The Business of Orphan Drugs is Booming, by Pharmaceutical Commerce

This article provides a comprehensive overview of the current state of the orphan drug market, which was launched with the enactment of the Orphan Drug Act (ODA) in 1983. Growing rapidly, the rare disease business has seen 49 drugs pass the FDA approval process in 2014 alone. Despite this growth, there is much more to do as there are currently 7,000 identified diseases and 95% of them still do not have an approved treatment. With the focus and growth of this market come challenges, including high costs, patient adherence and increased reimbursement scrutiny. There is an emerging dialog around whether payer contracts will become part of this market as some orphan drugs have become billion dollar blockbusters.  As the industry expands to fulfill the need and address the challenges, there are best practices emerging as biopharmaceutical companies look at their 30-year history under the ODA.  From early engagement of the FDA and payers to building connections with patients to supporting products in market with value-added services, biopharmaceutical companies are building a blueprint for how to find success in this growing, complicated orphan drug market.

The Road to Digital Success in Pharma, by McKinsey

Digital technology is rapidly changing the face of healthcare. Mobile, cloud, advanced analytics and the Internet of Things are transforming healthcare much in the way other industries have been transformed already. As pharmaceutical companies establish their digital technology strategies, they will need to determine how to prioritize these technologies to achieve the greatest value.

The article identifies four areas in which digital will drive the most value for pharma:

  • Personalized care: it is expected that in the near future many drugs will be part of a digital ecosystem that constantly monitors a patient’s condition and provides feedback to the patient and other stakeholders. Advanced data analytics mining EMRs and other data sources will inform optimal personalized treatments.
  • Better engagement by omnichannel conversations with physicians and patients using mobile phones, the Internet, apps, or social media. Anytime-anywhere virtual care will become a normality.
  • Data-driven insight: advanced analytics to increase pipeline and commercial value. Companies have huge opportunity to drive the value of their data, optimize the R&D process and to set their market and sales team for success. Pharma organizations which master the art of analysis of different sources of data can generate real-world evidence about the drug’s efficacy and streamline reimbursement and clinical practices.
  • Real-time responsiveness: Cloud and mobile technology and next-gen BI prove real-time transparency on progress, costs and business value.

Companies will need to determine how best to capture the value created through these digital technology opportunities.

Future Health: Transformation Through Innovation, by Deloitte Health

The eBook by Deloitte Health looks into different strategies that health care organizations can implement to respond to the transformation of healthcare and the shift from volume to value, among which:

  • Embracing technology to deliver better care, such as text messaging, telehealth, virtual health, etc. Mobile is key for enhancing patient engagement, lower costs, improve services and strengthen consumers’ loyalty
  • Analytics: the use of the large amount of data which the industry has collected will empower the healthcare’s transformation.
  • The shift to value-based care: to succeed in the new environment, organizations should leverage data to evaluate performance and innovate, improve care coordination, learn how to better meet the needs of the consumers and implement best practices.
  • The role of the CIOs in driving strategy, innovation and revenue growth
  • M&A: the ongoing changes trigger consolidation.

2015 Pharma and Biotech Half-Year Review, by Evaluate Pharma

The report by Evaluate Pharma takes the pulse of pharma and biotech evaluating major trends in the first half of 2015. The conclusion is that the sector is in a very good health. Some of the positive signs are:

  • The fast rate of approval of new treatments is sustained since 2014, aiming to replace products lost to patent expiry. The outlook is that 2015 will equal the 2014 results and possibly 50 new drugs will be approved by end of 2015.
  • All biopharma shares went up, where Eli Lilly is the winner with 21% rise.
  • VC funding: there are chances that the 2015 VC funding can beat the unprecedented $6.5bn raised in 2014.
  • The merger mania continues and much of it has been conducted by specialty pharma groups.

The report’s says that the outlook for the second half of 2015 is quite positive, should the trend set in the first half continue. One of the greatest challenges of biopharma will be to provide evidence to payers about the value to the patient of their high priced treatments.