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2018 proved to be a pivotal year, and as we look towards 2019, key foundational policies and recommendations still need to be fleshed out. Here is our look into some of the most critical issues affecting all players in drug and device development.
1. Brexit-need I say more?
As March is creeping closer and closer each week, the situation in the UK and MHRA’s future role doesn’t seem any clearer. While the “deal” was published, it contained only vague notions about the future collaboration and regulatory network. Meanwhile, the other European regulatory agencies are hiring and training new staff, yet it would be difficult to replace the know-how of the experts at the British agency immediately, and the rumors are that pressure is increasing at other agencies, too. The EU Regulatory Network has, however, found a new gear in increasing collaboration-the EU regulatory system optimization is on a whole new level.
2. Digital health market growing and regulators jumping on the ball to advance the use of the new technologies
A recent projection from Global Market Insights1has estimated the digital health market to be $379 billion USD by 2024. The technologies advancing digital health emergence range from regular mobile phones to sophisticated artificial intelligence applications. The FDA recently released their open-source mobile application to promote collection of real-world evidence for regulatory decision making. At the same time, medical imaging solutions continue to surpass the human eye in accuracy of diagnosing; however, the uptake of such tools will depend on the medical society endorsement. Apple Watch received a medical device classification in the U.S., not to mention the recent FDA approval of the joint Bayer-Merck venture, an AI-based diagnostic tool for chronic thromboembolic pulmonary hypertension affecting only five in a million. The recent solutions will only be a door opener for further technologies, which will emerge quickly through the device pathway.
3. Stock market-after a long run of rising share prices, including for biotech and pharma, it looks like we now enter a period of regression
After some concerns in the U.S. that the incoming President would address prices of medicines, it feels that the situation has returned to business as usual. This coupled with some very exciting and promising new approaches such as the CAR-T and CRISPR-CAS9 technologies, immunotherapies, and promising new therapies for diseases with a high unmet medical need such as Alzheimer’s, enabled stocks of biotech and pharma companies to soar over the last two years. However, with continuous concerns over drug prices, the economic outlook (inflation, trade disputes, debt of countries like Italy), some important clinical failures (e.g. Alzheimer’s) and doubts about certain technologies, such as CRISPR, it seems we are entering a period of downward trends for pharma and biotech stocks.
4. Scientific discoveries and breakthroughs continue to shift innovation landscape
There are almost 300 cell and gene therapies in development currently, report PhRMA.2Many build on science that was established long ago but as development and commercialization takes a while to achieve, there are already new technologies emerging particularly in gene editing. The CRISPR story and its potential to create “designer humans” took a leap forward with the surprise announcement from a Chinese researcher having edited genes of two babies. With the hunger to become world leader in driving innovation and scientific discoveries, using loop holes for enabling such studies was not well regarded by neither Chinese authorities nor the global science community. Needless to say, the science will move forward faster in the emerging countries than in the countries with rigid regulatory frameworks-more headlines are on the horizon.
5. Medicine prices will continue to be an issue
Approval of a new, potentially curative, treatment for a spinal muscular atrophy (SMA), foreseen in May after having won a priority review with data demonstrating superior efficacy on 15 patients, has already reignited the debate on pricing. With the estimated price of 4-8 million USD, it is still argued to be cost-effective against the current treatment of $500,000 USD annually. This will continue to further drive the debate as to what constitutes value for society and patients, while payers across the globe are struggling to find new payment models to afford such treatments, often addressing high unmet medical needs. In Europe, the HTA process and value frameworks have been judged as not fit for curative therapies by leading academics, and countries are now developing new frameworks. However, as with some earlier examples, the expectation is that most European countries can’t afford purchasing the treatment to the patients in one go, as rare as the disease would be. Uptake of new contracting models (value-based, cost- or risk-sharing) are expected to rise with curative therapies coming to the market.
6. Acquisitions-trend of big pharma buying small biotech to fuel the pipeline will continue
The large pharma companies continue to optimize their portfolio. Under the new CEO, Novartis divested some of its businesses and streamlined operations further. At the same time, GSK, which also sold some of its portfolios under new leadership, have taken a new turn to focus on oncology with its Tesaro acquisition. New portfolio shifts will come with changes in infrastructure and skill set: the diagnostic tool developed by Bayer and Merck require employee profiles and a business model that looks very different to conventional pharma. With the recent change of Pfizer CEO, more shake up is expected in the coming year.
7. Data privacy, transparency, and confidentiality-what’s the perfect mix?
Last spring no one with an email or social media account could escape from seeing updates on GDPR. While the intentions are valuable, the impact is still being detangled in areas such as clinical trials, as it goes against the trend of being more transparent with data sharing. Both EMA and FDA have geared up their clinical data publication, albeit on hold in Europe at the moment, but it has not delivered more information to lay persons as wished for. Additionally, Professor Ben Goldacre.3made the effort to review rate of reporting clinical trials and outcomes, revealing that industry is doing rather well and hospitals rather poorly. Amidst the debate, particularly European payers still complain that they received the data too late and call for a better and earlier data sharing model. It is expected that EMA will prioritize it to some extent when out of business continuity.
8. Devices under a magnifying glass with investigative journalists-“Panama scandals” expected?
The journalist group that revealed Panama papers4are now digging into the medical device market. The initial articles published in December weren’t too nice to read; however, they are in line with the European policymakers’ attempt to clean the market with the new regulation. It is yet to be seen whether there are bigger scandals emerging in media or whether the steps that have already been taken will suffice to kill any potential fire before it takes off.
1Global Market Insights, Inc., Digital Health Market Size to exceed $379 Bn By 2024, Published: October 8, 2018
2Medicines in Development | 2018 Report, Medicines in Development for Cell Therapy and Gene Therapy, Accessed via http://phrma-docs.phrma.org: December 2018
3 EU Trials Tracker, accessed December 2018, eu.trialstracker.net
4International Consortium of Investigative Journalists, International Medical Devices Database, Accessed December 2018, https://medicaldevices.icij.org/
Thomas Bols, Senior Vice President & Managing Director, DIA Europe, Middle East & Africa