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

ASCO GI Preview - Heads Up for GI Cancer Drug Development

Posted by Marne Wessner on Tue,Jan 15, 2013 @ 08:00 AM

ASCO GI Cancer Drug Development

Abstract titles are up for ASCO GI, and here’s a list of the abstracts we are looking forward to seeing next week.  (Note that full abstracts won’t be available until January 22, so it is impossible to comment on the significance of these results until then.)

Abraxane positive in phase III for pancreatic cancer

The Abraxane study in pancreatic cancer (abstract 67) is likely to be the major news from this year’s meeting. At ESMO in October, KOLs were indicating that the results might be positive, and indeed, Celgene announced in November that this trial met its primary endpoint of overall survival with a statistically significant improvement over gemcitabine alone. Now we will finally see the data. There have been so many notable failed trials in this disease (axitinib and ganitumab, for example) that it’s exciting to finally see something positive!

New biomarker and safety data for Stivarga (regorafenib)

Bayer’s Stivarga was just approved for third line CRC in September 2012, and presentations at this meeting include biomarker and safety data (abstracts 318 and 467). Hopefully the new data will shed light on which patients are most suitable for Stivarga and how oncologists can deal with toxicities.

Ramucirumab data notably absent

We also note that the phase III gastric cancer trial of ramucirumab (VEGF inhibitor, Lilly), which was announced as positive a few months ago, will not be presented. The next opportunity to present the data at an important venue will be at the main ASCO meeting in June.

New GI cancer drugs on the horizon at last

KOLs have been complaining that there hasn’t been anything new in CRC for a long time, but it looks like the tide is finally turning with two new approvals in 2012, and some promising new agents on the horizon. All of these developmental agents could eventually make an impact in standards of care for CRC.

In HCC, there are a few developmental agents with early-phase data that will be presented. Of note is the Pfizer CDK inhibitor, PD-0332991, in HCC. Last month at SABCS, this agent reported very promising phase II efficacy data in ER+ breast cancer, as well as a mild toxicity profile. Efficacy combined with good tolerability could make it an attractive compound in HCC.

 

Key presentations of interest for drug development at ASCO GI

  1. Final results of a randomized phase III study of weekly nab-paclitaxel [Abraxane] plus gemcitabine versus gemcitabine alone in patients with metastatic adenocarcinoma of the pancreas (Von Hoff, et al, abstract LBA148)
  2. Mutational analysis of biomarker samples from the CORRECT study: Correlating mutation status with clinical response to regorafenib (Jeffers et al, abstract 381)
  3. Time course of regorafenib-associated adverse events in the Ph III CORRECT study (Grothey, et al, abstract 467)
  4. Ph II study of PD-0332991 in advanced HCC (Littman, et al, abstract 321)
  5. Patient-reported outcomes from a phase III multicenter, randomized, double-blind, placebo-controlled trial of gefitinib versus placebo in esophageal cancer progressing after chemotherapy: Cancer Oesophagus Gefitinib  (Dutton, et al, abstract 6)
  6. Ph II study of vismodegib, a hedgehog pathway inhibitor, combined with FOLFOX in patients with advanced GaCa and GEJ: A NY cancer consortium led study (Cohen, et al, abstract 67)
  7. A randomized, double-blind, placebo-controlled trial of trametinib in combination with gemcitabine for untreated metastatic PnCa (Infante, et al, abstract 291)
  8. Ph I/II study of golvantinib in combination with sorafenib in patients with advanced HCC: Ph I results (O’Neil, et al, abstract 294)
  9. Long-term efficacy and pharmacodynamic parameter analysis in pretreated KRAS-mutant mCRC patients treated with RG7160 (GA201), an ADCC-enhanced monoclonal anti-EGFR antibody (Delord, et al, abstract 379)
  10. Second-line therapy of KRAS-mutated mCRC with the MEK inhibitor selumetinib in combination with irinotecan: An AGICC study (Hochster, et al, abstract 380)

 


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: Drug Development, Biotech Consulting, Competitive Intelligence, Gastrointestinal Cancer, GI Drug Development

Welcome to Pharma & Biotech Today

Posted by Tom Penrice on Mon,Jan 07, 2013 @ 04:59 PM

Welcome to Pharma & Biotech Today, the blog for pharmaceutical and biotechnology industry professionals. Our goal is to provide you with thought-provoking articles and opinions to assist you in moving your pharma or biotech firm to the forefront in today's competitive market and ever-changing landscape.

Trusted pharma and biotech consulting industry services for over 20 years - Pennside Partners.20 Years Experience -
A Wealth of Information to Share 

With over 20 years in the pharma and biotech consulting business, we have a wealth of information and experience to share with you, our valued readers and subscribers.

In the weeks and months ahead we plan to devote considerable energy to addressing key biotechnolgoy and pharmaceutical industry topics of interest, including:

    • Prelaunch and launch planning and best practices
    • Launch analogs
    • Business development
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    • ... and many more!

Pharmaceutical and Biotechnology consulting experts - Pennside PartnersWe Want to Hear From You!

Tell us about any topics that interest you in today's fast-paced and ever-changing pharma and biotech fields. We'll look forward to addressing many of them in upcoming articles. We'd love to hear from you soon - just click to contact us any time.

Finally, please bookmark this site and check back often for the latest in pharma and biotech news, information, and thought leadership. 


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, Pharma Consulting, Biotech Consulting, Pharma Launch Planning, Competitive Intelligence, Pipeleine Analysis, Pharma Launch Analogs

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