< PreviousRARE DISEASES 20 Pharma Business International www.pbiforum.net F ollowing years of price gouging controversies and continuing cases of corporate greed in big pharma, it’s hard not to see a culture of putting profits over patients. Few could argue that blockbuster drugs are financially advantageous for their patent holders. Indeed, the world’s best-selling drugs have collectively earned tens of billions of dollars. But these are drugs predominantly meant to treat and mitigate the effects of common health conditions, viruses and illnesses and are therefore profitable to produce. However, there is a plethora of rare diseases for which it is not profitable to produce treatments and aren’t often likely to deliver returns on investment. The National Institutes of Health estimates that as many as thirty million people in the US have one of 7,000 rare diseases. Only five per cent have approved treatments. The figures speak for themselves and, more often than not, the cost of developing these drugs is higher when compared with those intended for more widespread conditions. In these circumstances, drug makers require government assistance to, essentially, make it worth their while. These rare Disrupting innovation The outbreak of COVID-19 has seen investment and research shift away from rare diseases, putting the lives of patients at risk. 22 Á 20-23.qxp_Layout 1 09/06/2020 11:09 Page 1Pharma Business International 21 www.pbiforum.net RARE DISEASES © Shutterstock /unoL 20-23.qxp_Layout 1 09/06/2020 11:09 Page 2RARE DISEASES 22 Pharma Business International www.pbiforum.net diseases are thusly designated as ‘orphan diseases’ and treated with ‘orphan drugs’. Although government incentives are required to encourage and facilitate the development, production and marketing of these orphan drugs, the market is rapidly growing. According to Allied Market Research, the global orphan drugs market is expected to bring in $169 billion from $106 billion and register a CAGR of 6.8 per cent during 2016-2022. This increase is due largely to a surge in the prevalence of rare diseases. There’s also been an overall rise in awareness among the populace regarding rare diseases, as well as an increased in R&D investment and drug development. Looking ahead, the market will be also be driven by growth in novel indications for known orphan drugs and untapped emerging economies, providing new market opportunities to orphan drug makers. However, this future growth has been hamstrung by the outbreak of COVID-19. Throughout the continuing pandemic, many labs have either been closed or are operating at low capacity. Given the severity of the situation, medical research any disease other than COVID-19 has been massively impacted. Untold numbers of experiments have been abandoned and clinical trials have been suspended or postponed. Not only will this delay breakthroughs, developments and advances for conditions such as cancer, but will seriously threaten R&D in rare diseases. Experts have cautioned that the industry might never fully recover from this loss. Despite some resumption of work on research outside of coronavirus, medical institutions say it will be difficult to make up from the lost time. For example, some researchers have to rebuild their colonies of specially bred animals. Then, of course, labs and other research facilities have to comply with social distancing measures such as staggering work shifts. “I don’t see how we can maintain the levels of activity we had in the past,” said Craig Jordan, a leukaemia researcher at the University of Colorado Anschutz Medical Campus, quoted by The Washington Post . One of the most insidious aspects of COVID-19 isn’t only the lives it claims directly, but indirectly. But shifting so much of research focus and investment away from rare diseases, patients will likely suffer. At present, the industry is taking steps towards widening the development focus, but it will be months – probably years – before things even approach pre- pandemic levels. It’s never been more important to focus on orphan diseases and the drugs that treat them. 20-23.qxp_Layout 1 09/06/2020 11:09 Page 3Pharma Business International 23 www.pbiforum.net RARE DISEASES © Shutterstock /unoL 20-23.qxp_Layout 1 09/06/2020 11:09 Page 4OUTSOURCING 24 Pharma Business International www.pbiforum.net O utsourcing production used to be a risky prospect, something that was only considered as a last-minute resource in a situation where an influx of demand, or a problem with your own production line had taken place. Nowadays however, with the increased technological development – and as a result the more affordable access to technology – outsourcing pharma production is not as risky a prospect as it once was. In fact, it’s now a booming industry. Mostly handled by CMOs (Contract Manufacturing Organisations), the healthcare CMO market is rapidly expanding. This has led to many major pharmaceutical firms making the choice to utilise CMOs for one hundred per cent of their production capabilities, greatly reducing staff costs and other overheads. When it comes to the CMO industry, major plays are the US and Europe, who have a large number of healthcare CMOs within their territory. Germany, Italy and the UK are particularly active in the EU – with Germany having a clear lead in terms of the sheer number of CMOs that specialise in the pharmaceutical industry. The story is much the same in the rest of the world. In 2012 Passing on production Although outsourcing production carried inherent risk for pharmaceutical companies, it is becoming industry standard for many companies. 27 Á 24-27.qxp_Layout 1 09/06/2020 11:09 Page 1Pharma Business International 25 www.pbiforum.net OUTSOURCING © Shutterstock /Parilov 24-27.qxp_Layout 1 09/06/2020 11:09 Page 226 Pharma Business International www.pbiforum.net © Shutterstock /sergey kolesnikov 24-27.qxp_Layout 1 09/06/2020 11:09 Page 3OUTSOURCING Pharma Business International 27 www.pbiforum.net the global pharma CMO market stood at an estimated $97 billion, while figures suggest that this has now ballooned to an incredible $246 billion. This is expected to be a compound factor of pharma giants making increased use of CMOs. But the figure is also strengthened by the ending of a number of patents which will likely see a flurry of suppliers seeking to provide now un-patented drugs at much lower rates. Of course, there are risks associated with making use of CMOs, with a major one being failure in on-time deliveries, which can cost the original pharma company in terms of both client confidence, and consumer confidence. And, of course, there are also concerns to be raised on the quality of products, and compliance to complex hygiene regulations. In many cases CMOs rise above such issues, as they are legitimate companies who adhere to any and all legislation, but there will always be dangerous elements who are willing to take risks. As always, it’s best for contractors to look into CMO associations, as membership of one of those often means the company has been made to pass some kind of accreditation check. The delay of a male pattern baldness drug from GSK highlights the risks in selecting a CMO, after a production halt at their French CMO meant they could not meet the launch date for Zagallo (dutasteride) in Japan. Of course, there are other forms of outsourcing that companies can consider, from contract research organisations (CROs) through to Contract Sales Offices or Agents. CROs are a popular choice due to the high levels of expertise they can often secure in recruitment. High quality scientists and researchers are always limited in the industry, with many being quickly snapped up either by pharmaceutical giants or other CROs. It’s not uncommon for CROs to also work closely, if they don’t already offer, quality-control and testing services for pharma products. This limits potential for cross- contamination or damage in shipments, and helps to keep information protected from any possible rivals. In an industry where intellectual property can be worth far more than anything else, it’s certainly something to consider. And IP has long been one of the reasons why outsourcing can be considered risky. While patents can help protect a company’s finished product from theft, the same can’t be said for the research process – which is still very much a race to the finish line. CROs are of course dedicated to protecting the interest of their clients, as they have a legal and moral obligation to do, but there will always be the associated risk where valuable information is sent from one location to another – and that is just another aspect pharmaceutical companies need to keep in mind. 24-27.qxp_Layout 1 09/06/2020 11:09 Page 4FUNDING 28 Pharma Business International www.pbiforum.net A lthough the European Medicines Agency (EMA), the European Union agency for the evaluation and supervision of medicinal products, doesn’t fund research or drug development directly, it can and does provide support to medicine developers that may be helpful for researchers applying for funding under the European Commission’s Horizon 2020 Programme. The largest ever EU funded programme for research and innovation, Horizon 2020 has a budget of almost €80 billion and runs until this year with the aim of trying to find and fund new, safer and more effective human medicines and interventions. An organisation that does provide direct funding is Innovate UK, a public body driving science and technology innovations to grow the economy. Since 2007, the organisation, billed as the UK’s innovation agency, has invested £1.5 billion in innovation, a figure that has been matched by a further £1.5 billion in partner and business funding. During this period, it has helped more than 5,000 companies across the industrial spectrum, medical, pharmaceutical and life sciences companies among them. It does this through direct investment in organisations but also through innovation competitions. Yet it’s not only companies Innovate UK provides funding for, but other innovation orientated organisations – including Medicines Discovery Catapult. This independent not-for-profit organisation is a national facility connecting the UK community to accelerate innovative drug discovery. It provides scientific capabilities and acts as a gateway to specialist facilities, technology and expertise, supporting SMEs to dive development and the widespread use of new approaches for the discovery and early development of new medicines. Part of the organisation’s literature is that traditional models of development Funding discovery Even the biggest pharmaceutical players are required to secure funding to bankroll drug development or to move a novel new substance into clinical trial. But for research firms and small-scale developers, funding is the very lifeblood of their research and development programmes. Fortunately, there’s a number of different avenues open to companies, researchers and other organisations, whether they get the required amount from one source or from a combination. 30 Á 28-31.qxp_Layout 1 09/06/2020 11:10 Page 1Pharma Business International 29 www.pbiforum.net FUNDING © Shutterstock /steved_np3 28-31.qxp_Layout 1 09/06/2020 11:10 Page 2Next >