prescribe cheaper drugs are also affecting physician acceptance of biosimilars in some northern European countries. For example, in Germany prescription quotas for generic drugs are imposed on physicians as a way to keep within allocated budgets. It is also helpful that Germany has declared copycat EPO’s to be the equivalent of the originals. Although not legally binding, these quotas in Germany have contributed to copycat EPO penetration growing from 30% to 39% in the past three years.[2]

Biosimilars - Impact on Price and Profits

The downward pressure on price due to the growing adoption of biosimilars has resulted in innovators making deep price cuts in order to retain market share, including with hospitals. As a result, payers are enjoying the savings.  Savings in Germany are estimated at over €1 billion per year by 2017.

Changing Definitions and Perceptions

EMA is increasingly accepting biosimilars due in part to recognizing that analytical science gains have made it possible to supply proof of similarity without requiring the time and expense of clinical data.  Last year EMU changed it’s description of biosimilars from products deemed “similar but not identical” to one stating, “the active substance of a biosimilar and the reference medicine is essentially the same biologic substance, though there may be minor differences”, thereby making it more difficult for innovators to make the case for their originals. 

FDA More Open to Biosimilars?

The FDA, while arriving late to the game, may be more open to biosimilars than EMA in terms of an easier acceptance of the analytical science argument for determining similarity. By comparison, the EMA appears to rely more on a clinical trial mindset. The US approach has not been without its rewards, as competition and savings can be significant. According to IMS Health, the US health care system has saved $1.6 billion since launch on Sandoz / Momenta Pharmaceuticals Inc.’s m-enoxaparin, the first of two competing complex copies of Sanofi’s Lovenox (enoxaparin).

Long Term Impact of Biosimilars on the Pharma & Biotech Industry

Now that payers are alert to the cost savings potential of biosimilars, there can be little doubt that the pressure to drive down prices will continue unabated. Indeed, the payers, especially the government payers, have the capability to change the game very quickly.

If prices and profits were to drop sharply and frequently for originals due to growing inroads made by biosimilars, one must wonder to what degree investment in research by innovators may suffer.


[2] http://www.elsevierbi.com/publications/the-pink-sheet/75/6/germany-may-need-to-jump-start-its-stagnant-biosimilars-market 


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They are headquartered in the United States, the United Kingdom and Switzerland.

Tags: Pharma Brand Strategy, FDA, Drug Developers, Biosimilars, Health Care Costs, EMA

NCI Improvements: Drug Development Opportunity for Pharma and Biotech

Posted by Judi Space on Tue,Mar 12, 2013 @ 06:00 PM

Three years ago the Institute of medicine declared that the nation’s cooperative group system for conducting clinical trials was so moribund as to not be an effective vehicle for the generation of scientific data in a timely fashion. In a report generated in 2010, the IOM recommended that the number of cooperative oncology groups be reduced from 10 down to 5[1], improve speed and efficacy of the trial processes as well as incorporate new scientific discovery into clinical protocols. Along with this, the report said it was critical to create a data platform that would assist in the administration of trials across all of the remaining cooperative groups. This would be a system to which both large academic centers and community cancer centers could adhere.

NCI Less of a Barrier; More of a Facilitator

To a large extent, the NCI was  a barrier to the conduct of clinical trials due to their role of providing oversight. The IOM report urged that NCI become part of the solution by helping to prioritize and design trials as well as directing patients to relevant studies so that enrolment could proceed more rapidly. In addition, CMS was tasked with removing barriers to enrollment by supporting appropriate costs associated with patient treatment during the trials.

Efforts Bearing Fruit

Now, nearly three years later, we can see the effects of this report. In a two day workshop[2] that took place in Washington last month, improvements in the national cooperative group trial system were documented[3]. Among the improvements noted:

  • The time between concept and activation of phase II trials sponsored by NCI has shrunk by more than a third. Trials not meeting timelines will be terminated!
  • Time to activation for phase III trials has been halved since the overhaul process began
  • The number of cooperative groups has actually been cut in half – now there are only 5
  • A common enrollment and data management system has been put into place
  • Patients can enroll 24/7 and they may participate in ANY Ph III trial in the system regardless of origin
  • There are now  standardized agreements for industry collaborations
  • A new  review process has been launched for grading the quality of trials
  • Integrated biospecimen banks are being created

Still to come are more innovative Ph II trials based on molecular biology of the tumors and the extensive use of biomarkers and genomic profiling.

Ecology of Clinical Trials

Community Cancer Centers are Key

The key to moving clinical research forward more rapidly will be enhanced participation of community cancer centers. Investigators at these sites are often hesitant to take part as many of the costs associated with the trials are not covered.  After more provisions of the Affordable Care Act go into effect, next January, this may no longer be a barrier. Federal law will require insurers cover the cost of routine patient care in clinical trials.

Design and Conduct of Trials Improving in UK As Well 

Interestingly, the US is not the only place that has had a sea change in the way it conducts clinical research for cancer. Over the same period, the UK’s National Health Service has recognized that clinical research is an important part of its mission, and therefore has provided incentives for patients to participate in trials. The key here is that cancer patients do not get access to novel agents unless they participate in the trials. The result is a trials network that rivals anything in the US but existing in a much smaller country.

 

Implications for Drug Development:

Drug approval process improving in the UK as well as the US

Pharma  and biotech companies can harness the power of the NCI to get things done faster than they have become accustomed to in the past, and the improvements will continue into the foreseeable future. This is also good news for cancer patients who will be able to access more cutting edge clinical trials no matter what part of the country they are from.

 


[1] http://www.iom.edu/~/media/Files/Report%20Files/2010/A-National-Cancer-Clinical-Trials-System-for-the-21st-Century-Reinvigorating-the-NCI-Cooperative/NCI%20Cancer%20Clinical%20Trials%202010%20%20Report%20Brief.pdf

[2] http://www.iom.edu/~/media/Files/Activity%20Files/Disease/NCPF/2013-FEB-11/Agenda%20-%20IOM%20ASCO%20Implementation%20Workshop.pdf

[3] http://www.nap.edu/catalog.php?record_id=18273


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They are headquartered in the United States, the United Kingdom and Switzerland.

Tags: FDA, Drug Developers, Oncology, Predictive Biomarkers, Targeted Therapy, Data Standardization, NCI, Cancer Drug Development, Oncology Clinical Trial Design

FDA-Mixed Response to Proposed Faster Drug Development Pathway

Posted by Tom Penrice on Thu,Feb 07, 2013 @ 04:00 PM

Does the US Need Another Expedited Drug Development Pathway?

The FDA’s meeting to discuss a proposed new pathway aimed at expediting the development of “medicines intended to treat serious or life-threatening conditions with unmet medical needs”, met with a mixed response on February 4th.  The proposal was prompted by a report last fall from the US President’s Council of Advisors on Science and Technology(PCAST), which said a “special medical use”(SMU) pathway could improve drug evaluation in the US.

FDA receives mixed results on proposal for faster drug development pathwayThe Public Weighs In

Participants weighing in on the issue included representatives from Cubist Pharmaceuticals, Trius Therapeutics, Biotechnology Industry Organization, The National Research Center for Women & Families, and Antibiotics Working Group (AWG).

Is There a Need for an Alternate Drug Development Pathway?

Creating an alternative approval pathway could "greatly enhance" the prospects for successful clinical development of novel antimicrobials against the ESKAPE pathogens and would complement the GAIN Act's incentives (Generating Antibiotic Incentives Now (GAIN) Act).

However, there are already six other expedited drug development pathways in place and 50% of the medicines approved in 2009 and 2011 were through a priority pathway. Additionally, various other proposals from the anti-infective development community and the Infectious Diseases Society of America (IDSA) are already on the table, with an assortment of names and acronyms, such as "special population limited medical use" (SPLMU) and "limited population antibacterial drugs" (LPAD).

Some Concerns Brought to Light

There were a few concerns expressed during the discussion, including:

  • Could another expedited drug development pathway create potential congressional intervention resulting in added demands on the FDA?
  • If the FDA pursues its new alternative pathway, could the expedited approval pathways in the Food and Drug Administration Safety and Innovation Act, such as the breakthrough therapies designation, get short shrift?

Suggestions to Enhance Potential Advantages:

Suggestions made for enhancing the potential advantages of the proposal included:

  • Adopt a tiered regulatory framework for the new alternative regulatory pathway, which would allow either disease- or pathogen-based label indications, along with labeling that encourages the most appropriate use of the new medicines commensurate with the approved indications.
     
  • Expand the new initiative to include later stage products, and include indications for which there currently is no guidance, such as prosthetic bone and joint infections, osteomyelitis and bacteremia.
     
  • For antibacterials, the FDA's definition of unmet medical need should not only focus on current needs, but also future needs.
     
  • Balance the dual priorities of expediting clinical development through smaller and more targeted studies and use of authorities that promote responsible prescribing for specific sub-populations through appropriate labeling and restrictions of use.
     
  • Provide clarity about mechanisms or processes to expedite the clinical development of limited use products if it is to include postmarket restrictions of use.
     
  • Make any SMU designation available early in drug development so that manufacturer can design appropriate clinical studies for use under the pathway, for example by conducting trials based on only the most severe manifestation of the disease without having to progress through more moderate disease populations first.
     

You Decide

Is there a need for yet another expedited drug development pathway? As mentioned in our prior post, a streamlined clinical trial process for drug development could have significant strategic planning and competitive intelligence implications for pharmaceutical and biotech firms. Please share your thoughts.


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They are headquartered in the United States, the United Kingdom and Switzerland.

Tags: FDA, Drug Development, Competitive Intelligence

FDA to Consider Faster Drug Development via Smaller Clinical Trials

Posted by Tom Penrice on Fri,Feb 01, 2013 @ 12:00 PM

FDA Hearing on Faster Clinical Trial Process

FDA considers faster drug development process. Implications for Pharma and Biotech Drug Development and Competitive Intelligence.In January the FDA announced it will be holding a public hearing on February 4 and 5, 2013 to gather input on what may lead to a faster clinical trial process for drug and biological products development for "serious or life-threatening conditions that would address and unmet medical need". If approved, an expedited drug development process will be permitted for smaller subpopulations of patients with "more serious manifestations" of a condition. Such a process will lead to faster clinical trials vs. typical studies involving a broader group of patients with a wide range of clinical manifestations.

New Labeling Required

Labeling for approved drugs under the proposed process must clearly show that the drug is "narrowly indicated for use in limited, well-defined subpopulations in which the drug's benefits have been shown to outweigh its risks." Labeling requirements may include an appropriate logo to appear on the container.

Obtaining Public Comments

The purpose of the public hearing is to obtain information and comments on the "need for and feasibility of this pathway and its potential advantages and disadvantages".

Other Highlights

The FDA recognizes that the resource-intensive programs required to approve drugs treating a "broad condition with a wide range of clinical manifestations" requires large study populations which can prevent a drugs from becoming available on a timely basis to patients with "important and unmet medical needs".

It is important to explore the benefits and feasibility of a drug development process for smaller sub-populations of patients with serious or life-threatening conditions, including infections caused by antibiotic-resistant bacteria.

The approval of a narrow indication could be broadened if future data demonstrate the effectiveness and safety in treating a "broader condition or patient population".

For further information, go to http://www.gpo.gov/fdsys/pkg/FR-2013-01-15/pdf/2013-00607.pdf

Impact on the Drug Development Industry

If approved, a streamlined clinical trial process for the development of drugs and biological products may have significant implications for pharmaceutical and biotech firms. Strategic planning and competitive intelligence will take on an added dimension. Forward-thinking strategists would do well to thoroughly consider the implications and the potential to gain a competitive advantage should approval be granted for an expedited drug development process.


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They are headquartered in the United States, the United Kingdom and Switzerland.

Tags: FDA, Drug Development, Competitive Intelligence

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