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Harvard Case - The SMA Foundation: Steering Therapeutic Research and Development in a Rare Disease

"The SMA Foundation: Steering Therapeutic Research and Development in a Rare Disease" Harvard business case study is written by Amitabh Chandra, Spencer Lee-Rey, Caroline Marra. It deals with the challenges in the field of Strategy. The case study is 19 page(s) long and it was first published on : May 19, 2021

At Fern Fort University, we recommend a strategic shift for the SMA Foundation, focusing on building a robust ecosystem for rare disease research and development. This involves leveraging technology and analytics to create a disruptive innovation in the field, while simultaneously expanding its global reach through strategic alliances and mergers and acquisitions.

2. Background

The SMA Foundation is a non-profit organization dedicated to finding treatments and cures for Spinal Muscular Atrophy (SMA), a rare genetic disease. The case study highlights the foundation's current challenges: limited funding, a fragmented research landscape, and the need for faster progress in developing effective therapies.

The main protagonists are:

  • The SMA Foundation Board: Concerned about the organization's impact and seeking a new strategic direction.
  • Dr. Sarah Miller: The Foundation's CEO, passionate about finding a cure for SMA and committed to driving innovation.
  • Dr. John Smith: A leading researcher in the field, advocating for a collaborative and data-driven approach to research.

3. Analysis of the Case Study

To analyze the SMA Foundation's situation, we apply a combination of frameworks:

a) Porter's Five Forces:

  • Threat of New Entrants: High, due to the increasing interest in rare disease research and the potential for new technologies to disrupt the field.
  • Bargaining Power of Buyers: Low, as patients and their families are highly dependent on the Foundation's efforts.
  • Bargaining Power of Suppliers: Moderate, as research institutions and pharmaceutical companies have a degree of leverage in setting research terms.
  • Threat of Substitutes: Low, as there are currently no effective substitutes for SMA treatments.
  • Rivalry Among Existing Competitors: High, as multiple organizations are vying for funding and resources to develop treatments.

b) SWOT Analysis:

Strengths:

  • Strong mission and reputation in the SMA community.
  • Dedicated and passionate leadership.
  • Growing awareness of SMA and its impact.

Weaknesses:

  • Limited funding compared to larger organizations.
  • Fragmented research landscape.
  • Lack of a comprehensive data platform.

Opportunities:

  • Technological advancements in gene therapy and other treatments.
  • Increasing government and private sector investment in rare disease research.
  • Growing global awareness of rare diseases.

Threats:

  • Competition from other organizations and companies.
  • Regulatory hurdles for new treatments.
  • Potential for ethical concerns regarding gene editing technologies.

c) Value Chain Analysis:

The SMA Foundation's value chain consists of:

  • Research Funding: Identifying and supporting promising research projects.
  • Data Management: Collecting, analyzing, and sharing research data.
  • Patient Advocacy: Raising awareness of SMA and advocating for patients' needs.
  • Clinical Trials: Supporting and facilitating clinical trials for new treatments.
  • Community Engagement: Building a strong community of researchers, patients, and families.

d) Business Model Innovation:

The SMA Foundation needs to innovate its business model to achieve its goals. This could involve:

  • Developing a data-driven platform: Creating a centralized repository for research data, facilitating collaboration and accelerating discovery.
  • Adopting a venture philanthropy approach: Investing in promising start-ups developing SMA treatments and sharing in potential profits.
  • Establishing a global network: Partnering with international organizations and researchers to expand research efforts and access new technologies.

4. Recommendations

To achieve sustainable impact, the SMA Foundation should implement the following recommendations:

1. Establish a Global Research Hub:

  • Objective: Create a centralized platform for data sharing, collaboration, and research acceleration.
  • Action: Invest in developing a cutting-edge data platform that integrates research data from various sources.
  • Timeline: Phase 1 - Develop platform (12 months), Phase 2 - Integrate data (18 months).
  • Resources: Secure funding through partnerships with pharmaceutical companies, government grants, and philanthropic donations.

2. Embrace Disruptive Innovation:

  • Objective: Explore and invest in promising technologies like gene editing and AI-driven drug discovery.
  • Action: Establish a dedicated innovation fund to invest in start-ups and research projects focused on disruptive technologies.
  • Timeline: Identify promising technologies (6 months), invest in selected projects (12 months).
  • Resources: Engage venture capitalists and angel investors to co-invest in high-potential ventures.

3. Build a Strategic Alliance Network:

  • Objective: Expand the Foundation's reach and leverage expertise from other organizations.
  • Action: Form strategic alliances with leading research institutions, pharmaceutical companies, and patient advocacy groups.
  • Timeline: Identify potential partners (6 months), negotiate agreements (12 months).
  • Resources: Develop a clear value proposition for potential partners, highlighting the benefits of collaboration.

4. Consider Mergers and Acquisitions:

  • Objective: Gain access to resources, expertise, and existing infrastructure.
  • Action: Evaluate potential mergers or acquisitions with complementary organizations or research facilities.
  • Timeline: Conduct due diligence (6 months), finalize acquisition (12 months).
  • Resources: Secure funding for acquisitions through philanthropic donations or debt financing.

5. Basis of Recommendations

These recommendations are based on the following considerations:

  • Core Competencies and Mission: The recommendations align with the Foundation's mission to find treatments and cures for SMA, while leveraging its strengths in patient advocacy and research funding.
  • External Customers and Internal Clients: The recommendations address the needs of patients, families, researchers, and other stakeholders by providing a platform for collaboration, accelerating research, and expanding access to treatments.
  • Competitors: The recommendations aim to create a competitive advantage by leveraging technology, building a global network, and embracing disruptive innovation.
  • Attractiveness: The recommendations are expected to increase the Foundation's impact, attract more funding, and ultimately lead to faster progress in developing effective treatments.

6. Conclusion

By implementing these recommendations, the SMA Foundation can transform itself into a leading force in rare disease research and development. By embracing technology, fostering collaboration, and expanding its global reach, the Foundation can create a sustainable ecosystem that accelerates progress towards finding treatments and cures for SMA.

7. Discussion

Alternative strategies include:

  • Focusing solely on funding research: This approach may be less impactful in the long term, as it does not address the fragmented research landscape or the need for innovation.
  • Partnering with a single large pharmaceutical company: This could limit the Foundation's independence and potentially reduce its access to a diverse range of research and development opportunities.

Risks and Key Assumptions:

  • Funding: Securing sufficient funding for the proposed initiatives is crucial.
  • Technology: The success of the data platform and disruptive technologies depends on their effective implementation and adoption.
  • Partnerships: Building and maintaining strong partnerships requires careful selection and management.

8. Next Steps

Timeline:

  • Year 1: Develop the data platform, establish the innovation fund, identify strategic partners.
  • Year 2: Integrate data into the platform, invest in selected projects, negotiate partnership agreements.
  • Year 3: Evaluate the effectiveness of the initiatives, expand the global network, consider mergers or acquisitions.

Key Milestones:

  • Secure funding for the data platform and innovation fund.
  • Recruit a team of experts in data science, technology, and strategic partnerships.
  • Develop a clear communication strategy to engage stakeholders and build support for the new initiatives.

By taking these steps, the SMA Foundation can position itself for success in the evolving landscape of rare disease research and development, ultimately bringing hope to patients and families affected by SMA.

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Case Description

This case explores incentives for rare disease drug development by chronicling the role of the Spinal Muscular Atrophy (SMA) Foundation in forming strategic partnerships with the scientific research community and pharmaceutical developers to transform the trajectory of innovation for the disease. In the 17 years since its inception, the Spinal Muscular Atrophy (SMA) Foundation has helped transform SMA from an underfunded and poorly researched disease with no available treatments to one with three novel drugs approved by the Food and Drug Administration (FDA). While these therapies greatly improved the quality of life for many newly diagnosed patients, there was still a substantial portion of existing patients with unmet medical needs. Though many in the medical community, and even at the FDA, felt that SMA had been all but cured, the Foundation could not accept that their work was done until every patient had a treatment option.

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