Tag: health

With about 600 articles published in major dermatology
journals, almost 40 books authored, 70 chapters in various books, and a h-index
of 49, Dr. Robert Baran is one of the most knowledgeable dermatologists in the
world. He began publishing more than 67 years ago, is invited to many
international conferences every year, and his specialty is nails.

He’s the one to ask about nail diseases and their therapies, and is one of the more active researchers in this field (Scopus mentions 7200+ citations and 380+ co-authors). He has membership in many societies (not the least of which is the American Academy of Dermatology as Honorary Member), and serves on many editorial or advisory boards.

Literature to the rescue

Dr. Baran is also a practitioner and leads the Nail Diseases
Center in Cannes, France, balancing his life between research and patient care.
But he has a challenge: he has no access to research facilities, such as a
general hospital. How does he manage this issue? Literature is the answer. His
office, where he writes his publications, is a real library, devoted to
dermatology, with thousands of references: articles, reviews, pre-prints,
books, abstracts of congresses, notes, and so on.

Delivering the nail bibliography

For decades, Dr. Baran has relied on information
professionals to survey what happens in the field of several subjects that he
is interested in. And I’m one of them. I have been helping Dr. Baran for almost
20 years, ever since I was a scientific information expert at a pharmaceutical
company, devoted in dermatology. Now I serve as a customer consultant at
Elsevier.

Every month, I provide Dr. Baran with the ‘nail
bibliography’, a survey of the biomedical literature, focused on the nail and
its diseases. Dr. Baran wants it to be quite detailed, to be sure we do not
miss any important papers (clinical trials, opinions, new cases, etc.). To meet
his needs, I have created a broad search strategy that covers Dr. Baran’s
favorite subjects, such as onychomycosis. He receives an alert, every month,
and then selects the relevant references.

How Embase helps

In this effort, I have always used Embase and MEDLINE, and,
for 10 years now, Embase.com. This is for several reasons, the first one being
the coverage. Dr. Baran needs a deep understanding and coverage of the
literature. He has an impressive comprehensive memory and understanding of the
literature in dermatology, and is still eager to learn more and more—that is
how he produces such a high-level work.

That is why combining the coverage of MEDLINE and Embase is mandatory. On average, in Embase.com, the unique Embase content represents about 28% of the database, MEDLINE stands for 28%, and 44% of the content is covered by both databases.

For the ‘nail bibliography’, 34% of the references are from the unique Embase content, which is meaningfully more than the expected average. That is why covering Embase and MEDLINE is valuable. Embase.com allows us to address both with a single request, using the Embase vocabulary, the Emtree thesaurus. This is another reason why I utilize Embase.com: it enables me to create a single search strategy that combines Emtree terms and free text words to broadly cover the subject. Emtree terms focus on very specific concepts, such as onycholysis or paronychia. Free text words help to cover the records that were very recently added to Embase.com and that are still in process.

Getting automatic alerts

Once the search strategy is designed, Embase.com helps to
automatically manage email alerts. So, each month, I receive an email, and then
I log into Embase.com to screen the bibliography—a task that is easier with the
clipboard and export feature. The search strategy can be modified or improved
at any time, according to the needs of Dr. Baran.

This emblematic example illustrates the usefulness of a tool
with a very broad content and advanced search functions. In the hands of a
scientific information expert, it makes it possible to best meet the needs of
the most demanding researchers.

Check out Embase.com now for yourself, or contact me if you want to know more about Embase and how it can help your research.

Medicines Discovery Catapult (MDC) have welcomed a cohort of MRC-funded PhD students as part of a training, talent and skills agenda to help develop the next generation of drug discovery researchers.

Charlotte Criscuolo, University of Manchester; Rebecca Kelly, University of Liverpool; Michael Eyres, University of Oxford; and Tim Muntslag, University of Southampton have joined MDC to work on 4-6-month neuroscience and biomarker research projects.

The projects provide the next generation of innovative scientists with key training and exposure to industrial R&D, whilst increasing MDC’s capacity to carry out novel science in areas of benefit to the UK’s drug discovery industry.

Dr Nicola Heron MBA, Head of Collaborative R&D, Medicines Discovery Catapult and External Advisory Board Member, MRC DiMeN Partnership, said:

“Fostering the next generation of innovative scientists and budding entrepreneurs is crucial for the future of drug discovery. By hosting these students at MDC, we are helping to develop their commercial skills and industrial knowledge, plus the generic skills all researchers require to succeed – communication, time management, teamwork and critical thinking. The students also bring novel scientific approaches to MDC that are of potential benefit to the UK’s drug discovery community. The advantages of this Doctoral Training Programme are genuinely reciprocal, and it is a pleasure for MDC to be involved.”

Dr Martin Main, Head of Molecular Technologies, Medicines Discovery Catapult, said:

“Drug Discovery, like any scientific field, is constantly evolving. Hosting these students at MDC is invaluable. It gives us the opportunity to not only to pass on the scientific skills and knowledge my colleagues and I have developed through years of experience, but to also learn from these early career researchers who are incubators of novel science. It is a real privilege to play a part in setting them up for future scientific success through this Doctoral Training Programme.”

The positions were advertised through the Discovery Medicine North (DiMeN) Partnerships Doctoral Training Programme (DTP), and across the wider network of MRC-funded university DTPs, demonstrating a collaborative academic approach to nurturing talent and providing development opportunities to researchers throughout the UK.

The DiMeN partnership is made up of partner institutions including the Universities of Leeds, Liverpool, Newcastle and Sheffield, and aims to train the next generation of researchers to tackle the major health problems facing the population.

The partnership provides up to 30 fully funded studentships across the partnership per year, focussing on the complementary themes of:

• Genetic Influences on Health
• Ageing and Disease
• Bioinformatics and Personalised Medicine

For more information on DiMeN and the DTP please visit: https://www.dimen.org.uk/

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An EMA advisory panel has rejected expanding the use of Eli Lilly’s Emgality to include prevention of episodic cluster headaches.

The Committee for Medicinal Products for Human Use (CHMP) said the single 106-patient study filed by Lilly to support the new marketing application for Emgality (galcanezumab) “did not show clearly that Emgality is effective for preventing attacks” in people with cluster headaches.

The CGRP inhibitor has been approved in the US and Europe for chronic migraine prevention since 2018, and was cleared by the FDA for the cluster headache indication last June, becoming the first drug for this indication in the US.

Cluster headaches are distinct from migraine and less common, but are considered to be much harder to treat. They are characterised by severe pain, typically on one side of the head and around an eye, with attacks occurring during ‘cluster periods’ which can last weeks to months, notes the CHMP.

Lilly’s clinical trial – called GCAL – showed that over a three-week study period, patients given Emgality experienced 8.7 fewer weekly cluster headache attacks than they did at enrolment, compared to 5.2 fewer attacks for the placebo group.

That difference only just scraped above the threshold for statistical significance, however and the FDA is thought to have allowed its approval because of the lack of effective treatments for cluster headache, plus supportive secondary results in the trial such as the proportion of patients seeing a 50% or greater reduction in weekly attacks.

The CHMP has clearly taken a tougher line, and its negative opinion means patients in Europe will have to wait longer for a treatment option.

To give an indication of just how debilitating the condition can be, subjects in Lilly’s trial averaged more than 17 cluster headache attacks per week during the baseline period, equivalent to 2-3 every day. Attacks typically last between 15 to 180 minutes.

The CHMP’s verdict was also a blow commercially for Lilly, which was third to market after rival injectable CGRP inhibitors Aimovig (erenumab) from Amgen and Novartis – the market leader – and Ajovy (fremanezumab) from Teva.

Being the first CGRP drug to get a claim for cluster headache on the label in the US is viewed as a way for Lilly to carve out its own niche market, particularly as neither Aimovig nor Ajovy remains in development for this indication.

There is more competition in play for Emgality from Lundbeck’s new intravenous CGRP drug Vyepti (eptinezumab), which requires less frequent dosing of Emgality as well as Aimovig and Ajovy, but has to be given intravenously in a clinic rather than by subcutaneous self-injection.

Amgen reported $306 million from US sales of Aimovig last year, while ex-US licensee Novartis doesn’t break out its sales of the drug. Emgality leapfrogged second-to-market Ajovy last year, making $163 million compared to $96 million for Teva’s drug, thanks in part to the cluster headache approval.

GlobalData said last week – ahead of the cluster headache rejection – that it expects Aimovig and Emgality to lead the migraine prevention market in 2025 with sales of $1.8 billion and $1.7 billion respectively, with Vyepti making around $566 million in that year.

Meanwhile, there are challenges elsewhere in Lilly’s migraine franchise too, with two new orally-active CGRP blockers for acute migraine treatment – Allergan’s Ubrelvy (ubrogepant) and Biohaven’s just-approved Nurtec ODT (rimegepant) – expected to exert pressure on Lilly’s acute therapy Reyvow (lasmiditan).

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A first-in-class 5-HT1F receptor agonist, Reyvow was cleared by the FDA in October but only launched a few weeks ago – at a price of $640 for eight pills – because it had to wait for scheduling by the US Drug Enforcement Administration (DEA).

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On January 2, 2020, the FDA released the CDER annual report, stating that 2019 was “another strong year for innovation and advances.” The FDA cleared 48 new drugs for market, making it the second most productive year in the last decade (2018 approved 59 drugs). But given the fact that the total number of submissions have gone down, and that the American government was shut down for a lengthy period at the beginning of the year, the FDA delivered an impressive result in 2019.

I’d like to share some of my personal thoughts
after reading this report. Here are a few notable highlights and my takeaways:

1. 21 of CDER’s 48 novel drug approvals, or 44%, were orphan drugs

What does that tell us?

* As Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, puts it, “New drug therapies for patients suffering from rare diseases are often among the most important approvals. Patients with rare diseases frequently have few or no drugs available to treat their condition — and for them, approvals of so-called “orphan” drugs can mean new hope for an enhanced quality of life, and in some cases, survival.”

* Patient unmet needs and market value merges where the pharmaceutical companies pay attention and take practical approaches. One of the most promising avenues that big pharmas can take is to partner with non-profits focused on particular unmet needs, and to collaborate with small biotech companies that are specializing in tackling unmet needs. One such example is Beacon Discovery, which has partnered with several leading companies, focusing on helping them with early-stage research and discovery.

If 2019 was a good year for orphan drugs, 2020 is already getting off to a great start. In Q1, three more possible orphan drugs to be on the look-out for include: Oxbryta, the first treatment to address the root cause of sickle cell disease (SCD); Adakveo, an injectable treatment for SCD patients who suffer from vaso-occlusive crises (VOC); and Givlaari, a drug to treat the blood disorder acute hepatic porphyria (AHP). Learn more about these drugs here.

2. Innovative
new therapies

Some of these new drugs in development are built
on very exciting new innovations:

* The above-mentioned Givlaari is based on RNA interference (RNAi), a technique that silences abnormal gene expression. As BioSpace reports, Givlaari is only the second drug to be approved leveraging RNAi.

* The cancer drug Rozlytrek, for pan-tumor use, marks only the third time that the FDA has “cleared a cancer drug to treat tumors based on a specific genetic signature rather than by location in the body,” explains BioPharmaDive.

As evidenced by the emergence of innovative drugs like these, my personal opinion is that we are truly transitioning into the era of precision medicine and personalized medicine, when more and more oncology drugs are being developed based on the use of biomarkers and targeted delivery of medicine.

3. 10 new biosimilars

Biosimilars, which are drugs that are very
similar to already-approved biologics, had a busy regulatory year in 2019, with
many new approvals and new guidance documents published.

There were 10 biosimilar approvals covering
indications for numerous conditions, such as rheumatoid arthritis, plaque
psoriasis, breast cancer, metastatic stomach cancer, metastatic colorectal
cancer, non-squamous non-small cell lung cancer, glioblastoma, metastatic renal
cell carcinoma, cervical cancer, B-cell non-Hodgkin’s lymphoma, chronic
lymphocytic leukemia, granulomatosis with polyangiitis, and microscopic
polyangiitis.

“Biosimilars have great potential for both patients and the entire health care system. As patents and exclusivity protections for biologics expire in the United States, we can expect many more biosimilars to be submitted for approval. More products on the market means greater competition that can lead to increased access to therapies and lower costs to patients.”
– Janet Woodcock

4. The first-ever FDA novel approval drug from a Chinese pharmaceutical company

November 14, 2019  was a clear milestone for Chinese pharmaceutical companies, as it was then that Brukinsa from BeiGene received FDA approval for the very first novel therapy from China in history. Regulatory reform in China has enhanced innovative drug discovery and clinical development, encouraging innovation and speeding patient access to new drugs.

Studies showed that BeiGene’s drug has real promise. “The FDA’s decision is largely based on tumor shrinkage data from a phase 2 trial in 86 Chinese patients,” reports FiercePharma. “In that single-arm study, Brukinsa triggered an overall response rate of 84% with a median duration of response of 19.5 months. Some 59% of patients saw a complete response, meaning their cancers were undetectable after treatment.”

A mission to work together

To quote Janet Woodcock once more: “CDER’s mission goes well beyond
critically reviewing the safety and efficacy of drug applications we receive
from industry … We are working to develop more innovative and efficient
approaches for the development and study of the drug therapies that will emerge
from these technological advances.”

I believe this is also the dream and the
mission of most people working in the pharmaceutical industry: to work
collaboratively with partners across academia, industry, patients, caregivers
and regulators to provide true benefits for patients. We at Elsevier Life
Sciences solutions all share that same mission.

If you are interested in finding out more about collaborations to support drug discovery and development, please read more on our website.