Biotech companies are starting to accumulate Big Data, consisting of gene expression profiles, biomarker reliability, patient outcomes and numerous other measurable aspects of drug development. The hope is that the mass amount of data can help streamline R&D. During the JP Morgan Healthcare Conference, FierceBiotech Executive Editor Ryan McBride asked a panel of drug development executives about the potential applications and implementation of Big Data in drug development. The bioteh executives discussed the pros and cons of using Big Data to generate hypotheses, and how Big Data can be used to target the right patients for the right drug trial. Another issue discussed was the unstructured, handwritten notes taken by physicians and the challenge of merging and indexing this data with the newly collected high tech data. Nevertheless, the executives agreed at the conference that Big Data is a great source of information for R&D, provided one knows how to use it, and it is just a matter of time before R&D companies do what is necessary to merge all the data into one integrated system. To read the full article on FierceBiotech, click here.
The MWDG of the ASQ Biomedical Division is next meeting to discuss the topic of biocompatibility in regards to medical devices.
When: November 29, 2012 from 5:30-8:00pm
Where: University of Indianapolis, Schwitzer Student Center Building #7, Room 013, Indianapolis IN 46227
Cost: Buffet dinner, $12.50 for members and $15.00 for nonmembers
Click here to register for the upcoming event. This link will be available until 5pm on Monday, November 26th. If you are unable to use the link, contact Ann Stankiewicz at firstname.lastname@example.org
MWDG devision of ASQ Biomedical presents “Human Factors in Medical Device Development” in Indianapolis
The Midwest Discussion Group (MWDG) of the ASQ Biomedical Division presents Human Factors in Medical Device Development on Thursday, October 25, 2012. Jim Kershner from Eli Lilly will share his experience dealing with issues around human factors in medical device development. Kershner will share his extensive knowledge and hands-on experience on human factors investigations. The meeting will be held at the University of Indianapolis. Hot buffet, beverage and dessert will be served at this event.
When: Thursday, October 25, 2012
Where: University of Indianapolis, Schwitzer Student Center Building #7 Room #013 (on lower level)
Time: 5:30p.m. – 8:00p.m.
Cost: $12.50 for members, $15.00 for non-members
For full details of this event, click here.
The Midwest chapter of Biomedical ASQ presents the Process Validation Game!
When: Thursday, May 24th, 2012 from 5:30-8 pm.
Where: University of Indianapolis in the Schwitzer Student Center Building #7 Room #013 Indianapolis, IN 46227
Cost: Buffet dinner $12.50 for members and $15.00 for non-members.
For additional information and to register for this event, click here.
FDA recently issued new final guidance for medical device manufacturers navigating the premarket approval (PMA) and de novo decision pathways. The document, entitled Factors to Consider When Making Benefit-Risk Determinations in Medical Device Premarket Approval and De Novo Classifications was issued on March 28, 2012 and describes in detail how FDA will weigh the risks and benefits of a device during premarket review—both in the future and for devices currently under review.
Featured in the guidance is a worksheet designed to streamline the risk-benefit analysis of submissions by providing in advance several key features of the device’s potential to do both harm and good, including: the duration, type, and magnitude of risks and benefits; as well as risk likelihood, patient tolerance, reported value to patients, and available alternatives. According to the guidance introduction, “FDA believes that the uniform application of the factors listed in this guidance document will improve the predictability, consistency, and transparency of the premarket review process.”
For more information about the premarket process or for help with premarket device strategy, planning, and submissions, contact Pearl.
Dr. Bruce Booth, Partner at Atlas Ventures, recently published an article on Forbes outlining the various models (or “worldviews”, as he calls them) of startup and early-stage biotechs, and how those models are perceived and evaluated for investments among the Venture Capital community.
Booth takes us from the more traditional, brick-and-mortar, product-generating platform companies (see Amgen); to the current trend of virtual, single-asset entities built for acquisition (see FerroKin). He outlines 3 critical success factors for each model; though different, the success factors all revolve around a theme of recruiting experienced core management and making effective use of resources and environment (in-house vs. outsourced development, early partnerships with big pharma, etc…).
The article is a great read for Pearl’s biotech clients—or anyone else developing and revising corporate strategy and fundraising campaigns in the biotechnology space. To read the article, click here. Or contact Pearl for more information.
It’s a brave new world when it comes to financing startup and early stage biotechs, and particularly for pharmaceutical development groups. No longer are there hundreds of pharma-focused VC firms able to independently fuel and sustain the growth of drug development programs. These development paths are long-term and extremely risky investments that more often than not fail to generate sufficient returns to back startups.
Plus, it’s been tough for VCs all around since the economic crisis. According to the National Venture Capital Association, the number of VC firms in the last decade has dropped from 1,022 down to just 462; and the biotech sector—with its extended timelines and lower probability of success—was arguably hit hardest by the shrinkage.
But with all that said, new models are emerging that give small and startup drug developers a chance to secure early capital and survive, and more importantly to share more effectively with investors and partners in both the risks and the rewards of the drug development process. And don’t worry—VCs haven’t dropped out of the race (total VC financing is up 34% from Q1 last year, according to BioWorld). Because after all, there are still enormous rewards to be earned; and often the focused effort of a small biotech is the most effective way to reach them.
A FierceBiotech report from VC heavy-hitter Burrill & Co. tracked $2.6B worth of the most recent biotech investing, which revealed several interesting players, partnerships, and trends. What stands out is the dramatically enhanced collaboration, co-investment, and partnership among various players as compared to years past. VCs, for example, are playing more nicely than ever before with governments, big pharma, academia, hospitals, trusts and foundations—and the result is a landscape of shared responsibility that enables biotechs to continue doing business.
Of course, this model won’t allow for the development of the next Amgen—with R&D, manufacturing, packaging, labeling, marketing, and just about everything else under one roof supported by bricks and mortar. But partnerships with major research foundations, governments, and big pharma—and the allocation of resources, responsibilities, and revenue that go along with them—may very well be the way of the future start-up and early-stage biotechs.
For more information, check out an Xconomy article by Luke Timmerman which does a good job identifying and explaining some of the newest trends and models in biotech financing. For regulatory, clinical, or product development support with your biotech, contact Pearl.
An article published this month by Susan J. Schniepp on BioPharm International illustrates the difficulty for contract manufacturing organizations (CMOs) in factoring in the quality priorities and needs of multiple clients into their own planning and operations. The article attempts to identify a formula that could help define the quality relationship more clearly between CMOs and their clients, namely how to balance the quality commitments and regulatory obligations of both parties involved.
Schniepp identifies four basic variables (and no constants) that contribute to the landscape of the CMO quality relationship:
1) CMO’s needs,
2) compliance needs,
3) client’s needs, and
4) regulatory commitments.
Each of these four variables must be weighed and considered when drafting a quality agreement, and each must be clearly identified and thoroughly explained within the document. According to Schniepp, “The Quality Agreement should be a living document that is reviewed and revised as often as needed to clarify the responsibilities of the client and the CMO as the product progresses through its lifecycle.” In order to sustain a successful relationship, both the CMO and its clients must be very clear on the elements required for quality and compliance, and where those responsibilities fall in each case. To read the full BioPharm article, click here.
At Pearl, we partner with our clients to assist them in building and managing the strongest quality compliance processes, including clearly defined vendor management systems and oversight, GMP audits, and more. For a listing of our QA services, click here. Or contact us at email@example.com to discuss your 3rd party supplier selection and management needs today.
Last week, the Supreme Court issued a unanimous decision on Mayo Collaborative Services v. Prometheus Laboratories, Inc., in which Mayo had challenged Prometheus’ patent claims covering diagnostic biomarker methods for personalized medicine, specifically as a companion diagnostic for autoimmune disease treatment with thiopurine drugs. In a reversal of the Federal Circuit’s decision, the Court invalidated Prometheus’ patent claims on the grounds of “effectively claim[ing] underlying laws of nature”, stating that “laws of nature, natural phenomena, and abstract ideas are not patentable subject matter.”
The decision provoked strong reactions both supporting and opposing the ruling, represented by two starkly contrasting statements submitted to Patent Docs:
The American Medical Association (AMA) fervently supported the invalidation of Prometheus’ patent claims. Dr. Robert Wah, AMA Board Chair, issued a statement maintaining that the ruling “prevented irreparable harm to patient care”, arguing that upholding “exclusive rights over the body’s natural responses to illness and medical treatment” would stand in the way of physicians’ unbiased collection and analysis of diagnostic data. The AMA has garnered support from several other physician organizations and medical associations.
The Biotechnology Industry Organization (BIO) landed firmly on the other end of the spectrum, staunchly opposed to the Court’s decision. In a short statement, BIO Deputy General Counsel for Intellectual Property Hans Sauer said, “We are surprised and disappointed in the Court’s decision…to strike down these patents for biomarker-based diagnostic methods.” Sauer went on to explain that “it introduces new and confusing concepts into the traditional body of patent law, which…fail to appropriately recognize the importance of personalized medicine, and of the research and investment incentives needed to develop new individualized therapies for untreated diseases.”
BIO’s concern may stem from the fact that FDA requires parallel development testing and regulatory submission for companion diagnostics. What does this ruling mean for diagnostic companies and biopharma alike if they aren’t able to protect intellectual property, secure royalty streams, etc?
With very little guidance accompanying the Supreme Court’s ruling, it is difficult to predict what the long-term impact will be on the field of companion diagnostics in the wake of this patent invalidation. More reactions are expected, and the US Patent and Trademark Office (USPTO) has already distributed a short memorandum to patent examiners to help prepare for the implementation of changes in response to Mayo v. Prometheus. To say the least, the discussion on subject matter eligibility, natural phenomena in patent claims, and companion diagnostics has not yet concluded.
To read the Court’s decision, click here. To read the AMA amici curiae brief, click here. To read the BIO amici curiae brief, click here. For more on reactions to and impact of this ruling, check back with Pearl soon. Send us your comments!
Indianapolis-based pharmaceutical giant Eli Lilly signed a deal last week with the National Center for Advancing Translational Sciences (NCATS), providing for Lilly to screen over 3,800 currently approved and investigational new drugs for new potential therapeutic indications. The medicines, which are part of NCATS’ extensive pharmaceutical collection, will be screened over the next 12-18 months. Results will be posted online and publically accessible at the NCGC Pharmaceutical Collection website.
NCATS, having only been launched in January of this year, is the NIH’s youngest center. With a mission to enhance efficiency in the drug development and approval process, NCATS acting director Thomas Insel said of the partnership with Lilly, “Working together, we can make drug development pipelines more productive. The key is precompetitive collaboration to benefit all partners, ensuring broad access to the results.”
To read the press release from NIH and Lilly, click here.