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Top Trends in Orphan Drugs

Top Trends in Orphan Drugs.

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Top Trends in Orphan Drugs

  1. 1. Top Trends in the Orphan Drug Industry Your guide to the hot topics influencing the industry now
  2. 2. Orphan Drug has become the most valuable designation in the Pharmaceutical Industry. With each new rare disease focused on, the trends for the pharmaceutical industry continue to shift towards a more personalized approach. The winds of change are strong in Pharma – and that makes the trends that make up this movement even more important. We gathered information from top pharmaceutical companies, patient groups, regulatory agencies, and healthcare providers to find out what some of the top trends were within the Orphan Drug movement. Varied in scope, these 17 trends are where the industry is headed, and are a must-read for any company looking to enter the rare disease marketplace.
  3. 3. The price of Orphan Drugs is increasingly under scrutiny. In the United States, The Department of Health and Human Services is fighting to keep the price of Orphan Drugs discounted when they are not being used for their specific rare disease designation, citing rule 340B. Meanwhile, industry claims that the same rule means that all drugs as designated as Orphan by the FDA are free from discounted pricing. A recent court ruling has put industry’s opinion in the right – but HHS hasn’t backed down and continues to discount Orphan Drugs in certain cases. In the UK, NICE and payers alike have begun to ask for more of a basis on pricing. What is the 340B Drug Pricing Program? 1
  4. 4. Rare disease patient groups are forming registries. Looked at as almost a status symbol by the more established patient groups, these patient registries allow for a better understanding of the natural history of many rare diseases. This is essential for a more efficient development process, and industry agrees. The fact that many patient organizations are embarking on this form of data generation is good for the upstream development of these drugs. 2
  5. 5. Breakthrough Therapy Drug Designation is a boon for the orphan drug industry. Breakthrough Therapies are defined by the FDA as a drug that, either alone or in combination with another drug, treat a serious or life threatening disease and based on preliminary clinical evidence may be far superior to anything in the market. For Orphan Drugs, meeting those criteria are far easier that their standard counterpart. Recently, Novartis gained a coveted third Breakthrough designation – meant for treatment of a rare disease. Meanwhile, other companies, such as Pharmacyclics, have cited the rule with chopping 7 to 9 months off the regulatory process. 3
  6. 6. Rare diseases are increasingly being seen as a tool to cure the common ones. Common diseases are often complex, consisting of many pathways over several systems. Rare diseases, however, usually are far more limited in their scope – often one gene and one pathway or mechanism. In fact, many rare diseases account for subsets of common ones. By studying rare diseases, there is a new thinking in the field that we are in fact studying ways to cure the common ones one pathway at a time. 4
  7. 7. Pressure is increasing for early access to potentially lifesaving treatments via FDA Expanded Access and/or Right to Try laws. If someone’s prognosis is already fatal, are we justified in fast-tracking them a proclaimed cure that could have deadly consequences in an attempt to save their life? The government and industry seems to believe so – though in not so stark a situation. While Expanded Access and Right to Try laws do not eliminate regulation, they certainly help to bypass the bureaucracy when someone’s life could be saved by a risky treatment. 5
  8. 8. Patient engagement in Clinical Trials is increasing. Both industry and the FDA have been increasingly growing their desire to have patients actively involved in the clinical trials process, both through patient representatives and workshops aimed at educating the test candidates what, exactly, it is they are looking for. The advent of patient groups and registries is helping with this tremendously. 6
  9. 9. There is a shift toward more curative treatments like gene therapy versus chronic, lifelong treatments. Enabled by new technology, the days of taking a pill to live a normal life may be long gone, and the days of an actual cure may be on the horizon. Gene therapies and other treatments, such as CRISPR and RNAi, are proving to appear to live up to the hype. These therapeutics have the potential to treat the disease – and never have to treat them again. 7
  10. 10. Patient organizations are increasingly looking to shape policies for rare diseases. Patient groups have more and more influence, particularly as they form registries, and they can put more pressure on the industry than previously thought – and they know it. Patient groups have led to companies racing to develop therapies, developed guidance on risk-benefit, and are increasingly looking to lobby for more favorable terms to help patients in the long haul. See the AKU Society for a great example of a patient organization working hard to push for what they need. 8
  11. 11. Policies continue to impact the rare disease space. In 1982, nobody developed for orphan drugs. Starting with the passage of the Orphan Drug Act in 1983, the rare disease space will always be tied to regulation and legislative action due to the nature of the risk-benefit surrounding developing therapies for them. In 2012, the Breakthrough Therapy laws changed the face of the game again, and future laws will continue to shift the playing field in ways that we can’t even predict yet. 9
  12. 12. Governments are trying to further help companies to work on rare diseases. There are many means for governments to innovate to help spur growth in the rare disease space, and adaptive licensing in Europe is just one example. As the impacts of this research on the population as a whole increases, government interest will only grow. 10
  13. 13. Rare disease is increasingly seen from a global perspective. Disease doesn’t end at our borders – and regulatory agencies are starting to take note. The FDA and EMA are working together more on rare disease topics than ever before, and more countries are getting involved yearly. This will help spur orphan drug laws in countries where they do not currently exist. 11
  14. 14. Mergers will continue to be common. Just recently, it was revealed that AbbVie was looking to acquire Shire for their Orphan Drug focus – a merger that seems to be likely to take place. In an industry where one breakthrough drug can make or break your finances, expect to see mergers and acquisitions continue to be a major player in the marketplace, particularly as Big Pharma knows they can purchase a smaller Orphan Drug pharmaceutical manufacturer and limit their costs while reaping all of the rewards. Find out more about the merger between AbbVie and Shire here > 12
  15. 15. Drugs will be increasingly produced via Bioprocessing. Biologics are becoming more and more common every day, and the advantages can’t be ignored. The biologics space – and the cell culture field in general – is growing at a staggering rate, and as more and more pharmaceuticals shift to biologics production, so too will the orphan drugs. 13
  16. 16. Therapy areas are getting increasingly focused. Long gone are the days where pharma companies decided to target every single disease known to man. It is becoming more and more common for companies to have therapeutic “areas of focus”, specific diseases or areas that they are targeting for one strategic reason or another. This increase in focus coupled with the intrinsic advantage is driving an increase in Orphan Drug production, as companies explore every aspect of their core competencies. For example, if a company is focused on the rheumatics, rare disease rheumatics are a natural extension. 14
  17. 17. R&D Centers and Innovation Clusters will spur growth in the industry. Countries are recognizing the benefit to catering to pharma and biotech companies – and it is shown in the growth of R&D centers across the globe. This will help spur orphan drug development and cooperation among typical competitors. 15
  18. 18. As new countries pass orphan drug legislation, emerging markets will increasingly play a large role in the industry. The countries that currently have Orphan Drug legislation have spurred unprecedented growth, but there are still several huge untapped markets – including China – that will become an enormous boon to the industry as payers and patient groups in those countries begin to push for the same rules as in the US or EU. Attendance by regulators and payers from these countries at recent conferences, such as the World Orphan Drug Congress, signals interest on the part of those countries in expanding their legislation. This will open up a huge additional market for the already booming orphan drug producers. 16
  19. 19. Big Data is helping speed up clinical trials and promote faster access to information on all sides. Big data is helping the entire biotechnology industry, and Orphan Drugs is no exception. In fact, several big data and analysis companies attended last year’s World Orphan Drug Congress. Being able to properly analyze data rapidly is speeding up clinical trials as well as speeding up the opportunities to look into new challenges. Likewise, the advent of patient registries is bringing forward new challenges to be solved by Big Data as the field progresses. 17
  20. 20. Exploring all areas of rare disease- through strategic keynote presentation, focused tracks, collaborative roundtable discussions and abundant 1-2-1 partnering; the World Orphan Drug Congress gives you the opportunity to tailor your participation and experience to your needs. Taking place between April 24-25, 2015, the World Orphan Drug Congress remains the place to discuss the hot topics currently being discussed within the industry. Find out more at: www.terrapinn.com/orphandrugsus

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