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.
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.
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.
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
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.
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...
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
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...
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.
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.
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.
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.
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...
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).
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