This paper is part of the free Open Access archive of the Journal of Commercial Biotechnology

Technology transfer in the biomedical sciences: The Medical Research Council approach

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ABSTRACT: This paper describes why the UK Medical Research Council participates in exploitation (an integral part of its mission), the objectives for exploitation (primarily to serve public food through contributions to the health and wealth of the UK) and how the organisation of exploitation is structured...

The Journal of Commercial Biotechnology is a unique forum for all those involved in biotechnology commercialization to present, share, and explore new ideas, latest thinking and best practices, making it an indispensable guide for those developing projects and careers within this fast moving field.

Each issue publishes peer-reviewed, authoritative, cutting-edge articles written by the leading practitioners and researchers in the field, addressing topics such as:

• Management
• Policy
• Finance
• Law
• Regulation
• Bioethics

For more information, see the Journal of Commercial Biotechnology website

This is a guest post from Robert E. Wanerman, a member of Epstein Becker Green’s Health Care and Life Sciences practice. Do you have a response to Robert’s post? Respond in the comments section below.

For many biotech manufacturers, obtaining Medicare coverage is a significant milestone in commercializing an item or service. Although Medicare coverage for specific items or services that use biotechnology methods for their production, design or delivery can vary in different parts of the United States, a small number of them are guaranteed nationwide coverage if the Centers for Medicare and Medicaid Services (“CMS”) has issued a National Coverage Determination (“NCD”).  Obtaining a NCD is a difficult process for companies in the biotech industry, and depends on factors such as the quantity and quality of clinical data, including outcomes data, and a consensus of professional opinions or practice guidelines.

Since 2006, CMS has carved out a middle ground for covering items and services when it believes that the clinical data is insufficient to justify issuing a NCD, but believes that the item or service shows promise for improving outcomes among Medicare beneficiaries.  In these situations, CMS has the discretion to cover an item or service with conditions under its Coverage with Evidence Development (“CED”) authority.  If CMS agrees that CED coverage is appropriate, the sponsor then has to design a study that CMS will approve.  How CMS makes that second decision whether or not to approve a study design is often poorly understood.  On November 20, 2014, CMS released its latest guidance on CED coverage, which adds some additional clarification for interested biotech parties.

The new CMS guidance builds on its past CED approvals and typically contains several requirements for an approvable CED study:

• Medicare beneficiaries must be enrolled in a clinical trial approved by CMS;
• The study design must answer the questions framed by CMS in its approval;
• The study must comply with all human subject protection regulations;
• The study must be registered on clinicatrials.gov before any enrollment can occur (registry studies must also be listed in the Agency for Healthcare Research & Quality’s (AHRQ) Registry of Patient Registries (RoPR));
• The study protocol must specify the method and timing of public release in either a peer-reviewed journal or in a publicly-available registry of all identified outcomes to be measured, including release of outcomes if outcomes are negative or the study is terminated early, which must be made within 12 months of the study’s primary completion date (even if the trial does not achieve its primary aim);
• The study protocol must explicitly discuss beneficiary subpopulations affected by the item or service under investigation, particularly traditionally underrepresented groups in clinical studies, how the inclusion and exclusion criteria affect enrollment of these populations, and a plan for the retention and reporting of the subpopulations; and
• The study protocol must explicitly discuss how the results are or are not expected to be generalizable to affected beneficiary subpopulations.

What is new about this guidance is CMS’s focus on outcome measurements.  Two new elements stand out: first, it strongly suggests that in order to approve a CED study, there must be a comparator in a control group in order to minimize potential biases and to give CMS a better grounding to evaluate the effectiveness of the new item or service at the end of the study period.  The comparator in the control arm of the study may be a placebo or standard of care treatment.  Although CMS does refer to blinding as a technique to minimize the placebo effect, the Guidance does not address evaluating (1) the risks to Medicare beneficiaries of sham procedures, such as a spine procedure to relieve back pain as discussed in the Guidance, or (2) how to offset the disincentives for a Medicare beneficiary to enroll in a clinical trial if they know that there is a chance of receiving a sham procedure instead of treatment.

The second new element in the Guidance is CMS’s suggestion that the CED study design include interim analyses that would be shared with CMS.  Although this could potentially expedite a final NCD decision if the results are strongly positive, it may increase the burdens on the trial sponsor and could become problematic for the sponsor if the interim data is not promising and CMS in response changes its demands for data in order to make a final determination.

Although the Guidance is something less than a roadmap for biotech manufacturers and other stakeholders seeking Medicare coverage, it does provide a clearer idea of CMS’s expectations when reviewing a request for CED protocol. As in the past, interested parties are encouraged to meet with CMS’s Coverage and Analysis Group and to maintain a dialogue with the agency throughout the process, to exchange ideas and to fine-tune the proposed clinical trial in order to reach a consensus with CMS.

About the author:

Robert E. Wanerman is a member of Epstein Becker Green’s Health Care and Life Sciences practice. His practice concentrates on regulatory, reimbursement, and compliance matters affecting biotech and health care manufacturers, service providers, and investors in biotech and health care organizations. Robert has extensive experience counseling clients in matters arising under the Medicare and Medicaid programs. He can be reached at rwanerman@ebglaw.com.

This is a guest post from Susan K Finston, President of Finston Consulting. Do you have a response to Susan’s post? Respond in the comments section below.

At the start of a bright and shiny new year in 2015, I am thinking about my own New Years resolutions as well as one that I would wish for the BRICS and Emerging Markets specifically: to strive for an enabling and nurturing environment for R&D-intensive Micro-Small and Medium Enterprises (MSMEs).  In 2014 I was privileged to work with the Wadhwani Foundation, a non-governmental organization active in South Asia and East Asia to promote MSME entrepreneurship and innovation.  The following is adapted from an upcoming Wadhwani Foundation study:  The Innovation Blueprint:  Identifying, Adapting and Assimilating Best Practice for R&D Intensive MSMEs:

R&D-Intensive MSMEs drive innovation across sectors including information and communications technologies, social media and applications, life sciences, and many others. In India there has long been recognition of the importance of MSMEs for livelihood creation, where the MSME sector employs an estimated 80 million people, and accounts for nearly half of all industrial output. The challenges facing India’s innovative MSMEs are also common to small-scale companies in Brazil and China. In fact in some areas India leads its BRICS peers. Brazil has lagged India in developing both a Venture Capital (VC) and start-up culture. While outpacing India in the WIPO Global Innovation Index, China has been unable thus far to translate innovative capacities into successful start-up companies for creation of economic and social value. For example while China has a stronger VC sector, it continues to struggle with issues of financing for innovative SMEs. China figures prominently in the Wall Street Journal’s Billion Dollar Startup Club  though, with 10 Chinese startups now valued at over a billion dollars (USD) as compared to 2 Indian companies out of a total of 71 companies (as of January 2015).  Looking at the BRICS as a whole, we also see continuing challenges to provide adequate capital resources needed for R&D-intensive life sciences start-ups.

In OECD-level countries, by contrast, innovative MSMEs play a leading role in generation of innovative products and services through direct commercialization, co-development and/or out-licensing to larger companies.

The World Intellectual Property Organization (WIPO), has described Micro-Small-Medium Enterprises (MSMEs) as a “deep, broad, fertile forest floor that nourishes, sustains and regenerates the global economic ecosystem,” 

Within the OECD, Israel and the United States excel at innovation and job creation through MSMEs, with a strong ‘start-up culture’ that values risk and tolerates failure. The mantra of one well-recognized US technology cluster, Silicon Valley, California, is “Fail fast, fail often.” Israel also places a high premium on individual initiative and a willingness to challenge conventional wisdom, recognizing that the only true failure is not to try something new. In this context, the Weizman institute estimates that up to 24% of leading new biopharmaceuticals include Israeli-developed technologies (see table).  Acceptability of risk increase effective mobilization of capital for MSMEs through angel investors, seed funders and venture capitalists, as well as local and  centralized sources of funding for start-up ventures.

MSMEs are empirically the most productive engines of innovative technologies, products and services, and yet in the BRICS and most emerging markets, government support and funding favors larger, established and less innovative companies. Perhaps the hardest and most important adjustment of all is attitudinal.

For 2015 then, let’s hope that policy-makers will extend MSME support across sectors and regions for inclusive growth and to truly see the innovative potential of vibrant R&D-intensive MSMEs to become the next Facebook, Google, Amgen or Celgene.

About the author:
President of Finston Consulting LLC since 2005, Susan works with innovative biotechnology and other clients ranging from start-up to Fortune-100, providing support for legal, transactional, policy and “doing business” issues. Susan has extensive background and special expertise relating to intellectual property and knowledge-economy issues in advanced developing countries including India and South Asia, Latin America and the Middle East North Africa (MENA) region. She also works with governments, and NGOs on capacity building and related educational programs through BayhDole25. Together with biotechnology pioneer Ananda Chakrabarty, she also is co-founder of Amrita Therapeutics Ltd., an emerging biopharmaceutical company based in India with cancer peptide drugs entering in vivo research. Previous experience includes 11 years in the U.S Foreign Service with overseas tours in London, Tel Aviv, and Manila and at the Department of State in Washington DC. For more information on latest presentations and publications please visit finstonconsulting.com.

Patents require regular ‘maintenance’ payments to keep them in force. Because the cost of maintaining large patent portfolios can be substantial, many companies elect to abandon patents that they feel are not worth pursuing. Sometimes these patents are abandoned because they are outside a company’s focus, and in other cases they may represent markets too small for larger companies to justify targeting.

The upside of abandoned patents is that the inventions they describe enter the public domain prematurely, on abandonment rather than 20-years from filing, and they can allow outside parties to leverage the cutting-edge research from leading labs. Accordingly, some of the patents abandoned by large companies may nonetheless be very valuable for smaller enterprises.

Leveraging data I collect for DrugPatentWatch and PatentStat, I have launched a new site at PatentDrop.com that provides regular updates of patents which expired prematurely due to lack of maintenance payments, in 33 different industry sectors.

These abandoned inventions are in the public domain, and may provide opportunities for open-source projects or small and medium-sized companies to leverage the inventions which larger companies abandoned.

Biotechnology patents can be found in the biotechnology category, or by tracking individual companies, such as Agilent, DuPont, or Wyeth.

This paper is part of the free Open Access archive of the Journal of Commercial Biotechnology

Patenting human genes and mutations: A personal perspective

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ABSTRACT:

The Journal of Commercial Biotechnology is a unique forum for all those involved in biotechnology commercialization to present, share, and explore new ideas, latest thinking and best practices, making it an indispensable guide for those developing projects and careers within this fast moving field.

Each issue publishes peer-reviewed, authoritative, cutting-edge articles written by the leading practitioners and researchers in the field, addressing topics such as:

• Management
• Policy
• Finance
• Law
• Regulation
• Bioethics

For more information, see the Journal of Commercial Biotechnology website

Drug Patent Expirations for December 2014

This paper is part of the free Open Access archive of the Journal of Commercial Biotechnology

Biogenerics 2007: How far have we come?

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ABSTRACT: The recent approval of a follow-on version of Pfizer's Genotropin (recombinant human growth hormone) signalled the beginning of the end of an era in which biopharmaceuticals enjoyed immunity from competition even after expiration of their patent protection. This paper describes many of the key scientific challenges facing the nascent ‘biogenerics’ industry and the evolving regulatory framework that will shape its competition with innovator companies...

The Journal of Commercial Biotechnology is a unique forum for all those involved in biotechnology commercialization to present, share, and explore new ideas, latest thinking and best practices, making it an indispensable guide for those developing projects and careers within this fast moving field.

Each issue publishes peer-reviewed, authoritative, cutting-edge articles written by the leading practitioners and researchers in the field, addressing topics such as:

• Management
• Policy
• Finance
• Law
• Regulation
• Bioethics

For more information, see the Journal of Commercial Biotechnology website

As an offshoot of PatentStat, I’ve assembled a set of graphs of patent inventor locations, focusing in 33 industry sectors.

Here are the maps for biotechnology:

Click on the maps for links to the live maps, or visit PatentStat’s other maps.

This paper is part of the free Open Access archive of the Journal of Commercial Biotechnology

Kinik: Raising the stakes for importing products derived from US patented processes practised abroad

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ABSTRACT: This paper analyses the recent ruling in Kinik Co. v. Int'l Trade Comm'n, 362 F.3d 1359 (Fed. Cir. 2004), and the impact that decision may have on proceedings before the US International Trade Commission under the Tariff Act of 1930 (19 USC §1337(a)) – particularly those concerning the importation of products derived from practising US patented processes abroad.

The Journal of Commercial Biotechnology is a unique forum for all those involved in biotechnology commercialization to present, share, and explore new ideas, latest thinking and best practices, making it an indispensable guide for those developing projects and careers within this fast moving field.

Each issue publishes peer-reviewed, authoritative, cutting-edge articles written by the leading practitioners and researchers in the field, addressing topics such as:

• Management
• Policy
• Finance
• Law
• Regulation
• Bioethics

For more information, see the Journal of Commercial Biotechnology website

This paper is part of the free Open Access archive of the Journal of Commercial Biotechnology

The regulatory system in the EU and further afield

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ABSTRACT: The regulation of modern biotechnology began almost as soon as the potential benefits and risks became clear. In some countries a decision was made to use current law to address the new technologies, arguing that the changes that are able to be introduced into new products are not substantially different from those introduced by other techniques...

The Journal of Commercial Biotechnology is a unique forum for all those involved in biotechnology commercialization to present, share, and explore new ideas, latest thinking and best practices, making it an indispensable guide for those developing projects and careers within this fast moving field.

Each issue publishes peer-reviewed, authoritative, cutting-edge articles written by the leading practitioners and researchers in the field, addressing topics such as:

• Management
• Policy
• Finance
• Law
• Regulation
• Bioethics

For more information, see the Journal of Commercial Biotechnology website