Orphan Disease's Challenges
329 views | +0 today
Orphan Disease's Challenges
Global news about the Orphan Disease's market and it's evolutions
Your new post is loading...
Your new post is loading...
Scooped by Stéphane Campana
Scoop.it!

Genes Are Overrated

Genes Are Overrated | Orphan Disease's Challenges | Scoop.it
Their discovery wasn’t predestined, nor do they dictate our destinies—and current ideas about them may die.
more...
No comment yet.
Rescooped by Stéphane Campana from Social Media and Healthcare
Scoop.it!

Rare Diseases: The Role of Social Media in Patient Recruitment

Rare Diseases: The Role of Social Media in Patient Recruitment | Orphan Disease's Challenges | Scoop.it

Since the passing of the Orphan Drug Act in 1983, the rate of drug development in the United States has seen a significant increase. The act directly impacted funding - a major challenge associated with rare disease drug development. This in turn has led to a number of life-changing treatments for patients and an increasingly lucrative market for drug developers. However, there is still much more work to be done. Rare diseases impact 30 million people in the United States alone —more than the number of patients diagnosed with AIDS and cancer combined. Out of the 30 million patients, 95 percent do not have an FDA-approved treatment and at the current rate of drug development, it is estimated it could take over 450 years to treat every rare disease.

Mark Baglin, vice president and product strategy team leader at Shire Pharmaceuticals and Kevin J. Anderson, associate director of Global Clinical Operations at Alexion Pharmaceuticals, led a really interesting “Rare Disease” track during the recent Festival of Genomics in which they explained the unique challenges and opportunities in rare disease drug development. According to the speakers, unlike with big pharma, patient involvement plays a vital role in rare disease R&D and clinical trial design. Here we take a look at ways to best leverage physician and patient recruitment when it comes to rare disease drug development.

Patient Recruitment

The most obvious challenge facing the rare disease industry is recruiting patients and principal investigators (PIs) for clinical studies. Treating physicians don’t fall under a rare disease bucket, making it difficult to know what doctors should be involved. Because many of the PIs used for these trials are considered key opinion leaders within the field of rare disease, they can be expensive and harder to work with due to their demanding schedules. Furthermore, there are a limited number of experienced PIs outside of the U.S. and Europe, which limits the globalization capabilities of a trial.

Another major issue is it can be extremely challenging to find patients given the rarity of the disease. To gather meaningful data, patient recruitment requires the participation of several sites across many regions, as 50- to 75-percent of sites will not enroll a single patient and if they do, the average is typically only one patient. Delays in the recruitment process not only add costs but also create uncertainly around the feasibility of the program.

To leverage patient recruitment in clinical trials, traditional methods need to be altered to employ a more patient-centric approach. Patient recruitment should depend of the prevalence of a particular rare disease in a region, rather than launching sites across several regions waiting for patients to enroll. This not only decreases costs, but also is more efficient than the current method.

Patient Involvement

Historically, the patient’s greatest barrier to understanding the prevention, diagnosis and treatment of rare disease is an insufficient knowledge of the mechanisms and history of the disease. The key to developing patient knowledge is open communication. Doctors need to share the mission with the patient and help them understand all elements of disease research and treatment, including funding and patient services. Rare disease patients tend to be the most vocal and well informed and often have the support of active advocacy groups. Partnering with these groups and ensuring transparency among developers and site investigators is key to maintaining involvement.

SEO and Social Media

As enrollment rates continue to drop even as more clinical trials are introduced, it is clear that clinical trial coordinators need to develop new approaches to enrollment. Search engine optimization and social media offer the ability to alter the landscape of clinical trial recruitment. Social media is becoming increasingly important in filling the missing health information, particularly surrounding experiences using particular therapies and associated side effects. According to the Ogilvy Healthworld report “Connecting the dots: Which Pharma Companies are Succeeding in the Social Media Space,” the average number of tweets by pharma companies has increased by 530 percent since 2013.

But followers aren’t enough. The report shows that companies with the most effective engagement and that generate the most interest do not necessarily have the largest community. Smaller biotech companies need to take advantage of this opportunity and use social media and SEO for site and patient recruitment. Several companies have found recruitment success by reaching out to Facebook groups that may not have sufficient funding to form their own advocacy organizations and connecting with the organizers about becoming patient advocates. PatientsLikeMe is another online resource that many patient advocacy groups turn to and can serve as a useful outlet for recruitment efforts.

Developing a rare disease drug is no easy task, but the returns offer great rewards. Developers still face hurdles when it comes to funding and showing value to payers who now more than ever are looking for ways to save costs on drugs. For patient recruitment, patient advocates and social media can prove to be a valuable source when used effectively.


Via Plus91
more...
No comment yet.
Rescooped by Stéphane Campana from Biotech, Pharmas and ideas
Scoop.it!

Lancement du Guide interactif du parcours avec une maladie rare

Lancement du Guide interactif du parcours avec une maladie rare | Orphan Disease's Challenges | Scoop.it
A l’occasion de la journée mondiale des maladies rares, l’Alliance Maladies Rares a lancé le « Guide interactif du parcours avec une maladie rare » pour accompagner les patients dans leur parcours de soin.
Via Herve Ansanay
more...
No comment yet.
Scooped by Stéphane Campana
Scoop.it!

FDA Awards Research Grants for 18 Rare Diseases, Including HPV-Related Oropharyngeal Cancer

FDA Awards Research Grants for 18 Rare Diseases, Including HPV-Related Oropharyngeal Cancer | Orphan Disease's Challenges | Scoop.it
Eighteen new research grants, totaling more than $19 million, have been awarded by the FDA for product development in rare diseases such as HPV-related head and neck cancer and recurrent glioblastoma.
more...
No comment yet.
Scooped by Stéphane Campana
Scoop.it!

Roche met la main sur la biotech marseillaise Trophos

Roche met la main sur la biotech marseillaise Trophos | Orphan Disease's Challenges | Scoop.it
Le groupe pharmaceutique suisse a annoncé vendredi investir jusqu'à 470 millions d'euros pour acquérir cette biotech française qui développe un traitement contre une maladie neuromusculaire rare, l'amyotrophie spinale.
more...
No comment yet.
Rescooped by Stéphane Campana from Alzheimer's Disease R&D Review
Scoop.it!

Nobel discovery opens window onto Alzheimer's disease

Nobel discovery opens window onto Alzheimer's disease | Orphan Disease's Challenges | Scoop.it
LONDON (Reuters) - The discovery of cells in the brain that act as the body's internal global positioning system, which won three scientists the Nobel Prize for medicine on Monday, opens an intriguing...

Via Krishan Maggon
more...
No comment yet.
Rescooped by Stéphane Campana from Melanoma BRAF Inhibitors Review
Scoop.it!

Roche’s cobimetinib plus Zelboraf® (vemurafenib) provided significant benefit in advanced melanoma over Zelboraf alone

Roche’s cobimetinib plus Zelboraf® (vemurafenib) provided significant benefit in advanced melanoma over Zelboraf alone | Orphan Disease's Challenges | Scoop.it

Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced positive data from the coBRIM Phase III study. The results showed that people with previously untreated BRAF V600 mutation-positive, advanced melanoma who received the MEK inhibitor cobimetinib plus Zelboraf (vemurafenib) lived significantly longer without their disease worsening or death (progression-free survival; PFS) compared to Zelboraf alone.1

 

The combined therapy reduced the risk of disease worsening or death by half (hazard ratio [HR]=0.51, 95 percent confidence interval [CI] 0.39-0.68; p<0.0001), with a median PFS of 9.9 months for cobimetinib plus Zelboraf compared to 6.2 months with Zelboraf alone. The safety profile was consistent with a previous study of the combination. The most common adverse events seen in the combination arm included diarrhea, nausea, rash, photosensitivity, and lab abnormalities.1

 

The coBRIM results were statistically significant across multiple secondary endpoints. The median PFS by independent review committee (IRC) was 11.3 months for the combination arm compared to 6.0 months for the control arm (HR=0.60, 95 percent CI 0.45-0.79; p=0.0003). The objective response rate (ORR) was higher in the combination compared to the control arm (68 vs. 45 percent; p<0.0001). Overall survival (OS) data are not yet mature.1

 

The late-breaking coBRIM data will be presented at ESMO 2014 today during the Presidential Symposium by Professor Grant McArthur, Peter MacCallum Cancer Centre, Australia (Abstract #LBA5_PR, Monday, September 29, 2014, 16:00-17:20 CEST) and are also part of the official press programme. Additionally, the study was published online today in the New England Journal of Medicine.1

 


Via Krishan Maggon
more...
Krishan Maggon 's curator insight, September 29, 2014 8:40 AM

Cobimetinib plus Zelboraf reduced the risk of disease worsening by half compared to Zelboraf alonePhase III coBRIM study results will be presented today during the Presidential Symposium at the European Society of Medical Oncology (ESMO) 2014 Congress and published in the New England Journal of MedicineRoche has submitted an EU marketing authorisation application for the combination for the treatment of BRAF V600 mutation-positive advanced melanoma.

 

http://

Abstract LBA5_PR - Phase 3, Double-blind, Placebo-controlled Study of Vemurafenib Versus Vemurafenib + Cobimetinib in Previously Untreated BRAFV600 Mutation-Positive Patients With Unresectable Locally Advanced or Metastatic Melanoma (NCT01689519)

James Larkin, Paolo A. Ascierto, Brigitte Dréno, et al. Combined Vemurafenib and Cobimetinib in BRAF-Mutated Melanoma. NEJM 2014; published online September 29. DOI: 10.1056/NEJMoa1408868

www.esmo.org/Conferences/ESMO-2014-Congress/News-Articles/Cobimetinib-in-Combination-with-Vemurafenib-Improves-PFS-in-BRAF-Mutant-Unresectable-Locally-Advanced-or-Metastatic-Melanoma

 

 

Rescooped by Stéphane Campana from Melanoma BRAF Inhibitors Review
Scoop.it!

BMS and Merck Race to Melanoma Treatment Market

BMS and Merck Race to Melanoma Treatment Market | Orphan Disease's Challenges | Scoop.it
Merck’s Keytruda, recently approved in the US to treat patients with advanced melanoma that have stopped responding to other cancer therapies, will soon face strong competition from Bristol-Myers Squibb’s (BMS) Opdivo,  an analyst from research and...

Via Krishan Maggon
more...
Krishan Maggon 's curator insight, September 16, 2014 1:17 PM

BMS will take the lead in the melanoma market with its immuno combo grugs.

Rescooped by Stéphane Campana from PARP Inhibitors Cancer Review
Scoop.it!

Personalized medicine: Special treatment

Personalized medicine: Special treatment | Orphan Disease's Challenges | Scoop.it
Therapies targeted at the specific genetics of a patient's lung cancer have proved harder to realize than expected.

Via Krishan Maggon
more...
Krishan Maggon 's curator insight, September 11, 2014 12:10 PM

NATURE | OUTLOOK

Personalized medicine: Special treatmentMichael EisensteinNature 513, S8–S9 (11 September 2014) doi:10.1038/513S8aPublished online 10 September 2014
Scooped by Stéphane Campana
Scoop.it!

FDA Okays Avastin to Treat Aggressive and Late-Stage Cervical Cancer

FDA Okays Avastin to Treat Aggressive and Late-Stage Cervical Cancer | Orphan Disease's Challenges | Scoop.it
The FDA panel gave a nod to Avastin - a drug to treat patients with persistent, recurrent or late-stage cervical cancer.
more...
No comment yet.
Scooped by Stéphane Campana
Scoop.it!

Roche to buy Danish biopharmaceutical company Santaris Pharma for up to $450 million

Roche to buy Danish biopharmaceutical company Santaris Pharma for up to $450 million | Orphan Disease's Challenges | Scoop.it
Roche will pay an initial $250 million and further payments of up to $200 million for the Danish biopharmaceutical company, which specializes in so-called RNA-targeting therapeutics
more...
No comment yet.
Scooped by Stéphane Campana
Scoop.it!

AstraZeneca Initiates CDx Partnerships with Roche & Qiagen

AstraZeneca Initiates CDx Partnerships with Roche & Qiagen | Orphan Disease's Challenges | Scoop.it
Pharma will co-develop diagnostics for two NSCLC drugs, investigational compound AZD9291 and EC-approved Iressa
more...
No comment yet.
Scooped by Stéphane Campana
Scoop.it!

Gene therapy drug approval granted to GSK - BBC News

Gene therapy drug approval granted to GSK - BBC News | Orphan Disease's Challenges | Scoop.it
Regulators have given one of the world's largest drug companies approval to sell a new gene therapy.
more...
No comment yet.
Scooped by Stéphane Campana
Scoop.it!

How patients with rare diseases can scour the world for a cure

How patients with rare diseases can scour the world for a cure | Orphan Disease's Challenges | Scoop.it
TED Fellow Jimmy Lin shares how patients suffering from a rare genetic disorder can find the equally rare researcher who can help.
more...
No comment yet.
Scooped by Stéphane Campana
Scoop.it!

12 activists share what they want you to know about living with a rare disease

12 activists share what they want you to know about living with a rare disease | Orphan Disease's Challenges | Scoop.it
Living with a rare disease can be trying. Here's what activists want you to know about the realities, obstacles and triumphs of the rare disease community.
more...
No comment yet.
Rescooped by Stéphane Campana from Rare diseases & Orphan drugs
Scoop.it!

Journée des Maladies Rares Vidéo officielle 2016

http://www.rarediseaseday.org/

Via Gabriel PICART
more...
No comment yet.
Rescooped by Stéphane Campana from Biotech, Pharmas and ideas
Scoop.it!

Rare disease companies' existential challenge: Patient enrollment

Rare disease companies' existential challenge: Patient enrollment | Orphan Disease's Challenges | Scoop.it

“ Three companies are recruiting patients with Nieman Pick Type C (NPC), a very rare and deadly disease, for clinical trials.”


Via Herve Ansanay
more...
No comment yet.
Rescooped by Stéphane Campana from healthcare technology
Scoop.it!

Electronic Health Record Data Mining -- Is It a Dirty Word?

Electronic Health Record Data Mining -- Is It a Dirty Word? | Orphan Disease's Challenges | Scoop.it

Data mining has received its fair share of negative press, but the use of electronic health record data for secondary purposes could be a boon for health care research.

 

With the broader availability of data from electronic health records, the secondary use of this rich clinical data presents the opportunity for data mining. However, data mining has received negative press when used by pharmaceutical companies to monitor physician prescribing patterns.

 

In many industries, mining of Big Data has become a profitable source for business intelligence. Everything from financial trends to social media sentiment analysis is game. With the ability to search personal data through new tools like Facebook's Graph Search and increasingly targeted marketing based on huge databases of personal data, the concept of data mining is becoming synonymous with invading privacy.

 

In health care, the expanding use of EHRs creates opportunities for secondary use of health data collected at the point of care. Such data are unique in that they enable data mining of real-world clinical practice on millions of patients in large health systems. Unlike data collected in the carefully structured setting of a clinical trial, large groups of patients can be studied retrospectively as were treated for a variety of conditions.

 

 


Via nrip
more...
No comment yet.
Rescooped by Stéphane Campana from PARP Inhibitors Cancer Review
Scoop.it!

Perjeta (pertuzumab, Roche) extends overall survival by 15.7 months in HER2+ve metastatic breast cancer Phase III CLEOPATRA study. ESMO 2014

Perjeta (pertuzumab, Roche) extends overall survival by 15.7 months in HER2+ve metastatic breast cancer Phase III CLEOPATRA study. ESMO 2014 | Orphan Disease's Challenges | Scoop.it

Roche today announced final survival results from the Phase III CLEOPATRA study, which showed that adding Perjeta (pertuzumab) to Herceptin (trastuzumab) and docetaxel chemotherapy extended the lives (overall survival; OS) of people with previously untreated HER2-positive metastatic breast cancer (mBC) by 15.7 months compared to Herceptin and chemotherapy (median OS: 56.5 vs. 40.8 months).1 No new safety signals were observed in the study.1 These data will be presented today in the Presidential Symposium at the European Society for Medical Oncology (ESMO) 2014 congress in Madrid, Spain (Abstract #350O_PR; Sunday, September 28, 4:00 – 5:30 p.m. CEST) and are also featured in the official ESMO Press Briefing.

 

Perjeta in combination with Herceptin and docetaxel chemotherapy is approved in the United States and the EU for people with previously untreated HER2-positive mBC. The Perjeta regimen has also been granted accelerated approval as a neoadjuvant treatment (use before surgery) for HER2-positive early breast cancer (eBC) by the U.S. Food and Drug Administration (FDA). An application to update the Marketing Authorisation to include this indication has also recently been submitted to the European Medicines Agency.

About the CLEOPATRA Study2

CLEOPATRA (CLinical Evaluation Of Pertuzumab And TRAstuzumab) was an international, Phase III, randomised, double-blind, placebo-controlled study. The study compared the combination of Perjeta, Herceptin and docetaxel chemotherapy with placebo, Herceptin and chemotherapy in 808 people with previously untreated HER2-positive mBC, or with HER2-positive mBC that had come back after prior therapy in the adjuvant or neoadjuvant setting. The primary endpoint of the study was progression-free survival (PFS) as assessed by an independent review committee. Secondary endpoints included OS and safety profile.

 

The risk of death was reduced by 32 percent for people who received the Perjeta regimen compared to those who received Herceptin and chemotherapy (HR=0.68, 95 percent CI 0.56-0.84; p=0.0002).People who received the Perjeta regimen had a 32 percent reduction in the risk of their disease worsening or death (PFS; HR=0.68, 95 percent CI 0.58-0.80) compared to people who received Herceptin and chemotherapy.With longer follow-up, the median PFS improvement of more than six months was maintained (median PFS of 18.7 months for people who received Perjeta, Herceptin and chemotherapy compared to 12.4 months for those who received Herceptin and chemotherapy).The most common adverse events (AEs, rate greater than 25 percent or greater than 5 percent difference between study groups) seen with the Perjeta regimen were diarrhoea, rash, mucosal inflammation, headache, upper respiratory tract infection, itching, low white blood cell count with fever, dry skin and muscle spasms.The most common Grade 3-4 AEs (rate greater than 10 percent) were low white blood cell count, low white blood cell count with fever and a decrease in a certain type of white blood cells.

 


Via Krishan Maggon
more...
Krishan Maggon 's curator insight, September 28, 2014 8:00 AM

This is the largest OS gain observed in Phase III oncology trials and may benefit all HER2+ve advanced metastatic breast cancer patients. ESMO presentation

 

Final data from Phase III CLEOPATRA study showed people with previously untreated HER2-positive metastatic breast cancer who received Perjeta, Herceptin and docetaxel chemotherapy lived a median of 56.5 months compared to 40.8 months for people who received Herceptin and chemotherapy1Median overall survival of almost five years is the longest observed to date in people with this aggressive type of advanced breast cancer1
Rescooped by Stéphane Campana from Pharma Biotech Industry Review (Krishan Maggon)
Scoop.it!

Japan stem-cell trial stirs envy

Japan stem-cell trial stirs envy | Orphan Disease's Challenges | Scoop.it
Researchers elsewhere can’t wait to test iPS cells in humans.

 

 a Japanese woman with visual impairment had become the first person to receive a therapy derived from stem cells known as induced pluripotent stem (iPS) cells.

 

A lot rides on this trial. If the procedure proves safe, it could soften the stance of regulatory bodies in other nations towards human trials of iPS cells, and it could pave the way for treatments for other conditions, such as Parkinson’s disease and diabetes. It could also cement Japan, recently plagued by a stem-cell scandal, as a frontrunner in iPS-cell research.


Via Krishan Maggon
more...
Krishan Maggon 's curator insight, September 17, 2014 5:35 PM

open access

 

Japan stem-cell trial stirs envy

Researchers elsewhere can’t wait to test iPS cells in humans.

Sara Reardon& David Cyranoski16 September 2014
Rescooped by Stéphane Campana from Alzheimer's Disease R&D Review
Scoop.it!

AstraZeneca and Lilly alliance to develop and commercialise BACE inhibitor AZD3293 for Alzheimer’s disease

AstraZeneca and Lilly alliance to develop and commercialise BACE inhibitor AZD3293 for Alzheimer’s disease | Orphan Disease's Challenges | Scoop.it

AstraZeneca and Eli Lilly and Company (Lilly) today announced an agreement to jointly develop and commercialise AZD3293, an oral beta secretase cleaving enzyme (BACE) inhibitor currently in development as a potential treatment for Alzheimer’s disease.

 

Under the terms of the agreement, Lilly will pay AstraZeneca up to $500 million in development and regulatory milestone payments. AstraZeneca expects to receive the first milestone payment of $50 million in the first half of 2015. The companies will share all future costs equally for the development and commercialisation of AZD3293, as well as net global revenues post-launch.

 

AstraZeneca and Lilly aim to progress AZD3293 rapidly into a Phase II/III clinical trial in patients with early Alzheimer’s disease. Lilly will lead clinical development, working with researchers from AstraZeneca’s Innovative Medicines Unit for neuroscience, while AstraZeneca will be responsible for manufacturing. The companies will take joint responsibility for commercialisation of AZD3293.

 


Via Krishan Maggon
more...
Krishan Maggon 's curator insight, September 16, 2014 7:20 AM

Lilly agrees to pay upto $500 million in milestone, R&D, clinical trials and marketing costs for a risky shot at another BACE inhibitor for AD?

 

Astra Zeneca so called much touted smart management reduces its R&D risk as Lilly has more Phase III experience in the failed and still ongoing Phase III solanezumab AD trials. 

 

AZD3293 is an oral, potent and selective small molecule inhibitor of BACE that has been shown in Phase I studies to significantly and dose-dependently reduce levels of amyloid beta in the cerebro-spinal fluid of Alzheimer’s patients and healthy volunteers.

Rescooped by Stéphane Campana from ReactNow - Latest News updated around the clock
Scoop.it!

Roche to Buy Drug Maker InterMune for $8.3 Billion - New York Times

Roche to Buy Drug Maker InterMune for $8.3 Billion - New York Times | Orphan Disease's Challenges | Scoop.it
Wall Street Journal Roche to Buy Drug Maker InterMune for $8.3 Billion New York Times Roche said the acquisition would strengthen its portfolio of drugs for respiratory diseases, which has not been a major business for a company mainly known for...

Via ReactNow
more...
No comment yet.
Rescooped by Stéphane Campana from Pharma Biotech Industry Review (Krishan Maggon)
Scoop.it!

The Recovering Americans and the 'Top Secret' Ebola Treatment

The Recovering Americans and the 'Top Secret' Ebola Treatment | Orphan Disease's Challenges | Scoop.it
Two U.S. doctors with Ebola have improved after receiving an experimental antibody.

Via Krishan Maggon
more...
No comment yet.
Rescooped by Stéphane Campana from PARP Inhibitors Cancer Review
Scoop.it!

Gazyvaro® (Obinutuzumab, Roche) approved in Europe for leukemia

Gazyvaro® (Obinutuzumab, Roche) approved in Europe for leukemia | Orphan Disease's Challenges | Scoop.it

 European Commission has approved Gazyvaro (obinutuzumab) in combination with chlorambucil chemotherapy for the treatment of people with previously untreated chronic lymphocytic leukemia who have comorbidities making them unsuitable for an intensive therapy (full-dose fludarabine based therapy).


Via Krishan Maggon
more...
Krishan Maggon 's curator insight, July 29, 2014 7:53 AM
Gazyvaro, the first type II, glycoengineered anti-CD20 monoclonal antibody is now approved in Europe for patients with previously untreated chronic lymphocytic leukemia (CLL)Approval is based on the phase III CLL11 study which showed that Gazyvaro plus chlorambucil chemotherapy significantly extended the amount of time people lived without their disease worsening while increasing the depth of their remissions compared to standard treatments such as chlorambucil or MabThera plus chlorambucil