New evaluation techniques to drive R&D sector: CPhI Report

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The R&D market is diversifying innovation, with increased out/in licensing of technology, partnerships, and mergers. Improvements in evaluation have also been credited with helping the sector grow at an impressive eight per cent, with long-term objectives now being considered at earlier stages within the development process, according to the findings of  CPhI Worldwide Pharma Insights report on R&D.

Furthermore, the industry is evolving its model, maintaining innovative output whilst also standardising approaches to measuring effectiveness, and crucially, returns on investment- with 40 per cent using QbD for analytical and tech, 18 per cent using 6 Sigma, 15 per cent stage gate and 12 per cent lean techniques to evaluate effectiveness.

Unsurprisingly, with the milestone nature of moving between clinical stages, balancing long and short-term goals was seen as a major challenge (53 per cent) and improving efficiency (38 per cent) also highlighted the growing efforts to improve ROI between stages. However, a clear trend has emerged from this with more and more companies involving commercial side at an earlier and earlier stage with 30 per cent beginning in pre-clinical and a further 30 per cent prior to phase iii.

Almost a quarter of respondents sighted cancer as a major focus area for 2014, with antibiotics (13 per cent), cardiovascular (12 per cent) and CNS (12 per cent) targets also featuring notably. Additional evidence for the cancer-focus also emerged from the novel areas companies are working on, with 37 per cent researching combination drugs and 20 per cent personalised medicines. Outside of cancer, it appears that improved drug delivery mechanisms are targeted as 17 per cent were investing in nanotechnology and 12 per cent directly in drug devices.

However, the source of innovation is increasingly diverse with growing partnerships (75 per cent), mergers 20 per cent, and out/in-licensing of technology (55 per cent and 60 per cent respectively). Clearly, a major factor in competitiveness both now and in the future is access to technology, and we are seeing a more collaborative approach. This is an important trend and one that should enable the industry to continually innovate, with cross-pollination of projects, where even the smallest biotech can access crucial technology that enable them to move projects forward.

“With product development processes being increasingly managed through quality and control methods, the cost of and access to technology will be the essential access point on many projects ultimate viability.” Chris Kilbee, Group Director Pharma.

Highlighting the need for access to new techniques and technologies, 33 per cent of respondents were partnering with research institutes, a further 15 per cent with research-focussed companies and 10 per cent with universities.

Drawing all these findings together the report concludes there is a clear desire amongst all companies to ensure future R&D spend is prudent and focused on bringing future products to market faster- and not just innovative targets. Monitoring processes more prescriptively and standardising approaches should enable a quicker transfer between phases. Most interesting for the future, we are now witnessing an ‘open innovation approach’ to technology- which will enable reduced R&D timelines and better results.

“What we are now seeing is the maturation of the R&D sector having gone through a period of change over the last few years with innovation moving down stream from big pharma. However, we are now at a point where innovation is coming in from new and more diverse fields as different areas of science collaborate and bring new techniques into pharma production. This trend coupled with a commitment to tackle long term project viabilities, alongside standardised methods for practice, should help reduce bottlenecks in the pipeline and bring more targets to market in a more cost effective manner. It is a really exciting period for the R&D sector with new ideas, nanotechnology and manufacturing techniques and process bringing a new rigour and commercial focus to future projects,”Chris Kilbee added.

Source: PharmaBiz


FDA Speeds Innovation in Rare Disease Therapies

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Patients often need advocates, and that can be especially true for people with a rare disease, who have unique problems and may have little or no support or available treatment.

The Food and Drug Administration (FDA) is committed to helping patients and advancing rare disease therapies through the development of “orphan” medical products, including drugs, biologics (such as a protein, vaccine or blood product), and devices used to treat a rare disease or condition. The Orphan Drug Act defines a disease as rare if fewer than 200,000 people in the United States have it.

FDA’s Office of Orphan Products Development (OOPD), in collaboration with the Center for Drug Evaluation and Research (CDER), is launching web-based educational resources for patients and industry on FDA-related rare disease topics. The first of these resources will debut Feb. 28, 2014, in recognition of International Rare Disease Day, and will cover topics that include how to interact with the agency and how to access therapies that are currently being studied.

Rare Disease Day, which is commemorated on the last day in February, is a global campaign to raise awareness of the more than 250 million people worldwide who suffer from rare diseases. About 7,000 rare diseases have been identified around the world; some have familiar names, such as cystic fibrosis and Lou Gehrig’s disease, but many don’t. Thirty million Americans have rare diseases, which can be chronic, progressive, debilitating, disabling, severe or life-threatening. About 80 percent of rare diseases are genetic, and about half of all rare diseases affect children.

FDA’s Office of Orphan Products Development

FDA is in a unique position to help those who suffer from rare diseases by offering several important incentives to promote the development of products for rare diseases, including:

  • granting orphan drug designation for drugs and biologics, which encourages companies to develop a product by giving them financial and other incentives;
  • providing grant funds to further the clinical development of drugs, biologics, medical devices and medical foods for the treatment of rare diseases;
  • granting humanitarian use device (HUD) designation for medical devices for rare diseases, which makes these products eligible to enter the market via a separate marketing pathway known as the Humanitarian Device Exemption (HDE) Pathway; and
  • providing grants to fund consortia to promote the development of pediatric devices, many of which are used to treat and diagnose rare diseases.

Gayatri R. Rao, M.D., J.D., director of OOPD, says 2013 was a record year for her office. The number of requests under FDA’s Orphan Drug Designation Program rose about 18% in 2013 over 2012. FDA received nearly 450 orphan-drug designation requests and designated 258 promising orphan drugs, a 40% increase over 2012, says Rao.

“While many factors may be contributing to the growth of orphan drug development, patients are continuing to drive the push for innovation and treatments,” she says.

In 2013, FDA approved 33 drugs for treating rare diseases. Since 1983, FDA has approved more than 450 drugs and biologic products for rare diseases. In the decade prior to the Orphan Drug Act, fewer than 10 treatments had been developed by industry for rare diseases.

“Last year, FDA funded 15 new orphan products grants for about $14 million, all supporting clinical research in rare diseases,” says Rao. “Many of the studies that we have funded have supported the approval of orphan drugs and devices for rare disease patients.”

On the device side, in 2013, FDA designated 16 medical devices for the treatment or diagnosis of rare diseases and approved two under the HDE pathway.

In addition, based on feedback from stakeholders, OOPD revamped its Pediatric Device Consortia (PDC) Grant Program.

“Now we focus more heavily on a consortium’s ability to provide more holistic advice on device development,” Rao says. “To bring a device to market, you need engineers, scientists, clinicians, business people and regulatory people collaborating for success.”

Consortia advise on all sorts of devices through various stages of development, from the prototype stage through animal testing, clinical testing and commercialization. OOPD received 14 PDC applications last year and funded half of them.

In addition to these incentive programs, last year, OOPD, in conjunction with CDER and FDA’s Center for Biologics Evaluation and Research (CBER), began administering the new Rare Pediatric Disease Priority Review Voucher Program to promote the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. In 2013, FDA received five requests for designation as a “rare pediatric disease” and designated three. In 2014, FDA awarded the first voucher under this program for the development of Vivizim to treat patients with a rare congenital enzyme disorder called Morquio A syndrome.

Patients Are Key to Success

FDA is committed to improving the lives of people with rare diseases, says Rao. “We can make progress only if we—patients, industry, researchers and FDA—work together to develop safe and effective products for rare diseases.”

Patients play a critical role in that success, and FDA wants to hear their important voice.

“Patients are best able to say what is difficult for them and what sorts of risks they are willing to take,” says Rao. “Because of that, FDA is focused on getting that perspective earlier in the process.”

This article appears on FDA’s Consumer Updates page, which features the latest on all FDA-regulated products.


GAAMA urges state govt to relax loan rate for ASU industry to 4%

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With a view to provide required impetus to the ayurvedic industry, Gujarat Ayurvedic Aushadh Manufacturers Association (GAAMA) recently urged the Gujarat government to take pro-active measures towards reducing the industry loan interest rate to 4 per cent. Experts feel that such incentive from the government is a must for the sustenance of the ASU industry, especially under the rapidly changing regulatory set up, which is increasingly putting more economic stress on the small scale sector.

In a representation sent to the state health ministry, GAAMA pointed out that the industry which manly comprises of small scale manufacturers are finding it very difficult to meet the regulatory requirements due to lack of adequate funds to sponsor the same. Making it worse is the huge interest rate of 13 per cent that the industry has to shell out on the loan, for them to comply with the regulatory requirements, an added burden on the already cash crunched sector.

The ASU sector mainly comprises small and medium scale companies, struggling to make their ends meet, with hardly any financial support from the government. Prabodh Shah, president, GAMMA pointed out that while the industry does appreciate the government’s role in strengthening the regulatory apparatus further, consideration should also be given on the fact whether the small scale industries in this sector are financially capable to deal with these changes as well.

“What we need is a good government policy or framework that is constituted after deliberating all the woes and issues of the industry. We accept that the steps taken by the government are for the greater good of the sector, as it will in the long run help us in getting acceptability and credibility globally. However, the question is, are we financially strong enough to handle this change right now, which comes with heavy investment, and are we provided with the required impetus to sponsor this changes,” Shah asked.

He further stressed that considering the current financial state of the sector, the government should provide with a helping hand by relaxing some norms like reducing the interest rate on loan for this sector, so that more companies can willingly participate in the same. As per the regulatory requirements now even the ASU sector has to comply with the Good Manufacturing Practices (GMP) and provisions of Schedule T, which deals with minimum regulatory requirements for the ASU industry.

Source: PharmaBiz

CDSCO to celebrate 2014 as patient & animal safety year

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Celebrating 2014 as ‘Patient and Animal Safety Year’, the Central Drugs Control Organisation (CDSCO) has decided to strengthen the regulatory system to ensure that the drugs imported into the country are checked for their compliance with the existing provisions of the rules and norms.

“The CDSCO is celebrating the year 2014 as Patient and Animal Safety year. In its endeavour to ensure the mission of CDCO to safeguard and enhance the public health by assuring safety, efficacy and quality of drugs, cosmetics and medical devices in letter and spirit, it has been decided that the drug products which are being imported in the country be checked for their compliance with the provisions of the Drugs and Cosmetics Act 1940 and Drugs and Cosmetics Rules, 1945,” a notice by DCGI Dr G N Singh said.

“As the patient’s safety is of paramount importance, it has been decided that the drugs for import into the country shall be allowed only if they are in compliance with the requirements of the Drugs and Cosmetics Act, 1940 and Drugs and Cosmetics Rules, 1945,” it added.

However, it does not mandate for creation of patient monitoring cells in line with the quality cells in the manufacturing units, as proposed earlier. The DCGI, a few months back, had informed Pharmabiz that the manufacturers would be asked to set up special cells to share the onus of patient safety also for the domestic market.

“We have to fix the onus of patient safety also on the manufacturers. These cells will have dedicated people and they will report to the chief executive officer of the company. We will also make several initiatives as part of the patients’ safety year,” he had told.

The move was also planned to create additional job opportunities in the pharmaceutical sector, especially for the trained pharmacists. The cells were expected to continuously monitor the quality of the products, apart from tracking the adverse reactions of medicines once in the market.

Source: PharmaBiz

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