EXECUTIVE SUMMARY INNOVATION IS THE KEY TO CHANGING THE PARADIGM FOR THE TREATMENT OF PAIN AND ADDICTION TO CREATE AN AMERICA FREE OF OPIOID ADDICTION The Bitechnlgy Innvatin Organizatin (BIO) and ur member cmpanies are cmmitted t unleashing the pwer f scientific innvatin t develp slutins that will allw fr an America free f prescriptin piid addictin. BIO believes this plan shuld rest n three legs. 1. ADVANCE OUR UNDERSTANDING OF THE BIOLOGY OF PAIN AND ADDICTION TO ENABLE THE DEVELOPMENT OF INNOVATIVE TREATMENTS FOR PAIN AND ADDICTION AND ENSURE APPROPRIATE AND OPTIMAL USE OF EXISTING THERAPIES Supprt a mre cmprehensive understanding f the preclinical pain and addictin envirnment Supprt investment in research at Natinal Institutes f Health (NIH)and ther majr centers fcused n the bilgy f pain and addictin 2. ENSURE PATIENTS SUFFERING FROM PAIN OR ADDICTION ARE ABLE TO RECEIVE THE RIGHT TREATMENT AT THE RIGHT TIME WITH THE RIGHT SUPPORT, WITHOUT STIGMA Remve cverage and reimbursement barriers that are preventing patient-centric decisins abut and access t the mst effective treatments fr pain and addictin Advance implementatin f and adherence t laws and regulatins prtecting and prmting access t resurces fr cmprehensive pain and addictin treatment 3. STIMULATE RESEARCH & DEVELOPMENT (R&D) OF INNOVATIVE TREATMENTS THAT EFFECTIVELY TREAT PAIN AND OPIOID ADDICTION AND PREVENT ABUSE Increase leadership engagement, expertise, and resurces within the Fd and Drug Administratin (FDA) and the Drug Enfrcement Agency (DEA) t prmte effective and efficient review f innvative treatments fr pain and addictin Mdernize and streamline drug develpment and review prcesses t enable effective and efficient drug develpment and review, and stimulate investment in innvatin Establish and utilize expedited apprval pathways and ther incentives t stimulate innvatin
Nvember 1, 2017 Page 2 f 6 ADVANCE OUR UNDERSTANDING OF THE BIOLOGY OF PAIN AND ADDICTION TO ENABLE THE DEVELOPMENT OF INNOVATIVE TREATMENTS FOR PAIN AND ADDICTION AND ENSURE APPROPRIATE AND OPTIMAL USE OF EXISTING THERAPIES Understanding the bilgy underlying pain and addictin are critical t supprting the discvery f nvel and safer treatments fr bth pain and addictin as well as fr ensuring that patients receive the right treatment at the right time, with the right supprt, and withut stigma. Unfrtunately, ur current understanding f pain and addictin bilgy is lacking. Supprt a mre cmprehensive understanding f the preclinical pain and addictin envirnment A mre cmprehensive understanding f mechanisms underlying different types f pain as well as bimarkers that allw fr the differentiatin f pain subsets, and identificatin f preclinical mdels that better translate t therapeutic utcmes fr peple are needed t infrm the develpment f nvel and safer treatments. Supprt research investigating ptential therapeutic targets with increased receptr/intracellular signaling selectivity Supprt research investigating nvel mechanisms fr preventing and/r treating piid addictin and verdse Supprt the identificatin and validatin f bimarkers that identify subsets f pain and mre accurately predict treatment respnse fr bth pain and addictin Supprt the identificatin and utilizatin f mre effective drug screening mdels and assays that better translate t therapeutic utcmes fr patients Supprt research fcused n elucidating the underlying bilgical mechanisms f different types f pain and addictin, as well as differences in pain perceptin Supprt a mre cmprehensive understanding f the clinical pain and addictin envirnment Cmprehensive methds fr evaluating pain in the patient, tls that allw fr better diagnsis f pain, as well as the establishment f clinical trial registries, clinical trial netwrks, and data sharing and analytics will be critical fr ensuring that patients are able t receive the mst apprpriate treatment. Encurage the develpment and use, in bth the clinic and clinical trial setting, f mre cmprehensive and bjective tls fr assessing pain that take int accunt acute versus chrnic pain, the pssible neurbilgical and psychscial mechanisms underlying pain, individual differences in pain perceptin, and distinctins between smatic and psychic pain Supprt research int the develpment f diagnstic tls that enable the identificatin f specific causes f pain
Nvember 1, 2017 Page 3 f 6 Prmte multi-disciplinary appraches t treating pain and addictin by enabling appraches fr determining risk r susceptibility fr individual patients, and assciate utcmes with the basic neurbilgy and/r pharmaclgical alteratins Establish clinical trial registries and clinical trial netwrks fr pain and addictin t help develp, validate, refine, and deliver new treatment ptins t patients Encurage public and private payers, academic researchers, and industry spnsrs t apply data analytic methdlgies t answer imprtant questins abut pain and addictin treatment, including but nt limited t: Which treatments wrk best fr whm? What is the ptimal duratin f specific treatments? When shuld a patient s treatment regimen be changed, and hw? Given the critical rle that treatment must play in ending the piid epidemic, every effrt shuld be made t maximize the use f data t guide plicy-making decisins that supprt the mst effective appraches. INNOVATION IS THE KEY TO CHANGING THE PARADIGM FOR THE TREATMENT OF PAIN AND ADDICTION TO CREATE AN AMERICA FREE OF OPIOID ADDICTION The Bitechnlgy Innvatin Organizatin (BIO) and ur member cmpanies are cmmitted t unleashing the pwer f scientific innvatin t develp slutins that will allw fr an America free f prescriptin piid addictin. ENSURE PATIENTS SUFFERING FROM PAIN OR ADDICTION ARE ABLE TO RECEIVE THE RIGHT TREATMENT AT THE RIGHT TIME WITH THE RIGHT SUPPORT, WITHOUT STIGMA Patients suffering frm pain r addictin shuld have knwledge f, access t, and cverage fr all available medicines in rder t make infrmed decisins abut their treatment. A patient-centric apprach t the treatment f pain and addictin will help prevent piid addictin and prvide better care fr thse wh are diagnsed with addictin. Remve cverage and reimbursement barriers that are preventing patientcentric decisins abut and access t the mst effective treatments fr pain and addictin The healthcare cverage and reimbursement system includes barriers t current treatment ptins fr pain and addictin, and presents ptential barriers fr access t advances in treatment. Addressing these cverage issues is imperative t delivery f the right treatment at the right time fr each patient suffering frm pain and addictin. Establish apprpriate payer cverage pathways fr current pain and addictin treatments and innvative treatment ptins (sme examples include but are nt limited t nn-piid treatments, practitiner-administered (r nn-ral), and abuse deterrent frmulatins) including:
Nvember 1, 2017 Page 4 f 6 Ensure that the rute r lcatin f administratin f specific pain r addictin treatments des nt hinder patient access t treatment Secure timely designatin f specific healthcare payment cdes fr each apprved therapy fr pain and addictin fr adequate reimbursement f therapies Eliminate insurance cverage barriers that impact patient access t the mst apprpriate r mst timely curse f treatment fr their given cnditin (i.e., step-therapy, fail first, cst sharing requirements, care limits n substance use disrder treatment) Prvide cverage fr medicatin assisted treatment (MAT) fr substance use disrders and fr all Fd and Drug Administratin (FDA) apprved substance abuse disrder treatments acrss health insurance access pints Withdrawal f cverage fr nn-abuse deterrent frmulatins when abuse deterrent frmulatins with identical patient utcmes are available Advance implementatin f and adherence t laws and regulatins prtecting and prmting access t resurces fr cmprehensive pain and addictin treatment Recent and future legislative and regulatry activities include critical refrms t ensure patient access t cmprehensive treatment fr pain and addictin and activities t cmbat the piid crisis. Ensure equitable cverage fr treatment f substance use disrders as mandated under the Mental Health Parity and Addictin Equity Act (MHPAEA) Supprt prper implementatin f the aligned directives and activities f the Cmprehensive Addictin and Recvery Act (CARA) Supprt future legislative and regulatry effrts t revlutinize the treatment f and respnse t pain and addictin STIMULATE RESEARCH & DEVELOPMENT (R&D) OF INNOVATIVE TREATMENTS THAT EFFECTIVELY TREAT PAIN AND OPIOID ADDICTION AND PREVENT ABUSE Steady investments in drug discvery are critical fr drug develpment and fr bringing treatments t patients that need them. Over the past ten years investments in treatments fr pain have been incnsistent. Several factrs influence the lack f investments int treatments fr pain and addictin including uncertainty in bth cverage and access f treatments and drug develpment and regulatry review requirements. Mdernizatin f drug develpment and apprval prcesses, increased engagement between the FDA and industry spnsrs, and incentives fr the develpment f new therapies will help mitigate sme f these issues. Increase leadership engagement, expertise, and resurces within the Fd and Drug Administratin (FDA) and the Drug Enfrcement Agency (DEA) t
Nvember 1, 2017 Page 5 f 6 prmte effective and efficient review f innvative treatments fr pain and addictin Currently, limited resurces within the FDA and a lack f cmmunicatin between the FDA, the DEA, and industry spnsrs has led t inefficient review f nvel and safer treatments fr bth pain and addictin and have prevented individuals frm receiving apprpriate access t medicines that treat pain and addictin. Increased resurces within the FDA and imprved cmmunicatin between the FDA and industry spnsrs will help clarify the Agency s expectatins and speed apprval and access t nvel and safer treatments. Increase engagement f senir FDA leadership t supprt transparency, cnsistency, and effective cmmunicatin between FDA and industry spnsrs, thrughut the drug apprval prcess Increase engagement f senir FDA and DEA leadership t supprt transparency, cnsistency, and effective cmmunicatin with spnsrs t enable effective and efficient apprval prcesses and apprpriate access t medicines that treat pain and addictin Increase resurces and expertise within the FDA t ensure efficient and effective review f abuse deterrent frmulatins f cnventinal piids, nvel and safer treatments, and innvative treatments that have, in general, lwer r n abuse liability Advance the develpment f FDA draft guidance and encurage clarity in existing guidance t address issues related t abuse deterrent frmulatins and analgesic indicatins in a way that supprts innvatin Ensure that best practices are adpted by FDA divisins respnsible fr reviewing treatments fr pain and addictin Ensure cnsistent applicatin f warnings and ther safety infrmatin regardless f diagnsis fr all piid medicatins apprved fr the treatment f pain and addictin Encurage glbal harmnizatin f regulatry requirements fr treatments fr pain and addictin Mdernize and streamline drug develpment and review prcesses t enable effective and efficient drug develpment and review, and stimulate investment in innvatin Current apprval requirements fr nvel and safer treatments fr pain and addictin are ften inefficient, redundant, and unsafe. Nt nly des this slw apprval but it is als prhibitive fr investments in pain and addictin treatments. The utilizatin f innvative clinical trial designs, and new data surces, as well as the validatin and quantificatin f bimarkers fr pain and addictin will allw fr mre effective, efficient, and safer drug develpment and review. Mdernize and streamline clinical trial requirements by streamlining the number f clinical trials required fr each type f pain and utilizatin f mdern appraches fr the cllectin f safety data. Encurage the utilizatin f innvative clinical trials designs and new data surces that allw fr the use f cmmn cntrl data t evaluate efficacy in multiple therapies and reduce the number f individuals expsed t cntrl interventins
Nvember 1, 2017 Page 6 f 6 Enable validatin and qualificatin f bimarkers that help identify subsets f pain r risk fr addictin Establish and utilize expedited apprval pathways and ther incentives t stimulate innvatin While there is a clear need t develp and apprve nvel and safer treatments fr pain and addictin, drug develpment is risky, and time and resurce intensive. Several accelerated apprval pathways and incentives have been successfully implemented t encurage innvatin and investment in ther areas that are in urgent need f treatments, including rare diseases (Orphan Drug Act) and antibitics that are effective against drug resistant bacteria (Generating Antibitic Incentives Nw (GAIN) Act). Similar appraches shuld be implemented in rder t encurage investments fr the develpment f nvel and safer treatments fr pain and addictin. Supprt the utilizatin f expedited apprval pathways fr nvel and safer treatments fr pain and addictin Supprt the utilizatin f additinal incentives fr nvel and safer treatments fr pain and addictin