Lecture 1 2 Fall2024 PDF

By: Menna Attef Ewida, Ph.D. Introduction to Entrepreneurship in the Pharmaceutical Industry  Learning Objectives (ILOs) By the end of this course, students will be able to: Understand the fundamentals of entrepreneurship, particularly within the pharmaceutical industry. Recognize t...

By: Menna Attef Ewida, Ph.D. Introduction to Entrepreneurship in the Pharmaceutical Industry  Learning Objectives (ILOs) By the end of this course, students will be able to: Understand the fundamentals of entrepreneurship, particularly within the pharmaceutical industry. Recognize the unique challenges and opportunities in the pharmaceutical industry and how to use it to develop startup ideas. Identify successful pharmaceutical startups and their strategies. Gain basic knowledge of business planning, marketing, and financial management. Develop leadership, teamwork, and communication skills. Recognize the ethical considerations in pharmaceutical entrepreneurship. Collaborate on the creation of a hypothetical startup in the pharmaceutical field. Week 1& 2 outlines Define entrepreneurship Understand the role of in the context of the innovation and business pharmaceutical industry. in pharmacy. Introduce case studies of Discuss the group successful project. pharmaceutical startups.  What is Entrepreneurship? Entrepreneurship is the process of starting, developing, and managing a new business venture, often with innovation and calculated risk. Emphasize the importance of entrepreneurship in transforming ideas into profitable businesses, creating jobs, and driving innovation. Key Characteristics: Innovation Risk-taking Creativity Perseverance Passion Entrepreneurship in the Pharmaceutical Industry Unique Challenges: Opportunities: High regulatory hurdles Growing global healthcare market Long development cycles Advances in technology Significant financial investment Aging population Intellectual property protection Emerging diseases Why is Entrepreneurship Important in the Pharmaceutical Industry? Innovation: New drug discoveries, technologies, and services. Market Impact: Entrepreneurship helps meet unaddressed health needs. Cost Efficiency: Startups may offer more cost-effective solutions. Global Reach: Expanding healthcare access through innovation.  Research & Development (R&D): The backbone of pharmaceutical innovation. Regulatory Pathways: Understanding Key Drivers of drug approvals and compliance. Pharmaceutical Entrepreneurship Venture Capital: Funding essential for launching and scaling. Market Demand: Addressing unmet medical needs or improving existing solutions.  Characteristics of a Pharmaceutical Entrepreneur VISIONARY RISK-TAKER: PROBLEM- LEADERSHIP: ADAPTABILITY: THINKING: WILLING TO SOLVING: BUILDING AND RESPONDING SEEING EXPLORE FINDING GUIDING A TO INDUSTRY OPPORTUNITIE INNOVATIVE SOLUTIONS STRONG TEAM. CHANGES AND S WHERE BUT FOR UNMET REGULATIONS. OTHERS DON’T. UNCERTAIN MEDICAL VENTURES. NEEDS. Successful Pharmaceutical Startups Moderna Gilead Science Moderna Founded: 2010 by Noubar Afeyan, Robert Langer, and Derrick Rossi. Mission: Revolutionize medicine through mRNA technology, focusing on rapid drug and vaccine development. Early Focus: Developing mRNA therapies for a range of diseases, including oncology, infectious diseases, and cardiovascular diseases. Moderna's Innovation: Uses synthetic mRNA to instruct cells to produce proteins that mimic disease-causing agents (like viruses), prompting the immune system to respond. Key milestones: Pre-COVID: Moderna was working on mRNA vaccines for infectious diseases such as Zika and cytomegalovirus. 2020 – COVID-19 Pandemic: Moderna became one of the first companies to respond to the COVID- 19 crisis by developing an mRNA-based vaccine. The mRNA platform allowed Moderna to design a vaccine candidate within days of receiving the SARS-CoV-2 genome sequence in January 2020. By December 2020, the FDA granted EUA (Emergency Use Authorization (EUA): for Moderna’s COVID-19 vaccine, making it one of the first COVID-19 vaccines approved worldwide.  Moderna Moderna’s Growth and Market Impact IPO and Financial Growth: Moderna went public in 2018, raising significant funds to fuel its R&D. The company’s valuation skyrocketed during the pandemic, crossing $100 billion at its peak in 2021. The company supplied millions of vaccine doses globally, becoming a key player in the fight against COVID-19. Partnerships and Collaborations: Moderna forged partnerships with governments, health organizations (like WHO), and pharmaceutical companies to distribute its vaccines worldwide. Moderna’s Role in the Future of Vaccines and Therapeutics Post-Pandemic Plans: Moderna is leveraging its success to expand into new areas such as personalized cancer vaccines and rare disease therapies. The success of the COVID-19 vaccine solidified mRNA technology as a versatile platform with the potential to disrupt traditional pharmaceutical development across multiple therapeutic areas. Moderna Lessons for Pharmaceutical Entrepreneurs Disruptive Innovation: Moderna’s success highlights the value of being a first mover with disruptive technology in the pharmaceutical industry. Risk Management: Pursuing cutting-edge, high- risk technologies like mRNA can lead to significant rewards when successful. Partnerships and Funding: Securing strong investor backing and strategic partnerships is essential for biotech startups aiming to scale quickly and develop transformative products.  Gilead Sciences Founded: 1987 by Michael Riordan, originally as a small biotechnology company. Early Mission: Focused on developing antiviral drugs to treat infectious diseases. Initial Challenges: long R&D timelines and securing funding in a competitive industry. Pioneering Antiviral Treatments: Gilead's breakthrough came from focusing on antiviral therapies for life-threatening viral infections. HIV Treatment Innovations HIV treatment with drugs like Viread (tenofovir disoproxil fumarate) and Truvada (combination of tenofovir and emtricitabine). Impact: Gilead’s HIV treatments improved patient outcomes by reducing the viral load to undetectable levels, making HIV a manageable chronic condition. Hepatitis C Treatment Revolution: Gilead’s biggest breakthrough came with Sovaldi (sofosbuvir): Launched in 2013, with cure rates of over 90% in hepatitis C patients with fewer side effects and a shorter treatment duration. Harvoni: A follow-up therapy combined sofosbuvir with another antiviral (ledipasvir), offering a single-pill, once-daily cure for many patients with hepatitis C. Gilead dominated the hepatitis C market, helping millions of patients and generating billions in revenue. COVID-19 Response Remdesivir Originally developed as a treatment for Ebola and other viral diseases, remdesivir became one of the first drugs authorized to treat COVID-19. In May 2020, remdesivir received Emergency Use Authorization (EUA) from the FDA for COVID-19 treatment based on its ability to shorten recovery times in hospitalized patients.  Gilead Gilead’s Journey from Acquisitions Financial Reinvestmen a Startup to a Global and Strategic Growth: t into R&D: Pharmaceutical Partnerships: By 2014, Gilead Gilead Leader had generated consistently Gilead expanded $12.4 billion in reinvested Early Struggles: In the early through strategic sales from its profits into R&D years, Gilead faced cash acquisitions, hepatitis C to maintain its flow problems and long including portfolio alone. pipeline of next- R&D timelines typical of Pharmasset in generation 2011, which brought The company’s biotech startups. antiviral success therapies for HIV, the hepatitis C drug hepatitis, and Key Turning Point: The drove Gilead’s sofosbuvir into its other infectious launch of Viread and Truvada market value to pipeline. diseases. in the early 2000s marked a exceed $100 major turning point, allowing This acquisition was billion, the company to establish a turning point, transforming it itself as a leader in antiviral leading to Gilead’s from a niche therapies. dominance in biotech into a hepatitis C global treatment. pharmaceutical giant.  Gilead Lessons for Pharmaceutical Entrepreneurs Focus on Innovation: Gilead’s relentless focus on developing breakthrough therapies for underserved markets, such as HIV and hepatitis, demonstrates the power of innovative solutions in healthcare. Long-Term Vision: Despite early challenges, Gilead’s long-term R&D investment paid off, highlighting the importance of patience and persistence in the pharmaceutical industry. Adaptability: Gilead’s ability to pivot from earlier failures and pursue emerging opportunities, like its acquisition of Pharmasset for hepatitis C treatments, exemplifies strategic adaptability. Partnerships and Acquisitions: Leveraging acquisitions and collaborations to bring new technologies into the company’s pipeline was key to Gilead’s growth and success.  Idea Generation: Identifying a unique business opportunity in pharmacy. Stages of Business Planning: Creating a business model and roadmap. Starting a Funding: Securing investment through venture capital or other means. Pharma Product Development: Moving from idea to tangible product (drug, service, technology). Startup Regulatory Approvals: Navigating the complex process of drug or device approval. Marketing and Launch: Bringing the product to market. Ethical Considerations in Pharmaceutical Entrepreneurship 1. Patient Safety Ensuring patient safety should always be the first consideration when developing new drugs or therapies. All pharmaceutical products must go through extensive preclinical and clinical testing to ensure safety before being approved for public use. Phase I: Test for safety in a small group of healthy volunteers. Phase II & III: Larger trials with patients to evaluate the drug’s efficacy and safety. Entrepreneurs must comply with strict regulatory standards set by agencies like the FDA (U.S.), EMA (EU), and others to ensure that patient safety protocols are followed. After approval, pharmaceutical companies must continue monitoring the safety of their drugs through pharmacovigilance programs, reporting any adverse effects or safety concerns. 2. Drug Efficacy and Safety A drug must not only be safe but also effective at treating the condition it is designed for. This involves demonstrating clear benefits over existing treatments or addressing unmet medical needs. Companies are ethically obligated to publish all clinical data, whether positive or negative, to ensure transparency. Withholding negative results can harm patients and undermine trust in the pharmaceutical industry. Every new drug should undergo a thorough risk-benefit analysis, weighing potential benefits against possible side effects or risks. Drugs should only be approved if the benefits significantly outweigh the risks for the target population. Ensuring that clinical trials follow ethical guidelines such as informed consent, proper patient compensation, and minimizing risks to participants.  Ethical Considerations in Pharmaceutical Entrepreneurship 3. Intellectual Property Rights Intellectual property rights (IPR), especially patents, allow pharmaceutical entrepreneurs to protect their innovations and recoup the massive R&D investments required for drug development. It typically lasts for 20 years from the date of filing, providing exclusive rights to the inventor to produce and sell the drug. There is an ethical dilemma between protecting innovations through patents and ensuring that life-saving medications are accessible and affordable. In cases of national emergencies, governments can issue compulsory licenses, allowing other companies to produce a patented drug without the consent of the patent holder. Ethical entrepreneurs should explore equitable licensing practices and global collaborations to ensure that life-saving drugs are available to those in need worldwide, especially in developing countries. 4. Pricing and Accessibility Drug pricing is one of the most sensitive ethical issues in pharmaceutical entrepreneurship. Companies must balance the need for profitability (to fund future R&D) with ensuring that their products are affordable and accessible to the general public. Adopt tiered pricing models, where drugs are priced according to the income levels of different countries to improve global accessibility. In low-income countries, entrepreneurs should explore generic options, subsidies, or philanthropic initiatives to ensure access to essential medicines. Entrepreneurs must be transparent and compliant with pricing regulations. It’s essential to justify pricing by clearly communicating the high costs of R&D, clinical trials, and regulatory processes, while also considering programs like expanded access or patient- assistance programs for affordability.  Ethical Considerations in Pharmaceutical Entrepreneurship 5. Environmental Impact The pharmaceutical industry can have significant environmental impacts due to the chemical processes used in drug manufacturing, packaging, and waste disposal to ensure sustainability. Entrepreneurs should invest in green chemistry practices to reduce hazardous waste and emissions during drug production. Effluent Management: Proper disposal of drug waste and by-products is crucial to avoid polluting water supplies and damaging ecosystems. Entrepreneurs can adopt biodegradable or recyclable packaging materials to reduce the environmental footprint of their products. Focus on energy-efficient production processes to minimize the carbon footprint of drug manufacturing facilities. Engage in Corporate Social Responsibility CSR initiatives aimed at minimizing the company’s environmental impact and giving back to the community through health- related or environmental programs. Questions for Discussion What motivates you to become an entrepreneur in the pharmaceutical industry? What are the biggest challenges you foresee in starting a pharmaceutical startup? How can you balance innovation with ethical considerations in your entrepreneurial journey? What role can universities and research institutions play in fostering entrepreneurship in the pharmaceutical industry?