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US and Global Regulatory Affairs (FDA (the beginnings of the FDA (1902--…

- - - - Intellectual property
        
        unless the science and or technology is captured and protected by intellectual property laws there is no viable business proposition: No intellectual property means no business
        
        examples: a set of US patents, own the data, own the software/algorithms
      - venture capital
        
        following (or fueling)scientific and or technological breakthroughs, it will be many years before venture capital sources are necessary over many years to fund the proposition
        
        examples: angel funding, formal venture capital funding
      - bioenterprise finance
        
        through the many years of development, many times with multiple candidates at multiple phases in their development, with multiple funding sources and sometimes revenues, the operational finances of the organization must me properly managed
        
        examples: IPO (initial public offering)--publicly selling stock; deals with major Pharmaceutical companies
      - bioenterprise law
        
        laws with pertain specifically to the bioenterprise, both in the legality of its research and its ultimate products, as well as adjusting business strategy given legislation and case law. Law are different for each country. Supreme court decisions can change the viability of business propositions
        
        Example: Supreme court decision--2013--Association for molecular pathology v Myrid genetics, inc
      - strategic market insights
        
        a business must understand the potential market, the prospective competition, the changing landscape of science and technology, changing societal norms, et al. to make a defensible argument that when the product is market-ready, the market will be ready
        
        examples: no new drug to replace Lithium for bipolar disorder in 50 years
      - regulatory expertise
        
        whether the extreme case of regulatory involvement in the case of biopharmaceuticals or the simple case of a simple device, which from a strategic standpoint the company may forego FDA or other regulatory agency involvement or approval, regulator expertise is essential through the effort
        
        examples: talks about a drug in phase II; talks about filling an IND (investigational new drug)
      - Bio-strategic Media Relations
        
        As surely as the bioenterprise can benefit from competent positive media coverage, it cannot thrive in the face of unanswered negative and or inaccurate media attention. less well understood is how the bio-enterprise relates to global media, what the bioenterprise can and cannot control, and how bio-enterprise management qualifies specific tactics, activities and postures recommended by media relations professionals, both internal and external to the organization
        
        examples: a publication in Nature; an interview in the New York times
      - Bioethics
        
        what are the ethics of your effort? Is what you are doing socially acceptable? Innately ethical or unethical? Conditionally ethical? And what does that mean for your business proposition and the ultimate success of your business
        
        examples: reports of protesters, failure to disclose pertinent information
      - Bioenterprise information systems
        
        from the mammoth undertaking of collecting and managing clinical trials data (Phase 1, II, and III) to the terabytes required to decode human genetics, to all aspects of any bioenterprise, including, for some, the data itself may be the basic asset of the bioenterprise
        
        examples: decoding genetic profiles as part of the product or service; information systems full of data
      - social policy
        
        societal norms support or obstruct your product, e.g., does the society provide their citizens with free in vitro fertilization? is in vitro fertilization acceptable in the society for religious purposes?
        
        examples: product was approved by medicare, president signs Executive order blocking science/technology
      - multinational expertise
        
        every biobuisness is potentially a global buisness. Who doesn't hvae DNA?
        
        Examples: plans for developing product in other countries; drug previously approved and selling in another country
      - technology
      - Science Sci/tech
        
        the science and or technology breakthrough which enabled the creation of the new product. In addition, the new science and or technology which is required to make the product viable
        
        example: a unique scientific approach or a software algorithm
    - - Journal of Commercial Biotechnology (2016) 22(2) 50-63
        
        Article: The BIEM verification Study: Experienced Venture Capitalists Assess a Biopharmaceuticals Innovation Expertise Model
        
        Abstract: BIEM is used to quickly asses the status of a bioenterprise, analysed 20 biopharmaceutical venture capatalists with 30 years average biotechnology industry experience the data varified the BIEM, minimum experience was 20 years
        
        all BIEM disciplines were confirmed and non new disciplines added
        
        interrelations between disciplines correlated
        
        IP with venture capital
        
        regulatory expertise with science
        
        Technology with information systems
    - - the experienced bio-pharmaceutical venture capitalists' held biotechnology industry positions...
        
        Venture Capitalist
        
        100%
        
        Board Chair
        
        90%
        
        Board Member
        
        100%
        
        CEO
        
        80%
        
        President
        
        65%
        
        University professor
        
        15%
        
        University Researcher
        
        15%
        
        CSO
        
        5%
        
        CFO
        
        5%
        
        VP, Sales
        
        5%
        
        Production Manager
        
        5%
        
        Legal-Internal
        
        5%
        
        Legal-External
        
        5%
        
        VP, R&D
        
        0%
        
        Corporate Researcher
        
        0%
    - - Venture Capitalist
        
        100%
      - Science
        
        85%
      - Ongoing Enterprise Finance
        
        85%
      - Intellectual Property
        
        80%
      - Regulatory Expertise
        
        70%
      - Bioethics
        
        70%
      - Biopharmacetical-Related Law
        
        70%
      - last--Technology
        
        50%
      - second to last: information system and social policy
        
        55%
  - - - while originally created to qualify biobuisness for media interviews BIEM also lets anyone evaluate
        
        the state of a bioenterprise
        
        why is that important
        
        the "science-to-Market-Ready-Product" Life cycle
        
        new biotech products start with a breakthrough in science or technology
        
        to become products they must transition through a set of successive/parallel stages which must be funded, which must meet a number of regulatory hurtles, and which form and prepare product for market
        
        the essential elements of which must be addressed...
        
        products are approved by country and/or by political region, e.g., the EU and/or individual EU countries
        
        individual countries/political regions have individual laws and regulatory policies
        
        1 more item...
        
        some have commonality--eg., Japan and EU
        
        the disciplines which are represented are essential in every form of bio business, but can differ in importance, interrelation and duration depending on the product and the buisness landscape
        
        independent of market sector, all the disciples play a role
        
        most information is known about in bio pharmaceuticals; biomedical device verification study is under way
- - - - only 12% of compounds entering phase 1 succeed and 3.2 billion dollar investment averaging all attempts
        
        only 1 out of 9 drugs which enter phase I ultimately get approved
  - - - pre-clinical engineering development (1-3 years)
        
        class 1
        
        510(K) exempt
        
        reimbursemnet assignmnet
        
        class 2
        
        510(k) approval (1-9 months)
        
        Reimbursement Assignment
        
        class 3
        
        clinical trials (1-3 years)
        
        PMA approval (9-36 months)
        
        Reimbursement assignment
    - - Step 1
        
        classify the device as class 1, class 2, or class 3
        
        class 1
        
        low risk to moderate risk; general controls
        
        class 2
        
        moderate to high risk; general and special controls
        
        class 3
        
        high risk; general controls and premarket approval (PMA)
      - step 2
        
        choose correct premarket submission
        
        501(k)
        
        New device is substantially equivalent to a predicate
        
        PMA
        
        valid scientific evidence assuring safety and effectiveness required
        
        De Novo
        
        classify new device without predicate as class I or II
        
        HDE (Humanitarian Device Exemption)
        
        class III device for patients with rare diseases or conditions
      - step 3
        
        prepare appropriate information
        
        design controls
        
        in accordance with quality system regulations
        
        nonclinical testing
        
        must comply with good laboratory practices
        
        clinical evidence
        
        may need to obtain approval of an investigation device exemption (IDE)
        
        labeling
        
        must be written according to labeling regulations
      - step 4
        
        send premarket submission and meet with FDA
        
        include user fee associated with 510 (k) or PMA submission and complete establishment registration and device listing
      - Device approved
        
        post market requiremnts include tracking systems, report malfunctions, serious injuries or deaths, register establishments where devices are produced or distributed
    - - 15 years --founded 2003
      - 400 plus million investment and technology sill does not work
      - FDA had prohibited founder from participating in medical device industry for 2 years
      - Walgreens and other major partners have withdrawn
      - founder and investor currently under indictment for criminal fraud
- - - - FDA mission
        
        the FDA is responsible for protecting public health by ensuring the safety, efficacy, and security of human and veterinary drugs, biological products, and medical devices; and by ensuring the safety of our nations food supply, cosmetics, and products that emit radiation
    - - near NIH (national institutes of health)
    - - protect humans (and our pets) from harm
        
        withing the US and US territires
      - rapidly approve beneficial products
        
        while guaranteeing that approved products are safe
        
        ...and get it right the first time--no mistakes
  - - - information did exist
    - - quinine and US soldiers in Mexico
    - - results of antitoxin tetanus injections in St Louis--10 children dies
      - created the US public health service
      - upton sinclair publishes "The jungle" about the meat-packing industry
    - - "false and fraudulent labeling" prohibited false and therapeutic claims
    - - 107 people died, mostly children
    - - ...which meant they had to check 3,400 approved drugs; DESI( drug efficacy study implementation) of 1966
      - also, 1972 they had to check 300,000 over the counter drugs (OTC)...this phase of DESI was completed in 1992
- - - - both regulatory authorities and pharmaceutical industry
      - goals
  - - - biopharmaceuticals is 160 billion which is the largest BIoMarket Sector
        
        but piopharma is part of the giant global Pharmaceuticals industry
        
        in pharma (700 billion in global revenues), biophrma makes 175 or 160 billion, less than a quarter
    - - innovation matters
        
        innovation driving the future of pharmaceutical industry is primarily in biopharmaceuticals
        
        the biopharmaceutical pipeline (innovative therapies in clinical development)
        
        74 percent medicines in clinical development are potentially first inn class medicines, meaning they represent new pharmacological class for treating a medical condition.
        
        mostly in cancer and Alzheimer research
        
        mostly in phase 2 or phase 1
    - - global biotech venture capital investment, the influx of venture capital was continued bright point, approaching 16 billion
      - fewer inital public offerings/ floats
      - stock market performance
        
        S&P 500
        
        standard and poor commonly listed in the NYSE
        
        NASDAQ biotech index
        
        Dow Jones
        
        industrial
        
        NASDQ
      - FDA approvals headed to the market
        
        biosimilars