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Biopharma 2018 Success Kit: 11 months to go, 6 rare disease trends to watch!

For the rapidly developing orphan drug market, change is a constant. From new technologies to discussions over pricing, the estimated $137 billion market will witness new challenges for biopharma companies in the rare disease space.

In a bid to unravel these barriers and with 11 months more to go in 2018, the team at Partners4Access pick the six main trends to watch out for.

  1. M&A Deals to intensify, says Sophie Schmitz, Managing Partner. Here’s why:

“January in the biotech world is a little like the transfer season in football: mergers and acquisitions are announced at a furious pace. As most years, the announcements start at JP Morgan. This year we’ve seen Celgene acquire Impact Biosciences in a $7 billion deal and Juno Therapeutics for $9 billion. Novo Nordisk’s unsuccessful attempt to acquire Ablynx for €2.6 billion, was rejected in favour of Sanofi’s bid of €3.9 billion.  This is the second Sanofi deal announced within a week; after Sanofi acquired Bioverativ for $11.5 billion.

In 2017, biotech M&A deals reached $200 billion. This year I predict this could be even higher for three key reasons. Firstly, driven in part by the recent tax reform in the US, secondly the need for large pharmaceutical companies to access new orphan drug growth opportunities and thirdly, a continued trend to reduce R&D risk and acquire companies with innovative technologies and late stage pipelines.

There are many cash-rich companies who have not yet announced any deals. The Pfizer will-they-won’t-they buy BMS discussion is still ongoing, and we haven’t heard yet from J&J, Amgen or Merck of their planned acquisitions. 2018 is going to be a very busy M&A year!”

  1. Rare oncology drug approvals will rise, according to Joanna Fernandes, Analyst

“Over 198 rare cancers affect 4.3 million people in the EU, mostly children.  This accounts for ~22% of all cancer cases in the EU each year. However, there is a small number of marketed treatments for rare cancers. Hope remains for the future as various programmes in the U.S. and E.U. enable the approval of drugs for rare cancers faster than ever before.

In 2013, ~60% of the FDA breakthrough therapies were for rare cancers, and 40% of orphan drug designated treatments target rare cancers. This percentage has steadily increased since these orphan drug programs began (1999 E.U., 1983 U.S.) and this trend is likely to continue.

The approval of new drugs for rare cancers benefits not only patients but also manufacturers, with oncology drugs expecting $100-120 billion in sales in 2020. Celgene’s Revlimid for myelodysplastic syndrome is expected to remain one of the top selling orphan drugs in 2020, earning revenues of over $10 billion per year. This high revenue stream will continue to attract more manufacturers to the rare oncology space further boosting approvals.”

  1. Christina Poschen, Associate Consultant talks of a bright future for new gene-therapy technologies

“Recently I interviewed a leading gene-therapy professor and asked about his thoughts about the future developments. He replied: We are optimists, we had to wait for so long and didn’t give up. Now this is an exciting time and will continue [to be so].

We agree! After the exciting launches in 2017, we expect 2018 to be even bigger and with more technologies to come to the market. Following CAR-T (2017), CRISPR a gene-therapy in its widest sense seems to be on the rise for 2018 offering hope for patients with a variety of rare genetic diseases and research the long-awaited success story.”

  1. Societal burden data will gain increasing prominence, according to Nader Murad, Senior Analyst

“When negotiating the price and access for a new treatment with payers, a manufacturer must demonstrate the incremental value offered by the therapy relative to available treatments. The same holds true for Orphan Drugs, albeit with less stringent data requirements. But what constitutes value?

Traditionally, efficacy, safety and budget impact on the healthcare system have been the primary focus of HTA appraisals; but in reality, the true value of a drug transcends these parameters and can have a positive impact beyond the patient’s condition and the healthcare system. These can include increased productivity of the patient’s carer, as their time would have otherwise been spent looking after the patient had the treatment not been available; reducing the strain on social care and disability benefits’ budgets etc.

While some HTA bodies such as NICE have begun to formally patient perspectives into their appraisals, there is little clarity as to how much impact they have on the outcome. As more high-value treatments such as gene and cell therapies make their way to the clinic, in 2018 we will witness more discussion and consideration given to the societal burden of a disease and the impact a therapy might have on it into HTAs. Healthcare systems are not mutually exclusive to the wider economy in which they exist, and high value therapies may just be the spark required to ignite this approach to evaluating the true value of a health technology.”

  1. Emphasis on planning Real World Evidence will grow, Jaineel Mistry, Market Access Specialist opines

“As the investment in rare diseases continues to increase, so does the acceptance for Real-World Evidence (RWE) within the space.

RWE has matured and now presents a great way to support adaptive and conditional approval and early market access processes by supporting regulatory decision-making. It enables adaptive value-based contracting centered on mutually agreed outcomes.

Moving forward into 2018, it is important that companies strategically plan the generation of real-world patient centered evidence by considering key questions raised by stakeholders. Questions will typically include the methodology, reliability and the usefulness of RWE in decision-making, particularly regarding treatment effects.

A core part of planning ahead will be to consider seeking scientific and HTA advice and potentially utilizing the various incentives and adaptive pathways which leverage real-world evidence.”

  1. Maximus Rex, Analyst highlights the emerging war on drug prices in the U.S.

“An overarching story from the first year of the Trump administration was the battle over the future of U.S. healthcare. Despite its best efforts, the Republican-controlled Congress was unable to “repeal and replace” Obamacare (Affordable Care Act) as promised. The intensity of this debate resulted in lawmakers benching another key healthcare-related issue- pharmaceutical prices.

On the surface it seems there is rare agreement between the two parties in Congress at the moment that prices are too high. Big pharma has been routinely criticized by the president on this subject, while Democrats have announced plans to regulate prices and fine companies for “exorbitant” price rises. Whether the current administration is serious about cutting prices is unclear, with the appointment of a former pharmaceutical executive to the Cabinet left some with raised eyebrows.

Pharma must be wary, especially since Spark charging $850,000 for Luxturna will push this topic further to the front of public debate.”

Please contact the P4A team on contact@partners4access.com

About Partners4Access:

Partners4Access is a global consultancy specialising in orphan drug access. We support the biotechnology and pharmaceutical industry along their launch journey to help secure successful price, reimbursement and access for orphan drugs. Partners4Access has solid partnerships with clients supporting their strategy and operations to effectively ensure launch success.