Bringing Innovation to Neglected Disease R&D

The mission of the nonprofit organization BIO Ventures for Global Health (BVGH) is to save lives by accelerating the development of novel drugs, vaccines, and diagnostics coming from the biopharmaceutical industry that address the unmet medical needs of the developing world. In March 2012, BVGH, in conjunction with the Biotechnology Industry Organizaton (BIO) released a report, Developing New Drugs and Vaccines for Neglected Diseases of the Poor: The Product Developer Landscape, which included information about of the industry's participation in R&D for neglected diseases at the global level (1). The report showed that small- to medium-sized biotechnology companies are participating in a high percentage (41%) of projects targeting neglected diseases of the developing world. The findings of this report were surprising, given the financial risks these companies take on and the little to no promise of a return on their R&D investment.

(BIOVENTURES FOR GLOBAL HEALTH)

Intrigued by the level of participation already among industry and stakeholders, BVGH joined forces with BIO to learn more about the biotech sector and its contributions to neglected-disease product development across drugs, vaccines, and diagnostics. Using updated product development data from the BVGH Global Health Primer, BVGH and BIO have authored a new report, Biotechnology: Bringing Innovation to Neglected Disease Research and Development, which not only reinforces the findings of the March Product Developer Landscape, but also sheds light on the mechanisms driving biotech participation in global health and makes recommendations for fully leveraging the innovation emerging from the biotech sector (2).

The following sections highlight the report's key findings and are based on the full BVGH and BIO report snapshot.

CURRENT INDUSTRY ENGAGEMENT

The following points summarize the work the biotech industry is currently doing in neglected disease R&D:

Partnering is an important driver. Sixty-four percent of all products in development by biotechnology companies across the pipeline of drugs, vaccines, and diagnostics for neglected diseases involve partnering.

Product development partnerships (PDPs) and government agencies are driving biotechnology company partnering for neglected diseases. PDPs are a unique public–private partnering mechanism to increase biopharmaceutical participation in neglected disease R&D. Biotechnology companies partner with academia, PDPs, government agencies, and other industry, to advance neglected disease R&D projects. PDPs are the second most frequent partner, after academia—PDPs are involved in 52% of partnered projects. When a PDP is not involved in a project, biotechnology-government partnerships increase, especially for vaccines and diagnostics.

For tuberculosis and dengue fever, biotechnology companies are often working independently. Across all neglected diseases, biotechnology companies work alone 36% of the time. For tuberculosis, 49% of projects are conducted alone and for dengue, 53% of projects are completed alone. Companies more frequently overcome barriers and leverage some market potential to work alone. For the other neglected diseases that have little or no market, such as leishmaniasis, African sleeping sickness, and Chagas disease, biotechnology companies can take advantage of strategic partnerships to increase their participation. Working alone is not encouraged per se, but this observation is aligned with the idea that the market barrier is significant for many of the neglected diseases that suffer from an insufficient market.

As noted in the report, products for tuberculosis and dengue fever may have some return on investment, especially in the private sector of emerging market countries based on demand and market analyses, which may allow the company to pursue the opportunity without partners.

PROPOSED FUTURE COMMITMENT AND INVESTMENT

The following points summarize the proposals of BVGH and BIO going forward in terms of continued work among the industry and stakeholders in neglected disease R&D:

For companies

• Increase R&D participation in partnering mechanisms and seek collaboration opportunities.

• Pursue partnerships as a way to access non-dilutive financing to engage in global health R&D.

• Seek out existing incentives and innovative financing mechanisms that are available to companies working in neglected diseases.

• Expand R&D efforts beyond HIV, malaria, tuberculosis, dengue fever, and leishmaniasis to other neglected diseases.

• Invite key global health stakeholders to industry events and meetings to increase the neglected disease dialogue in traditional biotechnology circles.

For stakeholders (academia, governments, nonprofits, foundations)

• Actively target small to medium-sized biotechnology companies through existing industry engagement initiatives to increase engagement with the sector.

• Continue to fund existing part-nering mechanisms, such as PDPs, and increase specific targeting of biotech for participation.

• Bring forth new financial incentive proposals that specifically meet the needs of small to medium-sized biotechnology companies and that have the endorsement and buy-in of the biotechnology sector.

• Continue support of existing incentives and collaboration mechanisms, such as the FDA Priority Review Voucher, Orphan Drug Act, and WIPO Re:Search programs, that are compatible with biotechnology company participation.

• Engage the biotechnology sector leadership at the highest level by involving CEOs and top executives.

CONCLUSION

Data sets and figures aside, the most important finding of this report is not necessarily what has been accomplishd to date, but rather what is possible moving forward. To begin to solve the unmet needs of the developing world, both biotechnology companies and global health groups must ramp up commitment and involvement and seek to engage each other through partnerships.The biotech sector's capacity for innovation, not to mention willingness to take financial risks, is incredibly encouraging. But with only 5% of all biotechnology companies involved in neglected disease R&D, imagine what engaging the other 95% could accomplish.

REFERENCES

1. BVGH and BIO, "Developing New Drugs and Vaccines for Neglected Diseases of the Poor: The Product Developer Landscape" (March 2012), www.bvgh.org/LinkClick.aspx?fileticket=h6a0cJK9drg%3d&tabid=39.

2. BVGH and BIO, "Biotechnology: Bringing Innovation to Neglected Disease Research and Development" (June 2012), www.bvgh.org/2012-biotech-report.aspx.